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Medicare Coverage Policy ~ MCAC

Executive Committee

Transcript for March 1, 2000 Meeting
(For more information, contact the Executive Secretary.)

Note: The language on this website comes directly from the 
            transcribed testimony taken at this panel meeting. The views and 
            opinions are those of each of the experts and not those of the 
            Health Care Financing Administration. HCFA does not edit these 
            transcripts and makes no assertion as to their accuracy. 

  10        Medicare Coverage Advisory Committee
  11             Executive Committee Meeting
  15                    March 1, 2000
  19        Health Care Financing Administration
  20                   Main Auditorium
  21               7500 Security Boulevard
  22              Baltimore, Maryland 21244
   1                      PANELISTS
   2          Chairperson:  Harold C. Sox, M.D.
   3          Vice-Chairperson:  Robert H. Brook, M.D.
   5                   Voting Members:
   6       Thomas V. Holohan, M.A., M.D., F.A.C.P.
   7           Leslie P. Francis, J.D., Ph.D.
   8               John H. Ferguson, M.D.
   9               Robert L. Murray, Ph.D.
  10             Alan M. Garber, M.D., Ph.D.
  11           Michael D. Maves, M.D., M.B.A.
  12         Frank J. Papatheofanis, M.D., Ph.D.
  13                Ronald M. Davis, M.D.
  14             Daisy Alford-Smith, Ph.D.
  15                Joe W. Johnson, D.C.
  17                    HCFA Liaison:
  18            Hugh F. Hill, III, M.D., J.D.
  19            Jeffrey L. Kang, M.D., M.P.H.
  20               Consumer Representative:
  21              Linda A. Bergthold, Ph.D.
  22               Industry Representative:
  23              Randel E. Richner, M.P.H.
  24                 Executive Secretary:
  25                Sharon K. Lappalainen
   1                  TABLE OF CONTENTS
   2                                               Page
   3  Welcome and Introduction
   4       Harold C. Sox, M.D.                       5
   5  Conflict of Interest Statement
   6       Sharon Lappalainen                        7
   7  Opening Remarks
   8       Jeffrey L. Kang, M.D., M.P.H.             8
   9       Hugh F. Hill, III, M.D., J.D.             9
  10  Subcommittee Report/Chairperson's Remarks
  11       Harold C. Sox, M.D.                      12
  12  Open Public Session and
  13  Scheduled Commentaries
  14       Guido Tricot, M.D., Ph.D.                39
  15       Richard Justman, M.D.                    43
  16       Morgan Downey                            53
  17       Donald S. Baim, M.D.                     61
  18       Wayne I. Roe                             69
  19       Vicki Gottlich, Esquire                  80
  20       Larry M. Weisenthal, M.D., Ph.D.         88
  21       Sandy Sherman                            98
  22       Thomas Meskan                           100
  23  Recitation of Letter from ACP-ASIM
  24       Sharon Lappalainen                      110
   1              TABLE OF CONTENTS (Cont'd)
   2                                               Page
   3  HCFA Response to Subcommittee Report
   4       Jeffrey L. Kang, M.D.                   115
   5       Hugh F. Hill, III, M.D., J.D.           117
   6  Open Committee Deliberation                  124
   7  Lunch Recess                                 164
   8  Open Committee Deliberation                  164
   9  Open Public Comments
  10       Stephen J. Northrup                     258
  11       Thomas Meskan                           264
  12       Steven Lascher, D.V.M., M.P.H.          266
  13  Closing Comments
  14       Jeffrey L. Kang, M.D., M.P.H.           271
  15  Conclusion                                   276
   1                  PANEL PROCEEDINGS
   2            (The Executive committee meeting was
   3  called to order at 8:11 a.m., Wednesday, March 1,
   4  2000)
   5         DR. SOX:  I'd like to welcome everybody to
   6  this meeting of the Executive Committee of the
   7  MCAC.  The purpose of this meeting is to discuss
   8  the recommendations of the subcommittee that
   9  developed recommendations for all principles and
  10  procedures for the panels, and we'll be hearing
  11  from a number of representatives of the public
  12  today as well as from HCFA as well as from the
  13  subcommittee.
  14            We're going to start off by introducing
  15  the members of the Executive Committee who have
  16  made it already.  And I'll start on this side,
  17  and hopefully people will show up before we get
  18  around to the other side.
  19            Randel, will you introduce yourself and
  20  say where you're from.
  21            MS. RICHNER:  Randel Richner, Boston
  22  Scientific, industry representative.
  23            DR. BERGTHOLD:  I'm Linda Bergthold,
  24  and I'm the consumer representative.
  25            DR. MURRAY:  I'm Bob Murray from the
   1  Laboratory and Diagnostic Services panel.
   2            DR. HOLOHAN:  Tom Holohan, Chief of
   3  Patient Care Services, VA, headquartered in
   4  Washington.
   5            DR. HILL:  Hugh Hill, HCFA.
   6            DR. SOX:  I'm Hal Sox.  I'm from
   7  Dartmouth Medical School and Chairman of the
   8  Executive Committee.
   9            Jeff, will you introduce yourself.
  10            DR. KANG:  Hi.  Jeff Kang, Health Care
  11  Financing Administration.  I'll introduce myself
  12  later on also.  I apologize.  I'm a little under
  13  the weather here, as you can tell from my voice.
  14            MS. LAPPALAINEN:  Hello.  I'm Sharon
  15  Lappalainen with the Health Care Financing
  16  Administration.  I'm the Executive Secretary for
  17  the panel.
  18            DR. BROOK:  Robert Brook from RAND,
  19  UCLA.
  20            DR. GARBER:  Alan Garber, Department of
  21  Veterans Affairs, Stanford University.
  22            DR. DAVIS:  Ron Davis from the Henry
  23  Ford Health System in Detroit.
  24            DR. PAPATHEOFANIS:  Frank
  25  Papatheofanis, University of California in
   1  San Diego.
   2            DR. SMITH:  I'm Daisy Alford-Smith.
   3  I'm the Director of the Summit County Department
   4  of Human Services in Ohio as well as the
   5  Chairperson of the DME panel.
   6            DR. FERGUSON:  I'm John Ferguson, Chair
   7  of the Laboratory and Diagnostic Services panel
   8  as a consultant in healthcare.
   9            DR. SOX:  Now we're going to hear from
  10  Sharon with some procedural matters.
  11            MS. LAPPALAINEN:  Good morning and
  12  welcome to the panel, chairperson, the Executive
  13  Committee and members of the audience.
  14            The committee is here today to hear
  15  reports from its subcommittee and will discuss
  16  and consider the levels of evidence and types and
  17  presentation of information that it believes
  18  should be considered by the medical specialty
  19  panels at future MCAC meetings.
  20            For the record, I will read the
  21  conflict of interest statement for this panel.
  22            Conflict of interest for the Executive
  23  Committee meeting, March 1, 2000.
  24            The following announcement addresses
  25  conflict of interest issues associated with this
   1  meeting and is made part of the record to
   2  preclude even the appearance of an impropriety.
   3  To determine if any conflict existed, the agency
   4  reviewed the submitted agenda and all financial
   5  interests reported by the committee participants.
   6  The conflict of interest statutes prohibit
   7  special government's employees from participating
   8  in matters that could affect their or their
   9  employer's financial interests.
  10            The agency has determined that all
  11  members may participate in the matters before the
  12  committee today.  With respect to all other
  13  participants, we ask in the interest of fairness
  14  that all persons making statements or
  15  presentations disclose any current or previous
  16  financial involvement with any firm whose
  17  products or services they may wish to comment
  18  upon.
  19            And at this time I'll turn the panel
  20  over to Dr. Sox.
  21            DR. SOX:  Thank you.  First we're going
  22  to hear some opening remarks from Dr. Jeffrey
  23  Kang, who is Director of the Office of Clinical
  24  Standards and Quality.
  25            DR. KANG:  Dr. Sox, thanks a lot.
   1  Given my voice, I actually have some remarks that
   2  I really want to make at 10:30, 10:40, and I'm
   3  going to ask Hugh to read those for me.
   4            I just want to say in addition to being
   5  the director of the office, I am HCFA's chief
   6  clinical officer, and coverage is one of several
   7  responsibilities that I have.  I am greatly
   8  appreciative of the efforts of the Medicare
   9  Coverage Advisory Committee on coverage
  10  decisions.
  11            DR. SOX:  Thank you.
  12            DR. HILL:  If I can say Jeff's prepared
  13  remarks, thank you.  Good morning to you all.
  14  And on behalf of him, I would welcome you all and
  15  indicate that the office of clinical standards
  16  and quality are the folks that this committee and
  17  through you the other MCAC panels advise.  He's
  18  had a chance to meet many of you personally, but
  19  he wanted to welcome you and the members of the
  20  public that are here to the second meeting of the
  21  Executive Committee of the Medicare Coverage
  22  Advisory Committee.
  23            Jeff wanted me to express our
  24  appreciation to all those present for your
  25  participation in this process, and on behalf of
   1  HCFA's administrator Nancy-Ann DeParle, we want
   2  to especially thank the members of the committee
   3  for their service.
   4            Involvement in the initial phase of
   5  anything can be challenging and perhaps even more
   6  so when the government makes a change.  This
   7  seems to be true even when that change is
   8  universally applauded as an improvement in the
   9  way HCFA fulfills its responsibilities to our
  10  beneficiaries and the American public generally.
  11            Since the Medicare program began a
  12  little over a third of a century ago, some things
  13  have changed, and many have stayed the same.  We
  14  continue to see our mission as beneficiary
  15  focused.  While we strive for leadership in
  16  improving the health of all Americans, our goal
  17  remains assuring access to healthcare for the
  18  Medicare-eligible population as we increase our
  19  concern for planning in the access of future
  20  beneficiaries as well as today.
  21            We have moved towards working with
  22  providers of all types as customers and partners
  23  in delivering care in recognition of the
  24  continued central role of the care professional
  25  in assuring our beneficiaries' health.  My
   1  office, Jeff's office, has important new tools
   2  and programs for measuring and improving quality,
   3  but our eyes remain firmly fixed on Medicare's
   4  original and continued goal, better health.
   5            Let me tell you -- myself as well as
   6  Jeff would like to tell you -- although there are
   7  those that would say otherwise, making good
   8  beneficiary-focused coverage decisions is not a
   9  new goal for HCFA.  Yes, we've shifted from the
  10  role of processor and payer to the role of
  11  prudent purchaser.  And yes, we are more attuned
  12  to projections of future Medicare costs than we
  13  were at the program's beginnings, but coverage
  14  questions have been with us from the beginning.
  15            Congress gave us some guidance in the
  16  original statute.  Told us not to pay for
  17  anything that wasn't reasonable and necessary.
  18  You are, I think, aware of our renewed efforts to
  19  define what we think those terms mean.  But
  20  clearly, unarguably, science should have a role
  21  when we decide whether or not something is
  22  reasonable or necessary.  We think science should
  23  have the most important role.
  24            We recognize that the critical
  25  examination of the scientific literature is
   1  complex in every case and difficult in many.
   2  That's why we need your very expert help, and we
   3  are profoundly grateful for it.  Thank you.
   4            DR. SOX:  Thank you.  The next agenda
   5  item is the subcommittee report.  I'm going to
   6  deliver the subcommittee report, and if I could
   7  ask for the first transparency, we can get
   8  started.
   9            First let me introduce the members of
  10  the subcommittee, Randel Richner, Linda
  11  Bergthold, myself, Bob Brook, Alan Garber, and
  12  David Eddy was also a participant.  Dr. Eddy,
  13  because of the extreme press of other businesses,
  14  had to resign from the MCAC, but he nonetheless
  15  has substantial input into this document.
  16            DR. BERGTHOLD:  No, he hasn't.
  17            DR. SOX:  I beg your pardon?
  18            DR. HILL:  We're still talking.
  19            DR. SOX:  Oh.  We're still talking?
  20            DR. HILL:  We're hoping to keep him
  21  involved one way or another.
  22            DR. KANG:  He's resigned actually from
  23  being a chair of the panel but would like to stay
  24  on as a member of the MCAC.
  25            DR. SOX:  Wonderful.  Thank you for
   1  that correction.  I appreciate that.
   2            So our document has two purposes.  The
   3  first is to provide general guidance to the
   4  panels in the form of suggestions -- general
   5  suggestions, not detailed suggestions -- about
   6  how to evaluate evidence and focus on two
   7  characteristics of the evidence.
   8            The first is is it adequate to draw
   9  conclusions?  And the second is how big is the
  10  benefit of the intervention?
  11            So in fact, we asked these two
  12  questions.  Is the evidence concerning
  13  effectiveness in the Medicare population adequate
  14  to draw conclusions about magnitude of the
  15  effectiveness relative to other items or
  16  services?  And then secondly, if the evidence is
  17  adequate, how does the magnitude of effectiveness
  18  of the new medical item or service compare with
  19  that of other available interventions?
  20            Then the second major purpose of our
  21  document is to suggest specific procedures that
  22  the panels should follow in trying to draw
  23  conclusions about the adequacy of the evidence
  24  and the magnitude of the effect.  And these
  25  procedures are drawn from the collected
   1  experience of the members of the subcommittee in
   2  doing this sort of work in other venues.
   3            So the goal basically of our document
   4  is to make the evaluation process more
   5  predictable for the proponents of technology so
   6  they know what's going to happen and can prepare
   7  for it and therefore avoid unnecessary delays in
   8  getting an effective intervention through the
   9  coverage process, to make sure that our panels
  10  are consistent from one panel to the other and
  11  from one technology to the other, to make our
  12  decisions, or rather, our recommendations, more
  13  understandable to the proponents of the general
  14  public, and finally, to make sure that the panels
  15  are accountable both to each other and the
  16  Executive Committee for the quality of work that
  17  they do, but also more accountable to HCFA and to
  18  the public.  So the whole notion is to try to
  19  make this process more transparent so that both
  20  proponents and the public understand the basis
  21  for coverage decisions that HCFA would make based
  22  on our assessment of the evidence.
  23            So let's turn to the next transparency
  24  where we deal with what is probably the most
  25  difficult problem, which is deciding whether the
   1  evidence is adequate.  Our statement is that the
   2  panels must determine whether the scientific
   3  evidence is adequate to draw conclusions about
   4  the effectiveness of the intervention in routine
   5  clinical use in the population of Medicare
   6  beneficiaries.
   7            And that statement really can be broken
   8  down into two substatements.  The first is is the
   9  evidence valid?  Do the conclusions really
  10  represent what actually happened?  And secondly,
  11  is the evidence applicable to Medicare
  12  beneficiaries, the population of interest?  So
  13  let's spend some time talking about each one of
  14  those.
  15            Now, the first question you have to ask
  16  when you're comparing the effects of a new
  17  intervention to an old established intervention
  18  is are the two populations of patients that
  19  you're using to make that comparison truly
  20  comparable so that the only difference between
  21  them that might affect the outcomes that you're
  22  trying to measure is the intervention itself?  So
  23  when we ask about bias, we ask whether the study
  24  systematically overestimates or underestimates
  25  the effect of the intervention because of
   1  possible bias or other errors in assigning
   2  patients to either the intervention group or the
   3  controlled group.
   4            An example might help here.  Suppose
   5  there's a surgical procedure of unknown
   6  effectiveness, but pretty risky.  It's the sort
   7  of thing that you wouldn't do on somebody who was
   8  real sick for fear that they would die
   9  prematurely as a result of the intervention
  10  rather than of the disease for which the
  11  intervention is intended.
  12            In an observational study in which you
  13  try to compare the outcomes of using this
  14  intervention with the previous intervention,
  15  which is let's say less dangerous, but possibly
  16  less effective as well, the problem would ensue,
  17  when the surgeon looks at a patient and says this
  18  patient is simply too sick to go through this
  19  procedure, so I'm going to assign this patient to
  20  the controlled group, it's not going to get the
  21  procedure.  And through a series of such
  22  decisions, you end up with the study population
  23  that gets the intervention, who's basically
  24  pretty well because they're well enough to get
  25  through the procedure safely, and the controlled
   1  group, which are all the sick patients, who look
   2  like they wouldn't be able to get through the
   3  procedure.
   4            So a year later when you look at the
   5  outcomes, sure enough, the people who got the
   6  procedure, many more of them are still alive and
   7  functioning well as compared with the controlled
   8  group, but because the two groups are very
   9  different in their composition, you can't tell
  10  whether it was the intervention that led to them
  11  being more healthy after the intervention or
  12  whether it was the fact they were healthier
  13  before the intervention as a result of assignment
  14  on the basis of their ability to survive the
  15  procedure.  So that's an example of biased
  16  allocation of patients to intervention and
  17  controlled group that could lead to a very
  18  misleading interpretation of the outcomes at one
  19  year.
  20            So how do you avoid bias?  Well, the
  21  best way to avoid bias is simply to allocate
  22  patients randomly to the controlled group or to
  23  the intervention group.  Random allocation
  24  eliminates the type of systematic bias that I
  25  described in my example, although it's still
   1  possible that the two groups could be unbalanced
   2  because of just the random allocation process,
   3  which doesn't necessarily assign people to the
   4  two groups in equal numbers if the numbers in the
   5  two groups are relatively small.
   6            Now, in an observational nonrandomized
   7  study such as the one I described in my example,
   8  it's often very difficult to decide whether the
   9  results were due to bias or due to the
  10  intervention.  And so we're advising the panels
  11  to be very alert to the possibility of systematic
  12  allocation bias and observational studies by
  13  considering, first of all, the comprehensiveness
  14  of the available data, how the patients were
  15  selected to receive the intervention and the
  16  extent of disease in intervention and controlled
  17  groups.
  18            And it's possible, using statistical
  19  methods, to control for the variables that you
  20  know about if you've measured them carefully.
  21  The big problem is that you can't control for the
  22  variables you don't know about.  And that's the
  23  beauty of the randomized approach is that the
  24  intervention and the controlled group are
  25  equivalent, not just for the variables you know
   1  about, but also for the variables you don't know
   2  about.  It's a very powerful idea,
   3  randomization.
   4            In some cases the panel may decide that
   5  it can't draw firm conclusions about the
   6  effectiveness of an intervention without
   7  randomized trials.  And you can see how that
   8  might be the case from the example I described.
   9  But in some other cases, perhaps many cases, the
  10  panel will determine that observational evidence
  11  is sufficient to draw conclusions about
  12  effectiveness.
  13            When they do that, it's really the
  14  panel's obligation to describe potential sources
  15  of bias that they perceive and to explain why
  16  biased allocation as the result of those factors
  17  doesn't account for the results.  So in other
  18  words, there's a substantial burden of proof on
  19  the part of the panel to show that it was really
  20  the intervention that made the difference rather
  21  than some other difference in the two study
  22  populations.
  23            Finally, the subcommittee made, I
  24  think, a very strong statement saying that a body
  25  of evidence that consisted only of uncontrolled
   1  studies, whether based on anecdotal evidence,
   2  testimonials or case series or disease registries
   3  without adequate historical controls, is never
   4  adequate.  So we really feel strongly there needs
   5  to be some form of control even if it's only
   6  historical controls.
   7            So let's move on then to the question
   8  of external validity basically asking the very
   9  simple question, do the results apply to the
  10  Medicare population?  Do we expect that we will
  11  see these results in the Medicare population if
  12  they receive the intervention?
  13            For a long time randomized studies
  14  tended to deal with populations that did not
  15  include the elderly.  Part of the reason for that
  16  is that the older people have other diseases that
  17  may cause their death before the disease for
  18  which the intervention that you're testing is
  19  intended.  And so it's much better if you get a
  20  population of patients who have only the disease
  21  that you're trying to evaluate as the potential
  22  cause of death.  And so as a result, until
  23  relatively recently, elderly patients were not
  24  included in randomized trials.
  25            For example, there are no women over
   1  the age of 75 in randomized trials of screening
   2  for breast cancer despite the fact that the
   3  incidence of breast cancer continues to rise
   4  through the 70s.
   5            Now, increasingly, randomized trials
   6  are including elderly men and women.  However, if
   7  elderly men and women are included in those
   8  studies only in proportion to their numbers in
   9  the population as opposed to a study that's only
  10  including elderly people, there may be too few
  11  older people in the study to draw firm
  12  statistical conclusions about the effect of the
  13  intervention.
  14            There's also a concern if the study
  15  population is not the same as the general
  16  population, the Medicare beneficiaries, then you
  17  have to decide that results in a particular
  18  subsection of Medicare beneficiaries apply to all
  19  Medicare beneficiaries that might eventually
  20  receive the intervention.
  21            So we call upon the panel to explain
  22  its reasoning in deciding that the findings of a
  23  series of studies really apply to all Medicare
  24  populations.  And in fact, the panel might
  25  conclude that they don't, and it would be up to
   1  HCFA then to decide on coverage based on that
   2  conclusion.
   3            Finally, interventions vary from site
   4  to site.  What works at Johns Hopkins or at Mass
   5  General may not work in a community hospital.  So
   6  the panel has to explain whether the results that
   7  are published are going to apply to all
   8  healthcare settings and explain why they think
   9  that would be the case.
  10            So far we've talked about how you
  11  evaluate the adequacy of the body of evidence.
  12  And the issues, again just to repeat them, are,
  13  first of all, biased allocation of patients to
  14  the intervention group and the controlled group
  15  as something that interferes with the ability to
  16  draw a conclusion about whether it's the
  17  intervention that really made the difference,
  18  and secondly, the general applicability of the
  19  results to the Medicare population.
  20            So let's now turn to talk about the
  21  size of the health effect.  And our statement is
  22  that evidence from well-designed studies that
  23  meet the first criterion -- that is to say
  24  adequate evidence -- must establish how the
  25  effectiveness of the new intervention compares
   1  with the effect of established services and
   2  medical items.
   3            And we think that we've helped HCFA
   4  with its assignment to make coverage decisions by
   5  placing both the size of the effect and the
   6  direction of the effect as compared with
   7  established services or medical items into one of
   8  these seven categories.  And by the direction of
   9  the effect, I mean is it better or is it the same
  10  or is it worse?
  11            So one category would be a breakthrough
  12  technology.  This is something that we all want
  13  to see a lot more of, something that causes such
  14  a large improvement in healthcare outcomes that
  15  it becomes overnight standard of care.
  16            The second category would be more
  17  effective.  The new intervention improves
  18  healthcare outcomes by a definite significant,
  19  albeit small, margin as compared with established
  20  services or medical items.
  21            The third category would be as
  22  effective, but with advantages.  So the
  23  intervention has the same effect on healthcare
  24  outcomes as established medical services or
  25  items, but it has some advantages that would be
   1  important to some if not all patients, such as
   2  convenience, rapiditive effect, fewer side
   3  effects and so forth.  So some people might
   4  prefer it over existing interventions.
   5            Then there's a category called as
   6  effective, but with no advantages, an
   7  intervention that basically has the same effects
   8  on healthcare outcomes as existing services and
   9  doesn't have any substantial advantages.
  10            A fifth category is less effective, but
  11  with advantages.  So it's certainly possible that
  12  an intervention could be somewhat less effective
  13  than existing alternatives, but it would have
  14  some advantages that would be so important to
  15  some patients that they might choose it even
  16  though it might not have the same effect on their
  17  health status as existing interventions.
  18            The sixth category is less effective
  19  with no advantages.  The intervention is less
  20  effective than established alternatives, but more
  21  effective than doing nothing, and doesn't have
  22  any significant advantages.
  23            The last category is not effective.
  24  The intervention has no effect or has deleterious
  25  effects on healthcare outcomes when compared with
   1  doing nothing, such as treatment with placebo or
   2  patient management without the use of a
   3  diagnostic test in the case of a diagnostic test.
   4            So let's then move on from two
   5  principles by which the panels can hopefully
   6  provide consistent, understandable advice to HCFA
   7  about the quality of the evidence and the
   8  magnitude of the effect on healthcare outcomes.
   9            Now we're going to get into operational
  10  procedures, how the subcommittee feels the panel
  11  should operate in order to provide consistent
  12  results from panel to panel and from intervention
  13  to intervention.
  14            And the first basic principle is that
  15  the panel must explain its conclusions in
  16  writing.  And this requirement is clearly aimed
  17  at trying to improve the transparency of the
  18  process and the accountability to the public as
  19  well as to the proponents of the technology.
  20            We've also put it in the hands of the
  21  panel chair to be responsible for writing the
  22  explanation of the panel's conclusions.
  23            The next procedural recommendation has
  24  to do with structuring the evidence so that the
  25  panels can function effectively.  So we recommend
   1  that the panels should receive well-organized,
   2  high-quality background information before they
   3  begin their deliberations about the adequacy of
   4  the evidence and the size of the effect.  And we
   5  recommend that the evidence should be summarized
   6  in a report, which we call an evidence report,
   7  not simply presented as a collection of data or
   8  primary studies.  And there's ample precedent for
   9  this in the technology evaluation efforts of many
  10  other organizations.
  11            So our basic principle is the integrity
  12  of the coverage decision process begins with
  13  complete critical evaluation of the literature.
  14  And we feel that the standard for HCFA should be
  15  the best that's out there in other settings, such
  16  as the private sector where Blue Cross Blue
  17  Shield has a long track record of doing
  18  evaluations of the evidence and making coverage
  19  decisions in what is a process that's both
  20  efficient and I think highly regarded by
  21  professional organizations such as the ACP-ASIM
  22  and by other federally sponsored panels.  The
  23  Agency for Health Research and Quality has a
  24  series of evidence-based practice centers in
  25  various universities, and I think there are a
   1  couple of private settings around the country,
   2  and they provide technical support for the U.S.
   3  Preventive Services Task Force on which I serve.
   4            Now, evaluating the evidence carefully
   5  and providing a balanced, well-organized report
   6  of it to the panels is a task that inevitably is
   7  going to take some time.  It's the opinion of the
   8  subcommittee that it should be possible to do
   9  these reports in six months or less.  Those of
  10  you who are experienced in doing this work know
  11  that that's fast for doing an adequate evidence
  12  report, but we think that HCFA should meet that
  13  standard.
  14            The next procedural recommendation is
  15  basically that members of the panel should be
  16  actively involved in the process of reviewing the
  17  evidence, and that's based on quite a lot of
  18  experience with other health technology
  19  programs.
  20            So for example, we think that the chair
  21  of the panel and perhaps others -- but certainly
  22  the chair -- should work with HCFA to establish
  23  which are the most important questions that the
  24  evidence report should address, and then
  25  ultimately the panel must answer as part of its
   1  deliberations.
   2            Secondly, we feel that several members
   3  of the panel should be active participants in
   4  designing the evidence review and preparing the
   5  evidence report that the panel will consider.
   6  And that's based in part on what we feel is the
   7  need to have real expertise on the panel on the
   8  topic in question.  And the best way to get that
   9  expertise is to participate in the design of the
  10  evidence review and the writing of the report.
  11            Finally, we feel that it's very
  12  important that each evidence report be given an
  13  extremely careful review.  We expect that all
  14  members of the panel will read the report very
  15  carefully, but we also recommend that one or two
  16  members of the group be assigned to be what are
  17  called primary reviewers, and we expect those
  18  people to really dig into that report, do their
  19  best to find any potential problems with the
  20  report so that the panel will know that the
  21  report has been given sort of the ultimate in
  22  very close scrutiny.
  23            Finally, we recommend that there be
  24  expert review of the evidence report.  To ensure
  25  that the evidence report is complete and free
   1  from bias, the Executive Committee recommends
   2  expert review of the evidence reports.  This is
   3  going to mean in general subjecting the reports
   4  to external review.  And the purpose of that is
   5  to assure everybody, the public, the proponents
   6  and the panel, that the evidence report is
   7  complete and that it's fair.
   8            That external review should take place
   9  before the panels meet, and the evidence report
  10  as well as the comments of expert reviewers will
  11  be part of the public record of the panel's
  12  deliberations.  We envision a relatively small
  13  number of expert reviewers, perhaps a half dozen,
  14  and we will require them to complete their review
  15  in a timely fashion, within a month.
  16            Now, the last transparency is not part
  17  of our report, but it's based on what you could
  18  read in the report as a possible time line for a
  19  typical MCAC evaluation.  So times zero is the
  20  time that HCFA decides to go to MCAC for an
  21  opinion about the adequacy of the evidence.  Then
  22  in the first month HCFA and the panel chair would
  23  decide on what are the key questions that the
  24  panel needs to address and what are the key
  25  requirements of the evidence report.  In
   1  addition, HCFA would decide who would do the
   2  evidence report.
   3            Month two to seven would represent the
   4  time during which the evidence report would be
   5  prepared.  And again, it might not be month two
   6  to seven.  It might be month two to five if the
   7  topic was one that led itself to a more speedy
   8  conclusion of the review of the evidence.
   9            In month eight the report is out for
  10  external review.  It's out to members of the
  11  panel for review.  And at the end of that month
  12  there's a meeting of the panel that leads to a
  13  report to the Executive Committee.  And certainly
  14  in the ideal world, the timing of the Executive
  15  Committee meetings would be closely tied to panel
  16  meetings, so the Executive Committee could sign
  17  off on the recommendations of the panel within a
  18  month after the completion of the panel meeting.
  19  And then it will be up to HCFA to decide on its
  20  own time schedule about coverage policy.
  21            So that concludes the report of the
  22  subcommittee.  And I think it would be good now
  23  for members of the subcommittee to say anything
  24  that they wish about my report to be sure that it
  25  reflects the views of the members of the
   1  subcommittee.
   2            So would anybody on the subcommittee
   3  like to comment at this point on my review?
   4            MS. RICHNER:  I have something.
   5            DR. SOX:  Randel, please.
   6            MS. RICHNER:  I actually wrote
   7  something last night.  I wanted to write them all
   8  down so that I didn't forget anything.  So excuse
   9  me while I load up here to get something.  If
  10  anybody else has anything to say -- I didn't know
  11  that this was my time to talk.
  12            DR. SOX:  Randel, is it okay if John
  13  makes a few remarks?
  14            MS. RICHNER:  Sure.
  15            DR. FERGUSON:  Just a few.  First of
  16  all, I think that this is a very nice road map.
  17  It's an idealistic road map in my view.  And I
  18  guess my overall view is although I think that
  19  this is something that we all might like to shoot
  20  for, that the end result following this totally
  21  might tie the process so that it wouldn't work,
  22  and I would not like to see that happen.
  23            A couple of specifics.  Point one on
  24  the adequacy of the evidence.
  25            DR. SOX:  John, actually, if you don't
   1  mind, I think I'm going to interrupt you.  We're
   2  going to have an opportunity later on in the
   3  morning to present our concerns about the
   4  report.  I think maybe it would be better to do
   5  that later and just have the members of the
   6  subcommittee comment on whether I have given the
   7  report as they think it is.  Is that okay?
   8            DR. FERGUSON:  Sure.  You meant from
   9  the members of the subcommittee?
  10            DR. SOX:  Yes.
  11            DR. FERGUSON:  Excuse me.
  12            DR. SOX:  If you wouldn't mind holding
  13  it.
  14            DR. FERGUSON:  That's fine.
  15            DR. SOX:  Has that given you enough
  16  time to get your thing up on the computer?
  17            MS. RICHNER:  Once again, I'm sorry to
  18  have to do it this way, but I decided to write
  19  this on the computer last night, so I didn't have
  20  any way to print it.
  21            DR. KANG:  We can print it for you.
  22            MS. RICHNER:  That's okay.  I'll just
  23  read it.
  24            In my work to date with MCAC, I have
  25  attempted to bring views on the impact of our
   1  coverage and process recommendations on the
   2  industry, on technology development and
   3  innovation, and first and most importantly, of
   4  the impact of these recommendations on patient
   5  access to new technology.
   6            My views are derived from years of
   7  practical experience and applied research from
   8  being a nephrology transplant nurse, public
   9  health research background, including health
  10  economics -- now comes research for the
  11  pharmaceutical industry -- and most recently, as
  12  the vice president of a large manufacturer of
  13  minimally invasive technology.
  14            I've always considered myself one who
  15  comes from a scientific and clinical perspective
  16  and passionate about what is important for the
  17  patient.  Having said this, I am certain that no
  18  matter what I say, it will not be to the liking
  19  of at least one if not several of the
  20  constituencies represented here today.
  21            While I was invited to participate in
  22  the subcommittee who has drafted this document, I
  23  can say that I am not completely satisfied with
  24  the final output of this draft.  First, I was
  25  particularly concerned with the tone, which
   1  implied a lack of flexibility in reviewing and
   2  assessing the information that is available for
   3  technology assessments.  I feel that overall the
   4  document assumes that new technology information
   5  is innately flawed, or another way of saying it,
   6  that all technology is guilty until proven
   7  innocent and that it is HCFA's responsibility to
   8  protect the public.
   9            Second, we do not take into account the
  10  availability and rigor of evidence that is
  11  available over time for a technology.  Depending
  12  upon when the technology is referred to MCAC, the
  13  life cycle of the technology can have a profound
  14  impact on the level and the types of evidence to
  15  be reviewed.
  16            Third, our primary task was to describe
  17  a process for which the panels could make
  18  efficient decisions.  I felt the draft was never
  19  clear on the who, what and when directions for
  20  the panels.  I also was concerned that we have
  21  added on time and many additional reviewers that
  22  would make the overall process arduous for any
  23  technology to overcome.
  24            However, I must strongly support that
  25  we, the industry -- and I assume that we're all
   1  the industry in some ways -- have a
   2  responsibility to the patient to ensure that the
   3  technologies we develop and expect to be covered
   4  and paid for will ultimately produce some
   5  additional benefit to the Medicare patient.  This
   6  should be expected and demanded by consumers of
   7  healthcare services and products.
   8            Finally, I feel that HCFA should have
   9  provided MCAC more guidance for the Executive
  10  Committee on content and process.  I feel that
  11  the lack of published guidelines could have
  12  provided clearer guidance on criteria for which
  13  the technology should be assessed.  They've
  14  essentially left it de facto to the committee.
  15            I'm very committed to the MCAC
  16  process.  We have an incredible resource of
  17  dedicated, highly talented individuals from which
  18  we can freely draw and use their expertise for a
  19  technology assessment process that is workable,
  20  doable, predictable and fair.
  21            The committee should have had
  22  instruction on the goal of coverage evaluations
  23  in a divided, fragmented coverage and payment
  24  system that no one can possibly understand who is
  25  not intimately involved with the inner workings
   1  of HCFA.  I even wonder if those inside HCFA
   2  really understand how one system affects
   3  another.  It's very important.
   4            As a quick example, how many times have
   5  I heard recently from very educated individuals,
   6  why can't we simply get them, HCFA, to increase
   7  the DRG payment to cover the new technology?
   8  J&J did it with stents.  I hear that one all the
   9  time.
  10            In conclusion, all the dialogue has
  11  been particularly useful to move this to the
  12  point where I believe we can now successfully
  13  design a process and criteria that will work for
  14  fair technology assessments.  With some open and
  15  frank discussions I expect we'll have today, I
  16  hope that we can enable a definitive coverage
  17  process for promising therapies and
  18  technologies.  Thank you.
  19            DR. SOX:  Thank you very much.
  20            Would any other member of the
  21  subcommittee wish to make any remarks?
  22            Well, since there are no further
  23  remarks from the subcommittee, it's now time for
  24  us to go into open public session.  And let me
  25  just briefly lay out the ground rules.  We have
   1  nine people.
   2            DR. BERGTHOLD:  I'd just like to say
   3  one thing for the record.
   4            DR. SOX:  Thank you very much.
   5            DR. BERGTHOLD:  I just wanted to
   6  comment on the process of the subcommittee for
   7  those of you who didn't have the opportunity to
   8  be involved, including people here around the
   9  table, and that is that Hal as chair was very
  10  open to all kinds of our concerns about nuance,
  11  word and tone, and I believe this went through at
  12  least a dozen drafts and iterate of drafts trying
  13  to be sure that the tone was clear.
  14            And so while some may think that this
  15  looks negative, I think it is incumbent upon
  16  everyone, not only here, but in the audience, to
  17  really carefully read this document.  Almost
  18  every word was discussed and talked about at
  19  great length so that the tone would be clearly
  20  that while there's a gold standard for evidence,
  21  we understood, all of us, that not every new
  22  technology will meet that standard.
  23            So I just wanted to make that clear,
  24  that we had this level of discussion at the
  25  subcommittee level, and I wanted to thank Hal for
   1  being very receptive and open to everybody's
   2  comments.  Thank you.
   3            DR. SOX:  Thank you very much.
   4            Any other comments before we move on?
   5            In that case we'll go into open public
   6  session.  The plan is to have five speakers in
   7  the next hour, then take a 20-minute break, and
   8  then come back for the last four speakers, then
   9  move on to the HCFA presentation at approximately
  10  a quarter to 11:00.
  11            So five divided into 60 goes 12 minutes
  12  per speaker.  Excuse me.
  13            Could you approach the mic if you have
  14  to make a comment.
  15            DR. WEISENTHAL:  My name is Larry
  16  Weisenthal, and I just have a protest concerning
  17  the allocation of time to the speakers.  I
  18  noticed that your five speakers for the first 60
  19  minutes have 12 minutes a piece, and that leaves
  20  four speakers in 20 minutes for five minutes a
  21  piece.  So the first speakers get 12 minutes.
  22  The second speakers get five minutes.
  23            I paid $900 of my own money to fly from
  24  California and miss two days of work, and I was
  25  told in advance I'd have ten minutes.  I can say
   1  it in ten minutes, but I'd really like to have
   2  12.
   3            DR. SOX:  Thank you very much.
   4  Everybody's going to have the same amount of
   5  time.  Let's see.  We've got basically an hour
   6  and -- I think what we'll basically say is ten
   7  minutes per speaker, which I guess is what you
   8  were led to expect, and we'll just let the time
   9  fall where it may.
  10            So I'm going to ask you to stop at ten
  11  minutes, and I will be impolite and tell you to
  12  sit down if you try to go over, just so you
  13  understand that's the way I am.  And I'll raise
  14  my hand with about a minute to go to give you a
  15  chance to wrap up.
  16            So let's start with Guido Tricot, who
  17  is Director of the Myeloma Transplant Center at
  18  the University of Arkansas.  Welcome.
  19            DR. TRICOT:  Thank you very much for
  20  giving me the time to bring up a few issues.  My
  21  name is Guido Tricot.  I'm the director of the
  22  myeloma program at the University of Arkansas.
  23            The first issue I would like to bring
  24  up is the age issue.  Although we assume that
  25  Medicare is mainly for patients over the age of
   1  65, when we reviewed the records of patients who
   2  had transplants for myeloma, approximately
   3  one-third of the patients were under the age of
   4  65.  That's one issue.
   5            The second issue about age is that most
   6  of the reasons why age has become a problem --
   7            MS. LAPPALAINEN:  Could you bring the
   8  mic closer to you?  It's wireless, so you can
   9  pick it up, if you'd like.
  10            DR. TRICOT:  -- why age has become a
  11  problem is because of the comorbid conditions
  12  that the patients may have.  And in most studies
  13  there are sufficient exclusion criteria to deal
  14  with the comorbid conditions.  And rather than
  15  making age an issue, because we all know that
  16  there is basically no difference between a
  17  patient who is 64 years and 11 months and
  18  somebody who is 65 years, and that there's a
  19  difference between calendar age and biologic age,
  20  I think exclusion criteria rather than age itself
  21  should be the main thing to exclude comorbid
  22  conditions.
  23            A second point that I would like to
  24  bring up is that in the explanation of panel's
  25  conclusion, the panel chair is responsible for
   1  writing the explanation of the panel's
   2  conclusion.  We need to make sure that there are
   3  mechanisms in place that the report is a
   4  reflection of the whole group of the panel and
   5  not necessarily mainly a reflection of what the
   6  chair's vision is.
   7            A third point is the external review by
   8  experts.  Although it states that this will
   9  become part of the public record, we need to make
  10  sure that this becomes part of the public record
  11  prior to the panel meeting and that there's
  12  adequate time to review and comment at the time
  13  that the proponents will make the report.
  14            A smaller comment is on the randomized
  15  studies.  Although we all would like to have many
  16  randomized studies all showing the same results
  17  and going in the same directions, we also need to
  18  be aware of the fact that once there is one
  19  randomized study that shows that one treatment is
  20  better than the other, it becomes difficult to do
  21  further randomized studies.  In principle you're
  22  only supposed to do randomized studies if as a
  23  physician you're not convinced that one treatment
  24  is better than the other and that you have no
  25  bias toward any of the treatment modalities.
   1            There's also a problem with referral
   2  patterns.  We at the University of Arkansas have
   3  tried to do randomized studies, but the patients
   4  that are coming to our institution come from
   5  everywhere, and they come because they want a
   6  certain procedure done, and we have never been
   7  able to do randomized studies because of that.
   8            And the last point I would like to
   9  bring up is that there is a tremendous time lapse
  10  between initiation of the process and the point
  11  in time the proponents are convinced that what is
  12  proposed is better than what has been available
  13  before and the ultimate approval.  And it's going
  14  to be at least nine months, and probably more
  15  likely, 12 months or more.  And I think there
  16  should be a mechanism in place that provides
  17  temporary approvals in between this 12-month
  18  lapse and that a committee of experts can be
  19  gathered to give temporary approvals until the
  20  final decision by HCFA is made.
  21            I think those are my main concerns.
  22  Thank you very much for giving me this time.
  23            DR. SOX:  I should remind the members
  24  of the Executive Committee that we're going to
  25  have about an hour to ask questions of the people
   1  who are going to speak.  So take notes and be
   2  ready to ask some questions during the hour that
   3  will be reserved for discussion with them.
   4            With that, we'll move on to Richard
   5  Justman, who is medical director of United
   6  Healthcare and the American Association of Health
   7  Plans.
   8            DR. JUSTMAN:  Thank you.  Good
   9  morning.  My name is Dick Justman, and I do not
  10  have any financial connection to technology or
  11  device manufacturers.  In my current position
  12  that would be very difficult.
  13            My name is Dick Justman, and I'm the
  14  national medical director of United Health Group.
  15            DR. HILL:  Excuse me, Dr. Justman.
  16  Would you do the same thing with your
  17  microphone?  Folks in the back are indicating
  18  they can't hear.
  19            DR. JUSTMAN:  Is that better?
  20            DR. HILL:  Thank you.
  21            DR. JUSTMAN:  I'm the national medical
  22  director of United Health Group, and I'm here
  23  today speaking on behalf of the American
  24  Association of Health Plans.  AAHP represents
  25  more than a thousand health maintenance
   1  organizations, preferred provider organizations
   2  and other similar network-based health delivery
   3  systems that provide healthcare to more than 150
   4  million Americans.  AAHP member health plans are
   5  dedicated to the philosophy that we put patients
   6  first by offering them benefit packages offering
   7  coordinated comprehensive healthcare.
   8            United Health Group, the company for
   9  which I work, has 40 health plans around the
  10  United States serving approximately 14 million
  11  commercial enrollees in HMO, PPO point of service
  12  and exclusive provider organization products.  We
  13  also have approximately 400,000 Medicare
  14  enrollees.
  15            As you may have read recently in the
  16  newspapers, United Health Group has recently
  17  embarked upon a program which we call care
  18  coordination, and this is a model of healthcare
  19  coverage which essentially allows physicians and
  20  patients to make healthcare decisions with
  21  minimal intrusion by the health plan subject only
  22  to the limitations of benefit design.  However,
  23  we feel very strongly that for this endeavor to
  24  work, we need to be covering procedures to
  25  biases, treatments and drugs that we know
   1  actually do work.
   2            We strongly endorse a rigorous,
   3  evidence-based approach to coverage
   4  determinations.  We applaud the establishment of
   5  the Medicare Coverage Advisory Committee to
   6  assist HCFA to evaluate the clinical evidence
   7  about the relative effectiveness of new medical
   8  devices, services and other technologies.
   9            The report of the Executive Committee
  10  working group to be discussed today will promote
  11  systematic and consistent evaluation of the
  12  clinical evidence by the panels that we believe
  13  should meet the needs of all the stakeholders.
  14            There is compelling evidence, including
  15  evidence cited by President Clinton's own
  16  advisory commission on consumer protection of
  17  quality in the healthcare industry, that
  18  Americans do not always receive the best possible
  19  healthcare.  In many instances they do not
  20  receive important healthcare services that they
  21  should, and yet in other instances they receive
  22  services of uncertain value, and unfortunately in
  23  yet other instances they receive services of
  24  questionable quality.
  25            Also, too often medical treatments are
   1  widely disseminated before they have been proven
   2  to be effective putting patients potentially at
   3  risk of harm, and this also discourages for
   4  further research.
   5            Both of these problems, the variation
   6  and the use and quality of healthcare services
   7  and the proliferation of unproven treatments,
   8  illuminate the importance of promoting a delivery
   9  care that is based upon robust, scientific
  10  evidence.
  11            To give you an example, a recent study
  12  showed that between 1987 and 1991, only 21
  13  percent of eligible elderly patients were treated
  14  with beta blockers for ischemic heart disease,
  15  myocardial infarction and related disorders and
  16  that the subsequent mortality rate for those who
  17  did receive the treatment was 43 percent lower
  18  than for those who did not receive the
  19  treatment.  This translates into, in that study
  20  group, 18,000 potentially avoidable deaths that
  21  would not happen because the appropriate
  22  treatment was not given.
  23            What is really stunning in this case is
  24  that in the words of the American Medical
  25  Association, beta blockers are one of the most
   1  scientifically studied and substantiated medical
   2  therapies.  There is a plethora of published
   3  evidence about them.  The American College of
   4  Cardiology and the American Heart Association
   5  have brought guidelines and physician statements
   6  promoting their use.  And despite this and
   7  despite voluminous evidence, there are many
   8  eligible people who potentially would have
   9  benefited from beta blockers who have not
  10  received them.
  11            A second problem undermining the
  12  quality of care is the proliferation of
  13  treatments that have been widely disseminated in
  14  the absence of proof that they are effective.  In
  15  such cases patients may be harmed because they
  16  forego a standard proven therapy in favor of a
  17  treatment that may be less effective than the
  18  standard one.
  19            A most recent example is that of high-
  20  dose chemotherapy and bone marrow transplantation
  21  for women with breast cancer.  An assumption was
  22  made many years ago that if women are partially
  23  responsive to standard dose chemotherapy, that
  24  high-dose chemotherapy coupled with bone marrow
  25  or peripheral stem cell rescue would be even more
   1  effective.  Unfortunately at the time this
   2  assumption was made, there was little evidence to
   3  support this, little robust scientific evidence.
   4  And in fact, this became widely disseminated as a
   5  treatment that women must have.  Well-intentioned
   6  advocacy groups promoted its use.  Many states
   7  actually passed laws mandating coverage for
   8  this.  And this essentially became a
   9  self-fulfilling prophecy.
  10            Women assumed that if states were
  11  mandating coverage for this, this must be a
  12  preferred and effective treatment.  This
  13  essentially made it very difficult for women to
  14  randomize themselves into controlled trials
  15  because women were afraid that if they were
  16  randomized into the standard treatment group,
  17  they would miss out on treatment that might be
  18  effective.  So in fact, there was circular
  19  reasoning here.
  20            And as you know, there has been recent
  21  published evidence that says that if anything,
  22  high-dose chemotherapy bone marrow
  23  transplantation is no more effective than
  24  standard chemotherapy for women with breast
  25  cancer although the morbidity of high-dose
   1  chemotherapy is substantially greater.  So this
   2  is a very stunning example of a situation in
   3  which a therapy is rapidly proliferated in the
   4  absence of scientific evidence, and it is very
   5  difficult now to reverse that trend.
   6            Another example of a less life-
   7  threatening but equally pervasive disorder has to
   8  do with low-back pain.  Approximately a year ago
   9  in a national news weekly, a device was
  10  discussed, which presumably through a heat
  11  treatment, reduces significantly diskogenic
  12  low-back pain.  This was widely reported, and
  13  many providers in many regions of the country
  14  began to promote this treatment.
  15            At the time that this was done, there
  16  was almost no scientific evidence published at
  17  all.  All the scientific evidence that was
  18  available was available on a website.
  19            To make matters worse, there were yet
  20  other providers who began to use this device to
  21  treat neuropathic pain, for which the FDA
  22  indications never existed in the first place.  So
  23  this is yet another example where in the absence
  24  of scientific evidence, there can be rapid
  25  proliferation of technology that desperate people
   1  will try to use.
   2            Health plans have taken a prominent
   3  role in promoting evidence-based care.
   4  Increasingly, health plans are working with
   5  physicians to reduce the variation in practice
   6  patterns through the dissemination of chemical
   7  profiling tools and processes of care that guide
   8  physicians to provide their patients the right
   9  care at the right time and in the right setting.
  10            Health plans distribute and encourage
  11  the use of evidence-based processes of care by
  12  physicians and other healthcare providers.
  13  Health plans also provide feedback to physicians
  14  about how their treatment practice patterns,
  15  including underutilization and overutilization,
  16  compared to scientific evidence and also to the
  17  practice patterns of their peers.  Health plans
  18  make scientific coverage determinations based
  19  upon the best available evidence.  Through these
  20  and other activities, health plans actively
  21  promote the use of evidence-based care.
  22            Through technology assessment, health
  23  plans are working to approve coverage of new
  24  treatments supported by medical evidence and to
  25  avoid the coverage of treatments for which there
   1  is no scientific evidence and for which these
   2  treatments may actually harm patients.  In
   3  technology assessment organizations gather and
   4  evaluate the scientifically valid evidence
   5  available, including, but not limited to,
   6  surgical procedures, devices and drugs.
   7            First, they determine whether the
   8  evidence demonstrates that the treatment is
   9  safe.  Second, they evaluate whether or not the
  10  evidence demonstrates that the treatment is as
  11  effective or more effective than an existing
  12  treatment if an existing treatment does exist.
  13            Health plans use this information in
  14  determining whether or not the treatment should
  15  be a covered service.  By implementing a
  16  structured method for evaluating new or existing
  17  treatments and not covering treatments not proven
  18  to be effective, health plans are working to
  19  reduce the proliferation of unproven and
  20  potentially unsafe treatments.
  21            However, health plans cannot solve this
  22  problem alone.  We need the help of others within
  23  the system, including Medicare, Medicaid
  24  providers, researchers and manufacturers.
  25  Increasingly, the healthcare community and policy
   1  makers recognize the importance of promoting
   2  evidence-based care and are working to change the
   3  current environment.
   4            In addition to health plans, others in
   5  the healthcare community understand the
   6  importance of promoting and providing evidence-
   7  based care, and in order to be valid, the
   8  evidence itself must meet certain criteria.
   9            We support very definitely the use of
  10  the best possible scientific evidence, and we are
  11  aware that randomized controlled trials ideally
  12  are the best evidence.  We recognize also,
  13  however, that those are not always possible,
  14  either due to the lack of availability of a
  15  control arm, the size of the cohort or other
  16  factors.  However, we believe very strongly that
  17  we must always seek the best scientific evidence
  18  that is available and the best methodology
  19  available in order to make coverage decisions.
  20            In conclusion, I would like to stress
  21  that the first goal of the healthcare system
  22  should be to provide quality healthcare
  23  services.  In our current system too often
  24  quality is compromised because the care delivered
  25  is not consistent with the best available medical
   1  evidence.
   2            Health plans are committed to improving
   3  quality care through reliance on medical evidence
   4  when making coverage determinations, when
   5  evaluating new therapies and in communicating
   6  with providers.  In order to improve the quality
   7  for all patients, however, all stakeholders in
   8  the healthcare system, not just the health plans,
   9  must be actively committed to the process of
  10  using evidence-based medicine.  Thank you.
  11            DR. SOX:  Thank you very much.  Just so
  12  that the speaker knows when there's one minute to
  13  go, I'm going to stand up, which hopefully will
  14  catch your eye.  Putting up my hand didn't seem
  15  to work very well.
  16            Our next speaker is Morgan Downey,
  17  Executive Director of the American Obesity
  18  Association.
  19            MR. DOWNEY:  Thank you, Mr. Chairman
  20  and members.  It's a pleasure to be here with you
  21  this morning.
  22            My name is Morgan Downey, and I am the
  23  Executive Director of the American Obesity
  24  Association.  This association is about four
  25  years old, and it was founded as an adequacy
   1  organization to promote research, treatment,
   2  prevention and intervention in the epidemic the
   3  country is going through, obesity.
   4            I'm very pleased to be able to address
   5  the complex issues of obesity in the Medicare
   6  program with you this morning.  For the record,
   7  the American Obesity Association is supported by
   8  several major companies, including Amgen Hoffman-
   9  LaRoche and all pharmaceuticals, Weight Watchers
  10  International, in dues from professional and lay
  11  members.  To the best of my knowledge, no
  12  supporter has a specific coverage issue before
  13  the Medicare Coverage Advisory Committee at this
  14  time.
  15            At the outset I'd like to put our
  16  current and immediately foreseeable situation on
  17  the record.  Over half of the United States
  18  population is overweight, and about a quarter is
  19  obese measured as their body mass index of over
  20  25 and over 30 respectively.  According to 1991
  21  data, the percentages of the Medicare population,
  22  with the BMI of over 27.8 percent for males and
  23  27.3 for females, ranged from 23.8 percent for
  24  white males to 48.7 percent for black females.
  25            As you well know, obesity is a major
   1  independent risk factor for conditions such as
   2  Type II diabetes, hypertension, heart disease,
   3  stroke, several cancers, arthritis, end stage
   4  renal disease, gallbladder disease and sleep
   5  apnea, to name a few of the 30 or so conditions
   6  where associations have been found.
   7            We know that obesity is increasing
   8  rapidly in the population.  Jeffrey Copeland,
   9  Director of the Centers for Disease Control and
  10  Prevention, has likened its spread to that same
  11  in infectious diseases.  According to a recent
  12  article in JAMA in October, between 1991 and
  13  1998, the prevalence of obesity measured as a BMI
  14  over 30 among persons age 60 to 69 increased 44.9
  15  percent.  The prevalence among persons over 70
  16  increased 28.6 percent.  That is a rate of 6.4
  17  percent per year at a BMI level of 30 and four
  18  percent a year increase for a person over 70.
  19            We also know that obesity is a major
  20  generator of healthcare costs.  According to a
  21  study of the American Obesity Association
  22  commission from the Lewin group last year,  the
  23  direct healthcare cost of obesity exceeded a
  24  hundred billion dollars in 1999.  This figure
  25  does not include indirect costs or costs spent on
   1  treating obesity itself.  We did not ask for a
   2  breakdown by payers, but I think it's fair to
   3  assume that the Medicare program plays a
   4  significant if not majority component of those
   5  costs.
   6            So it's not without substantial
   7  justification that obesity is now listed as one
   8  of the nation's ten leading health indicators, as
   9  announced a few weeks ago by the surgeon
  10  general.
  11            We concede, therefore, that more and
  12  more Americans are becoming obese, which will
  13  dramatically increase their risk for diseases,
  14  which Medicare will pay for.  These people will
  15  come into the Medicare program, both as they age,
  16  and also as they become eligible for disability
  17  under Social Security disability procedures.
  18            The standards for the evaluation of
  19  obesity under Social Security is currently
  20  undergoing some changes, but we expect that the
  21  current number of 137,000 persons who receive
  22  Social Security disability under their obesity
  23  listing will continue to increase.  And as you
  24  know, after two years on disability, these
  25  individuals start receiving healthcare coverage
   1  under the Medicare program.
   2            Our interests today are twofold.
   3  First, we propose that the committee consider
   4  when evaluating new medical profits, be they
   5  laboratory tests, diagnostic procedures,
   6  preventative intervention or treatment, that a
   7  large portion, a quarter to a half of the
   8  Medicare population, is overweight or obese.
   9            Questions might be asked were the
  10  studies in support of the procedures conducted in
  11  a representative sample of the current population
  12  by weight?  Can Medicare beneficiaries who are
  13  obese access the new technologies?
  14            As an example, there are recent studies
  15  showing, for example, that obese women receive
  16  pap smears and mammograms with less frequency
  17  than do nonobese women.
  18            Last fall the representative of HCFA,
  19  speaking at a conference we had on public policy
  20  implications of obesity, indicated that the bone
  21  marrow transplantation protocols in this country
  22  exclude persons with obesity without medical
  23  justification.
  24            Second, we propose that the committee
  25  begin the process of clarifying Medicare coverage
   1  of obesity.  Paragraph 3526 of the coverage
   2  manual states, quote, obesity itself cannot be
   3  considered an illness.  The immediate cause is a
   4  caloric intake, which is consistent with a higher
   5  than caloric output.  Program commitment may not
   6  be made for the treatment of obesity alone since
   7  this treatment is not reasonable and necessary
   8  for the diagnosis and treatment of an illness or
   9  injury.  Yet under paragraph 3540, obesity
  10  surgery, bariatric surgery is covered if
  11  medically appropriate and necessary to correct an
  12  illness caused or aggravated by obesity.
  13            Clearly these two paragraphs are
  14  inconsistent.  If obesity cannot be considered an
  15  illness, the surgery to correct it can't be
  16  covered.  On the other hand, as a reduction of
  17  weight can correct an illness or injury
  18  aggravated by obesity, what possible
  19  justification is there for covering exclusively
  20  the most drastic and life-threatening
  21  intervention when other equally effective and
  22  less risky treatments are available?  Clearly
  23  3526 of the coverage manual is wrong and should
  24  be considered an embarrassment to the Health Care
  25  Financing Administration.
   1            Illness is synonymous with disease.
   2  Virtually every medical and scientific definition
   3  define diseases as, for example, does Stedman's
   4  medical dictionary, which is, one, an
   5  interruption, cessation or disorder of body
   6  functions, systems or organs, or two, a disease
   7  entity characterized by at least two of these
   8  criteria; one, recognized etiologic agent or
   9  agents, two, an identifiable group of signs and
  10  symptoms, three, consistent anatomical
  11  alterations.  Clearly obesity means all three of
  12  these criteria.
  13            Any analysis of the definitions of
  14  illness and injury disorder will demonstrate that
  15  obesity is considered an illness by the vast
  16  weight of modern, scientific and medical
  17  understanding.  Therefore, we'd like to suggest
  18  two issues for your consideration.
  19            First, given the increase in the
  20  overall Medicare population which is obese and
  21  the increases in medical technology, we want to
  22  be sure that all such advances are available to
  23  the obese Medicare population.  Therefore, AOA
  24  suggests that all future subjects for Medicare
  25  coverage determinations be evaluated with this
   1  population in mind.
   2            Second, we suggest the committee
   3  establish a subcommittee or working group to
   4  revise the current and incorrect coverage manual
   5  paragraph 3526.  There are many professional
   6  guidelines for the treatment of obesity in adults
   7  including that developed two years ago by the
   8  National Institutes of Health, which relies on
   9  literally hundreds of randomized controlled
  10  clinical trials and other studies which would
  11  meet the criteria earlier elucidated by the
  12  chairman regarding the considerations of this
  13  committee.
  14            The American Obesity Association would
  15  be pleased to provide whatever assistance or
  16  support would be helpful to the committee in
  17  these undertakings.  Thank you.
  18            DR. SOX:  Thank you very much.  Our
  19  next speaker is Donald Baim.
  20            DR. KANG:  Hal?
  21            DR. SOX:  Jeff?
  22            DR. KANG:  Mr. Downey, on your second
  23  issue, procedurally -- I think you got our April
  24  notice last year -- you really need to submit a
  25  coverage decision internally.  MCAC gets only a
   1  very small subset referred to by HCFA.  This is
   2  actually the first time I'm aware of that
   3  coverage manual issue, and we'd be happy to look
   4  at it, but maybe we can talk about that off line
   5  how to get that done.
   6            MR. DOWNEY:  Okay.
   7            DR. SOX:  Thank you very much.
   8            Our next speaker is Dr. Donald Baim,
   9  Chief of the Interventional Cardiology Section at
  10  the Beth Israel Deaconess Hospital, and he's
  11  speaking today on behalf of the Health Industry
  12  Manufacturers Association.
  13            DR. BAIM:  Thanks.  It's my pleasure to
  14  be down here.  HIMA asked me to speak about some
  15  of the real world applicability of technology
  16  innovation and adoption in the interventional
  17  cardiology area and specifically as it pertains
  18  to the coverage decisions by this group.
  19            Can I see the first overhead, please.
  20  I think we all share common goals in terms of
  21  encouraging industry to develop newer devices and
  22  device improvements and facilitate the rapid
  23  adoption of safe and effective new diagnostic and
  24  therapeutic technologies in healthcare to improve
  25  the well-being of our population.  We more than
   1  anyone endorse the use of robust-data-driven
   2  approaches and avoiding technologies that are
   3  less effective.  And I'll talk a little bit about
   4  where the FDA process has gone in interventional
   5  cardiology.
   6            But in reading the report of the
   7  committee, I'm concerned that we preserve the
   8  nimbleness and responsiveness of a system of
   9  coverage decisions both to allow rapid adoption
  10  of technology and avoid placing already strapped
  11  hospitals in further financial jeopardy by
  12  forcing them to buy effective new technologies
  13  without offsetting reimbursement.  And we'll talk
  14  about an example of that next.
  15            So I want to make three basic points in
  16  this ten-minute slot.  The first is that we
  17  really need a variety of evidentiary sources,
  18  randomized clinical trials being one of them, but
  19  also including registries, equivalence trials and
  20  OPCs to deal with different situations.
  21            The second is to point out that the
  22  trials that are currently being done for FDA
  23  approval are large and very methodical and should
  24  be the first points considered as new
  25  technologies emerge from the FDA process and are
   1  considered for coverage.  I'll talk a little bit
   2  about the fact that I do believe they're
   3  sufficiently generalizable to apply to the care
   4  of Medicare population by mainstream operators.
   5            And third, that delayed HCFA coverage
   6  approval restricts application of new and better
   7  therapies and adds financial burdens to hospitals
   8  with an expense reimbursement gap as well as
   9  industry.
  10            So I really want to cover that first
  11  point, the variety, the spectrum of evidentiary
  12  sources.  At different points in the development
  13  of new technology, pilot registries may be
  14  valuable for proof of concept and device
  15  refinement, although not for the coverage
  16  decisions you're talking about here, but broader
  17  registries that may contain thousands of patients
  18  may be adequate for approval of certain well-
  19  characterized devices.
  20            Third, randomized equivalency trials
  21  are now being used by FDA to approve new
  22  generation stents that we'll talk about in a
  23  second and demonstrate noninferiority relative to
  24  other established therapies.  The randomized
  25  superiority trials that the guidance document
   1  focuses on to establish superior outcomes or
   2  cost-effectiveness of high-volume, high-cost or
   3  high-risk procedures once they're mature versus
   4  the prior standard of care are not the only sort
   5  of valid evidence that needs to be considered in
   6  the coverage decision.
   7            And finally, the importance of post FDA
   8  approval collection of population-based outcome
   9  data to document the use, patterns and risk-
  10  adjusted outcomes of competitive procedures for
  11  certain conditions in the real world should not
  12  be underestimated.
  13            I just wanted to talk briefly about how
  14  this whole interventional cardiology got here,
  15  and it was through registries.  The NHLBI PTCA
  16  Registry 1, in 1977 to 1981, lead to the adoption
  17  of this therapy, and the Registry 2, in 1985 and
  18  '86, documented the improvement in devices and
  19  technique.  Katherine Detre from the University
  20  of Pittsburgh and I, with NHLBI funding, set up a
  21  third registry in 1989 that ended up enrolling
  22  some 4500 patients with seven new interventional
  23  devices and really still constitutes the largest
  24  series of patients with core angiographic
  25  laboratory evaluation of one-year follow-up for
   1  many of these devices.
   2            That type of registry approach,
   3  however, was not sufficient to lead to the
   4  approval of stents.  So in 1993 the first stent
   5  versus angioplasty randomized trials were
   6  performed within the NACI registry that use
   7  single indications, a full randomized clinical
   8  trial machinery and lead to the approval of the
   9  J&J stent in a rigorous FDA process in 1994,
  10  making the United States the last of the
  11  industrialized countries to receive approval for
  12  this device.  So it's a very slow process,
  13  randomized trials.  Particularly as new
  14  technology becomes accepted, there's emerging
  15  reluctance to randomize stentable patients to
  16  conventional angioplasty, and that leads to a
  17  very prolonged approval for the second stent to
  18  try to go through this randomized comparison to
  19  angioplasty.
  20            So how have the variety of stents that
  21  are now in interventional practice gotten through
  22  this FDA process?  It's really been by a change
  23  in paradigm.  And the change in paradigm that
  24  took place in 1996 was really to say we don't
  25  need to randomize stents versus angioplasty any
   1  longer, that documenting equivalency to approved
   2  stent designs would be also an acceptable
   3  approach.  And the last half a dozen stents to be
   4  approved have been done in that format, usually a
   5  thousand patients randomized to a new versus an
   6  old stent.  Recruitment is faster because
   7  everyone gets a stent, and it's a good solution
   8  to follow-on improvements and accepted
   9  technology.  It has the rigor of an RCT, but
  10  without a placebo group.  It can also monitor for
  11  improvements in stent designs, but it's a
  12  paradigm that's showing signs of age because
  13  showing equivalency to a first generation stent
  14  is probably not good enough, and it wastes the
  15  money of reconfirming the performance of the
  16  first generation stent in each successive trial.
  17            So where we're headed in this new
  18  device era in 2000 and beyond is to develop OPCs,
  19  objective performance criteria, that will collect
  20  registry data and document performance consistent
  21  with the OPCs for stent performance.  The reason
  22  I go through this series of evaluation paradigms
  23  is really we're right back now with registries,
  24  and each of these different formats for evidence
  25  collection has been appropriate for a different
   1  point in the development of the technology.  We
   2  can't just fixate on randomized clinical trials.
   3            I just wanted to show you what this new
   4  device era has meant in our own practice, and
   5  this one shows in stacked bars the different
   6  therapies used in our program over the five years
   7  from 1994, when the J&J stent was approved,
   8  through 1998.  Angioplasty is the bottom bar
   9  shown in red, conventional balloon angioplasty,
  10  which has now fallen to 21 percent in
  11  interventions.  Stenting over that period has
  12  risen, the yellow bar, from 29 to 68 and now 79
  13  percent last year in 1999 with two atherectomy
  14  technologies accounting for the final quarter.
  15            So this adoption of technologies has
  16  really revolutionized our field.  The J&J stent,
  17  as we said, was approved in 1994.  And Medicare
  18  decision about coverage and assignment to DRG
  19  116, however, did not take place until 1997.  And
  20  in those three years between FDA approval and
  21  Medicare reimbursement coverage, the hospitals
  22  were having to buy this effective technology from
  23  manufacturers without any incremental
  24  reimbursement, and it contributed in no small way
  25  to the financial deneument of many of the leading
   1  institutions.
   2            Now, one could say this rapid adoption
   3  of technology is just to appease technology-
   4  crazed operators, but this shows the
   5  corresponding incidence of major complications
   6  over that same time period.  And the adoption of
   7  these technologies has in fact cut major
   8  complications in half, so we need to keep
   9  facilitating this rapid adoption process.
  10            I just want to close by taking you
  11  through one of the trials, a Boat trial and
  12  atherectomy trial, to give you a flavor for the
  13  generalizability of the Medicare population.
  14  This trial enrolled a thousand patients over a
  15  one-year time frame, actually 16 months, to
  16  angioplasty versus atherectomy.  This was done at
  17  36 centers, and this shows that they are
  18  geographically distributed, and they're both
  19  active practice centers.
  20            One concern is the age of patients, and
  21  what I've shown on this is the cumulative
  22  distribution in yellow of our own interventional
  23  patients whose median age is 64 compared to the
  24  age in pink, I guess, of 12 trials with 8,000
  25  patients that have been run by our daily
   1  coordinating center showing the median age of 63.
   2  So the age distribution in the interventional
   3  trials is representative of about half the
   4  Medicare population of routine practice.
   5            The issue about few golden operators
   6  driving the results of these trials, I think, is
   7  addressed here showing the center-by-center
   8  performance in this trial.  There's a wide
   9  variety of operators and operator experience, and
  10  as you can see in the DCA results shown in the
  11  yellow bars, in terms of residual stenosis
  12  there's a wide variety of practice patterns.
  13  Thank you.
  14            DR. SOX:  Thank you very much.  Our
  15  next speaker is Wayne Roe, who is Chairman of
  16  Covance Health Economics & Outcome Services in
  17  Washington, D.C., and he's speaking on behalf of
  18  the Health Industry Manufactures Association.
  19            MR. ROE:  Good morning.  I'm glad to be
  20  here.  I'm actually speaking on behalf of
  21  myself.  I'm speaking at the behest of HIMA.  I
  22  have lots of reasons to have conquest in this
  23  business, and I do a little bit of consulting in
  24  the coverage policy area, very little bit from
  25  the old days.  I'm on the boards of six medical
   1  start-up copies in the California area, involved
   2  with three venture capital firms who fund life
   3  sciences companies, all of whom will have things
   4  that will come before HCFA someday, but maybe not
   5  for three or four years.
   6            I think HIMA asked me to be here
   7  because I spent the last 15 years getting gray
   8  hair by coming to HCFA and working on coverage
   9  policies for probably over a hundred different
  10  devices, drugs, diagnostic tests and surgical
  11  procedures.  I've learned a lot about the
  12  process, got a lot of headaches through the
  13  process, have a lot of respect for the people
  14  doing coverage, and I think this group has its
  15  work cut out for it.  This is incredibly
  16  complicated stuff, as you hear today.  It's not
  17  simple, it's not trivial, and it can be academic
  18  and inherently judgmental no matter what you do.
  19            I'll start out with just a few
  20  comments.  HIMA doesn't know what I'm going to
  21  say because I wrote this last night when I was
  22  helping my daughter do chemistry, having read
  23  your paper several times.  I want to commend the
  24  MCAC.  I think you've done some very thoughtful
  25  work.  I think in 11 or 12 or 13 pages there's
   1  lots of good stuff in there.  I'm not going to
   2  try to wordsmith it at all.  I congratulate you
   3  on seven categories on the size of health
   4  effects.  I think those are pretty novel, pretty
   5  creative.  I think they really importantly
   6  reflect the fact that most new technologies in
   7  medicine, like it or not, are incremental.  They
   8  have a whole wide range of possible effects,
   9  positive and negative.
  10            Unfortunately, we believe there are too
  11  few breakthrough technologies.  It seems to be
  12  the way things work.  I wish we had more of
  13  them.  I think we want to encourage people to
  14  have more of them.  But I think having those
  15  categories three or four that clearly ought to
  16  lead to positive Medicare coverage decisions is
  17  kind of a good way to kind of simplify the
  18  world.
  19            I spent the last ten years telling
  20  medical developers I think they should stop
  21  thinking about thinking about themselves -- and a
  22  lot of this comes out of reading the work of Dr.
  23  Brook and Hal Sox and David Eddy and so forth --
  24  stop thinking about themselves as making tools or
  25  making drugs, but think about themselves as
   1  changing outcomes or changing the practice of
   2  care.  And if they don't do the right kind of
   3  research or science to demonstrate a change in
   4  how their product has an impact on how the
   5  patient does or at least how the patient is
   6  managed, then they shouldn't be bringing their
   7  technologies to HCFA or Blue Cross Association or
   8  anyone else.
   9            I think by and large that kind of
  10  admonition, which lots of people have been saying
  11  is getting through in the overall level of
  12  science, in the life sciences world, is a hell of
  13  a lot better today than it was 10 or 12 years
  14  ago.  There's no question about it.  No one even
  15  thought about any kind of randomized study, even
  16  controlled study, 12, 14, 15 years ago when I
  17  entered the device industry and we had the old
  18  National Center for Healthcare and Technology,
  19  which said many of the same things we've said
  20  that you are trying to say to today.
  21            And I encourage you to appreciate
  22  really that the document you're writing here is
  23  going to be a sentinel of technology
  24  gatekeeping.  We don't like to think this
  25  sometimes, but the bottom line is it's going to
   1  get read by lots of people, the final document,
   2  and it's going to be used by lots of people to
   3  make decisions.  It's a gatekeeping signpost.
   4  Obviously HCFA is a gatekeeper, but you all are
   5  the experts.
   6            We have a luminary panel here, the best
   7  and brightest we have in terms of doing outcomes
   8  research, and I think it's appropriate and
   9  important for you to encourage better science, to
  10  challenge the innovators to do better scientific
  11  work.  And I think the tone of this should be to
  12  do that.  On the other hand, I think it would be
  13  very bad to discourage them, to tell them well,
  14  we want everybody to high jump eight feet, and
  15  less than eight feet was never going to be
  16  adequate, but you know, we really know behind the
  17  scenes six, five or six, six is going to be
  18  okay.  I think that's a discouraging kind of
  19  tone, and I encourage you to take a look at the
  20  tone again.
  21            HCFA staff and the care and medical
  22  directors, as we're here today, to private
  23  managed care medical directors, will read what
  24  you say, and they'll use it.  You don't want to
  25  give them the excuse to hide behind it, to not
   1  make decisions, to put everything on randomized
   2  controlled trials, because the bottom line is
   3  we're not going to have them all.  We're never
   4  going to have them all.  And it would be kind of
   5  an academic pipe dream to expect we're going to
   6  have it.  I don't think you should set the bar so
   7  high for people to use that as an excuse not to
   8  make tough decisions, not to allow progress in
   9  medicine.  So please be realistic.  You can't be
  10  academic in this exercise even though you want to
  11  be.
  12            I guarantee you I've been through
  13  this.  Somewhere in Menlo Park, California there
  14  is someone sitting down making a decision to fund
  15  $20 million for an Internet taco business versus
  16  some promising technology that will gather up
  17  plaque during cardiac endarterectomies that might
  18  save one of our lives someday.  You don't want to
  19  discourage those people who might get the money
  20  to do the atherectomy device or filtration
  21  technology with the idea that you have to have
  22  two huge randomized controlled trials in order to
  23  get coverage.  That is just a bad thing to send.
  24  But those decisions happen all the time with
  25  increasing frequency.  You've got your capital
   1  world and the pharmaceutical firms and so forth
   2  who are going to read this document and look at
   3  it, and they're going to look to you for some
   4  guidance.  Give them hope, give them a challenge,
   5  but don't let them feel like it's hopeless
   6  because they'll go and fund those Internet taco
   7  businesses, and I don't think we need that as
   8  much as we need things to deal with
   9  endarterectomy.
  10            Specific suggestions.  First, I find it
  11  quite amazing -- a little hyperbole in all of
  12  this, of course -- that there's no mention
  13  whatsoever -- maybe one mention -- of the FDA
  14  standard of evidence or labeling in this
  15  document.  Everything goes through the FDA to
  16  start.  I know we all in the coverage policy
  17  arena realize maybe it's not enough sometimes,
  18  but every new technology is studied with the FDA
  19  in mind.  And the FDA has very good outcomes
  20  researchers there, and they require sometimes
  21  randomized trials, sometimes not randomized
  22  trials, sometimes controlled trials, sometimes
  23  not, depending upon the product.  It seems to me
  24  there ought to be some recognition that the FDA
  25  is enough for certain things, particularly
   1  pharmaceuticals.
   2            The concept that people do
   3  well-controlled randomized trials, two of them in
   4  pharmaceuticals, for the purposes of
   5  demonstrating safety and efficacy and they're
   6  labeled to do and not to say hey, those things
   7  we're not going to take a look at and do a report
   8  on just seems to me to make your job more
   9  difficult and question what we have the FDA for.
  10  So I'd take a hard look what the FDA says.
  11            I had these discussions years ago with
  12  the Food and Drug Administration.  For whoever
  13  you talk to, the people I've talked to up there
  14  say when we approve something, be it a device,
  15  drug, diagnostic test, we're not approving it for
  16  Stanford, Hopkins or Cleveland Clinic.  We
  17  believe that if we let it in the marketplace,
  18  it's going to work when lots of people use it,
  19  everybody uses it, the average physician who is
  20  licensed and capable of using it.  You may
  21  question that, but the FDA doesn't say that.  If
  22  we think that only certain experts can use it,
  23  it's going to be effective there, then we're
  24  going to put that in the labeling and
  25  restrictive.  So take a look at that question.
   1            You heard this before.  The document in
   2  places, I think it needs more tone editing.  Far
   3  too much weight on randomized controlled trials
   4  as the desired level of evidence.  We're going to
   5  have them, we're going to have more of them, but
   6  they're going to be rare.  And we can't afford
   7  them all.  And we all know there are lots and
   8  lots and lots of reasons why we can't do them.
   9  And the FDA doesn't require them every time even
  10  for drugs.  So I think you have to recognize
  11  that.  There's lots of good science being done
  12  far better than before.  Overemphasis on
  13  randomized controlled trials is going to make
  14  other research seem inadequate, and I think it
  15  will lead to some research not being done, some
  16  good research not being done, and things not
  17  being developed.
  18            I think in the probably hundred things
  19  I've taken to HCFA over the last 15 years for
  20  national coverage evaluations or at least a peek
  21  at the national level without decisions being
  22  made to float down to the care level, maybe five
  23  technologies had very good powerful two or three
  24  randomized controlled clinical trials, but I
  25  never brought anything up here that wasn't pretty
   1  good scientific evidence that would lead someone
   2  to believe this is something that should have a
   3  good shot at being covered, and I'd say
   4  two-thirds of the time they were.  So I'd go back
   5  and recognize that there's a pragmatic end to
   6  this area, and if you put five or six clinical
   7  experts in a room before you to develop a
   8  technology, you can probably get to a scientific
   9  result that will make people feel that there's a
  10  benefit there.
  11            I think there's a serious source of
  12  bias in this document.  The bias is against new
  13  innovations.  Effectively what you're saying here
  14  is -- and Dr. Brook and others have published on
  15  this -- ten percent or less of all medicine that
  16  we have right now has any scientific controlled
  17  studies done on it.  This effectively says we're
  18  grandfathering all the old stuff.  We're not
  19  going to take a look at what we're comparing it
  20  to.  We want you to compare it to the old stuff.
  21  What if the old stuff's never been studied?  To
  22  me one of the biggest problems we have in
  23  technology evaluation of coverage policies is we
  24  can't get rid of the old stuff.
  25            For example, if the HMOs feel that ABMT
   1  for breast cancer is not any good, are they still
   2  covering it today?  We need to take a look at
   3  this.  We've got to get rid of the old stuff and
   4  question that before we just say the bar's higher
   5  now for everything new.  The science behind
   6  everything new is definitely better.
   7            Timing.  I worry about how long this is
   8  going to take.  Reports, consultants, et cetera,
   9  there's no way this is a six-month deal.  It's
  10  hard to believe.  There may not be enough top
  11  flight people with time who aren't publishing and
  12  doing research to be able to do this evaluation.
  13  I think MCAC should seriously take a look at
  14  talking with HCFA on provisional coverage.  If
  15  the data isn't quite right, but we think it's
  16  promising, then let's think about a situation
  17  where we set out these are the outcomes we'd like
  18  to have you take a look at.  We will cover for a
  19  fixed time period and stick to it, six months, a
  20  year.  This technology and other things that are
  21  being done require you, the person who's getting
  22  the benefit of having the thing covered, to
  23  collect the information, come back to us a year
  24  later because the clock stops, the coverage stops
  25  here till you give it to us.  I think you need
   1  some kind of innovative idea here which will
   2  allow research to be done.
   3            So in short, be realistic in what you
   4  ask for.  Use the FDA.  They've got to have a
   5  role here.  Don't ask for what you can't have.
   6  It's very discouraging.  Question the old stuff.
   7  Don't be advised against the new.  And time is
   8  money and opportunity.  I think you can
   9  incentivize better science with coverage, and
  10  we're not doing enough of it now, and I think
  11  that can be done even within the legal
  12  parameters.  Thank you.
  13            DR. SOX:  Thank you very much.  At this
  14  point we've earned ourselves a break of about 20
  15  minutes.  So be back at five minutes after 10:00
  16  o'clock.
  17            (Whereupon, recess taken -- 9:45 a.m.)
  18            (Whereupon, after recess -- 10:05 a.m.)
  19            DR. SOX:  If I could call the meeting
  20  back to order, please.  The first speaker is
  21  Vicki Gottlich, Center for Medicare Advocacy and
  22  Healthcare Rights Project.
  23            MS. GOTTLICH:  I'm Vicki Gottlich, an
  24  attorney with the Center for Medicare Advocacy
  25  and their Healthcare Rights Project in
   1  Washington, D.C.  The center is about 15 years
   2  old.  Our organization represents low income
   3  Medicare beneficiaries.  We currently have about
   4  60,000 open case files in which we're trying to
   5  get Medicare to pay for medically necessary
   6  services for our clients.
   7            I appreciate the opportunity to speak
   8  here today, and I particularly appreciate the
   9  opportunity to be representing beneficiaries
  10  before this committee.
  11            It is imperative for our clients that
  12  HCFA establish a mechanism for protecting the
  13  rights and interests of beneficiaries to receive
  14  medically necessary care and services authorized
  15  by their doctors.  The current processes
  16  available to beneficiaries, the claims and
  17  appeals process and the national coverage
  18  determination process under discussion today do
  19  not protect beneficiary rights.  Our clients and
  20  other beneficiaries have had limited success with
  21  the NCD process often because that process has
  22  not been open to them.  Few patients know they
  23  will need a procedure or technology when the
  24  process is underway, and even if they have timely
  25  knowledge, they generally do not have the
   1  resources to participate in the process.
   2            Of utmost importance, the current
   3  process for evaluating new procedures and
   4  technologies and for reevaluating previous
   5  coverage determinations is too slow.  Conditions
   6  deteriorate, and beneficiaries die, and I really
   7  want to emphasize that we have had clients die
   8  while waiting for HCFA to decide to cover
   9  services, technologies and devices covered by
  10  other insurers, including private industry, the
  11  Department of Veterans Affairs and state Medicaid
  12  agencies.
  13            We applaud the subcommittee for their
  14  efforts to clarify the national coverage
  15  determination process.  We are greatly concerned,
  16  however, that the process used by HCFA and under
  17  consideration today exceeds the agency's
  18  authority by depriving beneficiaries of services
  19  prescribed by their physicians for extended
  20  periods of time.
  21            Let me explain.  I really don't need to
  22  describe to this group what the Medicare statute
  23  says because you're all familiar with the
  24  Medicare statute.  And the statute provides that
  25  services will be covered as long as they are
   1  medically necessary or Medicare will not pay for
   2  services that are not reasonable and necessary.
   3            The key point to the exception that
   4  HCFA will not cover services is a determination
   5  by HCFA that a service is not reasonable or
   6  necessary.  In other words, Congress placed the
   7  burden on the agency to overcome the presumption
   8  that the service is covered.  Congress did not
   9  prohibit coverage of services prescribed by
  10  beneficiaries' doctors simply because enough or
  11  the right kinds of studies showing their positive
  12  value have not yet been amassed.  This
  13  interpretation is in keeping with the prohibition
  14  against controlling the practice of medicine or
  15  the manner in which medical services are
  16  provided.
  17            But the proposals today follow HCFA's
  18  practice of placing the burden of proof on the
  19  proponent to show why a service or technology
  20  should be covered and to produce evidence of a
  21  certain type in standard that is not always
  22  available or even appropriate to the
  23  beneficiaries who actually need the service.
  24            The proposals do nothing to assure that
  25  beneficiaries will receive quick access to the
   1  services their own physicians found reasonable
   2  and necessary.
   3            For example, the suggestion that
   4  outside experts be used in certain situations to
   5  evaluate the evidence exasperates the delay
   6  problem.  In addition to harming beneficiaries,
   7  such delays cause further disparities between
   8  Medicare and private insurance coverage and
   9  result in carriers having to deny Medicare
  10  coverage for services they cover in their own
  11  private insurance practice.
  12            The proposals also fail to address
  13  adequately the needs of the over five million
  14  beneficiaries under age 65.  Many members of this
  15  community are adversely affected by HCFA's
  16  failure to include new devices and technologies
  17  among Medicare's covered services.  Delays in the
  18  processing for approving devices and technologies
  19  result in beneficiaries with disabilities losing
  20  their independence or their ability to function
  21  to their maximum capacity.
  22            Beneficiaries with disabilities are
  23  also adversely affected by national coverage
  24  determinations that are based on evidence
  25  applicable only to the population over age 65.
   1            For example, the Office of Civil Rights
   2  of the Department of Health and Human Services
   3  last year worked on and assisted a Medicare
   4  beneficiary in her mid 40s who was denied
   5  coverage of a potentially life-saving cancer
   6  treatment because of a national coverage
   7  determination.  The national coverage
   8  determination was based on evidence that the
   9  treatment was not efficacious for women over age
  10  65.  Ample evidence existed, however, that the
  11  procedure was effective for younger women, and
  12  the Medicare HMO in which the woman was enrolled
  13  covered the procedure for its non-Medicare
  14  population.
  15            While the appeals process is not a
  16  concern of this group, it is really an important
  17  element for our clients because the appeals
  18  process provides no recourse for beneficiaries
  19  who seek to challenge the national coverage
  20  determination or to get Medicare coverage of a
  21  technology or device not yet approved by
  22  Medicare.  The Medicare statute makes it nearly
  23  impossible to challenge a national coverage
  24  determination rule upon which services were
  25  denied by preventing consideration of the issue
   1  at the administrative level.  If the claim
   2  reaches federal court, a federal judge who
   3  determines that the record is incomplete or
   4  insufficient to support the validity of the
   5  national coverage determination must remand the
   6  case for supplementation of the record.  The
   7  court may only determine that an item or service
   8  is covered after review of the supplemented
   9  record.
  10            So the individual who was adversely
  11  affected by the obesity ruling that was discussed
  12  earlier today would have to go through the whole
  13  national coverage determination process and
  14  couldn't go through an appeals process in order
  15  to change the ability to get coverage for
  16  treatment for obesity.  If the national coverage
  17  determination process is as lengthy as the
  18  appeals process, it is going to be years, and
  19  that's why we are very concerned about the
  20  delays.
  21            In sum, we are not advocating that
  22  Medicare pay for quack services, which have been
  23  shown to lack medical value.  We are advocating
  24  for an efficient coverage determination process
  25  that allows Medicare beneficiaries to receive
   1  Medicare payment for services and procedures,
   2  devices and technologies that have been approved
   3  by the FDA where appropriately are being covered
   4  by private insurers, the VA and Medicaid, and are
   5  found by the beneficiary's own physician to be
   6  reasonable and necessary for treatment of that
   7  beneficiary's illness or condition.
   8            We also seek an effective and
   9  expeditious appeals process that will allow
  10  beneficiaries to challenge a denial of coverage
  11  based on an NCD that is no longer supported by
  12  medical evidence and practice.  And while that's
  13  not within your jurisdiction, we do ask that you
  14  consider an expedited process to consider NCDs
  15  that don't have any support for them.  And there
  16  are a lot, as I'm sure that you are aware.  Thank
  17  you very much.
  18            MS. LAPPALAINEN:  Vicki, would you
  19  state for the record whether you have any
  20  financial interest in the --
  21            MS. GOTTLICH:  I'm sorry.  Our
  22  organization has no financial interest in any
  23  medical devices, and neither do I.  Thank you.
  24            DR. SOX:  Our next speaker is Larry
  25  Weisenthal from the Weisenthal Cancer Group.
   1            DR. WEISENTHAL:  My name is Larry
   2  Weisenthal.  I'm a medical oncologist in private
   3  practice, and I provide the service that I'll be
   4  describing.  I'm a medical oncologist from
   5  Huntington Beach, California.  I participated in
   6  the Medicare Coverage Advisory Committee meeting
   7  last November 15th and 16th.  My experience
   8  related to this meeting is what now compells me
   9  to offer comments concerning the structure and
  10  procedures for future MCAC reviews.
  11            My specific concerns involve, one,
  12  serious defects in the advanced draft outline of
  13  the proposed review process, and two, a lack of
  14  appreciation for special considerations related
  15  to laboratory testing in a draft proposal which
  16  seems exclusively directed toward the review of
  17  direct therapeutic interventions.
  18            Rather than speaking in a theoretical
  19  sense, I would like to use my own experience with
  20  the November MCAC meeting to convey my concerns.
  21  The draft proposal places heavy emphasis on a
  22  series of independent reviews by so-called
  23  experts in the field.  Essentially the process
  24  would be centered around a collection of up to
  25  six independent written reviews by these
   1  experts.  There would appear to be a relatively
   2  small role for the proponents of the technology
   3  under consideration as they would have no
   4  opportunity to rebut these reviews in advance of
   5  the meeting.  One can easily project proponents
   6  having to use their entire 15 or 20 minutes or
   7  less of allocated time at the meeting just to
   8  hurry through complicated rebuttals of complex
   9  and misconstrued data.
  10            The November MCAC meeting considered
  11  the issue of human tumor assays, which involved
  12  short-term cultures of fresh biopsies of human
  13  tumors in the presence and the absence of
  14  anticancer drugs.  Following cell culture, drug
  15  effects are assessed by one of two end points,
  16  either cell proliferation or cell death.
  17            Historically all work in this area was
  18  effectively abandoned in American universities in
  19  the mid-1980s.  The only major academic group
  20  continuing work in this area was the lung cancer
  21  group at the National Cancer Institute.  However,
  22  the NCI investigators had a primary focus on
  23  creating cell lines through passaging and
  24  subculturing.  I anticipated a major emphasis on
  25  three public studies arising from this work, and
   1  I quoted several pages of my proposal, submitted
   2  two and one-half months in advance of the
   3  November meeting, to a detailed rebuttal of this
   4  work.
   5            Fearful that this rebuttal would be
   6  overlooked, I was also forced to devote precious
   7  minutes of my oral presentation to this issue,
   8  which gave me no time to take the committee
   9  through the many important positive studies and
  10  prestigious peer-reviewed journals, which were
  11  included in my written proposal, but which were
  12  ignored by all the reviewers chosen by HCFA.
  13            The major reviewer of the cell death
  14  technologies proposed for coverage by me was Dr.
  15  Edward Sauceville, associate director of a
  16  developmental therapeutics program at the
  17  National Cancer Institute.  Dr. Sauceville did
  18  not attend the morning presentations by the
  19  proponents and their supporters.  This led to the
  20  following embarrassing statement, quote, you can
  21  tell a patient who has the unfortunate diagnosis
  22  of pancreatic cancer that they're likely not
  23  going to respond to a medicine chosen after
  24  having gone through an additional test to obtain
  25  tissue and then test it for assay resistance.
   1            This statement was embarrassing because
   2  one of the earlier speakers had been a pancreatic
   3  cancer patient who has been in complete remission
   4  for more than three years after presenting with
   5  liver and kidney metastases and then being
   6  treated with an assay-selective drug regimen,
   7  which everyone agrees would never have been
   8  chosen absent performing the test.
   9            Dr. Sauceville was also either not
  10  shown or did not bother to read my written
  11  proposal submitted two and one half months in
  12  advance of the meeting.  He showed his complete
  13  ignorance of the field by failing to even
  14  mention, much less consider, 80 percent of the
  15  studies, totalling more than 1500 patients,
  16  confining his review almost exclusively to
  17  studies published before 1987 and to the
  18  irrelevant studies that the NCI lung cancer group
  19  alluded to previously.  Neither did he nor any of
  20  the other HCFA reviewers review and describe most
  21  of the many studies correlating assay results
  22  with patient survival.
  23            Again, all these data references were
  24  provided to HCFA two and a half months in advance
  25  of the meeting.  Nonconsideration of these
   1  studies led to the following remark at the
   2  December Executive Committee meeting by one of
   3  your members, Dr. Ferguson, who related, quote,
   4  we had very little survival information.  There
   5  were some unsettled elements.  I don't remember
   6  that there were other ones.
   7            This remark forced me to make the
   8  following frustrated comment at the December
   9  Executive Committee meeting, quote, there were
  10  many misrepresentations made, such as the lack of
  11  survival data.  I showed a slide at the meeting.
  12  There are 15 studies showing strong correlations
  13  with survival.  This is not just based on
  14  response.
  15            That the above assessment of the
  16  inadequacy of the outside review process is not
  17  just a figment of my imagination was shown by the
  18  comments of the committee chairman Dr. John
  19  Ferguson again at the prior meeting of this
  20  Executive Committee in December.  Quote, another
  21  was that the NCI representative presented a paper
  22  which in my view I was a bit disappointed in
  23  coming from my former institution that it did not
  24  seem to me to be up to date and lacked in that
  25  aspect.  Dr. Ferguson went on to say so I am not
   1  certain that the protagonists were given all the
   2  critiquing information.  We didn't have it.  We
   3  tried to give the protagonists time to respond.
   4  I think that that could have been done a little
   5  bit better in the sense that if all the critiques
   6  of presented papers could have been given to the
   7  presenters in advance, they might have had time
   8  to prepare some rebuttal in response to the
   9  critiques.
  10            Even more egregiously misleading than
  11  Dr. Sauceville's inadequate review was the
  12  horribly misleading review of HCFA's Dr. Burken,
  13  which by objective evidence demonstrably and
  14  unfairly damaged the case put forward by the
  15  proponents.  By way of background, one of the
  16  technologies proposed for consideration of
  17  coverage was the cell proliferation assay based
  18  on measuring tritiated radionuclide incorporation
  19  as an assay end point.
  20            Data was presented to document the high
  21  specificity of this assay in identifying drug
  22  resistance.  In his review of the literature, Dr.
  23  Burken devoted considerable time to technologies
  24  which had been abandoned 10 to 15 years
  25  previously and which were not proposed for
   1  Medicare coverage by anyone in the November
   2  review.  One of these abandoned technologies was
   3  a radionuclide precursor incorporation assay
   4  measuring the incorporation of tritiated
   5  thymidine or uridine only three hours after the
   6  addition of anticancer drugs to freshly
   7  disassociate the tumor cells.
   8            This contrasts with the technology
   9  under MCAC consideration which measured thymidine
  10  incorporation five days -- not three hours --
  11  after drug administration.  Whereas the five-day
  12  assay predicted for drug resistance with very
  13  high specificity, the three-hour assay gave very
  14  poor results and was abandoned by its own
  15  proponents in the 1980s.  Yet Dr. Burken showed
  16  four different slides detailing the poor results
  17  with this assay.  This demonstrably confused and
  18  mislead the panel, as conveyed by the panel's
  19  industry representative, who showed us a table
  20  constructed and to specify the MCAC panel
  21  depicting the negative predictive accuracy
  22  reported in the various studies and prominently
  23  including the four studies with the long
  24  abandoned three-hour assay which showed such poor
  25  correlations.
   1            The verbatim transcripts of the MCAC
   2  panel's deliberations revealed the damaging
   3  effect which the inclusion of these irrelevant
   4  studies had on the MCAC enthusiasm for coverage.
   5  Although clear from the transcript that there was
   6  overwhelming support for HCFA developing a policy
   7  to include coverage of these assays in at least
   8  some clinical situations, this support would have
   9  clearly been less reserved in the absence of the
  10  misleading presentations by the reviewers chosen
  11  by HCFA.  This is crystal clear in the
  12  transcripts of the meeting.
  13            But the purpose of my comments here is
  14  not so much to complain about the past as to help
  15  the Executive Committee develop a better process
  16  for future reviews.  To this end we must begin to
  17  appreciate that we are working in a time when an
  18  increasing number of important advances in
  19  medicine are occurring outside the traditional
  20  NIH and university research system.
  21            In the case of human tumor assays,
  22  there are no experts at all in either American
  23  universities or at the NIH.  No investigator at
  24  these institutions has contributed in any way to
  25  the literature in the field I represent of cell
   1  culture drug-resistance assays with cell death
   2  end points.  In my 20 years of full-time work in
   3  this field, I've talked with hundreds of
   4  university and NIH-based investigators with an
   5  opinion about this field.  It's been more than
   6  ten years since I last had a discussion with a
   7  non-European and non-Japanese university-based
   8  investigator to be able to discuss the subject
   9  based on an intelligent understanding of concepts
  10  and literature.
  11            So HCFA must be very careful to ensure
  12  a central role of the proponents of the new
  13  technology in presenting and explaining data to
  14  the MCAC panels.
  15            Cutting to the chase, we propose the
  16  following modification in the overall outline of
  17  the proposed system.  First, the process begins
  18  with a formal request to HCFA for coverage
  19  consideration.  Once informed that HCFA agrees to
  20  consider the issue, the proponents are
  21  responsible for presenting a formal defense of
  22  their proposal centered around a description of
  23  technology and complete review of all relevant
  24  data and literature.  This proposal is then sent
  25  to each of the outside reviewers.  The outside
   1  reviewers then prepare their own independent
   2  reviews, which are then given back to the
   3  proponents for rebuttal.  The rebuttals go back
   4  to the reviewers who are allowed to have the
   5  final word in the pre-meeting written
   6  presentations and reviews provided to the MCAC
   7  panel.  The proponents should also certainly
   8  receive a copy of this final review while in
   9  advance of the meeting.
  10            The meeting itself could then take
  11  place with all the complicated and contentious
  12  issues having already been pre-argued.  The
  13  meeting itself would begin with relatively brief
  14  summations by both proponents and reviewers,
  15  followed by a devotion of most of the time to
  16  open discussion by the committee with committee-
  17  directed questions to both proponents and
  18  reviewers.  However, prior to final deliberations
  19  and votings, both proponents and reviewers should
  20  have the opportunity to make brief final remarks.
  21            I've got one page here which I won't go
  22  over the time, but could this be put into the
  23  record?
  24            DR. SOX:  Sure.  If you want to submit
  25  something in writing.
   1            DR. WEISENTHAL:  Thank you.
   2            DR. SOX:  Our next speaker is Sandy
   3  Sherman, Assistant Director of Division of
   4  Federal Affairs & Outreach of the American
   5  Medical Association.
   6            MS. SHERMAN:  Good morning.  I just
   7  have a brief statement from Dr. E. Radcliffe
   8  Anderson, who's the Executive Vice President and
   9  CEO of the AMA, regarding your discussion paper.
  10            After the first MCAC Executive
  11  Committee meeting in December, I wrote to
  12  Nancy-Ann DeParle to say that the AMA was
  13  impressed and gratified by the commitment of the
  14  advisors and HCFA to ensure that MCAC
  15  recommendations would be grounded in scientific
  16  evidence of clinical effectiveness.  I also said
  17  that the meeting made it clear that she had
  18  fulfilled her promise to create an open, timely
  19  and accountable process for making national
  20  coverage decisions.
  21            The discussion paper that the committee
  22  members prepared for today's meeting underscores
  23  the observations we made in December.  The
  24  recommendations for evaluating evidence clearly
  25  state the key issues to consider in assessing the
   1  state of the knowledge regarding medical
   2  interventions proposed for Medicare coverage.  We
   3  are pleased that in addition to recommending a
   4  critical review of evidence from clinical trials,
   5  the Executive Committee or the members who
   6  prepared this proposal recommend that the
   7  standard of excellence for the evidence report
   8  include work developed by the national medical
   9  specialty societies.  We also commend the
  10  advisors for recommending that panel members take
  11  an active role in framing the questions to be
  12  addressed by the evidence report, participate in
  13  the report's preparation and seek external review
  14  of the evidence reports.
  15            Prior to the MCAC's formation, the AMA
  16  had expressed concern that Medicare coverage
  17  decisions might be driven to a large degree by
  18  information presented by those with a vested
  19  interest in coverage instead of by the available
  20  scientific and clinical evidence.  The discussion
  21  paper developed by the advisors has allayed our
  22  concerns in this regard, and we encourage
  23  adoption of its recommendations.
  24            DR. SOX:  Thank you very much.
  25            Our last speaker is Thomas Meskan,
   1  president of Medical Alley.
   2            MR. MESKAN:  Good morning.  My name is
   3  Tom Meskan, president of Medical Alley.  In terms
   4  of your financial statement, obviously we have
   5  members who pay dues to our association, and I
   6  presume that a number of them have issues pending
   7  before the agency.
   8            For those of you who aren't familiar
   9  with Medical Alley, we're a 15-year-old not-for-
  10  profit trade association based in Minnesota who
  11  has members from all aspects of healthcare.  Our
  12  members include health plans, medical device
  13  manufacturers, hospitals, clinics, long-term care
  14  organizations and academic health centers.  Our
  15  mission is to serve as a collaborative form which
  16  promotes an environment to enhance innovation in
  17  healthcare.
  18            I appreciate the opportunity to share
  19  our perspective and thoughts as they relate to
  20  the discussion paper.  We think that the MCAC
  21  process is an important aspect of Medicare's
  22  decision making and want to acknowledge and
  23  express our thanks for the time and effort all of
  24  the people, both you as panel members and agency
  25  staff, are spending to try and make the MCAC a
   1  valued component of Medicare decision making.
   2            To help you get a sense of the
   3  orientation of our organization, I will point out
   4  that we believe that Medicare should be a prudent
   5  purchaser of services, and we think that it is
   6  important that the agency has appropriate levels
   7  of resources to do its job.  At the same time we
   8  believe that the environment surrounding
   9  Medicare, and for that matter, all of healthcare,
  10  should be dynamic so that patient care improves
  11  in a timely and continuous manner.
  12            With regard to our principles on
  13  generating evidence, they are that HCFA
  14  preferences for how evidence is presented should
  15  be transparent.  Any approach to decisions about
  16  coverage criteria should be administratively
  17  feasible for both the agency and the
  18  stakeholder.  It is desirable that stakeholders
  19  achieve the level of valid scientific evidence
  20  necessary to demonstrate that a service should be
  21  covered, and there should be a minimization of
  22  potential for bias into conduct, reporting and
  23  analysis of studies.
  24            Our comments today fall into two
  25  categories.  First, we want to offer some
   1  observations about the role of perceptions in the
   2  success of your efforts.  Second, we will offer
   3  some specific reactions to some of the text in
   4  the discussion document.
   5            It is clear by looking at the names
   6  which make up this committee and the impressive
   7  roster of individuals that make up the MCAC
   8  panels that there is a wealth of expertise
   9  available to the agency.  I had the opportunity
  10  to introduce myself to Dr. Sox during the break,
  11  and he, if I can paraphrase him, said what he
  12  liked about his involvement in this committee is
  13  its potential effect to a large number of human
  14  beings and their health condition.  And I think
  15  that that's a very accurate statement.  And the
  16  most important point is we must make sure that
  17  you guys do everything you can to maximize your
  18  potential.
  19            Obviously each of you are approaching
  20  your MCAC responsibilities in good faith and with
  21  a desire to achieve the goals of consistency and
  22  accountability.  Further, you have laid out the
  23  recommendations in a manner which strongly
  24  signals your interest in promoting the greatest
  25  possible degree of rigor in the methods used to
   1  generate evidence.
   2            We too want to encourage the
   3  development of a decision-making process that
   4  will be informed, and we also support the
   5  continued improvement in the way the supporting
   6  data is collected and utilized.  Nonetheless,
   7  this committee, the agency and external
   8  stakeholders must acknowledge the history of
   9  coverage policy development so that whatever
  10  process this committee decides upon enjoys
  11  support of the largest possible percentage of
  12  affected stakeholders.  In this manner you can
  13  ensure that your time and efforts are valuable.
  14            In brief, that history suggests that
  15  whatever approach is taken by the agency and
  16  those who advise it to create greater detail on
  17  the concept of reasonable and necessary will be
  18  subject to extremely close scrutiny.
  19            We know the examples, a coverage
  20  regulation that has been kicked around since
  21  1987, the fact that this committee is just
  22  starting to get off the ground two years after
  23  the GAO found the act to be in violation of FACA.
  24  We also know that frequently in coverage decision
  25  making it becomes subject to second-guessing by
   1  Congress.
   2            We raise this because we want to
   3  encourage you to get this process off on the
   4  right foot.  We want the MCAC process to succeed
   5  and be used.  And while I heard Dr. Bergthold's
   6  comments about the effort that you went towards
   7  submitting this, it serves no one's interest if
   8  your approach is perceived incorrectly or not as
   9  so academically grounded that MCAC becomes
  10  nothing more than another health policy center
  11  which provides insights that have little life
  12  beyond those who formulate and to make them
  13  internally.
  14            We believe it is fair to say that
  15  outcomes research and technology assessment are
  16  evolving disciplines.  Further, while the
  17  document does not say so, it is extremely rare
  18  that data is ever perfect.  Similarly, a number
  19  of decisions faced by panels are likely to
  20  inquire around one of the truisms that surround
  21  healthcare.  That is part art and part science.
  22            Therefore, we encourage you to modify
  23  your discussion document to acknowledge these
  24  factors and create the opportunity for our
  25  acceptance of your approach.  Similarly, it will
   1  enhance your opportunity to improve the
   2  effectiveness of the panels.
   3            We offer you the following language as
   4  an example of a kind of statement that you might
   5  make.  Evidence presented to support a coverage
   6  decision should be deemed acceptable if it is
   7  ethically appropriate, administratively feasible
   8  and if it meets the current generally accepted
   9  used requirements for evaluation of a health
  10  service typically found within a technology
  11  assessment literature that were in place at the
  12  time the study was undertaken.  This is not to
  13  say that the evidence is then accepted as meeting
  14  a case for coverage, but rather reflects a common
  15  sense approach to considering the practical
  16  implementation issues which surround the
  17  methodology options for generating data.
  18            It is simply the case that a majority
  19  of the people who are involved in generating
  20  evidence for decision making are well-meaning
  21  people who want to do the best job they can.
  22  This does not mean that they are at all as
  23  schooled and knowledgeable as you on the nuances
  24  of evidence generation.  Your document needs to
  25  implicitly acknowledge these individuals and to
   1  speak to them in a manner which allows them to
   2  see clear, feasible pathways to being
   3  constructive contributors to Medicare coverage
   4  decision making.
   5            We suggest that with that opportunity
   6  comes an obligation.  We would suggest that the
   7  document be modified to express the interest of
   8  panels in receiving from stakeholders the
   9  rationale which drove such things as the study
  10  design, data sources utilized, the rationale for
  11  what the service is being compared to, the time
  12  horizon that's chosen and the statistical
  13  analysis methods used to address random events.
  14  In addition, we think it's appropriate for
  15  stakeholders to describe this data from
  16  unpublished sources.  This will provide useful
  17  information to the panels as they seek to weigh
  18  the value of the evidence presented.
  19            Let me now move to our observations
  20  about the specific aspects of the document.
  21  First of all, we would note that the paper fails
  22  to acknowledge those stakeholders who have
  23  already completed or are currently in the process
  24  of carrying out efforts to generate data for a
  25  national coverage decision.  The paper needs to
   1  provide some guidance so that these stakeholders
   2  and/or the panels do not feel that an
   3  organization must necessarily go back to square
   4  one in generating evidence because of this
   5  document.
   6            Moving to another area, while we
   7  recognize the panel's purpose is to focus on
   8  issues of science and evidence, it's somewhat
   9  ironic that the words or concept of a patient do
  10  not appear until page 6.  While the document's
  11  failure in this regard could be seen as semantic
  12  window dressing, we believe it's important that
  13  we all keep front and center in the end.  This is
  14  what we're all about.
  15            That said, the committee has indicated
  16  its interest in the panel's making conclusions
  17  about health outcomes.  We would ask that the
  18  committee modify the text on page 7 or at least
  19  my Internet version on page 7, item 3.  This text
  20  addresses the need for the panel to explain its
  21  conclusions.  We suggest that the committee ask
  22  the panels to describe as specifically as
  23  possible how each of the various health outcomes,
  24  including, but not limited to, mortality,
  25  morbidity, functional status, quality of life and
   1  patient experience were factored into its
   2  decision making.  By making the reporting
   3  requirements more detailed, the goals articulated
   4  in this item will be better achieved.
   5            We also believe that significant
   6  thought should be put into the item on page 7
   7  about the evidence reports provided to the
   8  panels.  Although the ability of this proposal to
   9  operate in a timely manner is suspect, we are
  10  also very concerned that the document does not in
  11  any way provide affirmative action between the
  12  stakeholder and MCAC on what materials will be
  13  contained in the evidence report.  We think the
  14  document should provide a mechanism for dialogue
  15  between stakeholders and the appropriate panel
  16  representatives before submitting the report.
  17            Another area of concern is found on
  18  page 5, the last sentence dealing with bias.  The
  19  text can be read to require that the panels
  20  describe why bias does not account for the
  21  results.  Conversely, the subjectivity, if you
  22  will, in judgment calls which are involved with
  23  these issues, we believe that the panel should be
  24  empowered to describe why it's comfortable with
  25  its conclusions.
   1            Finally, on page 6, the last two
   2  sentences on external validity, the terms typical
   3  practice setting and general practice setting
   4  appear to be used interchangeably.  Because of
   5  the importance that the agency puts on
   6  appropriateness of making decisions, we believe
   7  it would be valuable to clarify what the terms
   8  typical and general mean.
   9            In sum, we believe that all Medicare
  10  stakeholders are benefited by the recognition
  11  that improving the Medicare coverage decision-
  12  making process is a long road.  We believe the
  13  MCAC process is an important resource for the
  14  agency and for external stakeholders, but at
  15  these early stages of this effort care must be
  16  taken to create conditions for success.  We know
  17  that the talent, insight and good efforts exist
  18  on this committee to achieve these conditions.
  19  We stand ready to assist you in every way we can
  20  and thank you for your attention and
  21  consideration of our views.
  22            DR. SOX:  Thank you very much.  Before
  23  we go on to the HCFA presentation, Sharon's going
  24  to read a letter that we just received today from
  25  the ACP-ASIM on the same day that AMA commented
   1  on our document.
   2            MS. LAPPALAINEN:  The letter is
   3  addressed Dear Ms. Lappalainen, the American
   4  College of Physicians-American Society of
   5  Internal Medicine (ACP-ASIM), representing over
   6  115,000 physicians who specialize in internal
   7  medicine and medical students, wishes to offer
   8  its comments and concerns on the draft report of
   9  the subcommittee of the Medicare Coverage
  10  Advisory Committee's Executive Committee
  11  entitled, Recommendations for Evaluating
  12  Effectiveness.  ACP-ASIM is generally supportive
  13  of these recommendations, but feels it critical
  14  that the MCAC strike a healthy balance between
  15  assuring a coverage review process which is
  16  credible and defendable from a scientific
  17  viewpoint, yet not so mired in technical detail
  18  that final coverage decisions are unreasonably
  19  delayed.
  20            ACP-ASIM is very supportive of the
  21  draft report's objectives; that important
  22  clinical coverage decisions be reviewed on the
  23  basis of sound and objective clinical evidence by
  24  the MCAC's six medical specialty panels, and that
  25  there be a standardized methodology and format
   1  for panels to present their recommendations to
   2  the MCAC Executive Committee, thereby allowing
   3  the Executive Committee to make uniform,
   4  high-quality and scientifically defendable
   5  coverage recommendations to HCFA.  We also
   6  support the draft report's recommendation that
   7  the MCAC only focus on the clinical and
   8  scientific questions around the medical
   9  effectiveness of new items and services and the
  10  comparative effectiveness of new items and
  11  services relative to existing alternatives, and
  12  that the MCAC not address questions about dollar
  13  costs of new items or services.
  14            We are impressed with the amount of
  15  scientific rigor the draft report proposes for
  16  assessing the adequacy of clinical evidence
  17  related to a new item or service and calculating
  18  the magnitude of the health benefit such coverage
  19  would have on the Medicare population.  We do
  20  wish to raise some technical concerns under the
  21  draft report's section on Evaluation of
  22  Evidence.
  23            On page 3 the discussion of potential
  24  sources of bias has some noteworthy ommissions,
  25  including double-binding, perfect compliance,
   1  adequate length of follow-up, distinct treatment
   2  separation and inappropriate statistical
   3  analysis.  Imperfections in any of these would
   4  permit bias to enter into a randomized controlled
   5  clinical trial and thus make the results less
   6  valid for the population under study and thus
   7  difficult from which to generalize.
   8            We also feel the draft report's
   9  recommendation on page 4, that MCAC panels be
  10  required to describe possible sources of bias and
  11  explain why a panel decided that bias does not
  12  account for the results, should be applied in all
  13  coverage decisions, not just the limited
  14  circumstance of uncontrolled studies described on
  15  page 4.
  16            Also, on page 5 where seven categories
  17  of size of health effect are presented, there
  18  appears to be one category omitted, which we
  19  would recommend the addition of, more effective,
  20  but with disadvantages.
  21            In summary, ACP-ASIM believes it is
  22  vital that coverage decisions remain in the hands
  23  of the medical experts comprising the panels of
  24  the MCAC and that the credibility of this body
  25  will depend on striking a balance between
   1  scientific rigor and decision making which is not
   2  bogged down in process.  Decisions reached by the
   3  MCAC must be based on the best mix of objective
   4  data and professional judgment possible and lead
   5  to coverage recommendations that have a
   6  compelling weight of evidence, yet are rendered
   7  in reasonable time frames to avoid work backlogs
   8  which might undermine MCAC effectiveness and
   9  credibility.
  10            ACP-ASIM supports the MCAC coverage
  11  decision process and welcomes the opportunity to
  12  contribute to its evolution.  We believe the time
  13  spent now will pay great dividends in the future
  14  and that the MCAC's evidence-based decision-
  15  making model will soon become one of which we can
  16  all be proud.  Sincerely, it is signed by Whitney
  17  W. Addington, M.D., F.A.C.P, president.  Thank
  18  you.
  19            DR. SOX:  We'll now move on to the HCFA
  20  presentation by Dr. Kang and Dr. Hill.  Jeff, go
  21  ahead.  Well, Bob, you had something to say.
  22            DR. BROOK:  I don't quite understand
  23  the transition here, and I'd like some
  24  clarification on the process.  Up to now we've
  25  had a description of the subcommittee report and
   1  then a public session with public comment.  What
   2  is this part?
   3            DR. KANG:  This is actually the HCFA
   4  comment.
   5            DR. BROOK:  Is this the response to our
   6  subcommittee report?
   7            DR. KANG:  Yes.
   8            DR. BROOK:  I'm wondering whether the
   9  process we ought to -- I mean since we are an
  10  advisory committee to HCFA, do we want to have
  11  some discussion of the committee before we hear
  12  what HCFA thought of the report in relationship
  13  to the public report or is this a process that's
  14  prescribed by law or something that we can't do
  15  this?  I'm just wondering which way we want to do
  16  this since we're advisory to HCFA anyway.  Do you
  17  want us to put all this together when we try to
  18  deliberate or just look at the public response
  19  first?
  20            DR. KANG:  I'm actually okay either
  21  way, quite frankly, because there's many of the
  22  issues here which have been raised which I think
  23  we can resolve through discussion.  So if we want
  24  to kind of cut to the chase here, that's fine
  25  with me.
   1            DR. HILL:  In the sense that the
   2  subcommittee asked for a comment and a report to
   3  be given, when something's presented to the
   4  panel, we also would like to be able to comment
   5  about the subcommittee report at this point and
   6  hope that you would take that into consideration
   7  in your mix.
   8            DR. SOX:  Alan, do you have a
   9  suggestion?
  10            DR. GARBER:  Just speaking for myself,
  11  I would like to hear HCFA's comments before the
  12  committee deliberates so we can deal with all of
  13  the comments as a whole.
  14            DR. KANG:  I'm going to nix my
  15  presentation then.  I actually had only one
  16  comment then.  Dr. Hill has a bunch.
  17            I wanted to note that when I was a real
  18  doctor -- I guess I'm no longer a real doctor --
  19  it's been awhile since I've practiced --
  20  practicing geriatrics, I had to make very
  21  difficult choices and/or recommendations for my
  22  patients almost every minute of the day which
  23  diagnostic test to order, should I recommend
  24  hospitalization or home care, what treatment
  25  options should I suggest et cetera.  Usually this
   1  involved choices amongst well-understood,
   2  commonly utilized possibilities.
   3            Sometimes, though, something new or
   4  something new to me was as an appropriate
   5  consideration.  Usually in these situations I
   6  turned to the medical evidence and the literature
   7  to help me make a choice in this decision.  I
   8  think I did that largely in part because I wanted
   9  to be sure before abandoning the old that using
  10  the new would be better.  I think in many ways
  11  this is what we're wrestling with, and this is
  12  what national coverage decisions are about that
  13  we face frequently with new technology.  What
  14  does the evidence or science say about the new
  15  technology?
  16            In practice, though, I must admit I
  17  also recall the patient's condition and the
  18  availability of alternatives had a lot to do with
  19  how I reviewed the evidence.  If our patient was
  20  in serious trouble and there was a lack of any
  21  other beneficial alternatives, it actually made
  22  me more likely to offer the service even if the
  23  literature was suboptimal.  I think this was
  24  especially true if the risk of the service or
  25  procedure was very small.
   1            So I just ask in your deliberations
   2  today that you discuss whether or not the
   3  patient's condition, the availability of other
   4  alternatives and the risks associated with the
   5  service should affect how we actually view the
   6  evidence.
   7            That said, I applaud and thank you for
   8  your efforts to deal with this in a consistent
   9  manner for all panelists on how we read the
  10  evidence.  I believe that actually you're off to
  11  a great start, and there's many things that can
  12  be resolved today.
  13            DR. HILL:  Thank you.  I'll be as brief
  14  as I can.  First of all, I want to say on behalf
  15  of our group within HCFA that the subcommittee
  16  report is both admired and appreciated by us.
  17  Nothing that I will say should be taken as a
  18  denigration or a disparagement of this important
  19  contribution to HCFA's efforts to improve our
  20  coverage decision-making process.
  21            The report's recommendations for an
  22  optimal process, speaking from the position of
  23  the people who are going to have to carry this
  24  out, appear to be well-challenging.  It may be
  25  that at least for some decisions, we will have to
   1  commit to all the steps you outlined, but that
   2  possibility causes us as well as others to have a
   3  care for the time required.
   4            This is the most open and accountable
   5  process for making national coverage decisions in
   6  the history of Medicare.  When we designed and
   7  started this new way of doing business, including
   8  the MCAC, we knew that the period required to
   9  reach a decision would often include required
  10  minimum components and time periods because of
  11  the steps.  For example, announcing the planning
  12  of MCAC panels' open public meeting means some
  13  time is needed.  As we talk today about how to
  14  prepare for and get the best advice from MCAC
  15  panels, we're thinking again about the time
  16  required.  But let me be plain.  We were not
  17  then, and we are not now, hiding behind the
  18  process to delay coverage, to delay getting the
  19  latest evidence-proven treatments to Medicare
  20  beneficiaries, and we do not want anyone else to
  21  either.
  22            Our intentions and success in meeting
  23  those intentions are and will continue to be
  24  clear.  We announce matters under consideration
  25  for coverage decisions on the web with due
   1  dates.  If we can't meet our self-imposed
   2  deadlines, we give our reasons, again posting
   3  them publicly.  This process must not be driven
   4  back into a black box by criticism of that
   5  process, including criticism of timing.
   6            Our goal is to reach well-reasoned,
   7  scientifically sound decisions as rapidly as can
   8  be consistent with that level of quality.  We
   9  believe that this committee shares that goal with
  10  us, and we appreciate its comments on how to keep
  11  things moving.
  12            Let me refer to a couple of specifics
  13  in the subcommittee report that may raise
  14  concerns for process duration.  The suggestion
  15  that each panel explain its conclusions in
  16  writing should not in our view delay a decision
  17  until a second panel meeting months later is
  18  voting on that right.  We should be able to
  19  address this commendable desire for
  20  accountability, as consistently expressed in this
  21  suggestion, without more time than is already
  22  contemplated to write up and post the summary of
  23  that meeting.  This is something we're already
  24  going through.
  25            The suggestions regarding the structure
   1  of the evidence presented to the panel should not
   2  delay.  We are committed to presenting high-
   3  quality and well-organized information as called
   4  for in the subcommittee report and doing so
   5  within the time frames previously contemplated.
   6  We will get help doing this in a timely way when
   7  necessary, and we are already doing this for the
   8  next planned panels.
   9            I'm pleased to see Dr. Deborah Zarin
  10  from our well-respected sibling, the Agency for
  11  Health Research and Quality, with us today in the
  12  audience.  Dr. Kang and I have met on multiple
  13  occasions with AHRQ's leadership, and we look
  14  forward to their involvement as an important
  15  resource for us in examining evidence and
  16  preparing for MCAC panels.  We'll be talking
  17  about the subcommittee's time frames with them.
  18            Finally, on the time frame issues I
  19  want to respond to the subcommittee's item number
  20  6, expert review of evidence reports.  At the
  21  present time we are not planning to do this in
  22  every case.  Even if time were not an issue --
  23  and it may not be if this added step can be
  24  accomplished within current expectations -- we
  25  still regard this as a quality control feature.
   1  If we're doing a good job with the presentations
   2  to the panels and the postings on the web, if the
   3  process seems to be working without this step, we
   4  do not presently intend to make additional
   5  external review part of the routine.
   6            The other major concern we have heard
   7  about the subcommittee report -- you've heard it
   8  too -- is that it seems to set some impossibly
   9  high hurdle to bar every new technology without
  10  any regard for type.  We don't read your
  11  statement that way, but this should not be a
  12  concern regardless because we continue to explain
  13  that we are not abrogating our responsibilities.
  14  We understand that we have to make the coverage
  15  decisions.  You advise us, and we decide in part
  16  basing our decision on your advice.  So we want
  17  to know the basis of your advice, your
  18  recommendations, your thinking.  We will want to
  19  know what's behind the MCAC panel's inclusion
  20  about evidence.  We don't expect the panel to,
  21  nor can we allow the panel to, decide for us
  22  whether or not there's enough evidence to allow
  23  us to cover it.
  24            For example, when the subcommittee
  25  report says uncontrolled studies are never
   1  applicable, I read, in the context of that
   2  section, that if a clinical experiment reported
   3  in medical literature carries the possibility of
   4  bias in selection of patients, we understand the
   5  difficulties of explaining away that bias without
   6  randomization or other forms of controls.
   7            Dr. Sykes gave a good explanation of
   8  bias in his presentation to the subcommittee
   9  report.  Does the risk of unaccounted for
  10  selection bias mean that we shouldn't give the
  11  experiments' results much weight in deciding
  12  whether or not to cover the tested treatment?
  13  Possibly.  Does it mean we automatically refuse
  14  to cover?  No.
  15            As the subcommittee report suggests,
  16  observations alone may sometimes allow a panel to
  17  make conclusions about effectiveness.  Such
  18  suboptimal evidence may allow us to conclude that
  19  Medicare should cover the service.  Deadly
  20  diseases without alternatives come to my mind
  21  immediately as such a situation, also logical
  22  consistency with general medical science
  23  understanding.  The proof required to allow
  24  applicability to the Medicare population might be
  25  less where the application makes sense than when
   1  it's counterintuitive or inconsistent, hard to
   2  explain in the context of the rest of the
   3  science.
   4            I also see no credibility in the
   5  assertion that the committee is threatening to
   6  tell HCFA that one threshold fits all.  No one
   7  should take seriously the suggestion that we
   8  might require unrealistic trials such as double-
   9  blind tests of surgically implantable devices as
  10  a dodge to avoid covering something.  We said,
  11  and I say again, that the sector-specific
  12  guidance documents are purely of our
  13  quality-oriented coverage plan, and they are the
  14  next step after a coverage regulation proposal in
  15  the federal register.  We have already
  16  demonstrated, in the coverage decisions made so
  17  far under our new process, that we are aware of
  18  and can properly include the flexibility
  19  necessary for the variety of situations we face.
  20            But the questions you ask are at least
  21  potentially constant, and the important questions
  22  you've asked of this document can't be ignored.
  23  We still want to know whether studies that do not
  24  focus on patients over 65 produce results that
  25  can be applied to the Medicare population of that
   1  age group.  It's possible that the answer can be
   2  no or even unsafe over 65, and we might consider
   3  still covering, but only for our disabled and
   4  ESRD beneficiaries who are within the age range
   5  where medical benefit is shown by the evidence.
   6            So to the subcommittee we say thank you
   7  for this important contribution.  Thank you for
   8  these questions.  To industry and those who want
   9  to cover our product or service, we say let's
  10  look together at these questions.  We understand,
  11  and you know we understand, that these questions
  12  do not control HCFA's coverage decision making,
  13  but they will help inform and improve the quality
  14  of those decisions.  And to our beneficiaries and
  15  the public generally we say we will be faithful
  16  stewards of your health and the health of the
  17  future beneficiaries.  We will ask these
  18  questions.  We will continue the work begun two
  19  years ago, always listening to the medical
  20  community, providers, consumers and manufacturers
  21  and promoters, the work of improving Medicare's
  22  national coverage decision process.  Let's keep
  23  going together.
  24            DR. SOX:  Thank you.  We now go into an
  25  open committee deliberation, and what I'd like to
   1  suggest is that we start our deliberations and
   2  perhaps spend as much of the next hour as it
   3  takes to ask follow-up questions of people who
   4  made presentations to us, both from the public as
   5  well as HCFA, and then, again depending on how
   6  much time it takes us, either proceed on to
   7  starting a round table discussion of this
   8  document and what we need to do to come to a vote
   9  to recommend to HCFA.
  10            So with that brief introduction, I'd
  11  like to focus for now on trying to ask questions
  12  of the various presenters and so forth.  Bob?
  13            DR. BROOK:  Panel, can I raise a
  14  process issue of what we're trying to accomplish
  15  today?  Let me tell you what I've heard.  I
  16  didn't hear anyone except maybe HCFA have a --
  17  I'll retract that.  I didn't hear anybody sort of
  18  say the document is out of bounds.  It should be
  19  burnt and thrown away.  I've heard a lot of
  20  wordsmithing in some places, a lot of questions
  21  about tone and other questions, but no wholesale
  22  disregard for it.
  23            The question I'm asking is should we
  24  consider on this committee a bifurcated process?
  25  We need something to help the next set of panels
   1  get started with.  We could say that we've gotten
   2  there with this document as getting started, and
   3  we could ask the people that presented as well as
   4  other people to take the document we have and
   5  actually instead of doing what we did here,
   6  require them to do what we did ourselves, which
   7  is to white out, edit, alter whatever they would
   8  like in that document and provide a justification
   9  and a reason for what they're trying to
  10  accomplish by doing that and then take this so
  11  that we would actually have a written record that
  12  basically would allow us to look at this
  13  paragraph by paragraph, sentence by sentence on
  14  the belief that both the people at HCFA and the
  15  people of the subcommittee and people of the
  16  committee will disappear sooner than we can
  17  probably imagine given our mortality.
  18            And I wonder whether that kind of a
  19  process would be one that we would then have a
  20  written record of what people really would do to
  21  this document if they were all part of the
  22  subcommittee.  And then the subcommittee would
  23  then take those, produce a written record of how
  24  we responded to that and in a document that then
  25  we would do and produce as a second version and
   1  continue to involve this process over time as we
   2  get experience with it.
   3            So the thought here is go with what
   4  we've got now as advice to the committees to do
   5  the next round of the panels, get written input,
   6  continue to revise, continue to deal with this
   7  kind of a document and make it an evolutionary
   8  document with a history behind it so that we can
   9  continue the process forward.
  10            And as we get feedback, both from how
  11  it worked in the panels, and what the public
  12  believes about this feedback, we could then
  13  continue to modify this document and do it as
  14  sort of that kind of an approach as opposed to us
  15  trying to ask questions, get off-the-cuff
  16  responses, some of them well thought out, but not
  17  sort of at the level of how would you change this
  18  sentence?  When you mean tone, okay, what do you
  19  really want done here?  So getting commitment in
  20  writing to what people really want done.
  21            I'm wondering whether that would be a
  22  process that would get us further along.
  23            DR. SOX:  Let's discuss that.  It's a
  24  reasonable proposal.  Let's have some serious
  25  discussion.
   1            MS. LAPPALAINEN:  Right.  We have the
   2  document available for projection, and we are
   3  prepared to have someone make edits now.  For the
   4  entire afternoon we have set aside a large amount
   5  of time today for the committee to make those
   6  kinds of suggestions to the document.  Because
   7  the subcommittee met in essence in private, the
   8  deliberation and the review of the document needs
   9  to be in public today in order to satisfy the
  10  Federal Advisory Committee Act.  And this is why
  11  we have called the meeting today so that the
  12  entire Executive Committee could deliberate and
  13  review in open public format this document.
  14            DR. SOX:  Okay.  Well, Bob, in essence,
  15  I think, has said that we need to get rolling
  16  with the process, that the document that we've
  17  generated so far doesn't have any deadly flaws in
  18  it, but at the same time we've had some very
  19  useful comments and perspectives that might
  20  strengthen the document if they were incorporated
  21  into it.
  22            And perhaps we could simply have a
  23  two-part process, which we would decide whether
  24  or not to use the document as it is now to help
  25  the panels in their deliberations that are on the
   1  schedule right now and meanwhile give the public
   2  an opportunity for input into the document and
   3  reframe it as seems appropriate, then come back
   4  at our next meeting to present what we've come up
   5  with for further discussion and options.
   6            DR. BROOK:  That's not what I said.
   7  It's close, Hal.
   8            DR. SOX:  Thank you.
   9            DR. BROOK:  I think that we could have
  10  open deliberation today at the level of a
  11  committee about do we think this is good enough
  12  to overcome some of the major problems with the
  13  running of the next set of panels?  And we ought
  14  to confine our discussion to that for us at this
  15  moment.  But at the same process, I've heard that
  16  there are people that really want significant
  17  written changes in this document that we all may
  18  think there's no problem with, and it would
  19  improve the document.
  20            And if we had a process of saying --
  21  and I don't know the timing of this here, but you
  22  have six weeks to take this document and to write
  23  down, not just the edits, but just the reason you
  24  want it changed, the justification, what you're
  25  trying to accomplish, and then have the
   1  subcommittee look at that and then try to
   2  incorporate as much as this into a revised
   3  document and bring it back to the Executive
   4  Committee so that we get closer to what people
   5  really want and go through the step before we
   6  meet again as an Executive Committee of actually
   7  looking seriously at those changes and
   8  incorporating them, then we would have a written
   9  reason, a written justification, and then we
  10  could respond as a committee and say yes, we
  11  agree with, no, we don't, for these reasons.  And
  12  this would be a different kind of a process.
  13            DR. SOX:  So we have comments.  I was
  14  looking this way.  So Alan, why don't you take
  15  the first one.
  16            DR. GARBER:  I'll be very brief.  I
  17  just wanted to remind everyone -- and correct me
  18  if my memory is incorrect -- that at our last
  19  Executive Committee meeting we said that the
  20  subcommittee would produce a document that's
  21  really intended to be interim to provide guidance
  22  to the panels until HCFA issues its regulations.
  23  So one thing to keep in mind, none of us, I
  24  think, have the intention of producing something
  25  that's going to be permanent.  If this does
   1  happen to coincide perfectly with the rules that
   2  HCFA eventually develops, that would be great.  I
   3  don't think we have the expectation that that
   4  will necessarily happen.
   5            So this is indeed an interim document,
   6  and I don't think the idea is to make this so
   7  pristine and perfect that it never needs to be
   8  changed because we are almost bound to change
   9  this in the course of the next year, year and a
  10  half, however long it takes.
  11            The second point is that I think we
  12  said at the previous meeting that we hoped that
  13  we would more or less wrap this up at this
  14  meeting, and I think it's premature to talk about
  15  longer term changes until we've heard from the
  16  members of the Executive Committee, who did not
  17  yet have an opportunity to comment on the
  18  document, to get some sense of whether this is
  19  very close to the right ballpark and just needs
  20  some technical revisions that can be handled
  21  today or if it needs very extensive revisions.
  22            So I think we need to discuss ongoing
  23  revision only after we've heard from the
  24  Executive Committee has a whole.
  25            DR. SOX:  So Alan, let me understand
   1  you correctly.  Are you saying that we can't act
   2  on Bob's proposals until we discuss the document
   3  as it currently stands looking at it as an
   4  interim document that's going to help us get off
   5  the ground in the next 12 months or so?
   6            DR. GARBER:  Exactly.
   7            DR. SOX:  That certainly seems like a
   8  reasonable suggestion.  But why don't we see if
   9  there are any other comments.
  10            Jeff, did you have your hand up?
  11  Leslie?
  12            DR. FRANCIS:  I wanted to comment that
  13  I think that we should go actually section by
  14  section with the idea of whether or not there are
  15  things in this document, using it as a general
  16  framework, that we think are problematic even on
  17  an interim basis.  One example might be the
  18  implication in the generalizability section to
  19  the Medicare population, that the Medicare
  20  population is only the elderly.
  21            DR. KANG:  Yeah.  I would actually
  22  agree with that.  I think we need some minor
  23  tweaks here and more along the line of tone or
  24  clarification, and I don't think we're that far
  25  apart.
   1            Listening to the comments, I read this
   2  document in a completely different way than many
   3  of the commenters are reading it, and that really
   4  suggests that we have somewhat of a problem.
   5            The first is I did not read in this
   6  document that there's an implication that
   7  everyone has to have a randomized controlled
   8  trial.  What this document in my mind says is
   9  that's the gold standard, but to the extent that
  10  you deviate from the gold standard, you have to
  11  explain biases, how you dealt with it et cetera.
  12            So clearly a case controlled trial
  13  where the biases let's say against device or
  14  service or whatever, someone can say well, that's
  15  okay.  All the biases are against it.  That's a
  16  good trial.
  17            The second observation I had was the
  18  same as Dr. Francis', and this really actually
  19  dealt with, I think, the Medicare beneficiary
  20  rights testimony and a couple of other
  21  testimonies.  I think we do have to clarify that
  22  the results associated with the study population
  23  are the results associated with the study
  24  population.  Now, it so happens that the study
  25  population excluded people under the age of 65,
   1  and if you want to broaden that coverage, you
   2  actually have to deal with whether you can get
   3  there or not.
   4            As it turns out, as the doctor with
   5  multiple myeloma from Arkansas was saying, if in
   6  fact the study didn't have age exclusion but
   7  actually had another exclusionary criteria, then
   8  the age probably goes away.  You just actually
   9  write a coverage decision that had the
  10  exclusionary criteria.
  11            The whole point, though, is you look at
  12  the study population, and you agree with the
  13  results.  And then to the extent that you want to
  14  cover beyond the study population, you actually
  15  have to justify why it had reason to do that and
  16  explain why that's an okay thing to do.
  17            So I would actually see that those two
  18  minor tweaks -- and maybe they're not minor, but
  19  I think what Bob is suggesting is they still
  20  require a fair amount of wording, but I think
  21  that gets to most of the problems that have
  22  actually been identified by the presenters that
  23  there are some process problems.
  24            DR. DAVIS:  Well, I agree with a lot of
  25  the comments that have been made.  And to pull
   1  them together, what I would like to see is I
   2  agree with Leslie that a section-by-section
   3  review would be appropriate today.  We're not
   4  going to do all the things that need to be done
   5  to the document, but we can do a lot to fix
   6  this.  So I think a section-by-section review
   7  would be good, and then by the end of the day
   8  approve it with the fixes that the committee
   9  agrees to, and then approve it as work in
  10  progress, then give it to the panels as a
  11  framework to guide their work in the coming
  12  months, and then continue to come back to the
  13  document and refine it as necessary, especially
  14  considering that when panels begin to use it,
  15  that will represent a pilot test, if you will, of
  16  how appropriate and practical the document is,
  17  but again coming back to it over time refining it
  18  as necessary.  And also, I'm sure we'll want to
  19  take into consideration more detailed comments
  20  from the public and from various stakeholders.
  21            DR. SOX:  Ron, maybe you could also
  22  speak briefly to the concept Bob has advanced
  23  about getting public input to this document.  To
  24  me it's kind of an attractive idea that we would
  25  really seek broad input.  We would have to make
   1  the final call on the wording, but it would give
   2  us an opportunity to make some changes in tone,
   3  and if it seems appropriate to do so, that may be
   4  very difficult to accomplish in the short-term.
   5            What do you think of the overall
   6  strategy of getting public input?
   7            DR. DAVIS:  Well, we've obviously had
   8  some already today, we had some before we came
   9  here today, and we'll have more later on this
  10  afternoon.  So my sense is let's try and improve
  11  it today.  Maybe we can go section by section and
  12  allow people to propose improvements, and maybe
  13  those can be approved as we go along by the
  14  committee or disapproved, then hear some more
  15  public comment from 3:15 to 3:30 or whenever that
  16  happens as listed on the agenda, and then leave
  17  the final approval by the committee to the end
  18  of the day as the agenda indicates.  Then there
  19  will be more detailed commentary after we adjourn
  20  today, and we'll take that into account when we
  21  reconvene in a couple of months.
  22            DR. SOX:  Other comments about the
  23  process?  I would like to advance a notion and
  24  see how it flies with you.  I'm a little worried
  25  that we're going to get into wordsmithing over
   1  tone that's going to kind of bog us down and
   2  would like to propose that we try to focus more
   3  on technical content and less on tone during our
   4  discussion, explicitly recognizing that we're
   5  going to get a fair amount of public input
   6  hopefully in writing, I would suggest, on how we
   7  alter the tone in a useful way.
   8            My guess is that as long as this
   9  document continues to be an interim working
  10  document in the next few months, these issues of
  11  tone probably aren't central to getting on with
  12  that work.
  13            Does that feel pretty comfortable to
  14  you all that we focus on technical content and
  15  recognize we have a process for modifying the
  16  tone in response to public comment both here and
  17  that we may receive later on?  Alan?
  18            DR. GARBER:  Well, I want to make sure
  19  I understand the implications of what you're
  20  proposing.  I just know my panel, medical surgery
  21  panel, is meeting in a little more than a month,
  22  and I suspect that members of my panel won't care
  23  much about the tone of the document and will care
  24  a great deal about content.  And if by technical
  25  issues, you mean the content -- that is how are
   1  you going to evaluate the evidence and so on --
   2  that's great.  That's what we need.  And I agree
   3  the wordsmithing about tone is not going to be
   4  the number one concern of our panel.
   5            So if we could end today with the
   6  consensus about content as in what are the
   7  specific directions that the panels will receive.
   8  And let's not forget that although this is a
   9  public document, its primary purpose is to guide
  10  work for the panels.  So that's really what we
  11  should be focusing on.
  12            If we can come to some consensus today,
  13  that would be extremely helpful to us and I
  14  suspect all the other panels.
  15            DR. SOX:  Bob, did you want to
  16  comment?
  17            DR. BROOK:  From a process perspective,
  18  I believe that the question we ought to ask the
  19  committee, as a guide for the first panel
  20  meetings, is there anything you find in the
  21  document that's objectionable that would allow
  22  you not to want to give this to the panel as
  23  guidance for the first meeting?
  24            If we limit ourselves to that question,
  25  then I think we could do the task that people
   1  have talked about, going section through
   2  section.  If we do anything else, I don't think
   3  we're going to succeed.
   4            I think that, however, this is
   5  basically not a technical document, but a
   6  political document written by a technical group,
   7  and I would urge that we view it as such and
   8  therefore insist that before we finally approve
   9  the document, I think we can say to the panels
  10  use it as a guidance for the first thing, that we
  11  get absolutely specific written comments from
  12  anyone in the public who wants to give it to us
  13  with a justification for what they're trying to
  14  achieve by that comment so that we can explicitly
  15  respond in writing, do the same thing we're
  16  asking the panel to do, to explicitly respond in
  17  writing why we believe that this word ought to
  18  stay the same, this word ought to change or that
  19  we consider this other thing, and then do this as
  20  an evolutionary process.
  21            So my concern is do we have enough
  22  discipline to hold ourselves for this
  23  conversation around the table to say what's in
  24  here that really the chair should not use at the
  25  first set of panel meetings, not what you think
   1  about the tone and structure and everything, what
   2  we think this eventual document will look like?
   3            DR. SOX:  So it's partly objectionable,
   4  but it's also unclear and confusing.  I mean if
   5  you don't understand the document, you can't
   6  instruct the panel about problems.  We've got to
   7  deal with those problems as well.  Okay.  I think
   8  we're all together.  Bob?
   9            DR. MURRAY:  I'd like to comment that I
  10  think it's inevitable that this is a guidance
  11  that is titled recommendations.  It's filled with
  12  words like should, it's expected to, would
  13  normally.  It's only a guideline.  It's not a
  14  prescriptive legal statute.
  15            Secondly, it's inevitable that it's
  16  going to be treated as such because we have only
  17  a month or six weeks before the next panel
  18  meeting, and one of the provisions calls for a
  19  six-month or anticipates a six-month time line in
  20  order to get to the panel meeting.  Well, of
  21  course, you're not going to squeeze six months'
  22  work into six weeks.
  23            My feeling is that we should approve it
  24  as is or with minor modifications because it's a
  25  guideline.  It's a recommendation.
   1            DR. SOX:  I think we're all clear.  My
   2  suggestion is that we take it section by section
   3  and we take a few minutes before starting the
   4  discussion for people to go back over and if they
   5  haven't already identified concerns, to do so.
   6  I'm not sure everybody has a comment.
   7            Have most people already marked it up?
   8  Great.  In that case we can go right into it.
   9            DR. HOLOHAN:  Since we're switching our
  10  agenda a little bit, we're going to ask questions
  11  or make comments on some of the public
  12  statements, there are a couple of things I'd like
  13  to comment on before we start just to get them in
  14  the public record.  The written comments that
  15  were supplied are, I presume, in the public
  16  record, and I think a few things have to be
  17  clarified.
  18            One is HIMA has a statement that says
  19  the six months that are suggested in the document
  20  is the length of the life cycle of some
  21  technologies.  I find that very difficult to
  22  believe.  So it doesn't square with Mr. Roe's
  23  interest in people investing money into a --
  24  stent versus medical technology.
  25            Secondly, there's a HIMA statement that
   1  says technologies have improved laparoscopic
   2  cholecystectomy -- would have difficulty in
   3  clearing the evidentiary hurdle.  Laparoscopic
   4  cholecystectomy was actually decided as a
   5  coverage issue by Medicare on the basis of the
   6  request for review by the U.S. Public Health
   7  Service.  Their standard, arguably lengthy
   8  procedure, that was extant in the early 1990s,
   9  and HCFA was able to make a coverage decision in
  10  a period of four months.  So it's in the public
  11  record, but it's not entirely true.
  12            The only other comment I'd like to
  13  make, Ms. Gottlich mentioned again VA coverage.
  14  I'm perhaps oversensitized to this because it
  15  came up four times at our panel discussion on
  16  treatment of multiple myeloma.
  17            I think, as the only VA representative
  18  here, it's inappropriate to make comparisons
  19  between benefits provided by Veterans Health
  20  Administration and benefits provided by Medicare
  21  for two reasons.  The major one is that HCFA's
  22  statutory requirements and the VA's statutory
  23  requirements are considerably different.  The
  24  Veterans Administration is required by law to
  25  provide clinical care to patients to do research,
   1  to provide medical education to medical students
   2  and house officers and to act as a backup for the
   3  Department of Defense, and I think it is
   4  misleading to see VA provision of medical care as
   5  some kind of a federal imprimatur about safety
   6  and effectiveness in part because of the fact
   7  that research is part and parcel of what VA
   8  does.
   9            The second is that the VA benefits
  10  package extends far beyond medical care to things
  11  that HCFA doesn't cover, for example,
  12  modification of vehicles for patients with spinal
  13  cord injury, modification of homes, a much more
  14  expansive long-term care program.  So I think
  15  it's simple to say well, since the VA does
  16  provide high-dose chemotherapy and stem cell
  17  support for some patients with multiple myeloma,
  18  that it's ipso facto or important to VA for the
  19  safe and effective therapy, and Medicare, as
  20  another federal program, should follow suit.
  21  It's deceptively simple, but it's in fact not the
  22  case.
  23            DR. SOX:  Let's begin.  Let me suggest
  24  some ground rules that you want comments on
  25  elements of the text that seem objectionable as a
   1  basis for your panel proceeding or the text is so
   2  unclear that you feel that you can't proceed, it
   3  doesn't give you instructions you can understand.
   4            I'd like to suggest that people who
   5  have a problem with it try to identify the
   6  problem, if possible propose a solution, and the
   7  process for getting agreement is going to be
   8  mostly me looking around the room and seeing nods
   9  or asking if there's objections.  Try not to take
  10  votes unless we go into something that's real
  11  controversial.
  12            DR. DAVIS:  Hal, can I ask a process
  13  question?
  14            DR. SOX:  Go ahead.
  15            DR. DAVIS:  I think what you've just
  16  outlined is fine, but I wonder if we go through
  17  it section by section and stick to the issues
  18  that you mentioned a few moments ago, and if we
  19  have time perhaps we can go back section by
  20  section and address tone again if there's time.
  21            Would that fit in with what you're
  22  trying to do?
  23            DR. SOX:  I agree with separating the
  24  two, and if we have time, it would be reasonable
  25  to address tone.  I'm mindful of the fact that
   1  there may be a few members who are going to have
   2  to leave a little early.  So I'm hoping we can
   3  get done a little bit before it was scheduled for
   4  the end of the meeting so we have everybody here
   5  at the end.  So I qualify it I guess.
   6            MS. RICHNER:  On that note I was
   7  wondering if it's possible to do process first.
   8  I think that's a critical component of what our
   9  mandate is here.  A lot of this is so theoretical
  10  in the sense that we may get bogged down, and I'm
  11  very concerned that one of the huge issues is the
  12  evidentiary reports, and that whole section is
  13  very unclear, and I would love to be able to
  14  focus on that first.
  15            DR. SOX:  How do other people feel
  16  about that?
  17            DR. GARBER:  I guess although I think
  18  it's very important to get there, I think we
  19  should proceed in order.  I think that there are
  20  two big issues that were raised overall, if I
  21  could summarize what the commentators said in the
  22  public testimony.
  23            One of them had to do with the
  24  impression some had that -- trials would be
  25  necessary, and the other issue was timeliness.
   1  So the first is in the first part of the
   2  document, and the second is in the process part
   3  of the document.  I think we need to get through
   4  both, so that will be the responsibility of Hal
   5  to get us through this in a timely manner.
   6            DR. SOX:  Responsibility on all of us.
   7  Jeff?
   8            DR. KANG:  Mr. Chairman, if I could
   9  just add, as Dr. Hill was suggesting, the process
  10  in many ways, a lot of the timing is HCFA's
  11  responsibility, and we really have to work out
  12  the logistics et cetera.  And during the
  13  presentation this is the first time I saw the
  14  time frame, and I quite frankly think we can do
  15  much better.  So to the extent that we don't get
  16  there, I really just wanted to signal that we
  17  will very work very aggressively with the MCAC to
  18  speed up the time frames et cetera.
  19            MS. RICHNER:  Preparation of the
  20  evidentiary reports was another issue as well as
  21  the reviewers.
  22            DR. KANG:  I think we can do that
  23  faster.  A lot of that responsibility, quite
  24  frankly, falls to HCFA because it's staff
  25  preparation.  So I just want to send that message
   1  loud and clear to the extent that we get bogged
   2  down.  I actually think we should get to the
   3  content of guidance.  And we are committed to
   4  working on the process issue and getting things
   5  done faster.
   6            DR. SOX:  I think we ought to focus on
   7  issues that seem really important to the panel
   8  chairs and co-chairs.  So perhaps there won't be
   9  any comments on the preface since it's not
  10  procedural.
  11            DR. BERGTHOLD:  I would like to make a
  12  suggestion that we consider what we heard from
  13  the public today, which I thought was a very good
  14  point, and that we put explicitly up front in the
  15  preface, even though we all understand that, that
  16  this is for the Medicare beneficiaries to better
  17  serve them, so something like after the first
  18  sentence, provide advice regarding coverage so
  19  that Medicare beneficiaries can be better
  20  served.  I can't make a vote, but if someone else
  21  would carry that vote.
  22            DR. SOX:  That's a tone thing.
  23            DR. BERGTHOLD:  I don't think it's a
  24  tone thing.  I thought about that really hard.  I
  25  think it's a substantive thing that we missed.
   1            DR. SOX:  Anybody have any problem with
   2  now saying observing Medicare beneficiaries?
   3            DR. FRANCIS:  I'd like to add an
   4  invitation to the panels -- this will be on the
   5  last paragraph in the preface -- to convey back
   6  to us concerns about the document as they work
   7  with it.
   8            MS. LAPPALAINEN:  Just a matter of
   9  helping our typist, when the committee makes a
  10  suggestion to modify the document, you can then
  11  ask yourself if it's all right.  If then the
  12  committee agrees that that change is fine, if the
  13  person could then dictate slowly, and we can make
  14  that change.  We don't have to necessarily do a
  15  vote for each individual change.  We're hoping to
  16  have the document modified and that at the end of
  17  the day the entire document can be endorsed, if
  18  you will.  Thank you.
  19            DR. FRANCIS:  My suggestion might be
  20  you just add the paragraph of the interim
  21  document a work in process.  We invite panel
  22  comments about your impressions of the document
  23  and what changes they might recommend to the
  24  Executive Committee.
  25            DR. SOX:  Let's go down to the next to
   1  last paragraph.  So you want some wording that
   2  might go on to have that paragraph, the last
   3  sentence, continue to say and in response to
   4  suggestions from the panel based on experience,
   5  something like that?
   6            DR. FRANCIS:  Sure.  The Executive
   7  committee invites comments from the panels based
   8  on their experience with this interim document.
   9            DR. BROOK:  Why don't we just say we
  10  will modify these recommendations in response to
  11  panel feedback and as needed to respond to the
  12  HCFA final rule -- in response to feedback from
  13  panel members or something like that.  We will
  14  modify these recommendations as reflected by
  15  input from the panelists and as needed in
  16  response from the panel members.
  17            DR. FRANCIS:  Alan, are you clear that
  18  that's an open invitation to your panel to give
  19  us feedback on how it will work?
  20            DR. GARBER:  Yes.
  21            DR. SOX:  Okay.  Any other changes to
  22  the preface?  No objections?  Okay.
  23            Let's go on to Evaluation of Evidence.
  24  I'd like to suggest we basically go through it
  25  paragraph by paragraph so we're not jumping
   1  around, and it will make it easier for the person
   2  who's trying to make the changes in the permanent
   3  record.
   4            Any problems with the first paragraph?
   5  The second paragraph?
   6            DR. DAVIS:  We're talking about
   7  substantive process, right?
   8            DR. SOX:  We're talking about
   9  objectionable for the basis of panel action or
  10  unclear.
  11            DR. DAVIS:  Fine.
  12            DR. SOX:  So first paragraph?  Second
  13  paragraph?  What about the statement in boldface
  14  about the adequacy of the evidence, does that
  15  tell you what you need to know?
  16            DR. MURRAY:  This is one of the few
  17  places where the word must appears, and perhaps
  18  this is tone, but in the prior paragraph the word
  19  should is used.
  20            Would this be inconsistent to change
  21  must to should or must to is expected to?  I'm
  22  trying to address some of the concerns heard in
  23  the comments that this is overly prescriptive.
  24            DR. SOX:  Anybody have any problem with
  25  substituting should for must?  Go ahead, Alan.
   1            DR. GARBER:  Well, I think this is the
   2  sine qua non of what panels do.  Details are
   3  shoulds, but I can't see how a panel will
   4  discharge its duty if it does not determine
   5  whether the scientific evidence is adequate.  So
   6  this is one place where I feel the word must is
   7  used advisably.
   8            DR. MURRAY:  We must use must?  I
   9  really don't have any objection to that.
  10            DR. SOX:  Any problem with using must
  11  here?  Other comments on adequacy of the
  12  evidence?  John?
  13            DR. FERGUSON:  Just a comment, and that
  14  is that it was my understanding that HCFA
  15  wouldn't send anything to the MCAC panels unless
  16  they had some pretty good indication that there
  17  was enough evidence.  Now, that doesn't abrogate
  18  the panel's responsibility for judging it, but I
  19  think HCFA has said in their previous generation
  20  that they would not send things to the panel
  21  unless there was some clear evidence base.
  22            DR. SOX:  Do you have a wording change
  23  suggestion?
  24            DR. FERGUSON:  I would say probably in
  25  the paragraph before, the quality of the evidence
   1  from these sources will vary, and the panels
   2  should weigh the evidence according to its
   3  quality, a portion of that weighing has been done
   4  by HCFA prior to sending the request to the
   5  panels or something like that.
   6            DR. BROOK:  Can we stay away from
   7  that?  We don't know how HCFA will want to use
   8  this process in the future.  Why don't we just
   9  write a document on what the panel should do, and
  10  HCFA can determine what it will do.
  11            DR. KANG:  I think that's correct.  You
  12  can't presume what will happen here.
  13            DR. SOX:  That process isn't written
  14  down.
  15            DR. KANG:  Quite frankly, I think that
  16  the, quote, slam dunks, we'll just deal with
  17  administratively.  And the reality is that on
  18  your broad shoulders we'll be getting the plain
  19  ones that are somewhat controversial, so I think
  20  that we have to be very careful there.  I would
  21  just encourage you to just go ahead and do what
  22  you think is right.
  23            DR. SOX:  Anybody here who doesn't find
  24  Alan and Jeff's point compelling?
  25            Other comments on the boldfaced
   1  adequacy of evidence?  Any specific wording
   2  changes?  I don't hear them.
   3            So let's move on to the first paragraph
   4  under comment.  I'm just going to expect you to
   5  holler.
   6            Let's go on to the second paragraph,
   7  the one that says many forms of evidence.
   8            Third paragraph, when several such
   9  well-designed trials, any changes to this?
  10            How about the next one, the Executive
  11  Committee believes?  Jeff?
  12            DR. KANG:  I hate to say that this is a
  13  tone also, but we say here in considering the
  14  evidence from any study, whether they're
  15  randomized clinical controlled trials or any
  16  other trials or whatever, you could say the MCAC
  17  now should try to answer these two main
  18  questions.
  19            DR. DAVIS:  Where are you?
  20            DR. GARBER:  It's the last paragraph
  21  before bias.  You want to insert whether
  22  randomized controlled clinical trial or
  23  observational study?
  24            DR. KANG:  Or other controlled trials.
  25            DR. GARBER:  Or other controlled study?
   1            DR. KANG:  Yeah.
   2            DR. SOX:  So it's really any controlled
   3  study.  It wouldn't apply to a noncontrolled
   4  study.
   5            DR. KANG:  Right.  Any controlled study
   6  including randomized controlled trials because
   7  you do want to deal with bias, and even in an RTC
   8  it's possible.
   9            DR. SOX:  So the suggested wording is
  10  that after any, we would put any controlled
  11  study, including randomized controlled trials.
  12            MS. RICHNER:  What about the issue of
  13  registries again?  I think that limits this.
  14            DR. SOX:  We speak later on to the
  15  issue of registries without any form of control.
  16            DR. GARBER:  Well, there are some
  17  changes we might want to make later on, but I
  18  think we have to make it clear that registries
  19  can be controlled, and they can be uncontrolled,
  20  and I have some suggested wording later.
  21            MS. RICHNER:  But this wouldn't then
  22  negate evaluation of that type of evidence later
  23  on?
  24            DR. GARBER:  Right.
  25            DR. SOX:  If there was a control, then
   1  it would fall into this.
   2            MS. RICHNER:  Okay.  I see what you're
   3  saying.
   4            DR. BROOK:  Jeff, just to be clear,
   5  you've made this more limiting than it was
   6  before.  The purpose by inserting all that
   7  nonsense, the purpose of this sentence, was
   8  basically to say this is not a rigid
   9  restriction.  This is a general.  And now by
  10  stating controlled trials in it, you've made it
  11  much more rigid.  Study is very vague.
  12            DR. KANG:  I agreed with that point,
  13  but I was surprised by the comments that we were
  14  getting.
  15            DR. SOX:  Actually I think there's a
  16  logical reason for sticking it in there because
  17  the bias to controlled group and intervention
  18  group doesn't apply to a noncontrolled study.  So
  19  in other words, the remark about bias isn't an
  20  issue unless you're comparing groups.  So I think
  21  it makes much more sense.
  22            DR. MAVES:  Hal, that may be true, but
  23  I again like the way it was worded beforehand
  24  because it was more open, and it was broader and
  25  less sort of proscriptive.  Unless Jeff has a
   1  good reason for putting it in there.
   2            DR. BROOK:  What about this?  In
   3  considering the evidence from any study, whether
   4  randomized or not, the MCAC should try to answer
   5  these two main questions.  There can be bias in a
   6  randomized trial study.  So why don't we say
   7  considering the evidence from any study, whether
   8  randomized or not.
   9            DR. KANG:  That's fine.
  10            MS. RICHNER:  Thank you.  That's
  11  better.
  12            DR. SOX:  Is that compromise agreeable
  13  with everybody?  Okay.  Any other comments on
  14  that paragraph?
  15            How about the next paragraph, the one
  16  that defines effectively bias?  Then we have a
  17  real long paragraph coming up, many opportunities
  18  for finding fault here.  Anybody want to make
  19  suggestions about how to change this next
  20  paragraph on potential sources of bias?
  21            DR. HOLOHAN:  The investigators cannot
  22  be sure that they have measured all of the ways
  23  in which treated patients differ from untreated,
  24  do you really want to put in the word measure?
  25            DR. SOX:  Can you tell us where that
   1  is, please.
   2            DR. HOLOHAN:  The fourth line down.
   3  It's talking about observational studies.  The
   4  investigators can't be sure that they have
   5  measured all the ways --
   6            DR. BROOK:  Are you saying measure to
   7  assess?
   8            DR. HOLOHAN:  Measured implies a
   9  quantitative evaluation which may not be possible
  10  in many instances.
  11            DR. MAVES:  How about considered?
  12            DR. SOX:  Alan?
  13            DR. GARBER:  The operational issue here
  14  is has it been recorded in some way that it can
  15  be incorporated into a study design?  And to
  16  observe is not sufficient.  To consider is not
  17  sufficient.  It has to be recorded.  Measure does
  18  not necessarily mean quantified in continuous
  19  terms.  It can mean it's a binary variable.
  20  Doesn't necessarily mean quantitative.  Measured
  21  means observed and recorded.
  22            DR. HOLOHAN:  Why don't we just say
  23  observed and recorded.
  24            DR. GARBER:  Well, fine.  I wouldn't
  25  have any objection to that.
   1            DR. BROOK:  That sounds fine.  Observed
   2  and recorded.
   3            DR. SOX:  Great.  Other comments on
   4  this paragraph?
   5            Now we turn to the one paragraph that
   6  starts random allocation of patients.  Any
   7  objections to this paragraph for lack of clarity?
   8            Then let's go on to the next paragraph,
   9  in an observational, nonrandomized study.
  10  Remember now we've got to focus on issues that
  11  are objectionable for the basis of panel action
  12  or unclear.  Ron?
  13            DR. DAVIS:  I guess some of these
  14  comments could address interpretation by panels,
  15  so maybe I'll offer this comment which could be
  16  tone, could be interpretation.
  17            At the very end where we say clinical
  18  trials of treatments for cancers that have an
  19  unpredictable natural history, for example, have
  20  repeatedly demonstrated that the results of
  21  observational studies are misleading, I wonder if
  22  we should say are often misleading.
  23            DR. SOX:  Yeah.  They aren't always.
  24  Fair?
  25            DR. BROOK:  It's not that they're
   1  misleading.  They're overly optimistic of the
   2  value of the therapy.
   3            DR. SOX:  How about frequently
   4  overestimate the size of the treatment effect?
   5            DR. BROOK:  That would be better.
   6            DR. SOX:  The results of observational
   7  studies frequently overestimate the size of the
   8  treatment effect, and delete often misleading,
   9  and go back to the --
  10            DR. BROOK:  Remove repeatedly at the
  11  first part of that sentence.
  12            DR. SOX:  One more wordsmithing change
  13  in that sentence, repeatedly on the left hand
  14  side, delete that.  Okay.  Good.
  15            Next paragraph, to detect important
  16  bias.  This one has a lot of operational
  17  implications.  Does it really do it for you?
  18  Okay.
  19            Next paragraph, although a body of
  20  evidence.
  21            DR. HOLOHAN:  Can I suggest that the
  22  phrase is never adequate be clarified a little
  23  bit?  And I think what was meant by the
  24  subcommittee was that it would never reach to the
  25  reliability of a probably done randomized
   1  controlled trial, but not that it is ipso facto
   2  inadequate.
   3            DR. SOX:  Alan, do you want to respond?
   4            DR. GARBER:  Well, I realize this is
   5  not a flash point, and I think we should be --
   6  the issue here that is I believe perhaps a
   7  semantic one -- I'm not certain -- and that is
   8  what do we mean by uncontrolled?  And from
   9  hearing the comments today, I think that some of
  10  the people may have been under the impression
  11  that what was meant by uncontrolled is not
  12  randomized controlled, and that's not the case.
  13            And I actually got some suggested
  14  rewording, and I don't know if this will do it.
  15  And Tom, I particularly appreciate your opinion
  16  about this.  That is the first sentence of the
  17  paragraph would begin although they do not have
  18  randomized controls, all well-designed
  19  observational studies include some form of
  20  control.  They may consist of an implicit or
  21  explicit controlled group or statistical
  22  controls, that body of evidence consisting only
  23  of uncontrolled studies.  And I think that's
  24  intended to make it clear that registries are
  25  probably assigned observational analyses,
   1  probably assigned controls, and the issue truly
   2  uncontrolled study, I think it's strictly true.
   3  If it is uncontrolled, it is not valid evidence
   4  by itself, yet there are plenty of studies that
   5  could have valid controls that are not
   6  randomized, and I would hate for the readers of
   7  this document to think that this paragraphs means
   8  you have to have randomized controlled trials.
   9            In fact, I was struck that some of the
  10  public comments seem to suggest that this
  11  document meant only randomized controls would be
  12  suitable.  We put a great deal of effort on the
  13  part of the subcommittee to try to make it clear
  14  that observational data would often be -- well,
  15  at least would sometimes be adequate, and it
  16  really depends on the characteristics of the
  17  studies that were being done.
  18            MS. RICHNER:  I still think that's
  19  missing the mark in a sense because I think why
  20  this is so controversial in a sense is that once
  21  again when you're looking at the technology curve
  22  when you have very little evidence in the very
  23  beginning of adoption, it's rare that you're
  24  going to have the kind of rigorous studies that
  25  you're interested in.  So I think what this does
   1  is we want to make sure that you're looking at
   2  the composite of all possible data that's
   3  available.  And this doesn't allow that.
   4  Essentially looking at perhaps unpublished data
   5  that might be available that would be
   6  interesting, case studies, et cetera, et cetera,
   7  and somehow this tone of this paragraph limits
   8  all of that.
   9            DR. SOX:  We've got to have something
  10  to vote on and some wording to vote on.
  11            MS. RICHNER:  And unfortunately I had
  12  wording that I sent to you that I thought was
  13  appropriate on e-mail that would have addressed
  14  that as well.  Unfortunately my computer has now
  15  just died.
  16            DR. BROOK:  Can I suggest some
  17  wording?  I want to suggest an alternative
  18  wording before we vote.
  19            DR. SOX:  I'm thinking that maybe what
  20  we need to do is to get -- this is a really an
  21  important issue, and that perhaps an approach
  22  would be that we delay the vote on this.  We can
  23  move on without this.  Each of you submit your
  24  wording that we get it up there and we actually
  25  wordsmith out.
   1            DR. BROOK:  Can I suggest an approach
   2  to this background before we do that?  I would
   3  like to suggest that we're limiting everything up
   4  to in some cases, and we start by saying in most
   5  cases given the current state of scientific
   6  evidence, panels will determine that well-
   7  collected observational evidence -- and then I
   8  think we ought to list in there what we mean by
   9  that -- will be sufficient to draw conclusions
  10  about effectiveness, and I think that that's the
  11  tone you want in this paragraph.
  12            MS. RICHNER:  Yes, that's much better.
  13            DR. BROOK:  Because with a large part
  14  of the technologies, that's what's going to
  15  happen.  So that's how I would alter that
  16  paragraph.  And I would then spell out in detail
  17  what we think are well-controlled observational
  18  kinds of studies, registries with historical
  19  controls, quasi experimental designs, et cetera,
  20  et cetera.  And I think I'd even add the point
  21  that Jeff came up with.  This would be especially
  22  true when we have breakthrough technologies and
  23  technologies dealing with people with severe
  24  diseases with no other recourse.
  25            DR. KANG:  That's good.
   1            DR. BROOK:  I think that's what the
   2  panels are going to do, and I think we might want
   3  to say it.
   4            DR. KANG:  May I make a suggestion
   5  since we're almost at lunch?  I don't think we're
   6  that far apart.  It actually strikes me that
   7  maybe Bob, Alan and Randel sit down at lunch and
   8  hack it out.  I hate to infringe on your lunch
   9  period.
  10            DR. SOX:  I think that's actually a
  11  very good suggestion.  We'll appoint a committee
  12  of three, and if any member of that committee is
  13  not satisfied with what you come up with, then
  14  that person will submit an alternative, and we
  15  can vote on it.  Does that sound reasonable?
  16  We've got about five minutes to 12:00.  Should we
  17  give ourselves a break at this point?  And we'll
  18  come back at 1:00 and continue the process.
  19            (Whereupon, recess taken -- 11:55 a.m.)
  20            (Whereupon, after recess -- 1:10 p.m.)
  21            DR. SOX:  Alan, do you have a report
  22  of the work group of the subcommittee?
  23            DR. GARBER:  We weren't able to locate
  24  one of the members of our subcommittee.  Randel
  25  and I went over some language that I think we
   1  agree on.  So if I could read that to the
   2  committee and the audience.
   3            DR. SOX:  Should we perhaps have it --
   4            DR. GARBER:  Let me read it once first
   5  because there's a lot of changes.  Okay.  This
   6  refers to the bottom of that page.  It's right
   7  above the subheading external validity, the last
   8  paragraph, and it currently starts although a
   9  body of evidence.
  10            The new language is as follows.
  11  Although if they do not have randomized controls,
  12  all well-designed observational studies include
  13  some form of control.  Controls may consist of an
  14  implicit or explicit controlled group or
  15  statistical controls.  A body of evidence
  16  consisting solely of studies with no controls
  17  whatsoever, whether based on anecdotal evidence,
  18  testimonies or case series, is never adequate.
  19  And then the last sentence reads, now that
  20  there's a change in the last part, when these
  21  circumstances apply, the panel must describe
  22  possible sources of bias and explain the basis
  23  for its decision that bias does not account for
  24  the results.
  25            Randel, does that reflect what we
   1  said?
   2            MS. RICHNER:  Yeah.  The key issue here
   3  is that any of the case series studies or
   4  composite of any of those sort of testimonials,
   5  anecdotal studies combined, can never constitute
   6  the proper evidence if it's only those types of
   7  studies.
   8            DR. GARBER:  Only studies without
   9  controls.
  10            MS. RICHNER:  Right.  Without some type
  11  of control.  So even in an observational study,
  12  you can use a statistical methodology in which to
  13  observe or have a control as part of that.  And
  14  that works.  What do you think, Bob?
  15            DR. BROOK:  My fault.  I didn't go to
  16  lunch, so I couldn't find you guys.  So my
  17  fault.
  18            DR. FERGUSON:  Can that be written down
  19  and circulated?
  20            DR. GARBER:  I just wanted to get it
  21  done in general first.
  22            DR. BROOK:  In general terms I don't
  23  believe a document ought to ever use the word
  24  never.
  25            MS. RICHNER:  Then never is a problem.
   1  I still don't like the never.
   2            DR. BROOK:  There is not a single
   3  testimonial that couldn't be put into historical
   4  context by some historian.  Whether you choose to
   5  do it or not makes it adequate or inadequate, but
   6  there is no case series that could not be put in
   7  some historical context no matter how bad.  And
   8  the panels are going to be left to judge how much
   9  effort and how good these controlled efforts have
  10  been.  That's why I would have simplified this
  11  just to say -- I mean that's their job in terms
  12  of what's going on.  That's okay.  It's my fault,
  13  as I said, for not being there.
  14            DR. SOX:  Okay.  Alan, do you want to
  15  read that one more time?  Then we can have
  16  discussion of it and maybe start to get it on the
  17  document as well.
  18            DR. GARBER:  Should I read this line up
  19  to it?  Insert at the beginning of the paragraph
  20  the following.
  21            DR. BERGTHOLD:  No.  She's just going
  22  to type it separately for now.
  23            DR. GARBER:  Oh, okay.  Fine.  Although
  24  they do not have randomized controls, all well-
  25  designed observational studies include some form
   1  of control.  Controls may consist of an implicit
   2  or explicit controlled group or statistical
   3  controls.  And then the next up is -- do you want
   4  to just retype the remainder of the paragraph?
   5            THE TYPIST:  Would that be here at the
   6  end?
   7            DR. GARBER:  It goes to the although.
   8  It's now the next sentence.  The word although is
   9  struck and then a body of evidence.  So you
  10  struck that.  The body of evidence consisting
  11  solely, and then strike only, and then strike
  12  uncontrolled.  And then after studies insert with
  13  no controls whatsoever.  And then after case
  14  series strike and disease registries without
  15  adequate historical controls.  Then it stays the
  16  same is never adequate.  And then insert however
  17  before in.  This is something I didn't mention
  18  that we changed also.  Strike some and replace it
  19  with many.  In many cases.  Then it goes to the
  20  last part of the paragraph.  Strike why it
  21  decided and insert the basis for its decision.
  22            MS. RICHNER:  Bob, you certainly still
  23  have a chance to comment.
  24            DR. SOX:  Well, it's time for comments
  25  or questions.  Actually I have a question.
   1  Statistical controls, could you explain what that
   2  means?
   3            DR. GARBER:  In other words, it's an
   4  observational study where they can collect data
   5  on a number of variables and basically look at
   6  patterns of outcomes, how they're explained by
   7  things like say age et cetera.  That can be a
   8  form of statistical control.
   9            DR. SOX:  Is that multivariant analysis
  10  essentially?
  11            DR. GARBER:  Yes.
  12            DR. KANG:  This is different or the
  13  same?  You do multivariant plus sensitivity
  14  analysis?
  15            MS. RICHNER:  I actually have some
  16  literature that is very recent from the
  17  pharmaceutical industry of which they do this
  18  type of methodology.  And once again, I can't
  19  articulate it well, but there are methods to do
  20  this in using observational data that is well-
  21  grounded.  I mean McMasters has done a lot of
  22  work at that.
  23            DR. KANG:  Could you take another
  24  attempt at trying to explain to me?
  25            DR. GARBER:  Let me tell you about some
   1  of the work we've done using Medicare claims
   2  files.  Let's say that you want to have an idea
   3  of whether revascularization in post MI improves
   4  outcomes.  You can take Medicare claims files
   5  which have extensive information about discharged
   6  diagnoses, age, location and a number of other
   7  individual characteristics, and there are various
   8  statistical methods you can use to determine
   9  whether the people who have treated with
  10  revascularization did better.  So you'll have Bob
  11  Brook saying that's all very hokey, but that's
  12  what statistical controls are, and the panels
  13  have to decide whether this type of evidence is
  14  adequate or not.
  15            DR. HOLOHAN:  It's retrospective.
  16            DR. GARBER:  Well, it's actually
  17  historical prospective.  The point is we're not
  18  going to determine right now whether any
  19  particular study in science is adequate.  The
  20  point is that there are methods, and there are
  21  cases where you can use that kind of a controlled
  22  group -- that is implicit statistical control --
  23  to draw conclusions.  The panels may decide yes,
  24  this is convincing or they may decide it's not on
  25  a case-by-case basis.
   1            DR. SOX:  Any other questions or
   2  comments about this?  Ron?
   3            DR. DAVIS:  Well, I like it.  I just
   4  wanted to suggest one other small change at the
   5  end.  Instead of saying that bias does not
   6  account for the results, to say that bias is
   7  unlikely to account for the results.  I think the
   8  panel would more likely say we don't think bias
   9  accounts for the results.  I don't think they'd
  10  say bias does not account for the results.
  11            DR. SOX:  Does that sound reasonable to
  12  you guys?
  13            MS. RICHNER:  We had that discussion as
  14  well.  Are you comfortable with that?
  15            DR. GARBER:  Yeah, I think that's
  16  fine.
  17            DR. SOX:  Any other comments?  So it
  18  goes.  We now go on to external validity, first
  19  paragraph.
  20            DR. FRANCIS:  There's a replacement
  21  effort.
  22            DR. KANG:  If you don't mind, Dr.
  23  Francis and I, in going through it ourselves as a
  24  group of two, took another crack at this.  So
  25  this is under external validity.  And maybe we'll
   1  read it.
   2            DR. SOX:  Is this suggested as a
   3  substitute for the paragraph?
   4            DR. FRANCIS:  Yeah.  For the first
   5  paragraph.
   6            MS. LAPPALAINEN:  I'll read it out
   7  loud.  Issues of external validity related to the
   8  study of population.  Medicare beneficiaries
   9  include elderly, nonelderly, and disabled
  10  people.  The Medicare population also may or may
  11  not include patients with comorbid disease.  That
  12  said, historically many controlled trials
  13  unfortunately excluded older men and women,
  14  people with disabilities and people with comorbid
  15  disease.  This means that even when a trial has
  16  adequate statistical power for the study
  17  population, that its results may or may not be
  18  generalizable to some portions or all of the
  19  Medicare population.  If the requester is asking
  20  for, or the panel is advising, coverage beyond
  21  the clinical and demographic characteristics of
  22  the study population, the panel should state that
  23  they believe the results of the trials are
  24  applicable to a broader population, define what
  25  that population is and explain its reasoning
   1  why.
   2            DR. SOX:  So Leslie and Jeff, perhaps
   3  you could explain what lead you to make this
   4  change so we all understand what's behind it.
   5            DR. FRANCIS:  One thing that was behind
   6  it was the recognition that Medicare population
   7  is not just the elderly.  And at least the way
   8  the myeloma panel was set up, the question that
   9  was posed to the panel was we've got a lot of
  10  data in there under 65s.  Can we extrapolate from
  11  65s and over?  And we wanted to take away any
  12  implication that that's the way stuff should be
  13  set up rather than focus on the question of what
  14  were the inclusion and exclusion criteria in
  15  studies and what that says about what are all
  16  portions of the management population coverage
  17  recommendations we are aiming for.  So that's
  18  what we're trying, however inartfully, to
  19  capture.
  20            DR. KANG:  Part of the problem with the
  21  tone of this paragraph is it assumes that all
  22  Medicare coverage decisions are for the general
  23  population.  We are now -- practically all of our
  24  coverage decisions are limited in some way, have
  25  exclusion criteria or inclusion criteria, and a
   1  lot of times we do it for the study population.
   2  That is something, quite frankly, that's been
   3  new.
   4            So I really think the issue here is is
   5  it a statistically valid study population -- then
   6  a request is for that study population.  And we
   7  should cover for that study population.  And if
   8  it so happens we only have three beneficiaries,
   9  that's okay.  It's still covered for those three
  10  beneficiaries.  That's more or less what we were
  11  trying to get to.
  12            DR. GARBER:  Well, Jeff, I guess you
  13  correctly guess that my concern is the last part
  14  of this.
  15            DR. KANG:  That's correct.
  16            DR. GARBER:  And the problem is
  17  probably semantic, but as I read this revision,
  18  it could be applicable to a broader population,
  19  but it doesn't necessarily mean it could pass
  20  that criterion and still not necessarily be
  21  applicable to any defined population of Medicare
  22  beneficiaries.  So the original language -- I
  23  mean I completely agree with the intent of this
  24  and with the rest of it, but the original
  25  language, just to remind people, is if the study
   1  population in the available trials is not the
   2  same as the general population of Medicare
   3  beneficiaries who would be candidates to receive
   4  the intervention, the panel must state whether
   5  the results of the trials apply to typical
   6  Medicare patients and explain its reasoning.
   7            And that language was really saying
   8  does this generalize to the relevant population
   9  of beneficiaries?  And I'm not sure the language
  10  that you proposed at the end actually gets at
  11  that.  So I would propose something like an
  12  amendment to the original language for the last
  13  part, and instead of saying typical Medicare
  14  patients, maybe two defined populations of
  15  Medicare beneficiaries so you cover ESRD,
  16  disabled et cetera.
  17            DR. BROOK:  Can I suggest changing
  18  broader population to the results of the trial
  19  applicable to any group of patients covered by
  20  Medicare?  So that would then allow you total
  21  flexibility since we're writing this for
  22  Medicare.
  23            MS. RICHNER:  Results in the study too
  24  rather than trials.
  25            DR. BROOK:  The results of the trials
   1  are applicable to any population covered by
   2  Medicare or can be applied to any population
   3  covered by Medicare.  Define what the Medicare
   4  population is and explain its reasonings why or
   5  what part of the Medicare population it applies
   6  to and explain its reasonings why.
   7            DR. KANG:  I'm not sure that gets it.
   8  I'm okay with it.
   9            DR. GARBER:  I like my wording better,
  10  which is defined populations of Medicare
  11  beneficiaries so you can say this is effective
  12  for ESRD beneficiaries, and this is effective for
  13  elderly Medicare beneficiaries, and this is for
  14  the disabled.  But the point is that the panel
  15  should explicitly say which population of
  16  beneficiaries if any they believe the results of
  17  these trials apply to.
  18            DR. SOX:  Alan, are you proposing we go
  19  back to the wording of that last sentence?
  20            DR. KANG:  Alan, I'm not sure I
  21  understand that because we actually -- our
  22  coverage decisions are now running like this is
  23  effective for ESRD patients who don't have heart
  24  failure or whatever it is.
  25            DR. GARBER:  That's what we're saying,
   1  that the panel should say what the trials apply
   2  to, some population like that.  Now, you could
   3  tell us look, we'll decide.  We don't want the
   4  panels to get in the business of determining
   5  whether the trials apply to populations of
   6  beneficiaries.  I think you'd be better off using
   7  panels to try and evaluate the evidence and see
   8  whether they think they can extrapolate from the
   9  trials to some population of interest to
  10  Medicare.
  11            DR. FRANCIS:  Why don't we just change
  12  the last sentence to say to populations or to
  13  groups covered by Medicare, define what those
  14  groups are, and explain the reason why.
  15            DR. GARBER:  Could you say the exact
  16  words?
  17            DR. FRANCIS:  Believe the results of
  18  the trials are applicable to some groups covered
  19  by Medicare, define what those groups are and
  20  explain its reasons why.
  21            DR. BROOK:  Define it in clinical terms
  22  if you want to.
  23            DR. GARBER:  No.  I think that's fine.
  24            DR. BERGTHOLD:  Does that allow
  25  Medicare to make, sort of, fine, sort of,
   1  distinctions within those populations though?
   2  Because that almost sounds like if you're an ESRD
   3  person, you get this treatment even if you do
   4  have heart failure or whatever.  No?  That
   5  doesn't mean that?
   6            DR. BROOK:  No.
   7            DR. KANG:  No.
   8            MS. RICHNER:  The other question I
   9  would have here about define it in terms of just
  10  trials, wouldn't you want to make it a little
  11  broader in terms of studies?  Because the whole
  12  part before was describing we're going to be
  13  looking at lots of different kinds of evidence,
  14  so therefore we don't want to limit ourselves to
  15  trials here.
  16            DR. KANG:  I was concerned this study
  17  has to be statistically -- so you could say --
  18            MS. RICHNER:  Well, yes, but that's
  19  covered in the part before.
  20            DR. KANG:  Okay.
  21            DR. HILL:  I don't think you meant,
  22  Leslie, to say that if the requester is asking,
  23  the panel should state.  That first phrase is in
  24  the alternative.  You only mean if they agree.
  25            DR. FRANCIS:  Right.
   1            DR. HILL:  So you state whether or not
   2  they believe.
   3            DR. FRANCIS:  Whether they believe.
   4            DR. HILL:  This way it's grammatically,
   5  if the requester asks, that they are being
   6  requested by you to state that they believe.
   7            DR. FRANCIS:  No.  They should state
   8  whether they believe.
   9            DR. FERGUSON:  I have a question.  Is
  10  it true that the sentence that says the study
  11  population results may or may not be
  12  generalized -- wait a minute.  If the requester
  13  is asking for, or the panel is advising,
  14  coverage, is HCFA comfortable with our panel's
  15  advising coverage?  Are coverage questions going
  16  to be asked specifically?
  17            DR. HILL:  We've answered that as we've
  18  gone along and repeatedly said that we understand
  19  we have the responsibility for deciding coverage.
  20  So I take that to mean if you want to clean that
  21  language up, I'd be grateful, but I don't want to
  22  slow you down.
  23            DR. FERGUSON:  Safe and effective or
  24  some other words.
  25            DR. KANG:  See, this is tough because
   1  by our federal register notice we are asking the
   2  requester to specify the population that they're
   3  seeking coverage for.  We get that with varying
   4  degrees of success.
   5            Maybe one of the ways we do that is to
   6  clean that up and really demand, before it gets
   7  to the panel, that they are very clear about what
   8  population they're looking for.  Then the panel's
   9  decision is whether or not the evidence supports
  10  that.
  11            The only thing that we get into
  12  somewhat of a problem is if it doesn't support
  13  it, then there's the question of well, what would
  14  it support?
  15            DR. FERGUSON:  But advising coverage
  16  and advising that the evidence supports coverage
  17  might be --
  18            DR. HILL:  May I suggest if the
  19  requester is asking for coverage or the panel
  20  concludes that medical benefit can be --
  21            DR. SOX:  I'd like to suggest -- I
  22  think we know what we're going to say here.
  23  Rather than try to wordsmith this thing in
  24  detail, I'd like to suggest that we take it down
  25  and somebody work on some language that doesn't
   1  have us recommending coverage, but still allows
   2  the requester to request coverage.  I think we
   3  know what we want to say.
   4            DR. KANG:  I think, John, advising
   5  support for will be okay.  Let's just get it over
   6  with.
   7            MS. RICHNER:  And the other part about
   8  trials versus studies.
   9            DR. KANG:  We took care of that.
  10            DR. BERGTHOLD:  It doesn't apply
  11  above.
  12            MS. RICHNER:  That sentence,
  13  historically many controlled trials
  14  unfortunately --
  15            DR. GARBER:  Yeah.  But that's true.
  16  It's much more common trials and observational
  17  studies to --
  18            MS. RICHNER:  Okay.  I see what you're
  19  saying.
  20            DR. KANG:  That's correct.  That's the
  21  ages within our society.
  22            DR. SOX:  I'd like to turn it over to a
  23  wordsmith to clean it up a little bit and make
  24  sure we're happy with the wording.  Who would
  25  like to volunteer to be the wordsmith?  Ron?
   1            DR. KANG:  I want to make sure you're
   2  okay with it.  I don't think this violates your
   3  original intent.
   4            DR. GARBER:  I think it's probably
   5  fine.  It's certainly not worth struggling over.
   6            DR. SOX:  Okay.  Let's move on.  We'll
   7  give this to Ron, he'll work on it, and we'll
   8  move on to issues of external validity also apply
   9  to the intervention.  Any objections or
  10  clarifications required here?
  11            MS. RICHNER:  This paragraph we also
  12  discussed at lunch briefly.  One of the issues
  13  here -- and I don't know if this example is the
  14  appropriate example in here.  I mean I guess we
  15  can go ahead and use it, but I'm concerned about
  16  the interpretation of this.  Certainly, once
  17  again, the technology, this skill of the surgeon
  18  over time improves, and the outcomes associated
  19  with time improve as well.  But once again, this
  20  is an example of external validity.
  21            DR. SOX:  Gets over the concept I think.
  22            MS. RICHNER:  Yeah, I think we're
  23  okay.
  24            DR. SOX:  Any other questions about
  25  this one?
   1            DR. SMITH:  I guess now that we have
   2  somewhat talked about the elderly and nonelderly
   3  and disabled, I guess my concern is I read where
   4  you have like demographics.  Have we lost or does
   5  that encompass let's say racial and ethnic
   6  inclusions or should there be, can there be, some
   7  consideration given to that particular area?
   8            DR. SOX:  Are you talking about --
   9            DR. KANG:  She's talking about the
  10  previous.
  11            DR. SOX:  -- the previous paragraph?
  12            DR. SMITH:  The previous one.  I mean
  13  it seems as if it's getting lost.
  14            DR. KANG:  Yeah.  I think the reason
  15  why -- and I'm not sure I'm aware of a trial with
  16  racial exclusion, but I could be completely wrong
  17  on this.  But I would not have any problems, I
  18  don't think, adding racial inclusion to the
  19  extent that it occurs.
  20            DR. SMITH:  I thought about it.  It may
  21  even be something that could be stated in the
  22  preface rather than just in one specific area,
  23  and then that automatically would speak to it
  24  with some consistency throughout the document.
  25            DR. KANG:  Actually this would be the
   1  place to deal with it I think.
   2            DR. SOX:  We need specific wording
   3  suggestions.  Daisy, do you want to take a look
   4  at this paragraph after Ron gets done with it and
   5  suggest some language?  Not all of us completely
   6  understand.
   7            DR. SMITH:  So when you have concerns,
   8  you just keep quiet, right?
   9            DR. SOX:  No.  We need something to
  10  look at so we know whether we like it or not.
  11            DR. HOLOHAN:  Just as an editorial
  12  comment, the best example I can think of recently
  13  of a trial that was dramatically racially
  14  imbalanced are the studies of -- and hepatitis C
  15  patients.  The patients tested do not represent
  16  the population of patients with hepatitic C in
  17  the United States today.
  18            DR. KANG:  So then probably we should
  19  add it along, and that would be the easiest way
  20  to deal with it.
  21            DR. GARBER:  Just to make maybe a
  22  substantive point because there will be a lot of
  23  interested parties here, we don't intend to imply
  24  that every study has to have adequate sample
  25  sizes of various ethnic groups and so on to draw
   1  conclusions.  Just the panel needs to decide
   2  whether they think the results of the studies
   3  apply to those populations.  We don't want to
   4  send a message gee, you're going to have to have
   5  an adequate number of Hispanics, adequate number
   6  of Asian Americans and so on.  That would be
   7  impossible.
   8            MS. RICHNER:  As a matter of fact,
   9  there's one more point I wanted to make about
  10  this, and that's foreign data.  I don't know how
  11  you're going to address that, but certainly there
  12  are many studies that are done outside the U.S.
  13  And how does that apply to Medicare populations?
  14  And in turn, we run across this all the time.
  15  The FDA now accepts foreign data.  So that is
  16  going to be an issue associated with this as
  17  well.
  18            DR. KANG:  By this language we're not
  19  excluding foreign.  This language says if it's
  20  foreign, then say that I believe this is
  21  generalizable to the American population for
  22  these reasons.
  23            MS. RICHNER:  As long as we're talking
  24  about methodology and study design, et cetera,
  25  and evidence.
   1            DR. HOLOHAN:  The issue is can the
   2  panels extrapolate?
   3            DR. FRANCIS:  One of the things that
   4  was very striking about the myeloma discussions
   5  was that although the incidence of the disease is
   6  much higher in African-Americans, the actual
   7  apparent access to the therapy in the testimony
   8  of the patients, who were all white, there were
   9  obvious issues of access that underlay the whole
  10  discussion, and I wonder whether there's a way to
  11  go back to the preface and put in something about
  12  equity and the importance of equity in the
  13  coverage process.
  14            DR. SOX:  Is that something that we
  15  could deal with after today and still operate
  16  as --
  17            DR. KANG:  We can.
  18            DR. SOX:  I want to move on now to Size
  19  of Health Effect.  Any problems with the way that
  20  is stated?
  21            DR. FRANCIS:  I have a clarification
  22  and a question.  The clarification is I want to
  23  be sure that category 2, more effective --
  24            DR. SOX:  You're getting ahead of us.
  25  We're going paragraph by paragraph.  First, just
   1  the stuff that's in boldface.  Any problems with
   2  that?  John?
   3            DR. FERGUSON:  Must we have must
   4  instead of should?
   5            DR. GARBER:  Yeah.  Because I think
   6  we're saying there's going to be a standardized
   7  way of reporting.  Each panel reports the
   8  evidence into these same set of seven categories.
   9  And if there's any reason these seven categories
  10  aren't right, we should probably change the
  11  categories now rather than saying should.
  12            MS. RICHNER:  Well, there was a
  13  suggestion by the audience for an additional
  14  category that was from one of the letters.  Not
  15  only that, I remember in our conference call that
  16  we had David Eddy suggested that there were
  17  perhaps 15 different categories.  So I think we
  18  do have to think carefully.
  19            DR. FERGUSON:  I withdraw my comment
  20  because I think what you're saying is the
  21  comparison is the must, and that's clear.
  22            DR. SOX:  Okay.  So we've dealt with
  23  the stuff in boldface.  Now let's go on to the
  24  first part of the comment, just that first couple
  25  of sentences.  Then we'll go through the seven
   1  categories.  No problems?  Then let's go to the
   2  seven categories.
   3            I'd like to suggest that modifying
   4  these may be the sort of thing that we do after
   5  we have a chance to use them a little bit, and we
   6  may find that these categories need to be
   7  expanded in order to deal with circumstances that
   8  will come up only when we actually do a study and
   9  try to classify its effect size and find we
  10  really can't do it properly.  It may work better
  11  than trying to wordsmith these categories or at
  12  least change significantly the categories right
  13  now.  John?
  14            DR. FERGUSON:  Just a comment.  And I'm
  15  sort of asking this.  One of the advantages might
  16  be cost, something would cost less.  And maybe we
  17  shouldn't put that in there, but it's certainly
  18  something that I would hope we sometimes are
  19  presented with as an advantage.  Is that a no
  20  no?  Can we list that as an example?
  21            DR. SOX:  Basically it's a no no.
  22            DR. KANG:  For the time being.
  23            DR. FRANCIS:  Can I just ask you about
  24  category 2?  Does that include small benefits for
  25  lots of people as well as relatively significant
   1  benefits for small numbers, but we don't know how
   2  to sort those out into identifiable subsets?
   3            DR. SOX:  Alan, do you want to respond?
   4            DR. GARBER:  The question comes down to
   5  whether they are prospectively identifiable
   6  categories of people who get substantial benefit.
   7  If they are identifiable, I would have
   8  interpreted this to mean they go in category 1
   9  and category 2 for the other groups.  And if they
  10  aren't identifiable, it's irrelevant.  There's
  11  always some people who will benefit, but you
  12  don't have any way to sort them.  You just have
  13  to go with the average benefit.
  14            So the question is can you identify a
  15  category with greater benefit?  Obviously if you
  16  give an intervention that's slightly better, what
  17  that usually means is that there's some people
  18  like you're measuring mortality, more people
  19  live, but you don't know for sure who's who.
  20  That's what subgroup analysis --
  21            So the other just quick comment, the
  22  ACP-ASIM talked about more objective, but some
  23  disadvantages.  I think that we discussed that in
  24  the conference call, and that would have gone
  25  into category 2.  So what they're talking about
   1  is subdividing category 2.  And the subcommittee
   2  was trying to get the smallest number of
   3  categories that we thought would do a good job of
   4  classifying people.  So it's up to the Executive
   5  Committee whether you think that should be
   6  expanded or not.
   7            DR. SOX:  I think we also want to get a
   8  sense from HCFA about whether those categories
   9  are likely to be beneficial to them in trying to
  10  make coverage decisions.  That's certainly the
  11  principle purpose of this system of categories.
  12            DR. KANG:  I actually think it would
  13  be helpful, yeah.  I mean obviously this is the
  14  place, quite frankly, where our final coverage
  15  criteria will interact, but at this point I think
  16  the better strategy is to go for more categories,
  17  whatever we can think of, and then to the extent
  18  that we're collapsing categories in the future --
  19            DR. SOX:  Debbie Zarin made the
  20  suggestion we've really got a three by three
  21  matrix for everything except for the breakthrough
  22  technologies, which would basically include every
  23  possible combination of effective on the three-
  24  point scale and advantages, no advantages or
  25  disadvantages.  So maybe we should simply use
   1  that and then collapse those categories if you
   2  find they're not useful.  Alan?
   3            DR. GARBER:  I guess my experience
   4  regarding the technologies per Blue Cross Blue
   5  Shield is that the vast majority of technologies
   6  have some advantages and some disadvantages, and
   7  I think that we would be telling the angels how
   8  to repent if we tried to decide whether or not
   9  they were more or less advantageous.  I mean some
  10  of these technologies have fewer side effects for
  11  the initial treatment, shorter duration of
  12  benefit.  Some have greater convenience, but less
  13  effectiveness.  And sometimes they trade off one
  14  side effect for another.  So I like our original
  15  classification because I thought this
  16  classification keeps us from spending too much
  17  time pondering the imponderable.
  18            DR. KANG:  I'm going to withdraw.  I've
  19  run into the same problems and gotten paralyzed
  20  from inaction.  So I like this just fine.
  21            DR. SOX:  We could in our explanation
  22  say why we put it in a particular category and
  23  actually list any factors that led us to do that,
  24  and that might be more valuable to you than the
  25  category itself for making a judgment.
   1            DR. KANG:  I think that's correct.
   2            DR. SOX:  Randel?
   3            MS. RICHNER:  I wanted to ask the
   4  overall panel if anyone has any concerns about
   5  how to identify what the established service and
   6  medical item is that you're going to be comparing
   7  the technology to or the item to.  Is that going
   8  to be an issue?  That's a question I have for
   9  everyone.  We've talked about that at length in
  10  the subcommittee about what an established
  11  medical service or item is and how do you
  12  determine what that is.  Is that going to be an
  13  issue?
  14            DR. HOLOHAN:  Can you be more explicit
  15  in what you mean by how do you determine --
  16            MS. RICHNER:  What's usual care, what's
  17  usual practice.  How are you going to decide that
  18  this technology -- what are you comparing it to
  19  for benchmarking this?
  20            DR. HOLOHAN:  You mean the term
  21  established services?
  22            MS. RICHNER:  Right.
  23            DR. SOX:  Originally we had it already
  24  covered, and we thought that would be too
  25  limiting.
   1            MS. RICHNER:  It is.
   2            DR. KANG:  Having thought about this
   3  problem a lot, I would actually suggest we're not
   4  going to be able to resolve this one today.  I
   5  think that we ought to wrestle with this as we go
   6  on and refine this one.  This really is a tough
   7  question.
   8            MS. RICHNER:  It's a tough question,
   9  but I think that the tumor assay issue sort of
  10  stems from all of that in terms of what is the
  11  comparison and what is the benchmark?
  12            DR. SOX:  I wonder whether it will vary
  13  from instance to instance.  And part of this
  14  series of things that you do during that first
  15  month when you're trying to get the chart set up
  16  is to decide what the comparison technology is
  17  going to be.
  18            DR. KANG:  This is actually why Dr.
  19  Hill referred to sector-specific guidance
  20  documents.  The reality is this is best addressed
  21  by the panels almost because this is going to
  22  vary from the sector that your talking about.
  23  Maybe we could indicate that the panel can at
  24  least in their context think about what the
  25  comparisons ought to be.  But this at this level
   1  is not a solvable problem.
   2            DR. SOX:  Or the panel chair in
   3  collaboration with HCFA staff is setting up the
   4  charts.  So I think Jeff has withdrawn his
   5  proposal that we expand the number of categories,
   6  and we can probably take that matrix down for
   7  now.  So we're still at 7, and we're going to
   8  stay with established.
   9            Are there any other comments about the
  10  categories before we move on?
  11            Hearing none, we'll move on to
  12  Suggestions for Panel Operations.  The first one
  13  is Explanation, A panel must explain its
  14  conclusions in writing.  I think the basic reason
  15  for this, the rationale is pretty clear, probably
  16  not likely to cause much push back, but maybe the
  17  implementation is an issue.
  18            DR. FERGUSON:  A comment, and I'm not
  19  sure how the wording needs to be changed, but the
  20  panel's conclusions will still be established by
  21  voting; is that correct?
  22            DR. SOX:  That's correct.
  23            DR. FERGUSON:  Those who oppose a
  24  motion are supposed to say why.  Those who vote
  25  yes, they presumably don't have to do that; is
   1  that correct, don't have to say why they're
   2  voting yes?
   3            MS. LAPPALAINEN:  Right.  The
   4  individual panel chair has the discretion at each
   5  panel meeting to go round robin after the vote is
   6  taken.  Generally a no will invoke a question of
   7  why you said no in order to make sure that any
   8  minority response gets to the record.  And the
   9  other is, of course, the majority of the vote.
  10  But this does not preclude the members from
  11  expressing their opinion or even a dissension in
  12  writing.
  13            DR. FERGUSON:  Okay.  So then the panel
  14  chair is responsible for summarizing the thought
  15  that went into the yes or no votes I guess.
  16            And again, it's a common question, how
  17  to handle it.  Maybe in case the panel chair, who
  18  does not vote unless there's a tie, would be
  19  responsible for writing this conclusion, and I
  20  might disagree with the conclusion, which has
  21  already happened once --
  22            DR. SOX:  It's the panel chair's
  23  responsibility to write the conclusion that
  24  reflects the majority regardless of his or her
  25  own preference.
   1            DR. KANG:  Or, John, you could delegate
   2  to a majority.  I mean it's really at your
   3  discretion.
   4            DR. SOX:  I would hope that panel chair
   5  is capable of writing a strong piece on something
   6  they disagree with.  That's part of the job.
   7            DR. DAVIS:  I wanted to propose a
   8  change on this last sentence which picks up on
   9  this issue we're discussing.  I wanted to suggest
  10  that we change it as follows.  The panel chair is
  11  responsible for drafting the explanation of the
  12  panel's conclusions, which should be circulated
  13  to panel members for their comments and/or
  14  approval.  I just don't think it should be solely
  15  in the hands of the chair without the opportunity
  16  of the panel members to see it.
  17            DR. SOX:  Sharon, did you want to say
  18  something?
  19            MS. LAPPALAINEN:  I just wanted to
  20  clarify something.  A summary of what happened at
  21  the panel meeting is required by the Federal
  22  Advisory Committee Act.  That summary is
  23  certified to by the executive secretary and the
  24  panel chair.  That is a legal requirement that we
  25  will continue to do, and this is in addition to
   1  that.
   2            DR. SOX:  Alan?
   3            DR. GARBER:  Well, I think Ron's
   4  suggestion kind of comes down to what this report
   5  of the conclusions is supposed to be in, and I
   6  guess in the course of our subcommittee's
   7  deliberations I had in mind saying it's going to
   8  be much more rapid, something like a one-page
   9  document that is approved at the time of the
  10  meeting.
  11            I think we have to be very sensitive to
  12  the ways that we might unintentionally create in
  13  this process, and I thought we should be brief
  14  and very rapid in summarizing the results of the
  15  meeting so that the panel can in real time
  16  approve the chairman's summary of the conclusions
  17  and the reasoning for the conclusions.
  18            I think in most cases this is only a
  19  summary.  It does not have to be an exhaustive
  20  review of what happened at the meeting because
  21  after all, transcripts will be available and the
  22  other materials that Sharon was talking about.
  23  So I had in mind something like a one-page report
  24  that is done at the meeting and wrapped up.
  25            MS. RICHNER:  But that's not clear.
   1            DR. GARBER:  I agree.  So I guess Ron
   2  has a much clearer way of stating the one model,
   3  which is a longer process, but my intent had been
   4  we do something in real time.
   5            DR. SOX:  I like Ron's approach better
   6  because I think it's very difficult to write a
   7  one-pager that is really good on the fly.  Maybe
   8  you can, Alan, but most of us can't.
   9            And the alternative would be to require
  10  the panel chair to write it, get it out for
  11  comment, and if you don't hear from somebody in
  12  48 hours, then you would assume to send and have
  13  a requirement basically that it be back in HCFA's
  14  hand in a week.  That would give a little bit
  15  more time to advise carefully and would give an
  16  opportunity for thoughtful review of what's been
  17  written.  And I would think of it not so much as
  18  approval, but comment.  And ultimately it's the
  19  responsibility of the chair to, in a just and
  20  fair way, take into account comments.  So that's,
  21  I guess, more an attempt to telescope it out in
  22  the interest of clarity.
  23            DR. GARBER:  Can I make a proposal that
  24  we approach with discretion and collect some
  25  experience?  Because it sounds like we're
   1  planning to adopt different approaches.
   2            DR. SOX:  But I think we ought to have
   3  a sense of the group.  Something ought to be back
   4  in HCFA's hands in ten days.
   5            MS. RICHNER:  It went from 48 hours to
   6  seven days to ten days.  That's too long.
   7            DR. SOX:  Does a week seem reasonable
   8  to get this done?
   9            DR. GARBER:  My concern is that there
  10  are discrepancies in the comments.  There's no
  11  problem if the only differences are points of
  12  clarification where there's no disagreement.  But
  13  as we've seen in some of these issues, there can
  14  be considerable disagreement.  And if you as the
  15  panel have to adjudicate between two members that
  16  say directly contradictory things, it's very hard
  17  to resolve that without having a conference call
  18  or face-to-face meeting.  And I assume things
  19  really have to be public.
  20            MS. LAPPALAINEN:  Presumably that would
  21  fall under an operational aspect because we had
  22  the public meeting, and the public transcripts
  23  are available.  The putting together of this
  24  document would be operational, so we could have
  25  another meeting to talk about the route.
   1            DR. KANG:  I actually have to agree
   2  with Alan.  While I'm sensitive to actually
   3  Daisy's concerns, we do want to try and make sure
   4  that the process does not slow down.  I think
   5  forcing a summary at the end actually forces
   6  people to agree on what they can agree on and
   7  disagree on what they can disagree on and
   8  actually get it up there.  The transcripts are
   9  available to HCFA and its staff, and the whole
  10  richness of the discussion is available.  And
  11  quite frankly, we would factor that in and look
  12  at that also and look at the summaries together.
  13  So I think forcing the summary before you go home
  14  is the way to go.
  15            DR. SOX:  Any other comments?
  16            DR. BROOK:  I can tell you what's going
  17  to happen here.  People will reach agreement and
  18  have very different reasons why they got there.
  19  And the chair will only figure out what he
  20  thought he heard, and it will not be what each of
  21  the individual panel members voted yes or the
  22  majority opinion agreed.  So we are stuck with
  23  either the panel chair trying to summarize the
  24  evidence saying they voted already and to
  25  summarize the reason for it or to ask each member
   1  of the panel before they go home to write a
   2  one-pager in support of their position, which
   3  then would be the summary.
   4            Instead of having this long transcript,
   5  you could have a situation where the panel chair
   6  is not responsible for summary, but each person
   7  who votes is responsible for defending their vote
   8  yes or no, and therefore, that would be part of
   9  the evidence that goes with the vote.  And nobody
  10  would try to reconcile that this person believed
  11  this because he liked that study, and this person
  12  believed this because that person wore a green
  13  tie, and this person believed this because they
  14  were tuned out and daydreaming.
  15            I mean it would motivate each panelist
  16  to pay a little bit more attention -- I think
  17  everyone would anyway -- but to pay a little more
  18  attention to the process if they knew at the end
  19  of it they would have to justify their vote.
  20            So I would change this to say that not
  21  only would this thing be voted on, but each
  22  panelist is responsible for explaining in writing
  23  at the panel's conclusion their individual vote.
  24            DR. BERGTHOLD:  There are seven
  25  questions sometimes or ten to answer, Bob, and
   1  that means that's a lot of stuff to write.
   2            MS. RICHNER:  I'm thinking of the FDA
   3  advisory panel process.  I mean it's been awhile
   4  since I've been there, but you decide that day,
   5  and you give your vote, and you say your
   6  explanation as to why you gave your vote, and
   7  it's on the transcripts, everybody knows it, you
   8  can use that data later on, and you don't leave
   9  that room until that's finished.
  10            Sharon, correct me, but that's what I
  11  remember.
  12            MS. LAPPALAINEN:  You're right.  The
  13  FDA process is as follows.  Any primary reviewers
  14  that are assigned to review prior to the panel
  15  meeting, those written recommendations are part
  16  of the administrative file for the particular
  17  matter in front of the committee.  At the
  18  advisory committee transcripts are taken,
  19  summaries are written and certified to.
  20  Panelists will often think about what happened at
  21  the panel, and subsequent to the panel meeting,
  22  will send to FDA in writing, if they feel
  23  compelled to do so, or if they feel that they had
  24  a minority opinion that was not properly brought
  25  forward.  Those things that are in writing are
   1  also part of the administrative record of what
   2  happened at the panel.
   3            DR. SOX:  Our goals here, I think, are
   4  twofold, mostly to serve HCFA's needs, and
   5  secondly, to turn out a product that you can
   6  understand and reads well.  And it seems to me
   7  that going around the room and explaining your
   8  vote really deals with Bob's issue, puts that on
   9  the record for HCFA to look at and say whoa,
  10  actually this person has a point, we'll do it
  11  this way instead of that way.  So I think it
  12  deals pretty well with that issue.
  13            I'm still, frankly, troubled, Jeff,
  14  with whether you're going to get the really good
  15  prose that you want to put on the Internet from
  16  trying to do it at the end of a long afternoon,
  17  but we'll try it and see how it goes the best.
  18            DR. KANG:  Sharon, I'm not familiar
  19  with the FDA process.  On the FDA panels do they
  20  actually try to do what Alan is suggesting with
  21  the one page?
  22            MS. LAPPALAINEN:  Well, if I can have a
  23  long response, the FDA asks particular questions
  24  regarding particular matters that come in front
  25  of the committee, and the panelists generally go
   1  round robin on those questions during the open
   2  committee deliberation.  However, the vote for
   3  either premarket approval in the device world or
   4  new drug application in the drug world or
   5  licensing application in the biologics world is
   6  actually approved.  And the panel has three
   7  choices, to approve, to approve upon a condition
   8  or to not approve.  And so the ultimate vote is
   9  really only on that issue and not the individual
  10  questions.
  11            DR. SOX:  Bob?
  12            DR. MURRAY:  I'm a bit concerned about
  13  trying to do it too quickly or in too frank a
  14  fashion.  Several points.
  15            Number one, if the purpose is to form a
  16  body of case law, then it has to be reasoned and
  17  organized, and I think doing it on a very short
  18  deadline before you leave in the afternoon would
  19  not serve that purpose.
  20            Secondly, I don't think it would serve
  21  the purpose of giving a concise, logical document
  22  to be used by other committees, by other panels
  23  or by the same panel subsequently, if instead of
  24  a single document, you had 10 or 15 separate
  25  opinions each scribbled hastily.
   1            And thirdly, if I were assigned to
   2  write the summary, I would like to look at the
   3  transcript because I would not want my summary,
   4  the words I use in my summary, to come back to
   5  haunt me if at a later meeting somebody had the
   6  transcript and were able to argue that I did not
   7  accurately summarize the expressions or the
   8  reasons for the vote.
   9            So I think that for the purposes that
  10  we're intending this summary to serve, we're
  11  simply going to have to live with a longer time
  12  line, that it will have to be a week or more than
  13  a week, at least until the transcript is
  14  available, so that we can have the document that
  15  will meet the needs that we've set forth.
  16            DR. HILL:  For our purposes, we're
  17  going to have to take some responsibility in the
  18  questions that we ask the panel because what we
  19  need more than why you voted like you did is what
  20  your scientific reasoning is.  So the points at
  21  which there's consensus of the panel and the
  22  recommendations, that's going to be most helpful
  23  to us, not so much the details of the dissent,
  24  but the whys.  And I think we can get at that
  25  with the questions.
   1            So Sharon's two-step process, we can go
   2  ahead and fulfill our obligations under the
   3  federal law with a summary.  And do you want to
   4  set a time frame today for how long you expect
   5  the panel to turn it around?
   6            And one last question, if I may.  May I
   7  take it that the Executive Committee is telling
   8  the panels that if the chairman of the panel
   9  disagrees as an individual with the findings of
  10  his or her panel, that they are tasked with
  11  writing or cooperating in the writing of the
  12  summary in the most favorable possible way
  13  against their own call, but in keeping with their
  14  panel's decision, rather than delegating?
  15            DR. SOX:  Well, that was my opinion,
  16  but others may disagree.  I just think we're
  17  professionals, and we ought to be able to do
  18  that.
  19            Jeff, can you give us a signal?  Your
  20  voice is saying, and your face is saying, you're
  21  not sure whether a week or the same day is really
  22  going to serve us well.
  23            DR. KANG:  I'm not sure I understand
  24  the recommendation that's on the table or on the
  25  floor or whatever.  I guess what I'm hearing is a
   1  summary that discharges our responsibility under
   2  FACA, but then a formal, kind of, more thought-
   3  out, well-reasoned document following it?
   4            DR. SOX:  Maybe we can do two things.
   5            DR. HILL:  I'm suggesting that in most
   6  cases I think we're going to be able to go ahead
   7  and use the committee's recommendations on the
   8  basis of the preliminary thing, and if people
   9  want to get their statement on the record for the
  10  record, to further the record later on, I don't
  11  think we're going to have to wait.
  12            DR. HOLOHAN:  I think you've just
  13  confused me.  You can make your decision on the
  14  basis of want while waiting for a more formal
  15  explanation, which makes it seem like the
  16  explanation is ipso facto redundant.
  17            DR. HILL:  No, sir.  I'm sorry.  I
  18  didn't mean to say that.  Thank you for pointing
  19  that out.  I mean to suggest that we can begin
  20  the process of working with the results of the
  21  panel's findings, getting it into a form that we
  22  can use.  We don't sit down the next day and say
  23  okay, that was it, here's the decision, and issue
  24  it.  We've got to go through some more work with
  25  it ourselves.  So if you take ten days, it's not
   1  going to slow us down.  We're going to begin our
   2  work right away.
   3            DR. BROOK:  Can I just make a
   4  suggestion here?  We have three things on the
   5  table.  Maybe we're just going too far.  There's
   6  to be a vote at the end of this.  There's this
   7  transcript.  Sharon said that HCFA has to write a
   8  summary of it.  Maybe we just ought to leave it
   9  at that and allow panelists the opportunity to
  10  submit within a couple of days any justification
  11  for their vote, if they so choose.  And then we
  12  get away from the chair having to summarize
  13  opinion without voting and doing all this kind of
  14  stuff.  But basically that they will have a vote
  15  on the issue.
  16            They're already going to have a summary
  17  of the transcript that HCFA has to prepare and
  18  which presumably is going to be done technically
  19  competently.  That will leave us with only the
  20  option that a panelist could offer, if they would
  21  like to explain their vote in writing, they could
  22  do it or not do it.
  23            DR. SOX:  The problem with leaving it
  24  as an option is that --
  25            DR. BROOK:  Then you come back to the
   1  answer that you require each -- I don't see how
   2  you can avoid requiring each panelist within a
   3  reasonable time period -- doesn't affect the
   4  vote -- to add anything else they want to add to
   5  the record.  That's what you're asking them to do.
   6            DR. SOX:  I think giving each panel
   7  member the opportunity or the obligation to say
   8  why they voted is going to help HCFA to --
   9            DR. BROOK:  So the panelists either
  10  orally or in writing will be given the
  11  opportunity, both orally or in writing, to
  12  indicate why they voted on a particular issue.
  13  And that discharges their responsibility.  And
  14  the panel chair's responsibility is to arrive at
  15  a vote on this subject, not to write the summary.
  16  And it's HCFA's responsibility, going over the
  17  transcript under whatever this law is, to
  18  basically write the summary.  And then we don't
  19  have a lot of redundancy.
  20            And I don't think any chair, believe it
  21  or not, is going to spend the next two days after
  22  getting the transcript reading the -- it takes
  23  two days to read it, right -- to read through the
  24  transcript to summarize it while HCFA is doing
  25  the same thing.  That doesn't seem to make a lot
   1  of sense.
   2            DR. SOX:  Alan?
   3            DR. GARBER:  Well, I want a stab at
   4  this.  I think a lot of this discussion is based
   5  on some unstated assumptions maybe I don't
   6  share.  I think unlike the two panels that met
   7  already, the way the future recommendations in
   8  this report are implemented, it will be a highly
   9  structured evidence review.  The issues the panel
  10  will have to deal with will be very sharply
  11  focused.  The staff has done its job in preparing
  12  these reports.  And it will boil down to a
  13  limited number of issues that the panel will have
  14  to make decisions about.
  15            And frankly, I don't think it's that
  16  difficult to write a brief summary in real time
  17  that talks about those issues.  It does not mean
  18  that you redo the work of HCFA staff as part of
  19  the report.  And I have the sense that people are
  20  talking about a very extensive redredging of the
  21  information and the arguments and so on, and I
  22  would suspect that will almost never be necessary
  23  if a good evidence report structured on the
  24  guidelines that this document states is
  25  available.
   1            I think this is actually pretty
   2  simple.  We're talking about what might amount to
   3  a handful of bullet points, to summarize it.  And
   4  I think a longer report, given all the other
   5  materials will be issued, is not going to be
   6  particularly useful.
   7            DR. SOX:  Maybe what we should do is to
   8  require a brief summary and then leave it up to
   9  the chair, if he or she wishes, to write
  10  something that would be somewhat longer, that
  11  would be literate, logical and so forth, and then
  12  just see what happens, what feels right once he
  13  or she has some experience with that.
  14            DR. HOLOHAN:  One of the purposes of
  15  this is to get uniformity and consistency, and it
  16  sounds like we're now drifting away from that
  17  again.
  18            DR. SOX:  But on the other hand, we're
  19  in a mode of trying to learn by doing.  And if we
  20  have an understanding that we're going to reach
  21  some final decision on this in a year, then we
  22  can have our cake and eat it too.
  23            DR. MAVES:  I actually support Bob's
  24  opinion on this.  And I think if you do want to
  25  write a summary, if the chair wants to do it, I
   1  think it would be fine as long as it was
   2  contemporaneously done, as Alan has indicated.
   3  I'd be very concerned about a report written a
   4  day or two after.  You sort of go home on the
   5  airplane, you think about this, you do the
   6  inevitable Monday-morning quarterbacking, and the
   7  report that Sharon writes and the report that the
   8  chair writes may have a little different spin or
   9  a little different angle.  Not much.  But that
  10  could be very, very important as time goes on.
  11            So I agree with Bob.  I think we've got
  12  two or three sort of summaries.  You have a
  13  transcript.  You have a HCFA-put-together summary
  14  of the meeting.  You have the testimony of the
  15  individual panel members as they give their votes
  16  on each one of these things.  I think that record
  17  should stand as is, and I think to do otherwise,
  18  except for perhaps a contemporaneously written
  19  document by the chair that's there, that we can
  20  see, that we can look at just like this, I would
  21  be very, very concerned about both panel members
  22  and the chair writing something after the fact
  23  that would potentially cause us more problems
  24  than fix them.
  25            DR. SOX:  It's pretty clear we're not
   1  going to reach a consensus on this, so I think we
   2  should have a motion and have a vote and move
   3  on.  Anybody want to make a motion so we can get
   4  off this one?  Mike, please.
   5            DR. MAVES:  I would move that the
   6  deliberative process that we use consists of the
   7  transcript, which is already being done by HCFA,
   8  the summary, which will be prepared by HCFA
   9  staff, the oral comments of the panel members as
  10  they testify, and that those three pieces of
  11  evidence suffice as the work product of the
  12  panelists.
  13            DR. JOHNSON:  Second.
  14            DR. SOX:  Any discussion of that motion?
  15            DR. KANG:  I have a modification.
  16            DR. SOX:  Please.  A friendly amendment?
  17            DR. KANG:  A friendly amendment.  I
  18  think what we want here is a summary, we want the
  19  transcript, and then we want the opportunity for
  20  dissent or whatever, which could always occur
  21  later.
  22            The summary could be done either way.
  23  I would suggest it could either be a HCFA-done
  24  with, as I understand, FACA, with agreement with
  25  the chair, or they can go ahead and do it right
   1  there and leave it up to the panel to figure it
   2  out.  But the end result is the summary, either
   3  HCFA-done, with approval of the chair, or Alan,
   4  kind of contemporaneously with whoever is doing
   5  it right there at the panel, the transcript, and
   6  then finally an opportunity for written further
   7  dissent, comments or whatever.
   8            DR. DAVIS:  And a vote tally.
   9            DR. KANG:  And a vote tally.
  10            DR. SOX:  Michael, is that acceptable?
  11            DR. MAVES:  I'll accept that.
  12            DR. SOX:  Okay.  Do we second it?  Any
  13  other comments?
  14            DR. FERGUSON:  Wait a second.  So let
  15  me understand this.  So this summary then,
  16  instead of being written by the chair, will be
  17  either written by HCFA and/or with the chair's
  18  input?
  19            DR. KANG:  The way FACA runs is by HCFA
  20  with approval of the chair.  So essentially the
  21  chair is delegated to represent the whole
  22  committee, or in fact, given the tone and
  23  everything, they can just go ahead and write it
  24  right there.
  25            DR. FERGUSON:  Done at the end of the
   1  meeting so that it's seen by all those present at
   2  the meeting; is that correct?
   3            DR. KANG:  Right.  Either one.  Up to
   4  the chair.  Either way would be acceptable.
   5            DR. SOX:  It wouldn't have to be done
   6  at the meeting.
   7            DR. KANG:  Right.
   8            DR. FERGUSON:  So that this third thing
   9  on our proposal here is sort of nixed at this
  10  point?  3.  Explanation:  A panel must explain
  11  its conclusions in writing.  We're now doing
  12  this --
  13            DR. SOX:  We've now operationalized
  14  that.  We probably should add this to the
  15  document, add Mike's motion to the end of this
  16  just to make it operational.
  17            DR. FERGUSON:  The transcript is done
  18  anyway.  That's a given.
  19            DR. KANG:  Right.
  20            DR. SOX:  Ready to vote?  Bob?
  21            DR. MURRAY:  I would just like to make
  22  a comment.  I'm inclined to vote against the
  23  motion.  The reason is that when we had our
  24  executive meeting in December, I believe we got
  25  all of the documents that were discussed.  We had
   1  the HCFA summary of what had happened at the
   2  previous two panel meetings, and we had
   3  voluminous information.  But in all of that, what
   4  I found most valuable was Tom's summary of his
   5  view.  And what I hear happening is that we would
   6  not ordinarily get that unless somebody like Tom
   7  chose to do that.
   8            I would rather see a reasoned summary
   9  done after the fact because that would make our
  10  job as an Executive Committee much easier and I
  11  think more effective.
  12            DR. SOX:  Other comments?
  13            DR. BROOK:  There are two ways of
  14  writing a summary.  You've now recalled my
  15  memory.  I believe the HCFA summary was day one
  16  began with this.  These people testified.  That's
  17  not a summary.
  18            When Sharon said that HCFA is required
  19  to write a summary, I understood that to be an
  20  executive summary of the 400 pages like the chair
  21  did up to now, which says here's the evidence,
  22  here's the major evidence discussed, here's the
  23  opinions, and here's the results, and that the
  24  panel would actually look at this 20-page
  25  executive summary of these 400 pages of material
   1  or maybe 30 pages of these 400 pages and have
   2  that kind of a document.  But if that's not the
   3  case, then somebody has to write that document.
   4            MS. LAPPALAINEN:  But the summary must
   5  reflect the agenda and what happened that day, so
   6  the construction of the last three summaries,
   7  which should have been available -- as a matter
   8  of fact, we have one as a public handout now --
   9  followed the agenda of December 8th.
  10            DR. BROOK:  But it didn't have any
  11  summary of the issues.  It did not have anything
  12  that said the scientific evidence was presented.
  13  The panelists basically looked at it.  The
  14  scientific consisted of this kind of information.
  15  In other words, it wasn't a contents summary.  It
  16  was a process summary.
  17            And I agree with you.  Somebody should
  18  write for the record a 20-page or so contents
  19  summary of this voluminous amount of material
  20  that only a very few people are going to read.
  21            DR. GARBER:  That's the evidence
  22  report.  I think looking back on the last panel
  23  meeting, this is a misleading -- because we will
  24  have evidence reports in place.  That is assuming
  25  that the recommendation goes forward.  So a lot
   1  of this would be superfluous.
   2            DR. KANG:  I think that's absolutely
   3  correct.  We can't look at the last two meetings
   4  as -- these are all interactive.  The reality is
   5  the first half of this discussion was setting
   6  guidance.  It's saying that these are the
   7  questions they'd have to answer.  Then the fact
   8  that -- good evidence report, this really tees up
   9  the issues, and I think that we're learning,
  10  quite frankly, as we're going along, and I really
  11  don't think that the first two will be
  12  representative of --
  13            DR. SOX:  I think we have a motion on
  14  the table.  We've had some discussion.  Is there
  15  anybody else who would like to offer discussion
  16  before we vote?
  17            DR. FERGUSON:  I guess my discussion is
  18  a question again.  The last sentence here, the
  19  panel chair is responsible for writing the
  20  explanation of the panel's conclusions, modified
  21  with what Dr. Davis did, that's different than a
  22  summary, as Dr. Brook said.  So we're not voting
  23  on whether or not the panel chair or a designee
  24  should write a summary of the panel's
  25  conclusions.  We're voting on something else.
   1            DR. SOX:  Why don't we vote on this,
   2  and then it seems to me that vote implies we
   3  ought to cross that out.
   4            DR. KANG:  How about the panel chair is
   5  responsible for writing the executive summary?
   6            DR. SOX:  But according to the motion,
   7  apparently not approved, it could be the panel
   8  chair or it could be HCFA staff with the panel
   9  chair.
  10            DR. KANG:  So HCFA staff or panel chair.
  11            DR. SOX:  I think we can basically
  12  delete that sentence and substitute the process
  13  that we voted on.
  14            DR. FERGUSON:  Delete this last
  15  sentence?  Is that what you're saying?
  16            DR. SOX:  That would be implied if we
  17  vote this in.  Any other questions?
  18            DR. HOLOHAN:  Can I ask for a
  19  restatement of the motion?
  20            DR. SOX:  Restatement of the motion,
  21  please.
  22            DR. MAVES:  I'll try.  The motion was
  23  that the operational documents that would result
  24  from the panel meetings would be -- the
  25  transcript will be number one.
   1            DR. SOX:  Not quite so fast.
   2            MS. LAPPALAINEN:  Operational
   3  documents.
   4            DR. MAVES:  From the panel meetings
   5  would be the transcript of the meeting, the
   6  summary of the meeting -- and I think you could
   7  put in parentheses prepared by HCFA staff -- and
   8  the explanation of each member's votes for the
   9  deliberations or the questions that are asked by
  10  folks.
  11            MS. LAPPALAINEN:  With an opportunity
  12  for dissension?
  13            DR. MAVES:  With an opportunity for
  14  dissension.
  15            DR. DAVIS:  If I could ask a question.
  16  If there are seven questions posed to the panel,
  17  then you'll have to go around the table and get
  18  an explanation from every panel member for each
  19  of the seven questions?
  20            DR. MAVES:  Yes.  And I think that
  21  mirrors the practice that goes on at the FDA, if
  22  any of you have been out there.
  23            MS. LAPPALAINEN:  As I have it written,
  24  operational documents from the panel meeting will
  25  consist of the transcripts, the summary that is
   1  prepared by HCFA and signed off by the panel
   2  chair, an explanation of each panel member's
   3  votes with an opportunity for panel member
   4  dissension.
   5            DR. MAVES:  Yes.  I want to make sure
   6  my seconder is here.  Jeff, you're comfortable
   7  with that?
   8            DR. KANG:  John, you were about to say
   9  something.
  10            DR. FERGUSON:  I'm not sure that's
  11  different than what we did before.  I mean we
  12  went around and voted on each question, and we
  13  were obliged to say why we voted against
  14  something, not really obliged for why we voted
  15  for, and that was all captured in the transcript
  16  and then HCFA's summary.
  17            DR. MAVES:  The reason for this is my
  18  sense was that we're getting to a point where
  19  we're going to have a third document, which would
  20  be the chair or his designee's interpretation
  21  being done at some point afterwards.  And my key
  22  concern about that was that you could have two
  23  different, if you will, interpretations of the
  24  same meeting.  And rather than that we have this
  25  as much as possible, either contemporaneously
   1  recorded and transcribed or as needs be done
   2  apparently through FACA, the HCFA summary of the
   3  meeting done as well so that we don't have
   4  situations -- and I think we had a little bit of
   5  that last time where the interpretation of the
   6  meeting and the HCFA document and the chair's
   7  recommendation or the chair's interpretation of
   8  the summary were two different things.
   9            DR. SOX:  I think there was one more
  10  comment.
  11            DR. BROOK:  I want to just be clear
  12  about the HCFA thing.  Sharon, when you write the
  13  HCFA summary, the last part of this is you're
  14  going to have the up-front evidence report, then
  15  you're going to have the explanation of the
  16  votes.  So you're going to look at this, the two
  17  pieces of this stuff.  Other than the process of
  18  the agenda, you're going to summarize something
  19  from the evidence report, a summary of the
  20  evidence report, what's available going in, and
  21  then the common themes across those whatever
  22  number of panel votes for each of those votes.
  23            So if Alan said the reason I voted yes
  24  on this was because there were six controlled
  25  trials and seven of these, the benefit was this,
   1  and I believe it could be extended, you're going
   2  to look at how they come across all the
   3  individual panelists and then summarize that in a
   4  factual manner so that it would be an aggregated
   5  factual summary across the vote.  That's the key
   6  of what would have to happen.  It would be
   7  factual, but the aggregate across the votes is
   8  based on reading the transcripts.
   9            Is that what I understand this summary
  10  is going to be?  If everyone has agreed or said
  11  the same thing, it could be one page?
  12            MS. LAPPALAINEN:  Right.  The
  13  requirement for this motion -- and that is an
  14  explanation of each member's votes -- will be
  15  added to the agenda as an agenda item for each
  16  panel, and that will be included in the summary
  17  if that is a required agenda item for each
  18  panel.
  19            DR. BROOK:  There are two issues here.
  20  You have ten people each saying a paragraph of
  21  stuff.  Somebody's going to look at the common
  22  themes and write a summary of that.  That's the
  23  key fact that has to be done.  And you're going
  24  to do that.  HCFA's going to do that.
  25            DR. SOX:  And the chair is going to
   1  approve it.
   2            DR. BROOK:  Now, does the chair have a
   3  right, if they're nonvoting, to actually give his
   4  or her summary on the record when you go around?
   5  After you've taken the vote, can we modify the
   6  process so that the chair just doesn't sit there,
   7  let's say at the end of this or at some point in
   8  this process, and say here's how I would have
   9  voted or something like that and here's my
  10  explanation?  Can that be done legally?
  11            MS. LAPPALAINEN:  Right.  Presumably
  12  after the voting period on the agenda and the
  13  agenda item, which has been added, which is the
  14  explanation of the vote, this also includes at
  15  the end of that an opportunity for the chair to
  16  express his or her opinion after the vote.
  17            DR. BROOK:  Why don't we require that.
  18  Why don't we state that the chair should on the
  19  record, after the vote has been taken, explain
  20  his or her explanation for what he would have
  21  voted or she would have voted, if he had the
  22  opportunity to vote, so that it becomes part of
  23  the record and part of the summary that you
  24  write.
  25            DR. SOX:  Does that sound reasonable?
   1            DR. BROOK:  So we don't get the problem
   2  with the chair saying something later because he
   3  or she never had the opportunity like happened
   4  last time.
   5            DR. SOX:  In just a minute Sharon's
   6  going to read the motion, but first, since there
   7  has not been a motion to vote, there's still an
   8  opportunity for people to comment if they wish
   9  to.  Hearing none, Sharon?
  10            DR. HOLOHAN:  I don't want to
  11  redundantly overclarify, but the HCFA summary
  12  will in fact be what's written in this paragraph
  13  as a -- and I'm quoting -- written explanation?
  14            DR. BROOK:  Yes.
  15            DR. HOLOHAN:  Okay.
  16            DR. SOX:  Ready to vote?  And you're
  17  going to reread it and then say who's eligible to
  18  vote and who isn't.
  19            MS. LAPPALAINEN:  The motion which we
  20  have on the table -- and we have a second I
  21  believe -- is the operational documents from the
  22  panel meeting will be the transcripts, the HCFA
  23  summary, including an explanation, an explanation
  24  for each panel member's votes at the panel
  25  meeting, with an opportunity of dissension.  The
   1  chair after the vote will provide their opinion.
   2            DR. SOX:  Ready to vote?  All in
   3  favor?
   4            DR. KANG:  I think it's a summary of
   5  the votes.  It's an aggregate explanation with an
   6  opportunity for dissension.  The point is it's
   7  got to be a content summary.  It's got to say we
   8  took a vote, here was 8 to 3, and on average this
   9  is why it went this way.
  10            DR. BROOK:  It could say in voting yes,
  11  that there were adequate controlled trials, three
  12  said there was this, and two said this, but you
  13  have to take that two paragraphs of that -- or
  14  that two minutes of what that person says and
  15  write a thoughtful summary.  And we're giving the
  16  HCFA staff the responsibility to do that with the
  17  chair's approval, with the chair looking over
  18  that part of the transcript, which will be much
  19  shorter than the bigger thing, to do that.
  20            MS. LAPPALAINEN:  The motion is
  21  operational documents from the panel meeting will
  22  be the transcripts, the summary that HCFA
  23  prepares, including a summary of the content and
  24  explanation of each member's votes at the
  25  meeting, with an opportunity of dissension.  The
   1  chair after the vote will provide their opinion
   2  as well.
   3            For today's meeting the members that
   4  are eligible to vote on this motion are Thomas
   5  Holohan, Leslie Francis, John Ferguson, Robert
   6  Murray, Alan Garber, Michael Maves, Frank
   7  Papatheofanis, Ron Davis, Daisy Alford-Smith, Joe
   8  Johnson and Robert Brook.
   9            Dr. Sox will vote in the case of a tie
  10  vote.
  11            DR. SOX:  All those who are in favor,
  12  please raise their hand and keep it up long
  13  enough for Sharon to tally the vote.
  14            DR. SOX:  Two against.  Abstentions?
  15  One abstention.
  16            MS. LAPPALAINEN:  I'm going to read the
  17  vote back.  For the motion we have eight for, two
  18  against and one abstention.
  19            DR. BERGTHOLD:  Now you need a written
  20  explanation of that.
  21            DR. SOX:  We now need to move on to
  22  talk about structure of the evidence provided to
  23  the panel.
  24            DR. FERGUSON:  We don't have to explain
  25  our no votes here?
   1            DR. HOLOHAN:  I think you should be
   2  free to express why.
   3            DR. SOX:  Why did you vote no?
   4            DR. FERGUSON:  I voted no because of
   5  some confusion on my part as to the timing of
   6  when these documents will occur.  My
   7  understanding is that the transcript doesn't
   8  occur to be finished until a week or more later.
   9  The summary before wasn't finished at the time of
  10  the meeting so that we could all look at it.  And
  11  I can't imagine that summary occurring at the end
  12  of the meeting in a fashion that can be seen by
  13  all of us.  So since I was not clear on when that
  14  could occur in a way that I could conceive of, I
  15  had to vote no.
  16            DR. SOX:  Ron, do you want to explain
  17  your abstention?
  18            DR. DAVIS:  I thought it was confusing
  19  and awkwardly written, and I liked the original
  20  with the amendment that I proposed.
  21            DR. SOX:  And Leslie, your no vote?
  22            DR. FRANCIS:  I would have preferred
  23  just the requirements in the Federal Advisory
  24  Committee Act and let panels explain it.
  25            DR. SOX:  Thank you very much.
   1            DR. HOLOHAN:  Could I explain why I
   2  changed my vote?  I thought that Bob Brook really
   3  nailed down the content, and I was comfortable
   4  with that.
   5            DR. SOX:  Does anybody else want to
   6  explain a positive vote?  Hearing no other
   7  comments, let's move on to number 4, structure of
   8  evidence provided to the panels.
   9            I guess before we get into this, I'd
  10  like to note that we have not at this point said
  11  what ought to go in those evidence reports.  And
  12  presumably if we approve this section, then we're
  13  going to have to get a group to get together
  14  perhaps to work in collaboration with HCFA to
  15  decide what will be the requirements for
  16  whoever's going to write the evidence report.  I
  17  think maybe that would be better to not try to do
  18  that together, but rather to do that off line
  19  since it's really in the area of operations.
  20            If anybody disagrees with that, I'd
  21  like them to speak up, but that's my take on it
  22  given the time.
  23            Alan, do you think that's reasonable to
  24  do it off line?
  25            DR. BERGTHOLD:  Mr. Chairman, it's
   1  2:35, and we had a break scheduled for 2:15.  I
   2  just want to check.  This next thing is going to
   3  be actually I think complicated, or maybe not.
   4            MS. RICHNER:  Yes.
   5            DR. BERGTHOLD:  So I was wondering
   6  could we take our break now?
   7            DR. SOX:  We're hard at work, and we've
   8  shown our ability to talk for quite awhile in
   9  trying to solve some of these operational issues.
  10  So my suggestion is if there are members of the
  11  panel who need to excuse themselves, they should
  12  do so, but I think we ought to just work straight
  13  on through.
  14            Okay.  So now we have number 4,
  15  structure of evidence provided to the panels.
  16  And what we're interested in hearing is -- again,
  17  just to remind you of objections to this as a
  18  basis for the panel's operations or lack of
  19  clarity that's going to interfere with your
  20  ability to work with your panel.  And if you have
  21  a problem with the language, we'd like you to
  22  propose a change so we'll have something specific
  23  to work on.
  24            With that, I'll open the discussion.
  25  Jeff?
   1            DR. KANG:  Could I just ask a question
   2  as to your opening question?  Because I missed
   3  the first MCAC meeting of executive counsel.
   4            I actually had thought the whole
   5  purpose of the preceding four or five pages,
   6  quite frankly, posed the evidence questions that
   7  the evidence may support needs to think about
   8  with this, so I was -- that was my -- and you're
   9  thinking now that that's not adequate?
  10            DR. SOX:  I guess for myself, I'm
  11  thinking that it provides the framework, but it
  12  will be my, for example, wanting to talk to the
  13  folks who are running the U.S. Preventive
  14  Services Task Force to find out what their charge
  15  has been to the evidence-based practice, for
  16  example, what their deliverables are, and then
  17  modify that as appropriate to meet the needs of
  18  this group.  I really think we need to define the
  19  deliverables of whoever's going to provide these
  20  reports, and those are specific.
  21            DR. KANG:  Let me suggest a strategy
  22  because that second issue you raised is more of a
  23  logistical issue.  It's an issue as to --
  24  whatever we want exists already or can we get it
  25  -- I'm kind of wrestling with why it would be
   1  fund -- I know that it meets the -- but why
   2  wouldn't we want the evidence-based report to
   3  take the first stab at answering the questions
   4  that we have posed here in the heart of the first
   5  five pages of this document?
   6            DR. SOX:  We might have some opinions
   7  based upon our expertise about what they would
   8  actually have to do to answer those questions
   9  operationally.
  10            DR. KANG:  But there you're trying to
  11  do deal with that in number 5.  Whoever's working
  12  on the evidence-based report who wanted
  13  interaction with the panel members back and
  14  forth, these things could get created -- some
  15  interaction back and forth.
  16            DR. SOX:  Maybe it would be useful for
  17  Alan, who's at least peripherally involved in --
  18  comment on what sort of things go into their
  19  report just so we have an idea of what we're
  20  really talking about.
  21            DR. GARBER:  I think actually if I can
  22  go to the prior question first, I think Jeff and
  23  Hal are talking about this real important
  24  operational issue, should the Executive Committee
  25  give a lot of detail about how the evidence
   1  should be structured to HCFA or should HCFA staff
   2  proceed.  And my relevant experience is actually
   3  as chair of the med-surg panel where we've been
   4  going over the agenda for our upcoming meeting,
   5  and I've seen the first draft of what would be an
   6  evidence report, and it's occurred to me from
   7  seeing that, which I might add so far seems to be
   8  very well done, that we might want to build up
   9  some experience with HCFA staff doing these
  10  before we make recommendations.
  11            So I actually think that what they've
  12  done so far is exactly the kind of thing that
  13  this committee would recommend anyway.  And maybe
  14  because there are some areas that are a little
  15  different, like diagnostic technologies, we might
  16  want to gain some experience before we the
  17  Executive Committee make any more specific
  18  recommendations.
  19            I'm actually very sympathetic to what
  20  Jeff has just said based on my experience in
  21  trying to prepare for our upcoming meeting.  That
  22  is it may not be suitable for us at this point to
  23  give very detailed information about what things
  24  like evidence tables should look like because
  25  right now what they're doing for the urinary
   1  incontinence studies is exactly what any EPC
   2  would do.
   3            DR. KANG:  Let me add.  It's
   4  unfortunate because I don't think the first two
   5  issues are representative.  Reality is we can
   6  contract out for some of this stuff, and we can
   7  have, whether it's the tech assessment group over
   8  at AHRQ or whatever, and then there's no reason
   9  why the panel member can't interact with
  10  whoever's doing that and interact in a fashion,
  11  take a quick look, say no, you forgot to ask this
  12  question or whatever.
  13            I don't think we should get into the
  14  logistics of how to do this.  I think we should
  15  just stick with we want an evidence-based report,
  16  here is the list of issues and concerns we are
  17  concerned about right now at this point, and
  18  start working to answer those questions, and then
  19  have number 5 there as an interaction to the
  20  extent that they're things that are coming up
  21  that we didn't anticipate.
  22            MS. RICHNER:  One of the discussions
  23  that we had in our conference call, if you'll
  24  remember correctly, was that the panels would
  25  have an opportunity to pose the questions for the
   1  evidence report before they were originally
   2  conceived.  So is that still the issue?  I mean
   3  that's still going to occur then?
   4            DR. SOX:  That will be number 5.
   5            MS. RICHNER:  My other problem and
   6  question once again, how does this fit?  I still
   7  don't understand how and where the evidence
   8  report fits in this Medicare coverage process
   9  that has been published.  So where and how is it
  10  triggered and where does it fit in terms of the
  11  panel receiving it?  I still don't understand
  12  it.
  13            DR. HILL:  In that flow chart you'll
  14  see where we have the opportunity to refer things
  15  to the Medicare Coverage Advisory Committee when
  16  we take in issues as part of the process of
  17  preparing the information for that committee
  18  between the time the intention is arrived at to
  19  send something to the panel and an accurate
  20  amount of time before the panel.  So they can be
  21  able to digest it, we either create or get some
  22  help in creating this evidence table.
  23            MS. RICHNER:  So the evidence reports
  24  as we know take approximately six months to do.
  25            DR. HILL:  Not always.  We've had
   1  indications from AHRQ that in some cases, many
   2  cases, they'll be able to do something for us a
   3  little faster than that.  We're working on our
   4  own process internally trying to gear ourselves
   5  up to be able to do those things faster.
   6            If you're concerned about the time
   7  frame that's involved, that's not stated on
   8  there, and it wasn't -- so this doesn't change
   9  that.
  10            MS. RICHNER:  You see, if we have the
  11  evidence reports being -- there has to be
  12  something written in here that when you, HCFA,
  13  trigger this to MCAC, the evidence report and the
  14  questions that need to go into the evidence
  15  report have to be decided by the panel at that
  16  particular moment.  You have to have some
  17  mechanism for the panel to get together to say
  18  these are the seven things I want the evidence
  19  report to reflect, and that doesn't say that in
  20  here.  I'm really grappling with this.
  21            And then you have the six-month time
  22  period where the evidence report would be
  23  prepared approximately four to six months.  Then
  24  it would come to MCAC.  We would then get the
  25  evidence report and review it and have all this
   1  time associated with reviewing it.  I mean I'm
   2  not tracking it with this document.
   3            DR. SOX:  Let me try to recall the end
   4  of my talk this morning.  HCFA decides to refer
   5  something to MCAC.  In that first month they work
   6  with the chair of the appropriate panel to define
   7  the questions, and that's a process that could
   8  include other members of the panel if the chair
   9  so designated.  And they decide who's going to do
  10  the piece of work and perhaps on the basis of the
  11  nature of the problem --
  12            MS. RICHNER:  Who's they decide?
  13            DR. SOX:  The chair and HCFA.
  14            MS. RICHNER:  Decide who it's going to
  15  be referred to?
  16            DR. SOX:  The decision about who's
  17  going to do it -- HCFA decision.
  18            MS. RICHNER:  Whether it's going to be
  19  ACRI or AHCPR or whoever is going to --
  20            DR. HILL:  Or internal.
  21            DR. SOX:  And then after that sort of
  22  month of preliminary work, whoever is going to do
  23  it gets the job, and they spend four to six
  24  months doing it.  They produce a report.  And
  25  then that goes out to the members of the panel to
   1  prepare for a meeting that will occur
   2  approximately a month after the report is
   3  completed.
   4            MS. RICHNER:  None of that is reflected
   5  in here.  You know that, right?  None of those
   6  times.
   7            DR. HILL:  That's correct.  As I said
   8  earlier, we didn't state those times.
   9            DR. SOX:  Leslie?
  10            DR. FRANCIS:  This is a clarification
  11  question.  As a member of the panel, I would want
  12  to get copies of the studies as well as the
  13  evidence report, right?  I don't want to just get
  14  somebody's summary of it.
  15            DR. GARBER:  You may have 200 studies.
  16  Again this is patterned on well-established other
  17  technology evaluation processes.
  18            And really, Randel, your questions are
  19  getting into point number 5.  But anyway, the
  20  idea is that combination of staff and the chair
  21  will identify interested panel members with
  22  appropriate expertise and will involve them in
  23  the process of helping to advise HCFA staff about
  24  the scope of the evidence report or advise the
  25  contractors or whoever it may be.
   1            And this is intended to make sure that
   2  the evidence report is the most suitable document
   3  for the panel's deliberations.  That means not
   4  the entire panel is involved.  The attempt is to
   5  bring in all the really interested members of the
   6  panel.  And if by some chance that group of
   7  people -- that is, the chair and the interested
   8  panel is identified to assist in setting the
   9  parameters on the evidence review, if by chance
  10  they really goof up and they give directions that
  11  some important studies were neglected or the
  12  scope of it was wrong, that would come up during
  13  the panel meeting.  And then perhaps the panel
  14  will conclude they didn't have the evidence they
  15  needed.
  16            But generally speaking, this kind of
  17  system works where you get all the interested
  18  parties to give input early in the process, and
  19  you don't have to go through actually convening
  20  two panel meetings, one to set up the evidence
  21  report and another to evaluate it.
  22            DR. SOX:  Leslie?
  23            DR. FRANCIS:  I'm not asking for two
  24  panel meetings.  I just would not feel, as a
  25  panel member, that I was in a position to
   1  evaluate the evidence unless I both had the
   2  studies on which the evidence report is based and
   3  the evidence report as an analytic summary of
   4  those studies.  What concerned me with the
   5  myeloma panel was that I had about 30 studies and
   6  nothing else.
   7            DR. SOX:  My take is that if an
   8  individual panel member wanted those studies and
   9  had the time to do it, they could get them and
  10  that the evidence report, if it focused on two or
  11  three really key studies, that those might be
  12  included as an appendix to the report so you
  13  could read it.
  14            DR. HILL:  Our intention at this point
  15  is when we identify, or the panel chairman
  16  identifies, key studies that should be sent to
  17  all panel members, they will be.  And when you
  18  get to the table, if there's something you read
  19  off there, then we'll send it to you.  And if you
  20  tell us ahead of time that you're the one person
  21  who wants to get the whole five crates, we'll
  22  talk about it.
  23            DR. SOX:  Any other comments about this
  24  section before we move on?  Jeff?
  25            DR. KANG:  I had to step out of the
   1  room, but I wanted to comment to Randel's issues
   2  on timing.  I said this earlier in the morning.
   3            The last slide we had, which was some
   4  time frames, was actually -- I don't know how to
   5  say -- was kind of Medicare Coverage Advisory
   6  Committee centric I guess.  The reality is that
   7  staff is really responsible for the logistics of
   8  the timing and the flow.
   9            I really would encourage you all in
  10  your deliberations to consider what is desirable,
  11  what do you want.  We then are responsible for
  12  the timing and the logistics and meeting what we
  13  said we were going to meet in the federal
  14  register notes.  And we're committed to trying to
  15  make that work.
  16            Now, it may turn out what you all
  17  believe is desirable is physically humanly
  18  impossible, and then we may have to rethink this.
  19  But I actually, quite frankly, think it is
  20  possible.  And this guidance that you've given
  21  staff is extraordinarily helpful because it will
  22  lead quickly to evidence-based reports to answer
  23  the questions or up front there's this
  24  interaction in step number 5.  I think this is
  25  very doable and still meeting the time frames
   1  that we said in the federal register notes.
   2  That's a commitment.  But our issue is to try to
   3  sort out the logistics, and we will do that.
   4            MS. RICHNER:  One more question.  The
   5  data.  There was a point when we had our
   6  orientation for the panel members.  As an
   7  industry representative, anyone can give me
   8  information that I would share then with the
   9  panelists.  That's one of my roles, which could
  10  be unpublished literature, it could be white
  11  papers, it could be FDA information, that would
  12  not have been provided in the evidence report.
  13            Where and how does that get
  14  considered?  I know that they may not be
  15  controlled studies, but it's information that can
  16  go into the decision-making process.  So where
  17  does that fit?
  18            MS. LAPPALAINEN:  Right.  The industry
  19  representative's role on the panel is to
  20  represent industry.  And if you believe that
  21  information needs to get to the panel, you need
  22  to give that to us at HCFA, not directly go to
  23  the panel and have the panel interact.
  24            MS. RICHNER:  But how does that fit
  25  into this?  Is it the only thing you receive is
   1  the evidence report?
   2            DR. KANG:  It's part of the evidence
   3  report.
   4            MS. RICHNER:  So that means I would
   5  have to give it to ACRI or AHCPR?
   6            DR. KANG:  You'll give it to us, and
   7  we'll figure it out.
   8            DR. BROOK:  The only problem with that
   9  is if the information is proprietary, then you're
  10  going to have a hard time because the evidence
  11  report, you're job should be -- everybody's job
  12  should be to get to the person at HCFA everything
  13  under the sun.  And that person should summarize
  14  that in an unbiased manner.  And so published,
  15  not published, we ought to be beating every drum
  16  we can find to get good information.  But if you
  17  send along a tag you can't use it or publish it
  18  because it's proprietary, then it won't be used.
  19            MS. RICHNER:  Of course.
  20            DR. KANG:  Randel, it's really not your
  21  responsibility.  It is the requester's
  22  responsibility.
  23            MS. RICHNER:  Right.  But it's not
  24  reflected here in this process, and so I just
  25  wanted to make sure that -- maybe the public now
   1  is aware that that is part of the process, that
   2  information can be provided to your industry or
   3  consumer representative that should be given to
   4  the panel or to HCFA as part of the evidence
   5  report.
   6            DR. KANG:  We'll make that clear, but I
   7  don't think this is the document to make it
   8  clear.
   9            DR. HILL:  We already do invite those
  10  sendings in our announcements.
  11            DR. SOX:  Any other comments on this
  12  section before we move on?
  13            The next section is about panel member
  14  involvement, the chair up front with appropriate
  15  other members of the panel, in framing the
  16  questions, and several panel members should be
  17  participants in the evidence review as a way of
  18  gaining familiarity with data and expertise on
  19  the topic, and finally, there should be a couple
  20  of primary reviewers whose responsibility would
  21  be to spend a lot of time going over the evidence
  22  report prior to the meeting and be in a position
  23  to summarize their take on the evidence as
  24  reflected in the report.
  25            So those three aspects of panel member
   1  involvement are now open for discussion.
   2            DR. HOLOHAN:  I think I'm asking this
   3  for Leslie.  It says panel members should take an
   4  active role in reviewing the evidence, a word
   5  that I believe is distinct from the evidence
   6  report.
   7            DR. FRANCIS:  It's not the evidence
   8  report.  It's the evidence.
   9            DR. GARBER:  I don't think that's
  10  realistic in some of these areas; that is to say
  11  to review all the evidence.  I mean this is
  12  basically reviewing all the evidence.  You do a
  13  serious job of it even without writing it up.
  14  It's a several-week, full-time job.
  15            DR. HOLOHAN:  I understand that.  I'm
  16  simply saying it --
  17            DR. GARBER:  Oh, okay.
  18            DR. SOX:  What do you think would be
  19  good language there?  Reviewing the evidence
  20  report?
  21            DR. KANG:  Preparing the evidence
  22  report.
  23            DR. SOX:  So it would be an active role
  24  in preparing the evidence report?
  25            DR. FRANCIS:  The reason I had made
   1  that is not equal to the evidence is I'm not
   2  going to know how to vote as a panel member
   3  unless I think I've been able independently to
   4  come to my own judgment.  I'm not around here to
   5  rubber stamp an evidence report.  An evidence
   6  report and other people's comments on it are
   7  helpful to me in trying to reach my own judgment,
   8  but if it's all just laid out and I can't in any
   9  way try to exercise my own critical judgment, I
  10  don't have any business being here.
  11            DR. BROOK:  First of all, there's no
  12  rubber stamp on this.  An evidence report just
  13  puts the evidence together.  And then you need to
  14  produce the judgment, based on the evidence, what
  15  to do.
  16            Now, if you're saying you want to redo
  17  the evidence report, what I think Alan and I are
  18  doing, having done a lot of these evidence
  19  reports, be it as it may, in areas which have
  20  lots of literature, we've reviewed 10,000 titles
  21  to come up with 300 articles to summarize, and
  22  we're struggling to get this done in six months.
  23            There is no question that HCFA, if
  24  you'd like, should be able to provide you all the
  25  original material that we work from, but I will
   1  tell you that unless you're the most
   2  extraordinary individual under the sun, you will
   3  not have the time to redo this what to do, but
   4  you ought to have the right to do it.
   5            And certainly I think any panel member
   6  ought to have the right to get the original
   7  evidence, and it ought to be stored in a manner,
   8  put together in a manner, and that ought to be
   9  sent out.  But you ought not to expect the
  10  average panel member to do that.  We ought to
  11  expect the average panel member to believe that
  12  the evidence has been synthesized correctly, and
  13  now you have to make a judgment about how it
  14  should be used and what it means.
  15            DR. SOX:  Do you want to add to that?
  16            DR. GARBER:  I think Hugh put it really
  17  well about how this would work.  I think, Leslie,
  18  the issue for us is going to be we have to look
  19  at the original data for some key studies, and
  20  all the panelists should get those key studies,
  21  but not the huge volume that Bob was alluding to
  22  that we usually start with.  So that's why this
  23  will never be -- I doubt that this will ever be a
  24  rubber stamp.  The panelists are going to read
  25  some studies, but they have to be whittled down
   1  somehow.  And that's all we're saying is be
   2  selective about it.
   3            DR. SOX:  I'd like to hear from the
   4  panel if there's objections to the concepts that
   5  are imbedded in the boldface number 5.  Does this
   6  look reasonable for panel members?  That's
   7  great.
   8            DR. BROOK:  Under the first boldface it
   9  should insert report.
  10            DR. SOX:  Panel members should take an
  11  active role in, I thought we said, preparing the
  12  evidence report.
  13            DR. KANG:  Preparing the evidence
  14  report.
  15            DR. SOX:  Not reviewing.  Change it to
  16  preparing and insert report after evidence.
  17            So let's discuss this section trying to
  18  pick out -- we don't have a lot of time now, so
  19  we've got to kind of focus again on problems with
  20  clarity, pieces that are objectionable.  John?
  21            DR. FERGUSON:  Just a suggestion.  This
  22  number 5 might better be put on one of the first
  23  under Suggestions for Panel Operations because
  24  this sort of explains what the beginning of the
  25  process is, which Randel was questioning about.
   1  Because this is the first part.  The evidence
   2  report, the panel chair and others working with
   3  the evidence, we're going to do the evidence
   4  report.  And the first thing you start out with
   5  is actually the end result.
   6            MS. RICHNER:  That would help a lot,
   7  just moving it to the first.
   8            DR. SOX:  The problem is that it does
   9  talk about the evidence report, which is defined
  10  in the immediately preceding section.  It can
  11  certainly be --
  12            DR. FERGUSON:  It sort of operationally
  13  comes after the fact.
  14            DR. SOX:  I think we can probably move
  15  the first one till later because it comes later
  16  in sequence.  That would work.  Okay.
  17            Other comments on this section?  In
  18  that case we're going to move on to number 6.
  19            MS. RICHNER:  One more thing.  To me,
  20  once again, it's the timing, but that's going to
  21  be clarified by HCFA and not by us?
  22            DR. KANG:  We're on the hook for
  23  timing.
  24            DR. HOLOHAN:  If I can offer an
  25  unsolicited comment.  There's been a lot of
   1  concerns first about how long these take.  If
   2  nothing else, the experience with the first two
   3  panels should have instructed us that doing it
   4  the right way is the fastest way.
   5            DR. SOX:  Just to quickly follow up on
   6  John's suggestion, consistent with John's
   7  suggestion that we try to get these operational
   8  things consistent with sequence, everybody happy
   9  with moving the first one, which is now number 3,
  10  a panel must explain its conclusions in writing,
  11  make that the last one?  Okay.
  12            Then let's move on to number 6, which
  13  is expert review of evidence reports.
  14            DR. KANG:  Before we discuss this, can
  15  I ask the subcommittee to explain why this is
  16  here just so I can understand?
  17            DR. SOX:  The opinion of experts is the
  18  best way to assure everyone that the evidence
  19  report is complete and fair.  So it's a notion
  20  getting said people that are competent experts to
  21  look at it and say it didn't miss anything, the
  22  report didn't distort the clinical facts as we
  23  know it.  It's something that, at least on all
  24  the other panels I've been involved with, outside
  25  review has been a key part of it if only to
   1  establish the credibility of the process, to say
   2  look, we've given the people who have an axe to
   3  grind the chance to sling their strongest arrows.
   4            DR. KANG:  Well, maybe I didn't read
   5  this closely enough.  The assumption here that
   6  the evidence-based reports are being done by
   7  nephrologists, internists or whatever and that
   8  the final analysis, if it's about some surgical
   9  procedure, that you'd also like to show it to a
  10  couple of surgeons?  Is that the issue here?
  11            DR. SOX:  That strategy -- namely,
  12  having evidence-based clinicians prepare the
  13  report, then have it reviewed by competent
  14  experts -- seems to really work well on the other
  15  side.
  16            DR. GARBER:  Jeff, one of the explicit
  17  precedents here is the evidence-based practice
  18  center's review process in which the external
  19  reviews come from actually a wide range of types
  20  of expertise ranging from pure methodology to
  21  pure clinicians.  I'd just like to emphasize the
  22  language here is committee recommends expert
  23  review.  I think we recognize this could be
  24  onerous in some circumstances and maybe not
  25  always is necessary in some circumstances as
   1  others.  So this is really truly advisory, but we
   2  do feel it's very important to do it to ensure
   3  the highest quality.
   4            DR. SOX:  Any other comments about this
   5  section?
   6            DR. FRANCIS:  I apologize for
   7  continuing to beat what's probably a dead horse,
   8  but I really do think whatever else you do, in
   9  addition to the evidence report, you ensure that
  10  panel members have the key studies.  It's okay
  11  from the evidence report to identify key studies,
  12  but I want to see them too.
  13            DR. SOX:  Ron?
  14            DR. DAVIS:  I actually scribbled out a
  15  sentence to address that, and if the Executive
  16  Committee feels it's important to make that
  17  explicit, panel members will have the evidence
  18  report at their disposal and will have the right
  19  to obtain any primary sources upon which it's
  20  based.  But I don't think there should be an
  21  affirmative obligation on behalf of HCFA staff to
  22  send us all those primary resources.
  23            DR. SOX:  That suggestion seems
  24  consistent with the discussion we've had.  Any
  25  objections to it?  Okay.  Then we have to go back
   1  and --
   2            DR. KANG:  I'm sorry.  I heard what
   3  Leslie was saying was key articles be part of the
   4  report and then that she also has access to the
   5  10,000 if she wants.
   6            DR. FRANCIS:  Exactly.  That's what I
   7  want.
   8            DR. SOX:  We've gone through the
   9  document once.  Now, let's start over.
  10            Ron has been given responsibility for
  11  marking up the transparency that Jeff and Leslie
  12  prepared during lunch.  Do you have a report to
  13  make?
  14            DR. DAVIS:  It's over there on the
  15  transparency.
  16            DR. SOX:  Okay.  Let's look at it and
  17  see if we like it.
  18            DR. DAVIS:  I tried to cut down words.
  19  The first line and a half goes in italics, I
  20  guess, because it's a subheading.  Should I read
  21  it now?
  22            DR. SOX:  Yeah.
  23            DR. DAVIS:  Medicare beneficiaries
  24  include elderly, nonelderly and disabled people.
  25  The Medicare population also may or may not
   1  include patients with comorbid disease.
   2  Historically many controlled trials unfortunately
   3  exclude older men and women, people with
   4  disabilities and people with comorbid disease.
   5  Thus these studies may have had adequate
   6  statistical power for the study population, but
   7  the results may or may not be generalizable to
   8  some portions or all of the Medicare population.
   9  If the requester is asking for coverage or if the
  10  panel believes there is a medical benefit beyond
  11  the clinical and demographic characteristics of
  12  the study population, the panel should state
  13  whether it believes the results of the studies
  14  are applicable to some groups covered by
  15  Medicare, define what those groups are, and
  16  explain its reasoning.
  17            DR. SOX:  Anybody have any changes
  18  they'd like to make to that masterful piece of
  19  rewriting?
  20            DR. FRANCIS:  Thank you.
  21            DR. SOX:  Great.  Thank you very much,
  22  Ron.
  23            DR. DAVIS:  Alan just suggested at the
  24  end to change it to say define the groups, and
  25  then we'll say --
   1            DR. SOX:  Daisy, are you ready with
   2  some suggestive language for the preface
   3  regarding --
   4            DR. SMITH:  Yes.  In fact, if you'll
   5  recall, initially we had discussed the
   6  possibility of inserting it under external
   7  validity.  And at that time when I was in that
   8  mindset, I thought we were going to say although
   9  the panel recognizes that adequate representation
  10  of every study may not be possible, consideration
  11  should be given to the applicability including
  12  race and culture when appropriate and necessary.
  13  Then I thought that would get into too much, but
  14  that's what I was charged to do in terms of the
  15  insertion.
  16            But instead I chose to suggest that we
  17  put it in the preface and add it to the amendment
  18  that had already been added, which stated so that
  19  Medicare beneficiaries -- you know, we said
  20  something about that.  I think Linda started.
  21  Then I just added to that -- can be better served
  22  regardless of race, ethnicity or socioeconomic
  23  status.  And that's a generalized statement
  24  without attempting an insert with limitations.
  25            DR. SOX:  Any objections to the way
   1  this is done?  Then we have one more suggested
   2  change.
   3            DR. BERGTHOLD:  Mr. Chairman, when will
   4  these changes be on the books?  I didn't take
   5  down every one.  Maybe I should be asking Sharon.
   6            DR. HILL:  By next week.  Maybe even
   7  sooner.
   8            DR. SOX:  So Ron, what is it that he
   9  just gave you?
  10            DR. DAVIS:  It's the sentence that I
  11  mentioned earlier about having the opportunity to
  12  review any of the primary sources upon which the
  13  evidence report is based.
  14            DR. SOX:  I'd like to move on now.  I
  15  think we're ready, are we not, Sharon, to have
  16  open public comments before we vote?
  17            DR. KANG:  I actually have one
  18  modification, that we put in a phrase that says
  19  based on feedback from the panels, this is a
  20  living document basically.  This has been
  21  modified.  I just wanted to say maybe it's
  22  feedback from the panels and other stakeholders.
  23  Obviously we have public comment period.  So it
  24  really is maybe the other way to say feedback
  25  from everyone.  It could come from public, could
   1  come from the advisory committee, the Executive
   2  Committee itself.
   3            DR. SOX:  Do you recall where that
   4  language was?
   5            DR. KANG:  It would be the last
   6  paragraph before Evaluation of Evidence, the
   7  section that begins Evaluation of Evidence.
   8            DR. SOX:  We're running out of time
   9  because some of our members are going to have to
  10  leave at 3:30, and I'd like, if possible, to have
  11  as many people here for the vote on this.  So we
  12  will put your suggestion in, Jeff.  Sounded like
  13  everybody was happy with it.
  14            We now have a 15-minute period when
  15  anybody who wishes to make a comment may do so.
  16  In order to assure that there be equitable
  17  distribution of the 15 minutes, I'd like anybody
  18  who wishes to make a comment to please raise
  19  their hand so I'll know how many people want to
  20  make a comment, and then I can decide how much
  21  time each person will be allotted.
  22            In the event that only a few people
  23  want to make a comment, I would hope that they
  24  could keep their remarks short because we would
  25  like to have a whole committee here if possible
   1  for final vote on this document.
   2            DR. KANG:  I apologize.  Just one
   3  procedural issue.  You recall earlier this
   4  morning I actually gave up some time to try to
   5  get on with the meeting.  I have an announcement
   6  I'd like to make with regard to coverage criteria
   7  for the public, and unfortunately I think the
   8  appropriate time would be right after the vote.
   9  I just wanted to alert people that I did want us
  10  to have maybe some closing remarks.
  11            DR. SOX:  Excellent.  We look forward
  12  to those.
  13            So Mr. Northrup is one person who's
  14  scheduled.
  15            Anybody else who wants to make a
  16  comment?  A total of four.  Three minutes each.
  17            Mr. Northrup?
  18            MR. NORTHRUP:  I want to thank you for
  19  this opportunity.  This is about as close to the
  20  last word as anybody outside the government ever
  21  gets on a public policy issue, so thank you very
  22  much.  I do want to thank all of you for what you
  23  are doing for Medicare beneficiaries, and that's
  24  why we're all here.  Before you close, I do want
  25  to also reiterate why what you're doing and why
   1  you're doing it --
   2            DR. SOX:  Excuse me.  I didn't
   3  introduce you.
   4            MR. NORTHRUP:  I'm Steve Northrup,
   5  Executive Director of the Medical Device
   6  Manufacturers Association in Washington, D.C.
   7            Again, I want to point out why what
   8  you're doing and how you do it is so important to
   9  the medical devices community and the patients
  10  we're trying to serve.
  11            A way of a little background, our
  12  association, MDMA, was created in 1992 by a group
  13  of medical technology entrepreneurs to represent
  14  and serve medical technology entrepreneurs.  And
  15  I do want this committee to keep in mind, and you
  16  probably already know it, but please keep in mind
  17  the foundation of innovation in medical
  18  technology is the entrepreneurial sector.  Most
  19  of the innovation in this industry comes from
  20  entrepreneurs, and in fact, I read recently one
  21  of the CEOs of a large medical technology company
  22  said that 60 percent of all the medical products
  23  sold in this country are less than 12 months old.
  24  And that seems like an impossible number, but
  25  that's the nature of innovation in this industry.
   1  It's incremental innovation fostered by
   2  entrepreneurs, entrepreneurs with lots of ideas,
   3  but limited time and limited cash.  And we need
   4  to be sensitive to that, and we talk about the
   5  type and amount of evidence HCFA's going to
   6  require and this committee is going to require
   7  and the amount of time it's going to take to
   8  reach a decision.
   9            And that brings me to the points I'd
  10  like to make briefly about evidence and about
  11  time.  With respect to evidence, I do appreciate
  12  the steps you've taken today to make some of
  13  these guidelines, I think, more reasonable with
  14  respect to evidence.  And ultimately HCFA's
  15  coverage criteria, which Dr. Kang will talk
  16  about, will provide that, quote, unquote, road
  17  map that manufacturers are looking for.
  18            Manufacturers are willing to jump over
  19  a reasonable bar, but if it's unreasonably high
  20  where we can't even see it, a lot of us smack our
  21  heads right into it.  And most importantly for
  22  your purposes, please keep in mind that most of
  23  the advances in medical technology that your
  24  panels will be considering are incremental
  25  advances and don't necessarily require a de novo
   1  review.  So when you're looking at incremental
   2  advances, let's look at the incremental
   3  evidence.
   4            With respect to time, still somewhat
   5  concerned -- and I do appreciate Dr. Kang's
   6  comments along these lines -- that some of the
   7  things you're considering will slow down the
   8  process of coverage decision making
   9  unnecessarily, and that will in turn slow down
  10  the pace of innovation in our industry.  The
  11  government will never be able to keep up with the
  12  pace of innovation in this or any other industry.
  13  That's just the nature of the beast.  But we need
  14  to try to keep the gap between innovation and the
  15  government's pace as small as possible.  And with
  16  respect to the comment that was made earlier
  17  about doing it the right way is the fastest way,
  18  to borrow a phrase, I'd like to say it depends on
  19  what your definition of right is, and we need to
  20  focus on doing it the best way.
  21            I do want to thank you for your time,
  22  and ultimately I'm not asking you to be sensitive
  23  to our companies or their needs or how they
  24  conduct their business.  That's my job and not
  25  yours.  What I would ask you to do is make sure
   1  that your actions and decisions don't hinder or
   2  discourage medical technology entrepreneurs from
   3  innovating because innovation is the key to
   4  improving the health of Medicare beneficiaries.
   5  Thank you.
   6            DR. SOX:  Thank you very much, Mr.
   7  Northrup.  Who's going to speak next?  Yes, sir.
   8  Please introduce yourself.
   9            MR. COOK:  My name is Ken Cook.
  10            MS. LAPPALAINEN:  Do you have any
  11  financial interest in any service?
  12            MR. COOK:  I have no financial
  13  interest.  My name is Ken Cook, and I'm a
  14  facilitator for a cancer support group at the
  15  University of Maryland Medical Center.  I just
  16  want to make a comment on two issues on the
  17  external validity issue and Medicare patient
  18  participation.
  19            Not only are Medicare patients excluded
  20  sometimes from clinical trials because of age,
  21  but because also of the financial problem.  Since
  22  Medicare will not pay for experimental protocols
  23  and since probably most patients or most
  24  beneficiaries of Medicare do not carry separate
  25  insurance, unless they are financially
   1  independent, they're basically precluded from
   2  being in the population that is undergoing a
   3  clinical trial.  So it's a catch 22.  You can't
   4  get into the clinical trials because you don't
   5  have the money.  That's the first item.  And so I
   6  would like to point that out for your
   7  consideration.
   8            The second issue is on the number of
   9  patients involved in any disease study.  If you
  10  are studying prostate cancer, there are many,
  11  many patients available for clinical trials.
  12  There is sufficient research money available.
  13  But if you are a patient with let's say multiple
  14  myeloma, which is less than one percent of the
  15  population, there is very little research and
  16  very little research money available or public
  17  interest in that issue.  And to try to require
  18  the same degree of rigidity in proof and making
  19  sure that the protocols are as perfect as can be
  20  possible may not be appropriate.
  21            So like there is the orphan drug law, I
  22  think that as we consider the various types of
  23  protocols and how they're applicable to the
  24  different groups, the same measures are not
  25  applicable to all.  One size does not fit all.
   1  Thank you ever so much.
   2            DR. SOX:  Thank you very much, sir.
   3            DR. KANG:  Chairman Sox, could I just
   4  respond to the first point?  I think that's a
   5  real issue.  I'm very aware of the IOM report.  I
   6  just wanted to say that I don't think this is the
   7  venue, the MCAC, but I just want to assure you
   8  that the issue on payment for -- clinical trials
   9  is very much on our screen and being reviewed
  10  here at HCFA.
  11            MR. MESKAN:  I'm Tom Meskan, Medical
  12  Alley.  The committee at one point was discussing
  13  its willingness to take comments about tone
  14  and/or substance of the document, and I tried to
  15  listen to the conversation closely, but never
  16  heard a complete resolution of whether you wanted
  17  to accept those remarks that would have any
  18  value, and what's your orientation for us.
  19            DR. SOX:  I think the sense of the
  20  group is that when we put this thing back on the
  21  website in its modified version, it will call for
  22  public comment very much on the spirit that Dr.
  23  Brook suggested of specific wording that we might
  24  change, specific changes in the wording that
  25  might improve the tone.  And it will, of course,
   1  be up to the committee to decide to accept those
   2  suggestions.  But I think that's the sense of the
   3  group.
   4            MR. MESKAN:  As it relates to tone, are
   5  you open to substantive changes or do you feel
   6  that where your document is now is kind of where
   7  it is and yes, it's an interim document that will
   8  be ongoing, but should we bother to spend the
   9  effort to make our points again in perhaps more
  10  compelling ways on substance?
  11            DR. SOX:  I think it would probably
  12  serve our group and your ideas best if you came
  13  back to us with them as we reconsider the
  14  document on a periodic basis.  Given the time, it
  15  probably isn't going to get the attention that
  16  maybe it deserves.
  17            DR. KANG:  I think I did hear Bob say
  18  though -- and I thought it was appropriate --
  19  that substantive changes would be considered, but
  20  then you have to justify why the substantive
  21  changes should be in that item.
  22            DR. SOX:  We want suggestions about
  23  tone and substance, and we'll take them up in due
  24  time, but we won't ignore them.
  25            The last speaker, please introduce
   1  yourself.
   2            MR. LASCHER:  Steve Lascher,
   3  epidemiologist of the Maryland College of
   4  Physicians.  I have no financial affiliation.
   5            DR. SOX:  What organization?
   6            MR. LASCHER:  ACP-ASIM.  Related to the
   7  overhead that was written related to the
   8  generalizability, I just wanted to mention that
   9  statistical power was mentioned, and perhaps in
  10  that respect it wasn't the appropriate term since
  11  statistical power relates to type two error, and
  12  perhaps you were thinking about sample size, and
  13  it might lead to some misunderstanding.
  14            DR. SOX:  Thank you.  It's now time for
  15  the committee to take a vote.  Sharon?
  16            MS. LAPPALAINEN:  At this time Dr. Sox
  17  would call for a motion, and he will be asking
  18  the voting members of the panel to vote
  19  concerning whether the report of the subcommittee
  20  should be ratified or ratified with modifications
  21  or not ratified.
  22            For today's panel, a forum is present,
  23  and the voting are Dr. Thomas Holohan, Dr. Leslie
  24  Francis, Dr. John Ferguson, Dr. Robert Murray,
  25  Dr. Alan Garber, Dr. Michael Maves, Dr. Frank
   1  Papatheofanis, Dr. Ronald Davis, Dr. Daisy
   2  Alford-Smith and Dr. Joe Johnson.  Dr. Robert
   3  Brook is absent.
   4            The panel vote may take one of three
   5  forms, ratification with no other modifications,
   6  ratification upon condition, for example,
   7  resolution of some clearly identified
   8  deficiencies which have been cited by you or by
   9  the HCFA staff.  Examples of deficiencies could
  10  include resolutions of some of the questions of
  11  wording or issues that you believe are necessary
  12  or you would like to see implemented.
  13            If you believe that modifications are
  14  necessary, then your recommendation should
  15  address the following points; the reason or
  16  purpose for the modification and the information
  17  that's required to change it.  And for
  18  nonratification, if you believe that the
  19  subcommittee report should not be ratified, we
  20  ask that you state for the record your reasons
  21  why the report should not be ratified and to
  22  identify those measures that should be taken in
  23  order for you to ratify it in your opinion.
  24  Thank you.
  25            DR. SOX:  Sharon, am I correct in
   1  saying that the only people that can participate
   2  in the discussion now are voting members?
   3            MS. LAPPALAINEN:  Yes.
   4            DR. SOX:  I've asked Ron to prepare a
   5  motion, and I'll read it on behalf of him.  Then
   6  there can be a second, then there can be an
   7  opportunity for discussion, and then amendment.
   8            Motion, that the Executive Committee
   9  approve the subcommittee's report and
  10  recommendations as amended and that the Executive
  11  Committee revisit the report and revise it as
  12  needed in response to comments from panel members
  13  and the public.
  14            So that's now open for a second.
  15            DR. GARBER:  Second.
  16            DR. SOX:  Second?
  17            DR. FERGUSON:  Second.
  18            DR. SOX:  Is there a discussion or
  19  modification?
  20            DR. FERGUSON:  The only modification
  21  that I would recommend on that would be to state
  22  the document be as it's approved, that it be used
  23  as an interim document so that HCFA could move
  24  forward in their process, that it be used as an
  25  interim document, recognizing that it is dynamic.
   1  And I would suggest that with the comments that
   2  we're getting from the public and from the panel
   3  members, that as part of the two-day meeting that
   4  we have scheduled next, that we make this an
   5  agenda item to revisit, at least at some point
   6  during that two-day meeting, part of the comments
   7  on this document.
   8            DR. SOX:  Why don't we get the wording
   9  up there.  And then it would be nice, if we could
  10  get the wording up there, then you can suggest
  11  how to --
  12            DR. FERGUSON:  It's essentially the
  13  same thing except that it would be approved as an
  14  interim document would be the only other addition
  15  with that and that we specifically make it
  16  revisited in the two-day meeting that's planned
  17  next.
  18            DR. GARBER:  I guess I have a
  19  question.  I agree with everything you said, but
  20  I take Ron's wording as meaning that it's interim
  21  when he says it should be revised and revisited.
  22  Is that acceptable?
  23            DR. FERGUSON:  As long as that's
  24  understood, yes.  I have no problem with the word
  25  interim not being in there as long as it's
   1  understood that HCFA's got something they can
   2  move forward with now as part of the process
   3  rather than having to wait.
   4            DR. SOX:  Would you like to say
   5  something to the effect of a new sentence perhaps
   6  that the panel shall consider possible revisions
   7  to the document at its next two-day meeting or
   8  something like that?  Would that capture the
   9  sense of what you'd like to have?  That would
  10  make it -- to do it --
  11            DR. FERGUSON:  Sure.
  12            DR. SOX:  -- as an agenda item.  I
  13  guess as the Executive Committee, right?  That's
  14  offered as a friendly amendment, Ron?
  15            DR. DAVIS:  Accepted.
  16            DR. SOX:  Any other comments or
  17  additional amendments?  It's now time for a vote.
  18            All those who are in favor, please
  19  signify by raising your hand.  Hold it up so the
  20  counter can tally the vote.  It's unanimous.
  21            MS. LAPPALAINEN:  Except for an
  22  absentee.
  23            DR. SOX:  We're now going to turn to
  24  hear briefly from Jeff with some announcement
  25  and benediction or something like that.
   1            DR. KANG:  Actually I believe these
   2  comments were made for the public and will be
   3  available outside.  They were meant as opening,
   4  and they're closing now.
   5            I would just, Chairman Sox, like the
   6  opportunity to reinforce and expand on HCFA's
   7  preface to the subcommittee's, which has now been
   8  the adopted subcommittee's recommendations as
   9  amended.  If people have that preface in front of
  10  them, I'd actually like to refer to the third and
  11  fourth paragraphs and just for the record read
  12  them in.  Actually now it's the current document
  13  below.
  14            We view the current document or the
  15  voted-in document as a list of suggested topics
  16  that should be considered and addressed to assure
  17  full and consistent discussion of issues by the
  18  MCAC panels.  HCFA itself will not view this
  19  report as a prescription of criteria by which we
  20  are to determine coverage or even an absolute
  21  standard by which we may judge the adequacy of
  22  evidence.
  23            In short, this document is a list of
  24  suggested topics that the MCAC and its panel
  25  should consider and address in evaluating
   1  clinical evidence in rendering advice to HCFA.
   2  Based on the advice in the record, HCFA will make
   3  its coverage decision.  We are confident that the
   4  MCAC and its process will be an enhancement, not
   5  a barrier -- the new document that you've all
   6  voted in -- not a barrier to the fair and open
   7  consideration HCFA will give to proposals for
   8  coverage.
   9            In summary, I think that we are
  10  interested in how good is the clinical evidence,
  11  what does it say, and what conclusions can be
  12  drawn from it?  And that's really what the
  13  evaluation of evidence is all about.
  14            Furthermore, as I stated in the fifth
  15  paragraph of that preface, we are not interested
  16  in asking the MCAC for advice on cost issues.
  17  You are really the clinical scientific experts,
  18  and that's what we're seeking your advice on.
  19            Finally, with regard to coverage
  20  criteria -- that's in the sixth paragraph here --
  21  we are diligently working on publishing a
  22  coverage criteria to further explain and
  23  interpret what reasonable and necessary means in
  24  discriminating cover from noncoverage services.
  25            I actually do want to point out today
   1  that today's effort deals with what is the
   2  evidence, what does it say and what conclusions
   3  can be drown from it, how we read it and how we
   4  interpret it.  That is distinctly different from
   5  criteria.
   6            Scientific evidence is in many ways the
   7  yardstick or the measuring stick while criteria
   8  is really how far you have to go, whether you
   9  have to go one foot or three feet or ten feet to
  10  get covered.  The evidence really is the
  11  measuring stick or the yardstick.
  12            To further the analogy to our current
  13  situation, HCFA could interpret in a rule that
  14  reasonable and necessary means many things.  For
  15  example, we could interpret it as meaning just
  16  safety, we could interpret it that a service has
  17  to be safe and effective, or we could interpret
  18  it as it has to be more effective, or we could
  19  interpret it as benefits must outweigh the risks,
  20  or it could be interpreted as being cost-
  21  effective, or we could interpret it as being
  22  cost-beneficial.  And there are other variations
  23  of the theme.
  24            The point here is irrespective of what
  25  we finally end up as criteria in the final rule,
   1  it should not change your work regarding what is
   2  good evidence, how do we read it, how do we
   3  interpret it, what does it say, and what
   4  conclusions can we draw from it?  Thus, your work
   5  is distinctly separate from our coverage criteria
   6  and can certainly go on in the absence of the
   7  criteria.  Of course, in the final analysis,
   8  today's work only guides your activity and your
   9  advice, and HCFA will be the final decision maker
  10  of what should be covered or not.
  11            Now, I would like to take this
  12  opportunity to briefly update you with where we
  13  are on the coverage rule.  On a personal note, to
  14  my chagrin, I've now figured out why the agency
  15  has struggled for over ten years to publish a
  16  rule.  However, the good news is that we actually
  17  do have criteria in mind and a framework for how
  18  they would be applied.  However, it does raise
  19  several operational and implementation
  20  questions.
  21            Given what is at stake and the
  22  considerable interest in this rule, I am pleased
  23  to report that we are expecting to publish soon a
  24  notice of intent for rule making in advance of a
  25  proposed rule.  In this notice we will share our
   1  current thinking and framework for coverage
   2  criteria, how it would work, and we would also
   3  raise some of the implementation questions that
   4  we are wrestling with internally.  Such a notice
   5  will provide ample opportunity for the public and
   6  other stakeholders or all stakeholders to have
   7  adequate input and assist us in our deliberations
   8  before we even propose a rule.
   9            And on that, today is not about
  10  coverage criteria.  I thought I'd take the
  11  opportunity to talk to you about coverage
  12  criteria.  But I would like to thank the advisory
  13  committee, the Executive Committee, for all of
  14  your efforts today to deal with, in a consistent
  15  manner for all panels, how we read the evidence.
  16  And I assure you we're working diligently on the
  17  coverage criteria, and I believe that you are off
  18  to a great start with regard to how we read and
  19  interpret evidence.
  20            DR. SOX:  Thank you, Jeff.
  21            Before we adjourn, for the record we
  22  had one absence.  Dr. Brook had to leave a few
  23  minutes early.  He left this note.
  24            I am happy with the report.  I would
  25  like to see the revised Section 6, signed Dr.
   1  Brook.
   2            Is there anything else that we need to
   3  do before we adjourn?
   4            MS. LAPPALAINEN:  Just to conclude
   5  today's panel meeting, I'd like to remind you
   6  that the next meeting of the Executive Committee
   7  is tentatively scheduled for June 6th through
   8  7th, the year 2000.  Please call the HCFA
   9  advisory committee line at 1-877-449-5659, which
  10  is toll free, or for local calls, 410-786-9379,
  11  and specify the Medicare Coverage Advisory
  12  Committee, or you may check our website for
  13  up-to-date information.  And again, I'd like to
  14  thank the committee.
  15            DR. SOX:  Before adjourning, I'd like
  16  to point out that copies of Dr. Kang's remarks
  17  are available on the table outside the door.  We
  18  want to thank everybody on the panel for their
  19  hard work and the audience for their patience.
  20  Thank you.
  21            (Whereupon, at 3:40 p.m. the meeting
  22  was concluded.)
  25 | Department of Health and Human Services | NMEP

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