[House Hearing, 108 Congress]
[From the U.S. Government Publishing Office]
SCIENTIFIC OPPORTUNITIES AND PUBLIC NEEDS: BALANCING NIH'S PRIORITY
SETTING PROCESS
=======================================================================
HEARING
before the
SUBCOMMITTEE ON HEALTH
of the
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED EIGHTH CONGRESS
SECOND SESSION
__________
JUNE 2, 2004
__________
Serial No. 108-131
__________
Printed for the use of the Committee on Energy and Commerce
Available via the World Wide Web: http://www.access.gpo.gov/congress/
house
__________
U.S. GOVERNMENT PRINTING OFFICE
95-440PDF WASHINGTON : 2004
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COMMITTEE ON ENERGY AND COMMERCE
JOE BARTON, Texas, Chairman
W.J. ``BILLY'' TAUZIN, Louisiana JOHN D. DINGELL, Michigan
RALPH M. HALL, Texas Ranking Member
MICHAEL BILIRAKIS, Florida HENRY A. WAXMAN, California
FRED UPTON, Michigan EDWARD J. MARKEY, Massachusetts
CLIFF STEARNS, Florida RICK BOUCHER, Virginia
PAUL E. GILLMOR, Ohio EDOLPHUS TOWNS, New York
JAMES C. GREENWOOD, Pennsylvania FRANK PALLONE, Jr., New Jersey
CHRISTOPHER COX, California SHERROD BROWN, Ohio
NATHAN DEAL, Georgia BART GORDON, Tennessee
RICHARD BURR, North Carolina PETER DEUTSCH, Florida
ED WHITFIELD, Kentucky BOBBY L. RUSH, Illinois
CHARLIE NORWOOD, Georgia ANNA G. ESHOO, California
BARBARA CUBIN, Wyoming BART STUPAK, Michigan
JOHN SHIMKUS, Illinois ELIOT L. ENGEL, New York
HEATHER WILSON, New Mexico ALBERT R. WYNN, Maryland
JOHN B. SHADEGG, Arizona GENE GREEN, Texas
CHARLES W. ``CHIP'' PICKERING, KAREN McCARTHY, Missouri
Mississippi, Vice Chairman TED STRICKLAND, Ohio
VITO FOSSELLA, New York DIANA DeGETTE, Colorado
STEVE BUYER, Indiana LOIS CAPPS, California
GEORGE RADANOVICH, California MICHAEL F. DOYLE, Pennsylvania
CHARLES F. BASS, New Hampshire CHRISTOPHER JOHN, Louisiana
JOSEPH R. PITTS, Pennsylvania TOM ALLEN, Maine
MARY BONO, California JIM DAVIS, Florida
GREG WALDEN, Oregon JANICE D. SCHAKOWSKY, Illinois
LEE TERRY, Nebraska HILDA L. SOLIS, California
MIKE FERGUSON, New Jersey CHARLES A. GONZALEZ, Texas
MIKE ROGERS, Michigan
DARRELL E. ISSA, California
C.L. ``BUTCH'' OTTER, Idaho
JOHN SULLIVAN, Oklahoma
Bud Albright, Staff Director
James D. Barnette, General Counsel
Reid P.F. Stuntz, Minority Staff Director and Chief Counsel
______
Subcommittee on Health
MICHAEL BILIRAKIS, Florida, Chairman
RALPH M. HALL, Texas SHERROD BROWN, Ohio
FRED UPTON, Michigan Ranking Member
JAMES C. GREENWOOD, Pennsylvania HENRY A. WAXMAN, California
NATHAN DEAL, Georgia EDOLPHUS TOWNS, New York
RICHARD BURR, North Carolina FRANK PALLONE, Jr., New Jersey
ED WHITFIELD, Kentucky BART GORDON, Tennessee
CHARLIE NORWOOD, Georgia ANNA G. ESHOO, California
Vice Chairman BART STUPAK, Michigan
BARBARA CUBIN, Wyoming ELIOT L. ENGEL, New York
JOHN SHIMKUS, Illinois GENE GREEN, Texas
HEATHER WILSON, New Mexico TED STRICKLAND, Ohio
JOHN B. SHADEGG, Arizona DIANA DeGETTE, Colorado
CHARLES W. ``CHIP'' PICKERING, LOIS CAPPS, California
Mississippi CHRIS JOHN, Louisiana
STEVE BUYER, Indiana BOBBY L. RUSH, Illinois
JOSEPH R. PITTS, Pennsylvania JOHN D. DINGELL, Michigan,
MIKE FERGUSON, New Jersey (Ex Officio)
MIKE ROGERS, Michigan
JOE BARTON, Texas,
(Ex Officio)
(ii)
?
C O N T E N T S
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Page
Testimony of:
Fauci, Anthony S., Director, National Institute of Allergy
and Infectious Diseases.................................... 21
Volkow, Nora D., Director, National Institute of Drug Abuse.. 26
von Eschenbach, Andrew C., Director, National Cancer
Institute.................................................. 23
Zerhouni, Elias A., Director, National Institutes of Health.. 10
(iii)
SCIENTIFIC OPPORTUNITIES AND PUBLIC NEEDS: BALANCING NIH'S PRIORITY
SETTING PROCESS
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WEDNESDAY, JUNE 2, 2004
House of Representatives,
Committee on Energy and Commerce,
Subcommittee on Health,
Washington, DC.
The subcommittee met, pursuant to notice, at 2:10 p.m., in
room 2123, Rayburn House Office Building, Hon. Michael
Bilirakis (chairman) presiding.
Members present: Representatives Bilirakis, Greenwood,
Shimkus, Pitts, Rogers, Barton (ex officio), Brown, Waxman,
Stupak, Green, Strickland, and Capps.
Staff present: Chuck Clapton, majority counsel; Cheryl
Jaeger, majority professional staff; Jeremy Allen, health
policy coordinator; Eugenia Edwards, legislative clerk; and
John Ford, minority counsel.
Mr. Bilirakis. The hearing will come to order. Good
afternoon. Today's hearing, which we have entitled ``Scientific
Opportunities and Public Needs: Balancing NIH's PrioritySetting
Process,'' is the fifth hearing that this subcommittee has held
during this Congress in order to highlight research activities
at the NIH. These bipartisan hearings have educated members and
others about the work that the NIH is doing so we can access
how to help the support and research entity better meet its
stated mission.
In many respects all of the hearings that we have
previously conducted have been leading up to today's hearing.
Now that members understand hopefully what the NIH is doing, we
need to understand how they choose what research to conduct and
how that research is funded.
The NIH is the world's leader in conducting important
research that will unlock critical information and lead to
discoveries beneficial to patients suffering from many
diseases.
Over the years the NIH has seen significant increases in
its funding and the development of many new programs.
What began as a one-room laboratory of hygiene in 1887 now
consists of 27 Institutes and Centers that have been created in
response to legislative or executive decisions.
When an institute is created, it has typically been
provided a separate annual budget from Congress. With this
individual budget comes the responsibility of setting
priorities and making budget decisions within the Institute's
domain.
I am very interested, as we all are, I think, in hearing
from today's witnesses about how they balance setting
priorities in their institute and then how they coordinate
those decisions across the entire NIH.
The priority setting process at NIH and within every
individual institute has drawn questions, God knows, from
Members of Congress as well as patient advocacy groups and
others. I believe that much of this criticism has arisen
because the priority setting process is extremely complicated,
especially the grant approval process, and because NIH lacks
transparency in many of their decisionmaking procedures.
Hopefully today's hearing will give members an opportunity
to really understand what criteria is used to determine which
grants are funded and why.
To address these issues, we have an excellent panel of
witnesses before us. I would like to welcome them.
First, Dr. Elias Zerhouni, Director of the NIH, who is here
today. Doctor, you have been so gracious with your time over
the past year. You have been here before us, what, the second
or third time, I think, over the past few months, and we
certainly appreciate all of your efforts to assist the
subcommittee's work on NIH.
In addition to Dr. Zerhouni, we have three very
distinguished institute directors: Dr. Anthony Fauci, who has
been the Director of the National Institute of Allergy and
Infectious Diseases since 1984. Dr. Fauci, we look forward to
hearing about your experience over these years.
Dr. Andrew von Eschenbach, the Director of the National
Cancer Institute; and Doctor Nora Volkow, the Director of the
National Institute of Drug Abuse, will also be able to discuss
the unique challenges they face. Thank you both for coming, and
I know that all of you have had to rearrange your schedules to
be here this afternoon, and you are some of the busiest people
in our world, and we certainly appreciate you taking the time.
In addition to hearing your individual testimony, I would
also like to initiate a dialog. I would like to see a dialog
between and among each of you as to how you have been working
together to ensure that the grants elected for funding measure
up in importance to not only your institute but the entire NIH
and the degree that you have been effective in your
collaborations.
Again, thanks for being here. I know that we have all been
very, oh, sometimes quizzical, if you will, concerned, whatnot,
in terms of how the NIH allocates their resources for research.
And I would hope that we could keep our opening statements as
brief as possible so we can give these gentlemen and lady an
opportunity to explain all of that to us.
I would now yield to the ranking member of the
subcommittee, the gentleman from Ohio, for an opening
statement.
Mr. Brown. Thank you, Mr. Chairman. I especially appreciate
Dr. Zerhouni being here and his terrific work, the terrific
work of all three of you on the panel. And I want to single out
Dr. Fauci for his excellent work on infectious disease and what
that means for Americans, and especially in the developing
world, especially on tuberculosis and malaria and all that you
have done that way.
I think all of us receive letters every day from
constituents urging support for specific--to deal with specific
illnesses, for cancer research, Alzheimer's, diabetes,
arthritis, lupus, cystic fibrosis. Every one of these health
conditions and thousands of others compromise equality and
length of our constituents' lives. It is incumbent upon Members
of Congress to ensure that NIH resources are allocated in a
manner that is reasoned, that is efficient and fair, but this
responsibility doesn't exist in a vacuum. We have a requisite
obligation to ensure ample funding overall for NIH.
A memo recently leaked to the press indicates that the
President plans to cut $600 million from NIH in 2006 to make
room for his continued request for tax cuts. Let me place that
in context. We have been increasing NIH funding. Mr. Bilirakis
has played a major role in the doubling of NIH funding, as we
all have bipartisanly, but we have been increasing funding by a
billion or so each year.
I am sure that, Dr. Zerhouni, that these cuts didn't come
from your desk, and I don't believe the Chairman or my fellow
colleagues on either side of the aisle would support that kind
of request from the President to make these kinds of cuts in
the National Institutes of Health, but we need to be aware that
NIH cannot evolve without the resources to do so. Prioritizing
research doesn't mean anything if you can't fund it, obviously.
You all don't have an easy job, nor does Congress. Both of
us walk a fine line in terms of how much influence to exert on
the general direction of medical progress and the priority
given to researching various diseases.
Should we invest in diseases that are most prevalent, the
most deadly or disabling to Americans, to the poor in the
developing world, or for those which have the greatest chance
of finding a cure? Should we focus more dollars on the here-
and-now concrete answers to concrete questions or in paradigm
shifts such as those represented by human genomics?
As a rule, I think most of us, and I will speak obviously
for myself, have tried to steer clear of any effort by Congress
to compromise the flexibility NIH has to allocate the tens of
billions of dollars it received. However, a few years ago I
made an exception. I raise the example here because it
illustrates, I think, three points:
One, that it is in fact important for NIH to revisit and
refind a way that allocates funding on a regular basis.
Second, that Congress as a representative of the public
must continue to play an oversight role and challenge NIH to
respond to public concerns about the agency's funding
decisions.
And third, what if NIH is not sufficiently responsive to
the public, it doesn't matter how the agency sets its
priorities, because those priorities will almost there, by
definition, be wrong.
In 2001 I cosponsored legislation that required NIH to pay
more attention to Duchenne muscular dystrophy. Specifically,
NIH was to expand and intensify research related to Duchenne
and other forms of MD and support centers of excellence that
would foster external muscular dystrophy research. It required
HHS to establish an interagency committee to coordinate all
Federal muscular dystrophy programs and activities.
I joined this effort because--and this is one of the few
times it has happened in a major way I think--it was abundantly
clear that Duchenne has somehow fallen through the cracks at
NIH, despite the fact it is the world's most prevalent
childhood killer. Still today, resources devoted to this
disease represent less than .0005 percent of the NIH budget.
The MD Care Act was signed into law by President Bush in
2001. The CBO scored it at $56 million in new spending over 5
years. My understanding is NIH has barely begun to fund the
research in surveillance programs established under the new
law, and it has been 3 years, and that the CDC surveillance
program is off to a sluggish start.
Congress is very reluctant, as I said earlier, and as Mr.
Bilirakis has said in the past, to be prescriptive in
appropriations report language with NIH, but the fiscal 2004
Labor-HHS appropriations bill calls for full funding in this
fiscal year of three additional centers of excellence.
I understand NIH has solicited proposals for, ``2 or 3
centers,'' and the funding won't be released until well in the
next year. The coordinating committee created under the new law
has met only twice since the bill's enactment.
It is my understanding the NIH has contributed to the
funding of only one clinical trial focused on Duchenne muscular
dystrophy. NIH has publicly stated it has received only three
clinical trial requests for Duchenne over the years. However,
scientists interested in this condition have told us that that
number is not accurate.
The NIH in the past, certainly, and in other diseases is
not just a passive recipient of research proposals. The agency
also obviously solicits proposals from the research community.
It is my understanding that no such solicitation has been made
in regard to Duchenne, even though established clinical trial
and research center networks exist and important research
opportunities have been identified.
I am confident, Dr. Zerhouni, in your dedication and
sincere interest in moving forward in NIH and the public
interest as you always have, but I also contend that NIH can't
ignore concerns raised by the public in Duchenne and other
things.
Mr. Chairman, if the public loses confidence in NIH, it
doesn't matter how we set our priorities and how you set your
priorities, Congress will be unable to secure the funding
ultimately needed to sustain this crucial agency, especially if
the President gets his way to slash spending in this agency.
We mustn't let that happen.
Mr. Bilirakis. I thank the gentleman.
The chairman of the full committee, Mr. Barton, good to
have you.
Chairman Barton. Thank you, Mr. Chairman. And I have a
formal statement that I would ask unanimous consent that it be
in the record.
Mr. Bilirakis. Without objection, it will be.
Chairman Barton. I am going to speak extemporaneously,
because I want to try to be very clear. This is the fifth
hearing that this subcommittee has held on the structure or the
goals of NIH, and Dr. Zerhouni has been cooperative in all of
these hearings, and I want to thank you.
Today we want to look at the mission statement of how NIH
sets its priorities. That is the official title, and I want to
thank our three institute directors for coming.
I had lunch with Dr. Von Eschenbach not too many weeks ago,
with former Congressman Archer, and it is good to see you
again.
But our oversight subcommittee is also holding hearings on
NIH, and the oversight function of this committee, and the
Congress is to kind of serve as a watchdog, and so we are kind
of on a dual track here. We are in the oversight function
looking at the way certain things are being done, and Dr.
Zerhouni is cooperating in that, but this subcommittee is
looking at the general structure of NIH and how we can maybe
reorganize, reprioritize, reform to make it better.
There are certain things that the committee is not at all
concerned about. We are not concerned about your peer review
process. You all have thousands of reviewers that do tens of
thousands, probably, of peer reviews every year, and I think
that is a good system. We are concerned that the NIH as it has
evolved--we now have 27 institutes and centers and they have
kind of grown up serendipitously.
We have one director appointed by the President, Dr.
Zerhouni right now, and he doesn't have a lot of control over
the centers and the institutes. And I would like to see if we
can, on a bipartisan basis, through these hearings come up with
a legislative package to reauthorize NIH. Most of the programs
at NIH have not been reauthorized in a number of years, and
that is a lack of discipline on the committee. That is not a
problem in NIH. That is our problem. We have not reauthorized
your functions, and we have thrown that on the appropriators;
in this case, Chairman Ralph Regula's appropriation
subcommittee.
So what we want to do in this subcommittee, with Mr.
Bilirakis's leadership and Mr. Brown's leadership, is see if we
can't work on a bipartisan basis to come up with some
legislative reforms that make it easier for NIH to do its
function. We are not opposed to the function. We are not
opposed to using the best scientific brains in the world to try
to find cures and treatments for all the many diseases and
afflictions that you folks deal with. But we are also not just
going to turn a blind eye and say, you know, business as usual
is okay, because the dollars are too big and the consequences
are too big. And, quite frankly, the assets at the disposal of
NIH are significant, and if we can channel them in a more
comprehensive, coordinated fashion, we are going to do great
deeds in the years ahead. So that is what these hearings are
about.
So I know it is--you know, we are beginning to see in the
press, because of what is happening on the oversight
subcommittee, you know, that the Congress is out to get NIH
or--nothing could be further from the truth. You know, it is
just the opposite. We want the most effective state-of-the-art
NIH for the 21st century, that gets the biggest bang for the
taxpayer bucks and the private sector dollars that are
coordinated with what NIH does. That is what these hearings are
about, and I want to thank Chairman Bilirakis for holding them.
And our goal is to have an NIH reauthorization package ready to
move through this committee in this Congress.
[The prepared statement of Hon. Joe Barton follows:]
Prepared Statement of Hon. Joe Barton, Chairman, Committee on Energy
and Commerce
Thank you, Chairman Bilirakis, for holding this hearing today. I am
pleased that the Energy and Commerce Committee continues to invest so
much time and energy in reviewing the operation of our most important
public health agencies.
Today's hearing is the fifth in a series that examines different
aspects of the National Institutes of Health. The more I learn in these
hearings, the more concerned I become about the existing NIH priority-
setting process. In particular, I am troubled by the relative lack of
authority possessed by the Director to set priorities and manage the
research portfolio of the entire agency.
Over the years, the organizational structure of NIH has been
arbitrarily expanded. This organizational structure largely determines
the priority-setting process at NIH. What we are here to figure out is
whether or not the current priority-setting structure at NIH is
adequate to meet the nation's medical research needs. Congress needs to
be able to evaluate if funding allocation decisions are made on the
basis of the best assessment of scientific opportunities and equity, in
light of existing public health priorities.
Let me be clear: we are not holding this hearing today to argue
that some of the science conducted by NIH lacks merit. The scientific
peer review process at NIH works remarkably well considering the volume
of grant applications reviewed each year. I'm certainly not interested
in picking and choosing among the thousands of diseases that afflict
Americans, to determine who should be the ``winners'' and ``losers'' of
NIH funding. But to ignore the directions that Congress has already
made in dictating how research priorities are set at NIH would be a
mistake.
Dr. Zerhouni, given the current problematic NIH structure, you
should be commended for your leadership. Your efforts to design an NIH
Roadmap to link all of the research activities at the 27 separate
institutes and centers towards shared research goals is a particularly
important step.
In addition to Dr. Zerhouni, the Committee is privileged today to
hear from three Institute directors, who are also some of the top
scientists in the world. Several of them rescheduled travel and other
commitments to attend today's hearing, which underscores the importance
of the topic. Dr. Fauci, for over two decades your leadership has
helped to control the rapid spread of infectious disease and prepare
our country to respond to potential bioterrorist attacks. In addition,
Dr. Von Eschenbach, and Dr. Volkow are nationally recognized experts in
the fields of cancer care and drug abuse, respectively. I look forward
to today's testimony and again want to thank all of the witnesses for
rearranging their schedules to attend the hearing.
Mr. Bilirakis. The Chair thanks Chairman Barton.
Without objection, by the way, the opening statement of all
members of the committee will be made a part of the record, and
now I recognize Mr. Waxman for an opening statement.
Mr. Waxman. Thank you very much, Mr. Chairman.
The NIH is the most important and successful medical
research organization in the world. It has produced more cures,
more breakthrough treatments, and more hope for millions of
patients around the world than any other group of scientists.
I think we can all agree that Congress and NIH must work
together to set NIH's research agenda and priorities in the
broad sense. Congress, with NIH's guidance, must decide how
much to appropriate to the general research areas covered by
each of the institutes and centers, and of course Congress must
exercise oversight.
After that, however, Congress should step back and allow
NIH scientists to decide what specific research projects will
produce the greatest gains for humanity. I am increasingly
concerned about congressional interference in NIH decisions to
fund specific research grants. In making those decisions, NIH
employs a rigorous and highly respected peer review process.
NIH uses over 11,000 scientific experts, all of which have
had--all of whom have had many years of scientific training and
are recognized in their fields, to staff 170 peer review
panels.
As the members of this subcommittee look into NIH's work, I
hope that we will all exercise self-restraint. In the past,
some Members of Congress have given in to the temptation to
substitute their scientific judgment for that of the peer
review process, and I think that is a very perilous activity.
The fact that social conservatives disapprove of certain
kinds of sexual behavior or drug use cannot be the basis for
deciding whether scientific research on that behavior is worth
funding. Funding decisions must instead be based on whether
such research will or will not help us learn how to stop the
spread of serious diseases and reduce human suffering.
And I am very pleased that Dr. Zerhouni has affirmed both
the scientific importance of research on sexual behavior in his
continuing support for the peer review process at NIH, and I
hope that from this subcommittee that we will have a
continuation of the policy to support a process whereby our
best scientists pursue the research that they have determined
offers the best chance to save many lives.
I know that we are looking at other issues with NIH in the
Oversight Subcommittee, and I am part of that subcommittee, and
those issues that have been identified ought to be dealt with
and we ought to make sure that the NIH lives up to the
requirements in the public interest of transparency and
accountability. But let's understand NIH is too important and
too successful and too valuable for us to in any way interfere
with the important scientific work that it is pursuing.
Thank you, Mr. Chairman.
Mr. Bilirakis. I thank the gentleman.
Mr. Rogers for an opening statement.
Mr. Rogers. Mr. Chairman, I am going to yield for questions
later.
Mr. Bilirakis. Thank you very much.
Mr. Green.
Mr. Green. Thank you, Mr. Chairman.
And, again, I would--like the chairman of the full
committee, I would like to welcome Dr. Zerhouni back to us, and
appreciate your patience.
But I also would be remiss if I didn't welcome my good
friend, Dr. Von Eschenbach, who we miss at M.D. Anderson at the
Texas Medical Center and your research successes you had in all
those years, but I told you earlier I will can our 95 degree
temperature and our 95 percent humidity and bring it to you
here in DC. Just in case you have missed it so much.
But, Mr. Chairman, I want to thank you and our ranking
member for this series of hearings so we can gain knowledge on
the important research NIH does, and I thank you for your
leadership. The work being performed at NIH has proved
invaluable. The groundbreaking research provided a lifeline of
hope to countless Americans living with diabetes, cancer and
AIDS and many other illnesses. As a testament to our support
for NIH, Congress has completed a 5-year effort to double NIH's
funding. Yet as NIH's authorizing committee, it is important
for us to understand how the NIH utilizes this funding, how it
determines its priorities, and which strategies it sets to meet
the goals.
I am particularly interested in NIH's research on diabetes.
I represent a district in Texas, it is over 65 percent
Hispanic, and nationwide 24 percent of Mexican Americans age 45
to 74 live with diabetes. Mexican Americans are also twice as
likely to have the disease as the Anglo population, which is
why diabetes treatment and prevention is so important to not
only myself but to the district I represent.
Diabetes research and treatment cannot be performed in a
vacuum, though, and that is what we know about research in
general. Obesity plays such a large role and genetics and race
play a role. The disease can lead to a host of other conditions
such as kidney failure and cardiovascular disease.
I want to make sure NIH's priority setting process includes
a truly collaborative effort--and I know we had this at other
hearings--with the myriad of institutes that we have involved,
to ensure that we are leaving no stone unturned in the research
to find treatments and a cure for this deadly disease.
Stem cell research also provides us with a great promise in
finding cures for devastating diseases, diabetes, Alzheimer's
and cancer. While it is no secret that NIH's stem cell research
is constrained by the President's policy on the issue, I
appreciate, Dr. Zerhouni, your candor when you recently wrote
that from a scientific perspective, more cell lines may well
speed some areas of human embryonic stem cell research. Stem
cell research offers a tremendous hope to patients with
diabetes, Alzheimer's, Parkinson's and cancer treatment.
Speedy research is exactly what these patients need and
this policy is simply a roadblock along the path of research
and discovery at the NIH that could lead to cures for many
illnesses.
Again, I want to thank the witnesses for being here today
and their effort for a number of years, and, again, Mr.
Chairman, thank you and our ranking member for your leadership
to make sure we continue to have this oversight, and I am glad
to hear the Chairman of the committee has the goal of doing the
reauthorization, so I am looking forward to that.
Mr. Bilirakis. I thank the gentleman.
Mr. Strickland for an opening statement.
Mr. Strickland. Mr. Chairman, I look forward to hearing the
witnesses, and I will forego an opening statement. Thank you.
Mr. Bilirakis. The Chair thanks the gentleman.
Mrs. Capps.
Mrs. Capps. Thank you, Mr. Chairman.
I also want to thank Director Zerhouni, Directors Fauci,
Volkow, and von Eschenbach for making yourselves available to
us today. We appreciate your time and the fact that you are
willing to share your expertise with us.
It is a matter of great pride, I believe, in a bipartisan
way that we have over the past few years doubled the funding
for NIH, and we in Congress often focus on the benefits that
the public gets from the billions of dollars that are devoted
to the NIH. This is a good work that we fund but that you carry
out. There is no doubt that this investment has paid off in
spades.
But there is an issue that I want to bring up today, hoping
that this committee and the NIH could begin to discuss, and
that is public access to the fruits of federally funded
research. It is complex, and I don't have a black or white
answer, but many results of federally funded research are
published only in very expensive journals that most of the
public has no access to. Many universities, libraries and
organizations such as the Howard Hughes Medical Institute, the
Welcome Trust, and the Susan G. Coleman Breast Cancer
Foundation would like to see this research opened up more fully
to the public.
To be sure, there are many questions that have to be
answered before this approach is endorsed by Congress or the
NIH. I believe that this is an appropriate place to carry out
some of this discussion. I hope we can do so in the near
future.
And again I want to address an issue that my colleague Mr.
Waxman has brought up. I have addressed it before, and maybe it
will come up today in some testimony. Some of our colleagues
who have little or no scientific or medical expertise--and
actually that has nothing to do with that--have raised
questions about NIH grants on human sexuality. Congressional
oversight is important, but it is critical, I believe, that we
be very serious about keeping politics from interfering with
science.
NIH was set up to dramatically improve the lives of
Americans by increasing the quality and amount of biomedical
research conducted, and NIH does this job admirably. We here
should not try to micromanage scientists about how to conduct
their research, and we should not engage in witch hunts to
discourage research into particular areas. I believe there is
no question that some Americans engage in self-destructive
behavior. If we want to help them to make their lives better,
we cannot pretend that that behavior does not exist. So we must
come to understand it and its effects on public health in order
that it can be addressed more scientifically and more
effectively.
I believe that is what scientific research is for. The
Congress should not hamper the excellent work of the NIH by
trying to impose any kind of ideology onto science.
So I look forward to hearing the testimony that you are
going to present to us, yield back the balance of my time.
Mr. Bilirakis. The Chair thanks the gentlelady.
I believe that completes the opening statements, and the
Chair is grateful for that so we can move right into the
witnesses' testimony.
I am going to set 10 minutes for each one of you, if I may,
and hopefully you can stay within it. Your written statement
is, of course, part of the record. And hopefully you will have
an opportunity for dialog among yourselves.
Dr. Zerhouni, please proceed, sir.
STATEMENTS OF ELIAS A. ZERHOUNI, DIRECTOR, NATIONAL INSTITUTES
OF HEALTH; ACCOMPANIED BY ANTHONY S. FAUCI, DIRECTOR, NATIONAL
INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES; ANDREW C. von
ESCHENBACH, DIRECTOR, NATIONAL CANCER INSTITUTE; AND NORA D.
VOLKOW, DIRECTOR, NATIONAL INSTITUTE OF DRUG ABUSE
Mr. Zerhouni. Thank you, Mr. Chairman. I am pleased to
appear in front of your subcommittee and I am pleased to be
accompanied today with my esteemed colleagues.
It is an important topic that I think you are addressing,
and that is reviewing the NIH research portfolio and discussing
our priority setting processes. I have submitted written
testimony. What I would like to do is summarize on slides what
I think are the salient components of what NIH does to allocate
resources to the best extent possible to address the disease
burden in our population.
There is no doubt that NIH has been and continues to be at
the leading edge of discovery. For example, in the area of
infectious diseases we identified the SARS virus in less than 3
weeks. In 1985 it took 3 years to identify the HIV virus, and
influenza took 30 years to be identified. This is due to the
investment that Congress and the administration have made over
the years in NIH.
Clearly, we can also see progress being made in other areas
of research besides infectious diseases. This year, for
example, we have now discovered over 12 genes which were never
suspected before to be the cause of mental illness, including
schizophrenia. We are now at the edge or the threshold of
understanding how schizophrenia develops in the human
population, reminding you that schizophrenia in young adults 25
to 44 years of age is one of the main causes of disability.
We continue to uncover basic principles of human biology.
The human genome is just one step in the process of
understanding how all of our molecules and cells are organized,
and we are continuing to make progress in this area at a very
rapid pace, which is being applied across all disease areas.
In addition, I think that life expectancy continues to
increase, and it is the result of many factors. Clearly, some
of the impact of the research of NIH has changed the landscape
of the diseases that we have to deal with and therefore changes
the mechanics and the strategies for priority setting in the
country.
For example, if you look just at the impact of our research
on cardiovascular disease over the past 30 years, it has been
remarkable. Mortality from heart disease and stroke has
decreased by over 50 percent.
How does that translate in numbers? If you looked at
coronary heart disease between 1950 and 2000, and if we had
projected in 1970 what the number of deaths would be in 2000,
it would be 1,329,000 deaths experienced in 2000 if we hadn't
discovered that hypertension was a risk factor, or high
cholesterol was a risk factor, or discovered all of the new
treatments and therapies that we are using in cardiovascular
disease.
So in fact you can say that 815,000 deaths have been
prevented in 2000 because of this 30-year investment in
cardiovascular disease with this rapid decrease in mortality.
What is the impact of that on life expectancy? Obviously
life expectancy increases, but at the same time this acute
disease, which used to kill many of our patients very quickly,
is transferred into a more chronic acute condition. And if
there is one word I can give you as to the scientific
priorities of NIH is this transformation of disease from more
short-term lethal acute diseases to more long-term chronic
conditions, including, for example, cancer where much progress
has been made to the extent that survivorship in cancer is
unprecedented at this time in history, and we have over 9
million individuals who survived cancer in this country.
So when you look at that, you obviously see a changing
environment, and the resource allocations and the budget
allocations have to reflect that changing environment in some
ways.
Just to go quickly over the NIH budget, there are four ways
to look at our budget. First and foremost, you can look at it
by mechanism: Do we fund grants, do we fund centers, do we fund
contracts?
The second is by research area: How much do we fund in
cancer versus infectious disease or pediatric research? The
other is to look at how we fund the structures of NIH that have
been authorized over the years by Congress, the institutes and
centers and offices, and how much funding is in each one of
these institutes.
And obviously we can also look at it in terms of scientific
effectiveness. In other words, what is the likelihood that we
can fund the research enterprise in the country and the
capacity that is committed to finding the treatments and
prevention strategies that we need?
If you look at that this way, I can show you very quickly
that about 85 percent of the NIH budget is spent outside of
NIH. About 10 percent of the budget is spent intramurally here
in Bethesda. We spend about 4 percent of our dollars on
research management and support, which is the administrative
expenditures, and about a billion dollars on activities like
the National Library of Medicine, education, and so on. But the
rest of it is spent on academic institutions, and we support
over 212,000 scientists in the country at over 2,800
institutions throughout the country.
So if you wanted to look at the budget by mechanism, where
is it intramurally, where is it extramurally, by institutes,
you can see the very large range of funded institutes and the
levels of funding.
NCI, my dear colleague Dr. von Eschenbach leads, is funded
at $4.87 billion. NIAID, under the direction of Dr. Fauci, is
$4.4 billion. And you can see the FIC at the bottom right is
the Fogarty International Center, which is our international
operation, $67 million. So you can see a wide range of
allocation of resources for different institutes with different
missions and different scope of missions. And this is the
complexity that you have to deal with as an institution, and
you have to balance that relative to the opportunities in
science and the good ideas that come from our scientists.
America is unique in the world, in that it uses a peer
review system that is fundamentally based on the ideas of
scientists, where investigator-initiated grants come in and we
review them and fund a small percentage--about a third of them
are funded. So if you look at our likelihood of success to be
funded, if you are a scientist in the United States and you
came up with a good idea and you came to NIH, in 1996 your
funding chances would be 28 percent. And then it went up during
the doubling period, all the way to 32 percent. It remained
very competitive. And currently in 2002, 2003, it went to 30
percent, and we project 27 percent for the 2004-2005 period.
Why is it that despite the doubling, the success rate has
remained very competitive? Most of the answer is in this graph.
We have had more and more scientists drawn to biomedical
research because of the long-term commitment and investment of
the American people to medical research. In 1996 we had about
23,800 research project applications that year. In 2003 we had
34,700, a huge increase in the research capacity of the United
States, which is obviously a good indicator that, A, there is
attraction to the field and, B, we remain very competitive in
who we grant and how we grant our research dollars.
When you look at priority setting, what I would like to do
is show you the entire wheel of how priority setting should
occur and occurs in practice. And as the Chairman said, it is a
complex process. It is not something that you can explain with
one parameter and say this is how we allocate our resources.
But at the top of the list is obviously our intent to reduce
both disease burden as we know it today, but also potential
disease burden as may occur in the future.
A good example is Dr. Fauci's investment in biodefense and
the increasing funding in biodefense. I mean, we are doing this
not because there is an existing disease burden. We are doing
it because of a potential disease burden, and other areas can
be illustrated.
So first and foremost, we try to evaluate the disease
burden that we are dealing with through a variety of
mechanisms. CDC has the National Center for Health Statistics.
The National Cancer Institute has a program called
Surveillance, Epidemiology and End Results to track cancer
progress. Other institutes have different mechanisms. We don't
have an established national way of measuring burden for all
diseases; however, we do it for the most important ones.
But let's say now that a disease burden is emerging, like
obesity, for example, as a new threat, or chronic disease,
which we did not experience in the past which are now becoming
very important. How do we then adjust the portfolio, adjust the
strategy?
First and foremost, we need to have, obviously, a public
investment in the area. What is the public investment used for?
First, we have to build scientific capacity. You cannot advance
in the field of research for any one disease if you do not have
the people, the resources, the ideas, the buildings, the
laboratories. And that is the first thing that NIH does. A good
example of that is what Dr. Fauci will talk to you about. Today
he is engaged in building scientific capacity for biodefense
research. They are building laboratories across the country at
NIH and in different areas.
Once that is done, the next step is to obviously build the
research capacity itself in the area of interest. Research in
tuberculosis and malaria, versus cancer, versus other areas,
requires different disciplines and different combinations of
disciplines. That research capacity then hopefully leads to
discoveries and breakthroughs that can be then used as new
opportunities.
So this is where we see scientific opportunities for
therapies occur, and these scientific opportunities then have
to be translated, and sometimes this is where I think a lot of
the discussion occurs. How much emphasis do we place on
scientific opportunity versus why don't we just go and create a
lot of clinical trials and do the translation? And the message
I like to give to everyone is you can't translate a language
you do not understand very well. To the same extent in science,
the timing of translation depends on how well you understand
the disease process and how much progress you have made at that
time and balance the research capacity with the opportunity and
with the ability to translate successfully. Hopefully once we
have translated that to practice, it is what I call the cycle,
lab to life, that essentially underlies every research
enterprise that you could analyze at NIH, and you will hear how
my colleagues do so within their own mission area. And this is
what we need to do also at the NIH level.
So let's talk about how well are we doing relative to the
disease burden. Let me show you an independent study that was
led by Dr. Cary Gross in 1999 where they evaluated the disease
burden, calculated with modern methods of computation of
burden, and the modern method is called disability-adjusted
life years.
Just to give you a simple example of how that works, if you
had a child 1 year old who had a disease and didn't survive,
that child would have lost their life expectancy. So that would
be 80 years of life lost. If somebody is 98 years old and has a
cancer, that is computed really maybe as 2 years of life lost,
depending on the actuarial tables. That is the computation that
epidemiologists make.
When you look at the diseases studied, the red line is
about where the average disease burden versus investment would
be. On the left-hand side is the funding in NIH dollars, and
the horizontal is the disability-adjusted life-year component.
And as you can see, by and large, the investment is around
the mid-life for these diseases. But you will also see
outliers, and question the outliers. For example, you can see
AIDS at the top of the curve, and this is a common question:
Why is AIDS, relative to the current disease burden, higher in
funding than other areas? Well, this is a judgment that is made
not on the basis of existing disease burden, but potential
future disease burden both here and in the world and the
potential impact this may have on the economy of the United
States.
And I will let Dr. Fauci comment on that. So there are
logical ways that we use to correlate, if you will, the disease
burden, the scientific opportunity with funding, but those are
not precise.
As you can see, there are variations across all diseases.
Now, how do we answer your question, which is, how do we
know that NIH has methods that allow it to not only do good
peer review but effectively manage its research portfolio? And
there are three factors there that we take into account:
science, public health requirements, and societal needs. And we
get inputs, as you mentioned, from many, many sources,
including the public.
One of the things I would like to attach to my testimony is
the last report of the Council of Representatives of the
National Institutes of Health who studied the transparency
issue and how can we improve our processes to have public input
at NIH to a greater degree than we have had in the past.
Mr. Bilirakis. Without objection, that will be the case.
[The report appears at the end of the hearing.]
Mr. Zerhouni. Thank you, Mr. Chairman.
A good example of adaptation is the example of obesity
research. So what mechanisms do we have to make sure that
nothing falls through the cracks? Clearly, the burden is going
up. CDC is reporting it now as the second preventable mortality
after smoking. So we have created a trans-NIH task force last
year to review all of NIH's strategies and portfolio. Then the
new plan was developed this year. And just to give you an
example, we decided to increase funding for obesity research by
10 percent, even though the rest of NIH grew by 2.6 percent. So
there are ways for us to use both disease-specific planning.
The second example I can give you is the neurological
institutes have now come together and are in the process of
developing a blueprint for neurosciences, which is another
mechanism where a cluster of like institutes can work together.
And last is a trans-NIH process which we implemented over 1\1/
2\ years ago called the NIH roadmap, which is a way for NIH to
explicitly analyze where it is on the horizon of research and
where it is that we need to make investments.
In this case we decided that the three areas would be
molecular understanding of biological systems, new research
teams and reengineering the clinical research enterprise.
So what are the next steps from my point of view that I
could share with the committee in terms of how do we improve
these processes that are there, that are operating? Can we make
them better? Obviously we can always make them better, and we
welcome all of the work that the committee is doing and that we
are doing in trying to improve the processes as much as
possible.
So how can we improve? One is we think--and all the
directors agree--that we need to create better information
systems to analyze the NIH portfolio of research. In one word,
I think NIH has world-class peer review. We can make some
advances if we invested in the information systems needed to
analyze not just each grant but the totality of the grant
portfolio both within institutes and across institutes.
The second recommendation would be that, just like the
roadmap which I think worked well, we need to institutionalize
a more regular process of trans-NIH priority review and
planning with a common pool of funds.
The issue I think everyone raises is that the rigidity of
the funding mechanisms do not allow institute directors, as
well as the NIH director, to jointly and aggressively fund
emerging areas of research. The fact that we have come together
for the roadmap shows it is possible. I think this is a
process; from my standpoint, I would increase tremendously the
effectiveness of the agency. But it has to also come with a
much more integrated governance and management system that
includes all directors. We have started to do this with a
transformation of how decisions are made at NIH.
And, obviously, we need to measure outcomes and have better
measures. So I wanted to quickly go over the rationale that we
follow, Mr. Chairman, and I would like to have my colleagues
continue the presentation. Thank you.
[The prepared statement of Elias A. Zerhouni follows:]
Prepared Statement of Elias A. Zerhouni, Director, National Institutes
of Health, U.S. Department of Health and Human Services
Mr. Chairman, Members of the Subcommittee, I am Dr. Elias Zerhouni,
the Director of the National Institutes of Health. I am pleased to
appear before you today to provide an overview of the NIH research
portfolio and discuss priority setting.
NIH's mission is to conduct research that will lead to better
methods of diagnosing, treating, preventing, and curing disease. The
research that we support has resulted in improvements in detecting
disease, better therapies, and more effective vaccines.
As one example, our ability to fight infectious diseases is greater
than ever before. Consider how research advances enabled the world to
quickly identify and contain the SARS virus. We will produce treatments
and vaccines for other diseases, such as West Nile Virus, in record
time. We have also developed a single dose, fast-acting experimental
vaccine to prevent one of the most feared viruses of all, Ebola. The
vaccine has proven successful in animals and human trials are under
way.
In the area of mental illness, NIH-supported researchers recently
discovered genes associated with schizophrenia, a tragic illness that
affects 1 percent of the adult population. This research, which brings
us closer to better treatments for this disorder, was cited by Science
Magazine as the number two scientific ``breakthrough of the year'' in
2003.
Much of our progress is attributable to basic research advances
furthering the understanding of human biology. NIH and its
collaborators have sequenced the human genome, one of the greatest
scientific achievements in history. Now we are moving forward with
research into molecules and proteins to gain knowledge leading to new
therapies that will alter the way medicine is practiced and result in
even greater improvements in public health. We are on the cusp of an
era of medical practice that will identify and prevent diseases before
the symptoms appear.
In short, we are living much longer and significantly better as a
result of biomedical research. And while we have come very far, we have
even farther to go. Despite our extraordinary successes, there is still
a great deal we do not know about human biology. In areas where we have
reduced death and suffering, we can do even more. Consider the case of
cardiovascular disease. One of the greatest public health success
stories of the last half-century is the dramatic reduction in mortality
from stroke and heart disease. In the year 2000, the number of deaths
from cardiovascular disease was nearly 40 percent less than we had
projected in the same year. Research identifying risk factors and new
therapeutic interventions to control the risks were largely responsible
for this remarkable lifesaving achievement. Yet cardiovascular disease
still accounts for about 38 percent of all deaths in the United States
every year. If we sustain our research effort, imagine how many more
lives we can save over the next 50 years.
The Nation remains committed to the support of biomedical research.
Congress and the President appropriated a little more than $28 billion
to NIH in FY 2004, and the President's Budget Request for FY 2005 is
$28.7 billion, a $729 million or 2.6 percent increase. Of the funds
appropriated in FY 2004, an estimated $5.6 billion will be spent on
cancer research and $4.9 billion on neurosciences research. We expect
to spend $3.6 billion on research affecting women's health and $3.2
billion on pediatric research. We plan to spend $1.6 billion on
biodefense research, and $2.4 billion on cardiovascular research.
Another major investment is aging research, which will receive $2.3
billion. Vaccine development research will total $1.4 billion.
There are several ways to view the NIH budget. The most relevant
picture is the snapshot of the individual Institute and Center budgets
because Congress appropriates funds on the basis of allocations to
these 27 organizations. In FY 2004, their budgets range from $4.7
billion for the National Cancer Institute to $65 million for the
Fogarty International Center.
Another common way to view NIH's budget is by the funding
``mechanisms'', such as grants, contracts, cooperative agreements, or
in-house programs. About 80 percent of the NIH budget is awarded to
extramural research institutions throughout the United States. The
largest grant mechanism is our Research Project Grants, which comprise
54 percent of NIH's budget in FY2004, or $15.1 billion. Another
important funding mechanism, research centers, supports groups of
investigators working on common disease or research areas. This
mechanism accounts for 9 percent in FY 2004, or $2.6 billion. Another
vital mechanism is our research training programs, which comprise 3
percent of our total budget, and will help ensure that we will have the
skilled workforce needed in the future to continue making progress in
research. Our intramural research program accounts for 10 percent of
our total budget, or $2.7 billion.
Our budget can also be viewed by funding of specific diseases,
several of which I have already mentioned. Research projects can often
contribute to advances in multiple diseases; thus, our estimates of
research expenditures by disease necessarily contain overlap and are
not mutually exclusive.
The public can also view the NIH budget from the perspective of the
success rate of grant applications. However, looking at the budget from
this perspective results in many misleading conclusions. Success rate
alone is not indicative of the number or size of grants being funded;
the number or quality of grant applications received in a given year;
and research mechanisms that NIH may be funding other than grants. In
addition, our estimates of the projected number of applications
submitted and the number actually awarded have undergone significant
revisions from earlier predictions, creating some difficulties in
making grant budget projections.
Over the decades, the allocation of NIH dollars has adapted to
public health needs. In fact, much of our spending focus recognizes the
shift in disease burdens that has taken place in recent decades. For
example:
NIH is increasingly targeting chronic diseases, which have overtaken
acute conditions as the Nation's leading health problem.
We are responding to a new epidemic--obesity--which, if continued
unabated, threatens to undermine our progress against disease
in similar ways to tobacco use. Part of this response has been
a proposed trans-NIH funding increase of 10 percent allocated
for obesity research in the FY 2005 President's Budget.
We are quickly expanding our research efforts to protect the Nation
against lethal bioterrorist acts by identifying the threats and
developing vaccines, diagnostics, and therapeutics to address
them.
We are committed to NIH's infectious disease research on problems such
as AIDS, SARS, West Nile Virus, influenza, malaria and
tuberculosis.
We remain committed to research on other long-standing problems, such
as the health disparities that exist among racial, ethnic, and
disadvantaged populations.
As the most influential force in the U.S. biomedical research
community, NIH exercises its leadership by continually surveying public
health needs and the scientific landscape to identify new biomedical
research areas that require attention. Simultaneously, we search for
emerging scientific opportunities. To maintain the vibrancy of our
scientific enterprise, NIH also actively supports strong basic and
clinical research training programs. Our programs are unique in both
igniting and complementing private sector research and development
efforts.
NIH undertakes studies for which the risks are too high or the
financial incentives too low to attract private investment. Tailoring
therapies for the special needs of vulnerable populations and
evaluating treatments for rare diseases are other NIH-led
investigations where the intervention of a public agency is essential.
With the massive responsibility of advancing knowledge across such a
wide landscape, whenever possible NIH marshals efforts of academic
institutions, industry, research organizations, disease foundations,
and patient groups to maximize its efforts.
This focus on vulnerable populations and rare diseases is an
essential part of NIH's mission, and must be a component of priority
setting. Instilled in all of us at NIH is the human dimension that
drives us to help the helpless, whether their suffering is from a
disease that affects millions or a disease that affects only a few.
This is why I became a physician, and it is why I was eager to come to
NIH.
To maintain a research portfolio that balances public health needs
and scientific opportunities, NIH seeks input through multiple
channels, including the Advisory Committee to the Director and the NIH
Council of Public Representatives. NIH uses an unparalleled peer review
system involving its Center for Scientific Review as well as separate
vetting programs within each Institute and Center. These programs are
part of a two-tiered system of advisory bodies and specialized review
committees that guarantees funding of the best applications from among
the nearly 50,000 research and training applications reviewed annually.
NIH's priorities are driven, in part, by the ideas and
opportunities presented to us through the grant applications we
receive. By placing most of our resources in investigator-driven
research, NIH ensures that federal dollars track the latest science.
But allowing the scientific community to drive research is only one
factor in how NIH sets priorities.
Determining research priorities is a complex, multifaceted process.
One cannot easily quantify the various factors and questions that
surround priority setting at NIH. Some of the variables in the
determination of resource allocations include public health needs and
the burden of disease, scientific opportunities, the quality of
research proposals, the experience of applicants, and the ability to
sustain research through adequate staffing and infrastructure. These
factors are often lost in the public debate about NIH funding, in which
the discussion is simplified by focusing attention on apparent funding
inequities between the toll of certain diseases and the amount spent on
research about those diseases.
Although burden of disease should not stand alone as a factor in
setting priorities, there are indications that NIH funding generally
tracks disease burden data. A study published in the New England
Journal of Medicine five years ago concluded that there is a
significant--although not absolute--correlation between the burden of
disease and NIH funding. The genome project, development of
instrumentation, training in clinical research, and new developments in
basic science all have high values in the treatment of specific
diseases, even though they lack a disease-specific orientation.
Nonetheless, the study is evidence that NIH resources reflect the
burden of disease in measurable terms.
Do these successes mean we are doing everything we can to ensure
that the NIH research portfolio is balanced, is focused on the most
urgent needs, and is based on irrefutable data? Let me answer that
question with the following observation: Great organizations can
maintain greatness only by continuous reassessment and adaptation.
I believe that we cannot be static. NIH must enhance the current
process for determining priorities and allocating resources as part of
a balanced research portfolio across the Agency and within each
Institute and Center. The system of funding research by allocating
resources directly to disease, organ, or special-population-based
Institutes and Centers has served NIH and the public well. We plan to
continue this approach to funding programs at the Institute and Center
level. But science is changing, driven by new technologies and
discoveries. Modern research is often best conducted by teams, which
may include mathematicians, chemists, physicists, engineers,
bioimagers, computer scientists, behavioral scientists, and physicians,
and which may cut across the expertise of many different NIH Institutes
and Centers. Several fertile areas of research--genomics, proteomics,
molecular engineering--serve all fields of endeavor and cannot be
pigeonholed according to specific diseases.
As the Institute of Medicine noted last year in its review of the
structure of NIH, consideration should be given to refinements in the
priority setting process and the management of our portfolio. There is
a particular need for new and sustained approaches to evaluating NIH's
crosscutting science. While maintaining the support for existing
Institute and Center research programs, I think we should consider ways
of using resources that may not be controlled by a single Institute or
Center, but by a priority-setting process with input from outside and
inside NIH. I am encouraging each Institute and Center to evaluate
their own priority setting and portfolio management processes and seek
best practices or other methods of enhancing their systems. I have also
asked the Institute and Center directors to strive to pool resources,
as they have done in research areas such as obesity and neuroscience.
An expanded approach to priority setting would enable NIH to ensure
balance in our research portfolio, identify appropriate cycles of
change, maintain proper turnover rates for grants and provide much more
accountability to Congress and the public. Under such processes, we
would identify crosscutting research that requires common investments
from the various NIH Institutes and Centers. This approach must include
a regular horizon scan of all research so that we can have sufficient
information to manage the NIH research portfolio.
Two years ago, soon after I arrived as the Director of NIH, we
convened a summit of the Nation's scientific experts to determine
obstacles to the advancement of research and methods to overcome them
that could not be addressed by any single Institute or Center. Teams
comprising the NIH's leadership, working with their counterparts in the
extramural scientific community, discussed new ideas. From these
deliberations, the NIH Roadmap emerged. The Roadmap is focused on three
goals: Identifying new pathways of discovery; Building the research
teams of the future; and Re-engineering the Clinical Research
Enterprise. As a modest but significant step forward, the Roadmap is
supported by voluntary funding from all of our Institutes and Centers,
with the goal of supporting research that will benefit all NIH programs
and research into multiple diseases.
As I said, the Roadmap is a modest attempt at progress. It has an
initial investment of less than 1 percent of NIH's total budget. The
Roadmap is an example of a better-integrated mechanism for priority
setting at NIH. My expectation is that we will build on the Roadmap,
and it will serve as a model for future determinations of resource
allocations.
In summary, I believe the confidence of the American people in NIH
to lead biomedical research has been and will continue to be deserved.
Our processes for identifying priorities and ensuring sound science
have worked well. But reassessment and adaptation should occur and lead
to a priority setting process that has greater public input, is more
transparent, and lead to a research portfolio that will keep NIH at the
leading edge of biomedical research.
I intend for the process to contain the following essential
elements:
A transparent process characterized by a defined scope of review with
broad input from the scientific community and the public.
A solid database of information, including uniform disease coding and
accurate, current and comprehensive information on burden of
disease.
An institutionalized process of regularly scheduled evaluations based
on current best practices to be used by Institutes and Centers.
The ability to weigh scientific opportunity against public health
urgency.
A method of assessing outcomes to enhance accountability.
Thank you for the opportunity to testify. I will be pleased to
answer your questions.
[GRAPHIC] [TIFF OMITTED] T5440.001
[GRAPHIC] [TIFF OMITTED] T5440.002
Mr. Bilirakis. Thank you very much, Doctor. Your general
but very well-done presentation, I think, sets up all the other
three, so I have allowed you to go well over your 10 minutes'
time.
Dr. Fauci, please proceed with your statement.
STATEMENT OF ANTHONY S. FAUCI
Mr. Fauci. Thank you very much, Mr. Chairman, and thank you
for giving me the opportunity to discuss with this committee--
and thank you, committee members, also--the priority setting
process of an individual institute that in some respects
represents all of the institutes but in many respects is unique
because of the mandate of our mission to respond to emerging
threats of infectious diseases.
On this first poster, as you see on your right, the mandate
for the NIAID, the National Institute of Allergy and Infectious
Diseases, as Dr. Zerhouni pointed out, the second largest
institute with a budget of about $4.4, $4.5 billion, is
research in immunology, microbiology and infectious diseases.
Now, if you look at the red arrows, these are the areas
that we are responsible for. We are responsible for diseases of
the immune system, infectious diseases in general, and then
there are other issues that are thrown upon us by events. For
example, the HIV-AIDS epidemic, emerging microbes, such as the
threat of a pandemic flu and, most recently, biodefense. We set
our priorities, just as Dr. Zerhouni mentioned, by the delicate
balance that we continue to fine-tune because it is a dynamic
process involving the scientific opportunities as well as the
public health needs.
I would like to point out that both of those can change
dramatically, because sometimes scientific breakthroughs create
opportunities that just a year or 2 ago we didn't have, and
sometimes public health needs like SARS comes along that you
had no way of predicting.
So let me show you the history, the funding history of this
institute, and how it has dramatically grown over the last
several years. Not only because of the doubling of the NIH
budget--because the scientific opportunities in immunology and
infectious diseases are rather dramatic--but also as I
mentioned, the HIV epidemic, the emerging and reemerging
diseases like SARS, and then finally the rather dramatic
increase that we experienced with the biodefense responsibility
that has been given to us to develop countermeasures.
Let me just show you the dramatic metamorphosis of an
institute that had to take place in the context of very serious
continuing priority setting. Let me go back now to 1980 when
things were, as we would say, stable. We had 60 percent of what
we did with infectious diseases, about 40 percent in
immunology, and that includes transplantation, asthma, and
others. The budget, looking at our budget now, was relatively
small, about $215 million.
Then let's fast forward to what we essentially say is the
middle of the HIV epidemic. You know, we started to get
significant funding right at the beginning of my directorship
in the early 1980's. But let's take 1999, where AIDS, just as
Dr. Zerhouni said, was something that was out of control. It
isn't necessarily the numbers that we have now but the numbers
that might occur. So it grew in a way that was in many respects
disproportional if it crowded out other areas; but because of
the increases in funding, the other areas, which we refer to as
``non-AIDS,'' also grew. But if you will look at that, more
than half of the institute then was AIDS, half non-AIDS.
September 11, 2001, the anthrax attack, and biodefense, and
here's what you have now; the institute in 2004, the current
fiscal year, where it is about roughly one-third each of
biodefense, AIDS and non-AIDS, non-biodefense.
Let me just spend a moment about emerging and reemerging
infections. This is a slide that is a favorite of mine because
I show it at many congressional hearings, as I am doing today,
and the reason I like this slide is because I change it each
year with one, sometimes two, and sometimes three additions.
And what it tells us is the dynamic nature of how we need to
prioritize. And, again, I gave you some examples. HIV-AIDS;
West Nile in New York, when that was well off our radar screen
many years ago; the ever-present threat of a pandemic flu that
we had to address this year with the cases in Vietnam and in
Thailand of bird flu jumping from a bird to a human. If it
developed the capability of going human to human, we would have
had to move very quickly. And in fact, we did. We made that
prioritization right in the middle of a fiscal year.
Next.
So what is this prioritization process that I talked about?
Well, as alluded to by Dr. Zerhouni, it is, again, a very
dynamic process with input, from scientists particularly, to
help us with understanding the scientific opportunity, but also
involving lay public, the administration as well as the
Congress, which is very sensitive--and we respect that--to many
of the needs of the constituency. So we take that into account.
We have a number of processes. We have meetings. We have
continual back-and-forth with blue ribbon panels. We have our
council. But what I instituted about 15-plus years ago was two
annual retreats, one a program retreat and one a policy
retreat, in which we continually reevaluate not only the new
initiatives--and I think this is important--but we do what Dr.
Zerhouni referred to as a programmatical portfolio review, so
that we don't get locked into something that is just getting
funded because we have traditionally funded that.
That ultimately leads to the priority setting and the
strategic planning and then the initiatives. What are
initiatives? Initiatives are requests for applications or
steering the field in a certain direction. We had to jump-start
biodefense. There weren't a lot of people out there that were
just dying to get into biodefense research. We had to cultivate
the infrastructure, both human capital as well as physical.
This is the fruits of that process, and this is available
on our Web site, but let me just point it out to you because I
think it is an example of how we have been able to move quite
rapidly. In the process of understanding that we were going to
assume the responsibility for biodefense, we put together a
strategic plan with input from many, many of our scientific
colleagues, not only NIH-funded colleagues, but colleagues in
the military and colleagues in other arenas.
We then developed the strategic plan-related research
agenda for the Category A and the Category B agents, and then
last summer we published our first progress report, and just a
couple of months ago another progress report; together they
encompass the Category A and B and C agents. Again, to
underscore what Dr. Zerhouni said, this required our building
the kind of infrastructure that years from now will allow us to
make use of the scientific opportunities, but also to create
scientific opportunities, which again, is a dynamic process
that we continue to fine-tune.
Finally, your staff had asked me to address an issue that I
briefed them on when they came to visit us at the NIH, and I
want to spend the last minute or so on that. And that is again
an example of the spectrum of going from fundamental basic
research to the expanded paradigm for the NIH, namely assuring
that we don't have a dead end with a very interesting
observation that doesn't get translated into something for the
public.
So if you look at basic research on the far left and look
at where we want to go, and that is, we want countermeasures
for biodefense, we want diagnostics, therapeutics, we want
vaccines, we want the same thing for HIV-AIDS, and we want it
for things like SARS.
We must take the initiative, and we are doing that now with
our prioritization, and pushing the process more toward the
preadvanced development so that we can meet industry halfway or
beyond.
On the one hand, we have created incentives. Let's take
biodefense as an example, with Project BioShield, with a secure
source of funding to buy products that we engage very heavily
in the concept development and basic research. Now we find in
our analysis of our budget--and we have had intensive
discussions with Dr. Zerhouni on this--now that the pipeline
for many things that we invested in basic research are getting
robust, we need to push that process forward. So we need to
reprioritize now the balance between the fundamental basic
research portfolio and how we push that to development so that
we wind up meeting the needs of the general public which have
put in our trust the money for the research that we are doing.
So this is a process that I think is an example of how the
NIH in a dynamic way continues to evolve to meet the
challenges, be it challenges of fundamental diseases that have
been around for a very long time, or the unexpected, like SARS,
like AIDS, like biodefense.
So I will end my comments there, Mr. Chairman, and be happy
later to answer any questions. Thank you.
Mr. Bilirakis. Thank you so much, Doctor. Fascinating.
Dr. von Eschenbach.
STATEMENT OF ANDREW C. VON ESCHENBACH
Mr. von Eschenbach. Thank you, Mr. Chairman and
distinguished members. It really is not only a great privilege
for me, but an important opportunity to address this
prestigious committee on scientific opportunities and public
needs and balancing those priorities.
As we sit here today, one American every minute is dying of
cancer. More than a half a million people will die from this
disease this year, and more than 75 percent of families are
affected. One in two men and one in three women will be told
during their lifetime that they have cancer.
Congress recognized the horror of cancer and the need to
make the conquest of cancer a national priority in 1971 when it
passed the National Cancer Act, which authorized the NCI
Director to build and lead our Nation's cancer program. Thanks
to the wisdom of Congress and your continued support, I am
pleased to follow up on the efforts of my predecessors and
report to you that scientific progress is now impacting on the
greatest public health concern of the American people, the fear
and problem of cancer.
During the past decade, for the first time ever, we have
seen mortality rates from cancer decline. This has been
especially true for the most prevalent cancers, lung, breast,
prostate, colon and rectal. In 1971, there were only 3 million
cancer survivors alive in the United States. As Dr. Zerhouni
indicated, that number today is almost 10 million. But the
greatest progress is yet to come.
Progress in cancer research that has been made possible by
the authorizations of the National Cancer Act of 1971 and the
continued appropriations provided by Congress has really
created an opportunity both scientifically as well as with
regard to the delivery of care that is really at this point
transformational. It is now making it possible for us to
envision a future in which no one will suffer and die as a
result of cancer.
The National Cancer Institute is committed to continuing to
fulfill the promise of bringing that reality about. We can do
that now, and we have established a goal of eliminating the
suffering and death due to cancer and making that a reality by
2015 because we now are beginning to understand cancer as a
disease process. We now recognize that there are steps at the
genetic, molecular and cellular level that are responsible for
our susceptibility to cancer, that are responsible for the
early premalignant changes that occur, and then those processes
continue to result in the overt development of a tumor, and
then that tumor's growth and dissemination and spread until
ultimately it takes a patient's life. Progress in biomedical
research that has come about because of the effort of the
National Cancer Institute in leading our national cancer
program is not only unraveling the steps in this process, it is
also providing the insights into the development of
interventions that can preempt this process. We now can
envision prevention, detection, elimination and modulation of
cancer in a way that people will either not develop cancer in
the first place; if they do develop cancer, we can detect the
disease early and eliminate it much more safely; or we are able
to treat and modulate established cancers such that people will
live with but not die from cancer.
In order to bring this goal about, in order to establish
the priorities and the investments that are necessary to
achieve this goal, we have created a priority-setting process
and a planning process that really defines, if you will, a
balanced portfolio, a portfolio of initiatives that are
involved in discovery, initiatives involved in development, and
those that are involved in delivery. Across the entire
portfolio of the National Cancer Institute, there are strategic
priorities and initiatives in all of these areas such that
through the process of discovery, development and delivery, we
will create those opportunities and deliver those opportunities
to patients in need to achieve that goal of eliminating the
suffering and death due to cancer.
In the process of establishing the portfolio, we engage in
a very elaborate and continuous process of providing and
obtaining input into the establishment of priorities and in
processes that review those priorities prior to implementation,
and then also processes that determine the impact of those
priorities. In order to give you an insight into that, I would
like to just lead you through how that ongoing planning and
budgeting process occurs.
It begins with a constant set of opportunities for input
into the establishment of those priorities. Those inputs come
from a variety of places and from a variety of organizations.
We have mechanisms in place that help us develop our annual
planning document and budget document that we call the bypass
budget. There are a series of targeted advisory groups and
disease focus groups that provide specific input, one of which
has been the ongoing series of the progress review groups which
has looked at opportunities and needs in areas like breast
cancer, prostate cancer and others. We have efforts that are
under way with regard to state-of-the-science meetings in which
we can look at some of those emerging scientific opportunities
that Dr. Fauci and Dr. Zerhouni alluded to, and we make certain
that we have significant input from the world community,
especially cancer survivor groups and organizations like the
American Cancer Society.
All of that input is then synthesized into an internal
planning document that then becomes the basis of both our
strategic plan as well as our business plan or budget. That
internal document is prepared by senior NCI leadership with
broad input from the entire NCI and is processed by our
executive committee. That is then reviewed on an ongoing basis
by initiative and by priority by formally chartered advisory
committees including our National Cancer Advisory Board, our
Board of Scientific Advisers and our Board of Scientific
Counselors as well as individual groups of expertise.
Once the programs have been vetted, they are then
implemented and approved by passage through the National Cancer
Advisory Board, which serves as our council, and then
disseminated and implemented throughout the entire cancer
community. With the implementation of those programs, we have
the opportunity for continued monitoring and surveillance that
gives us opportunities to determine measured outcomes and
results, which then feed right back into the ongoing planning
process.
It is through this decisionmaking process that is focused
on making strategic decisions about the balance in our
portfolio and making sure that that portfolio is constantly
being directed toward our mission that is the process by which
we establish and set priorities and balance the scientific
opportunities with the public need. Our ability to disseminate
that information through the professional judgment budget or
the bypass budget on an annual basis provides opportunities for
insight into both the strategic plan as well as the budget or
business plan that is required. This is not, however, our
budget submission process. That occurs directly through the
mechanisms that are available within the NIH and directly to
the Director of the NIH and then on to the Department of Health
and Human Services.
In addition to these formal processes, we have also looked
at opportunities to significantly increase our communications
with the community and have recently launched a weekly cancer
bulletin that is available on the Web as a way of communicating
to the entire community our scientific priorities and also the
scientific achievements that those investments are bringing
about. In doing so, we hope to continue to fulfill the mandate
and mission of the Congress to conquer cancer and eliminate its
suffering and death. Thank you, Mr. Chairman.
Mr. Bilirakis. Thank you very much, Doctor.
Dr. Volkow.
STATEMENT OF NORA D. VOLKOW
Ms. Volkow. Thank you very much, Mr. Chairman and members
of the subcommittee. It is a privilege for me to be here and
participate in this hearing.
I will not describe the process by which we set priorities
because it is similar to those described by my colleagues.
Instead I will share with you our research priorities and will
highlight the unique collaborations that NIDA has had to
cultivate in order to translate science to communities.
Like the other institutes, NIDA receives input about its
research priorities from a wide variety of sources, including
our National Advisory Council, scientific and health
professionals, and policymakers. However, unlike many other
medical diseases, addiction does not have many patient and
family advocacy groups. This is in part due to the fact that
drugs of abuse in most cases alienate the addicted person from
his family and his community rather than eliciting support.
This places additional importance on NIDA's ability to support
science that helps us identify national needs and emerging
priorities.
The disease burden attributed to drug addiction is
enormous. It is estimated to cost for both legal and illegal
drugs more than $484 billion a year. However, even as large as
this number may seem, it pales in comparison to the devastating
consequences of drug abuse to the individual and to society.
Drug addiction is a disease that targets the brain,
modifying its function in ways that limit the individual's
ability to make decisions on his or her behavior. The results
are widespread and devastating and can include family
disintegration, child abuse, loss of work and income,
accidents, criminal behavior, mental illness and suicide.
Moreover, because drug addiction develops during adolescence
and even sometimes in childhood, it can shatter the life of an
individual from its early beginnings. Drugs of abuse not only
affect the brain, but many organs in our bodies, thus also
contributing to the burden of many medical diseases including
cancer; cardiovascular, pulmonary, and infectious disease; even
obesity.
Research priorities at NIDA are set by the urgent need to
decrease drug abuse and its consequences while at the same time
taking advantage of scientific opportunities to increase our
knowledge about addiction.
Prevention and treatment of drug abuse and addiction are
NIDA's top priorities. Prevention is particularly relevant
since adolescents and children are the most vulnerable victims
for drugs of abuse. Moreover, research has shown that
prevention works, and this is illustrated on this poster from a
study that monitors, in teenagers, the perception of the
harmful effects of drugs versus the prevalence of drug abuse.
When students perceive drugs to be risky, their rate of drug
abuse drops. In fact, we are finding that through our
monitoring mechanisms, we can often predict the prevalence of
drug utilization on the basis of the perception of drug risk
detected the year prior.
Unprecedented scientific opportunities on prevention
research have emerged from the identification of genes that
affect the responses to drugs of abuse and also by the
development of technologies that now allow us for the first
time to evaluate the function of the human brain. We can now
investigate questions that were heretofore inaccessible, such
as how does early drug exposure affect the development of the
human brain, such as what is the relative contribution of genes
versus environment in drug addiction, such as how do
environmental factors and genes affect our brain and how that
in turn affects behavior.
In treatment our priorities include the development of
medications that can counteract the effects of chronic drug
utilization while at the same time developing research that
optimizes our ability to bring the science into the community.
Another priority in treatment is addressing the medical
consequences of drug abuse. Drug abuse is frequently comorbid
with mental illnesses and with other medical diseases. In many
instances this comorbidity results from the role that drugs of
abuse have as a contributing factor on the medical illness. For
example, drug abuse is one of the leading contributors to the
spread of HIV/AIDS in our country, not only by injection drug
use, which accounts for 36 percent of the new HIV cases, but
also by drug intoxication, which interferes with the judgment
of the person and increases the likelihood of risky sexual
behavior. Thus, treatment of addiction and prevention will have
an impact on the prevalence and the prognosis of other medical
diseases.
Scientific opportunities on treatment research have also
emerged from information derived out of the genome project,
which has allowed us to identify a wide array of new compounds
that in animal models interfere with drug administration.
However, notwithstanding the series of very promising
compounds, a major roadblock into their testing for clinical
utility has been the limited involvement of the pharmaceutical
industry on the development of medications. Issues such as
stigma, lack of reimbursement for drug abuse treatment and the
perception of a lack of a large enough market are some of the
variables that make companies reluctant to get into the
development of antiaddiction medications.
For science of prevention and treatment of drug abuse to
have an impact, NIDA relies on its collaborations with other
NIH institutes as well as its partnerships with other agencies
and organizations to help bring this knowledge into the
community. Indeed, the successful 11 percent reduction in teen
drug use during the last 2 years reflects the power of several
agencies working together toward a common goal. These
collaborations include not only the medical community such as
pediatricians and general practitioners for early drug abuse
detection, but also partnerships with agencies such as the
Substance Abuse and Mental Health Services Administration, or
SAMHSA, and the White House Office of National Drug Control
Policy, or ONDCP. It also reaches to the Department of
Education to bring prevention interventions into the school
environment and the Department of Justice to bring treatment
strategies that will minimize the chances of recidivism and
reincarceration once inmates with drug abuse problems leave the
jail or the prison system. We also work with State and local
agencies to bring science into the communities.
Though we have made significant progress in our
understanding of drug abuse and addiction, there is still much
more we need to know. Fast advances in knowledge and technology
provide us with opportunities to exponentially expand our
understanding of how our brain works and how it molds
behaviors. In the case of drug abuse where drugs directly
affect brain function and where the environment can play either
a permissive or protective role, new knowledge will help us
develop more effective prevention and therapeutic strategies.
I will be happy now to answer any questions you may have.
Mr. Bilirakis. Thank you very much, Doctor.
Let me ask you, I believe it was Dr. Zerhouni who addressed
obesity. Would the obesity research that is conducted be spread
throughout more than one institute?
Mr. Zerhouni. It is.
Mr. Bilirakis. It is, right?
Mr. Zerhouni. It is. The trans-NIH Obesity Task Force is
actually a multi-institute effort. It was led by Dr. Spiegel,
the head of NIDDK, diabetes and digestive disease institute and
heart and lung. The reason it is many is because it affects
children, so NICHD is involved. It has an impact, obesity, on
influencing the rates of cancer, so NCI is involved. There is
obviously a component of neurobiology, so the Neurological
Institute is involved. So it involves a large number.
Mr. Bilirakis. The reason I pick on it is sort of to help
me to try to get the picture. You indicated--I think you said
there was a 10 percent increase in obesity research funding as
against an average of 2.5 percent increase or something like
that. Did that 10 percent come about independently? In other
words, these institutes determined how much money should go
toward obesity research in these particular institutes so that
the ultimate total increases of all those turns out to be 10
percent?
Mr. Zerhouni. Both ways. The total portfolio in 2003 was
nearly $400 million of research. It grew from about $85 million
8 years ago to almost $400 million in fiscal year 2003 because
of the burden of the disease. The second is that obesity has
been declared an area of priority for all of medical research a
while back.
So how are the nearly $400 million distributed? Fifty-five
percent of that money on average, and I am not exactly accurate
about obesity, but 55 percent will be distributed because
scientists come to us with ideas about how to understand
obesity better. This is what we call the investigator-initiated
funding. About a third will come from clinical trials that we
are doing. For example, NIDDK conducted a trial in children
with obesity comparing diet versus exercise in the appearance
of diabetes in obese children. That was about a third of the
expenditures. Those tend to come from what we call initiatives.
So the institute, for example, NIDDK, issued what we call a
request for application to have people come forward and conduct
trials that we are interested in conducting.
As you can see, there are two components. There is a
directed component of the portfolio. This is what we call
initiatives that Dr. Fauci mentioned. And there is an
undirected component which responds to scientific proposals
that come to us. The 10 percent that we did by which we
increased the portfolio came from this planning process which I
insisted be done, asking the directors to come together and
look at the obesity portfolio across whole institutes, and it
was decided that there would be two priorities, two new
priorities. One, we think it is very important to accelerate
our research in obesity in childhood. All the evidence suggests
that obesity is determined very early in life. We thought we
didn't have enough investments in early childhood obesity, so
we increased our investment there. The second is obesity really
harms an individual not because of obesity itself, but because
it increases the chances of cardiovascular disease and diabetes
and other what we call comorbidities. So what we are thinking
is that research needs to be done to disconnect very quickly as
much as we can in the population obesity from the emergence of
diabetes and other comorbidities.
Mr. Bilirakis. So there was--I think you used the word
``we'' a number of times.
Mr. Zerhouni. At the end of the year when the budget came,
when we presented our budget to the Department, we set aside
$40 million; $22 million of that $40 million was dedicated to
these new areas that were deemed unserved at that point, the
childhood obesity and the comorbidity research.
Mr. Bilirakis. Would we say then that ``we'' was your
advisory council along with you that made those decisions? Did
you make them in coordination with the 27 institutes and
centers?
Mr. Zerhouni. That is correct. What we did since I became
Director, we have reorganized the way we make decisions at NIH.
We had 27 directors. It is very complicated to have that many,
so we created a steering committee of nine directors, a smaller
number that look over all the major corporate decisions that
NIH has to make. The budget is decided obviously between the
NIH Director and all of the directors that participated in this
NIH initiative, the trans-NIH obesity.
But it is limited. Our ability to move dollars from one
portfolio to another is limited. It is not something that you
can do arbitrarily, because programs tend to go over for 3, 4
years, and they are committed for that period of time. We need
to do it with the appropriate oversight. What I think needs to
be more encouraged, and we are encouraging it, and the
institute directors can comment, is more planning not within
the institutes, which is done very well, in most cases it is
the way to go, but planning across diseases that affect more
institutes, and areas of research that affect more institutes,
and areas that affect all of NIH with dollars attached to it.
Mr. Bilirakis. My time has expired, but hopefully we can
get back into that as time goes on.
Mr. Brown.
Mr. Brown. Thank you, Mr. Chairman.
Dr. Zerhouni, would you briefly comment on my opening
comments about Duchenne? They actually funded only one clinical
trial. Just give us a fairly brief answer to that, if you
would.
Mr. Zerhouni. Sure. First of all, I know that muscular
dystrophy has increased in funding. The funding is about $40
million, so it is about 15 percent of the NIH budget. I
understand that the MD-CARE Act is the mechanism, the vehicle
by which we are coordinating all of the portfolios of muscular
dystrophy.
You mentioned the issue of centers. I understand that three
were funded in 2003, and up to three will be funded, 2 to 3
will be funded in 2005.
I also can tell you that we have to be very careful when
you ramp up research capacity, you have to make sure you have
the people and the ideas there to make it happen. So review is
very important. In the previous cycle, our review was
indicating some reservations about the maturity of some of the
centers. But by and large what I think needs to happen is more
investments in a coordinated fashion in a set of centers that
would focus on that aspect.
I don't have information about what you said about clinical
trials and having three applications. I really can't comment. I
would like to get the information and forward it on to you.
Mr. Brown. Thank you.
Dr. Fauci, in Dr. Zerhouni's written testimony, he spoke,
and I am quoting, of NIH undertaking, quote, studies for which
the risks are too high or the financial incentive is too low to
attract private investment. A lot of us on the subcommittee are
concerned about a couple of things. One is that the lack of
research or the inadequate research on infectious disease,
especially in the developing world where it is hard to imagine
it would be very profitable for a prescription drug company, a
pharmaceutical company in this country; second, the emptiness,
if you will, of the antibiotic pipeline.
Could you comment on this? How much of the NIH budget
typically is devoted to that kind of research; how we can
assist you to do better, especially in the area of antibiotics,
with antibiotics, with antiparasitics, with antiretrovirals,
and especially with drug resistance in much of our antibiotic,
antiparasitic supply?
Mr. Fauci. That is an excellent question, Mr. Brown. We
take the responsibility in our emerging and reemerging diseases
program to address issues such as antibiotic resistance. This
is one of those areas where we absolutely need to deal on a
closer basis with our industrial partners. That is the delicate
balance that I was talking to you about, because they have
incentives to get into areas that are high profit margins for
them. That has to do not only with antibiotics, but also with
vaccines.
What we have been trying to do, and that is the reason I
showed that slide and why the committee staff wanted me to show
it at this particular hearing, was that we need to figure out
ways--and I can't give you a list of one, two, three things
that you can do vis-a-vis legislation or what have you, but I
would be very happy to work with you and your committee staff
to figure out ways how we might be able in a better way and in
a more facile way to deal with our industrial partners so that
we can get them interested in the things that we can do in the
normal interaction that we have.
One of the steps forward was the bioshield initiative was
very specific for biodefense. It created ways of dealing--in a
much more streamlined way of dealing with the industrial
partners, but also for giving them the incentive to get
involved in something even though it was not a guaranteed
profit margin for them. I think we need to look at that model
as it applies to all emerging and reemerging diseases,
particularly diseases that we refer to as the neglected
diseases.
We have a portfolio of research, but we need to get the
companies involved. We cannot do it all ourselves. That is the
reason why, as the months and years go by, we continue to
interact with the companies, and we are doing it much more now
than we have done years ago.
Mr. Brown. What are the neglected diseases, TB, malaria,
those that don't have much of a market in this country?
Mr. Fauci. There are two types of neglected diseases,
infectious diseases I am referring to now. There are those in
which the burden of disease is extraordinary, but there is not
necessarily a lot of research going on. Malaria and TB are the
two big ones on that. Do you know that we have, for example,
the vaccine for tuberculosis, BCG, which is quite ineffective
in preventing the adult type of infectious tuberculosis that
spreads from person to person, but is pretty effective in
preventing meningitis complications in children. Yet we now on
our own initiative--and this is one of the things that we
talked about--when we looked at the portfolio, there wasn't a
lot of action going on in TB vaccine research. So we seized the
opportunity of the capability of the sequencing of microbes
that we can do right now and the ability to use proteomics and
postgenomic function to develop a vaccine that we are now
testing in clinical trials which, believe it or not, it is
amazing to say this, the first new tuberculosis vaccine trial
in this country in 60 years, which we just started this year.
We did it because we were able to translate the opportunities
that we had with the new capabilities of sequencing the genomes
of microbes with the new modern-day molecular biology. It isn't
the old vaccine, based on the entire microbe, but a very small
molecular component that we call a fusion protein that will
allow us in a much safer way to do a vaccine trial for
tuberculosis. If that is successful, I think we are going to be
able to transform the entire landscape of tuberculosis.
Mr. Brown. What is an optimistic assessment or estimate of
how long from where you are now until it can be used in the
worst TB places in the world like India?
Mr. Fauci. When you talk vaccine development and ultimate
approval, you are always talking several years, 8 to 10 years.
I would imagine that if we accelerate the process, which we are
doing right now, we might be able to shave a year or 2 off of
that. But you are not talking next year or the year after. If
you are talking about full FDA approval, the kinds of things we
need to do for safety, it is going to take several years. It is
being tested now in our network of clinical trials.
Mr. Brown. Thank you.
Mr. Bilirakis. Mr. Shimkus to inquire.
Mr. Shimkus. Thank you, Mr. Chairman. Mr. Greenwood was
here way before I.
Mr. Greenwood. I am not sure that I was.
Mr. Bilirakis. That is not what the staff tells me.
Mr. Shimkus. Okay. I am ready. Thank you. Thank you, Mr.
Chairman.
I have great respect for my friends Ms. Capps and Mr.
Waxman and in their statements. They are noted health observers
and professionals in the field. But speaking from a very
conservative area of the country, we talked about this at the
last bicameral hearing we had on the Senate side last year,
that it helps us in rural America if the grants that are issued
pass the common-sense test.
The question is, is there a way that you can through this
evaluation process bring some sense or explanation on those
that don't? We don't have to go through them. They have been
publicly written about for years now. All of us get lobbied
strongly in support of the research done. It was Speaker
Gingrich who really pushed to double the size of NIH, and we
have made great investments. When we are asked for more and
more dollars in periods of tight dollars, we want to make sure
that those dollars are best spent. So how do we address again--
where is the common-sense application on some of the research
dollars?
Dr. Zerhouni, if you would answer that first, and then I
would probably like to follow up with Dr. Fauci.
Mr. Zerhouni. This is a very important question. We are
very concerned. In fact, the Chairman mentioned the term
transparency. I think in this area we found after our review
that we could do a lot better in making sure that we
communicate transparently and also fully about the importance
or lack thereof of the particular research. So one of the
things that I have done after reviewing this field, in
conjunction with all of the directors in our extramural office,
is to issue new requirements for explaining, in plain language,
both the public relevance as well as the importance of the
research scientifically. This information will be available in
clearly understandable language both to the public and to the
multiple review levels that we have in place so that there will
be more transparency and more explicit understanding of all
areas of research.
The common sense test that you rightly bring up is
something that we are quite concerned about because we depend
on the support of all taxpayers and we need to make sure that
whatever we do makes scientific sense and public health sense.
In that context I have asked all of the institute directors to
make sure that the two level of reviews are done fully. I know
it is a lot of work, but that there is a full discussion of the
grants at the advisory council level, because there are public
members in those mandated by law in these advisory councils,
and I think they should play their role.
This is why I had this report made by the Council of Public
Representatives called, ``Enhancing Public Input and
Transparency in the National Institutes of Health Priority-
Setting Process'' that addresses it, but I don't think that we
can weaken the peer review process in trying to answer the
concerns. We need to make sure that we accomplish both.
Mr. Shimkus. I applaud that. Somehow I would just hope that
as we move to more transparency, that that helps and doesn't
hinder. Again, as many of us would question the common sense of
the application of some of these grants, more transparency may
make it more difficult for us to defend the NIH.
Mr. Zerhouni. Although when I reviewed the grants, frankly,
the language was highly scientific with terms of art that were
not explained as well as they should be or could be. I think we
should do better and then obviously review the question.
Mr. Shimkus. I did mention Dr. Fauci, but I guess any of
the directors if they want to. It is up to you. My time is
almost out, so if someone else wants to add, you may do that.
The same area.
Mr. Fauci. We do the same thing. Obviously in areas such as
HIV/AIDS, it is a sexually transmitted disease, it is a disease
that is transmitted by injection drug use, by a variety of
other mechanisms. We cannot avoid addressing the issues that
are at the very foundation of why millions and millions of
people are getting infected. That is the reason why we are
sensitive to the issues that you bring up, really quite
sensitive, and I mean that sincerely. But we need to let the
science drive the questions if we are going to be able to get a
handle on this very devastating sexually transmitted disease.
Mr. Bilirakis. Mr. Strickland, you have 8 minutes.
Mr. Strickland. Thank you very much.
Dr. Fauci, I have read and have been concerned for a number
of years about what some say is the potential for a pandemic,
an influenza pandemic, occurring across the world that could
perhaps consume the lives of millions of people. I don't know
if what I have read is just reason to be concerned or not, but
the question I would like to ask you, is this a concern, is it
a possibility, and if it is, do you feel like we are doing
everything we can to be ready for such an occurrence?
Mr. Fauci. It is a possibility because it has happened in
1918; to a lesser extent in 1957 and 1968. It is of concern.
In science and public health, it is very rare that we can
say we are doing absolutely everything that can be done, but I
can tell you, Mr. Strickland, that we have put this at the very
highest priority. This is one of the things that I mentioned to
you in midstream we had to make adjustments in our priority
setting. For example, and I will be very brief on this, but it
is important because you are interested in this, and it needs
to be understood. There is what is called interpandemic
influenza, which the NIH and the CDC and the FDA have been
involved with for decades and decades, where you look at the
burden, and you look at the particular microbe that is
circulating, and you work together to have a vaccine for the
next interpandemic flu. Each year--it is very unappreciated:
36,000 people a year die from plain old flu, 114,000
hospitalizations. It is a very serious disease. I think it
suffers from the semantics of, oh, I have the flu, when you
don't really have the flu. You probably have a relatively
benign rhinovirus or something like that or a coronavirus.
What we are doing now in our preparation is that something
different happened over the last few years that started in 1997
when a bird flu jumped from a bird to a human. By killing and
culling the birds in Hong Kong, the lid was put on that. And
then successfully over the next few years until this particular
winter, 2003-2004, nine countries in Asia had the emergence of
a virus among flocks. In two countries, in Thailand and in
Vietnam, there was a total of 34 cases of which 23 died. That
is nearly a 70 percent mortality. The concern we in the
Department, particularly the NIH and the CDC, have is that that
microbe has the ability now to jump from chicken to human. The
reason it isn't a disaster is because it hasn't yet learned how
to go from human to human. So the potential epidemic has kind
of smoldered and stopped.
What we have been doing now is that we have been doing
basic research as well as developing a seed virus vaccine that
our grantees and contractors have developed. We have taken the
responsibility, even though we had to do midstream corrections.
This is something that I discussed in some detail with Dr.
Zerhouni and got his encouragement to move ahead with it, and
to now start making a pilot lot, which we are in the process of
doing.
Again related to the question that Mr. Brown asked, we had
to get very much involved in our industrial partners, in this
case it was inventors Pasteur and Chiron, in developing a pilot
lot and then to have that be able to scale up if necessary at
commercial levels. So we are doing everything within the
resources that we have right now because we put it as a very
high priority.
It is an example--again, just to get back to what Dr.
Zerhouni said a few minutes ago, the disease burden right now
in the United States for pandemic flu is zero, yet we are
putting resources into it, and we plan to do more next year
because we know the potential for that is enormous. We are part
of the whole Department. We have an HHS-based pandemic
influenza plan that is led at the level of the Department that
we, the NIH and the CDC and the FDA, are a very important part
of.
Mr. Strickland. Do you feel that the communication or that
the data-gathering infrastructure around the world is
sufficient to enable you to be alerted and to act as quickly as
possible based on what you currently have in existence?
Mr. Fauci. Yes and no. I will tell you what the yes is, and
then I will explain the no. The yes is that we have a number of
collaborating WHO centers of which the Department, namely the
FDA and the NIH and the CDC, play an important role at. We have
a grantee of ours who has a major program in Hong Kong. So when
you talk about flu, almost invariably it is going to emerge
from China, Hong Kong.
Mr. Strickland. Because of their agricultural practices?
Mr. Fauci. Because of the sociological and economic
conditions there. You have pigs and ducks and chickens and
people working on the farm together, a natural mixing bowl for
a virus that would jump from one species to another. That is
the yes. So we do have these people in communications. For
example, when the bird flu came out, we immediately dispatched
a person to Hong Kong to start working on it.
The no to maybe is that we have not had complete
transparency up to now, but it is getting better and better
with our Chinese colleagues. We saw that with SARS, which was
recognized months and months before in China until we knew
about it, and we only knew about it when it got to Hong Kong
where we had our people on the ground. With the flu now it is
getting better, but I don't have 100 percent confidence about
the transparency yet. But it is certainly much better than it
was before.
Mr. Strickland. I want to thank you for your answer. If the
potential consequences are so great, it is something that I
think we certainly should put all the resources that are needed
into it.
I would just like to say a word about the comment my good
friend on the other side made about the common sense test. It
seems to me that the common sense test is not relevant because
it is common, and that which is easily or readily understood or
appreciated is not, it seems to me, the major domain of the
scientific inquiry. You want to look at that which is not
common or easily or readily appreciated or understood. It seems
to me that is what the scientific inquiry is all about.
I have appreciated you being here. I wish we could spend
hours because there are so many issues. What you do, I think,
is as important as anything that we consider in this committee
or in this Congress, because you have cancer potentially being
cured in 15 years. I mean, with all due respect, I asked my
colleague if she thought you were maybe a little off to think
of that. I am speaking facetiously and trying to be humorous
here, I guess, but to think of that, it is overwhelming. How
great it would be. And then I said to her, it would absolutely
destroy our Social Security system. But what you do is so
integral to everything else we consider in this Congress,
economically, socially. We could talk forever about the social
implications of the stem cell research policy or of the
abstinence only education policy or the drug policy.
Mr. Chairman, I just wish we could do this more often and
for a longer period of time.
Mr. Bilirakis. You have had more time than anyone else.
Mr. Strickland. Thank you, sir. I appreciate it.
Mr. Bilirakis. Mr. Barton to inquire.
Chairman Barton. Thank you. My questions are going to be
more operational and structural and not going to be policy so
much. But my first question to you, Dr. Zerhouni, if you were
starting from scratch with a clean sheet of paper to create a
National Institute of Health, would you come up with 27
institutes and centers?
Mr. Zerhouni. No.
Chairman Barton. Is there a magic number?
Mr. Zerhouni. No. I think you really would like--if I had
my magic wand, I think what you would want is an evolving
structure that evolves easily and flexibly according to its
priorities. History dictates a tremendous amount of the
structure of NIH, history, congressional actions, legislation,
which really creates a degree of rigidity, which, from my
standpoint, needs to be thought through. A process is needed by
which that structure needs to be reviewed at regular intervals
to ask the obvious question, do we have structures that still
fit the reality of today. The rigidity, sir, is something that
I think would be a good topic of interaction.
Chairman Barton. Under current law, do you as the Director
have the authority to restructure, recombine institutes and
centers, or are they set by law and you have to go with what is
there right now?
Mr. Zerhouni. I would say it is almost completely limited.
I can do some restructuring within the structure. That is very
difficult to do. Remember, we have institutes and centers. In
my office, for example, we have program offices. Of the about
$290 million budget that you see within the Office of the
Director, there are mandated offices with their own budgets:
the Office of AIDS Research, Office of Behavioral Science,
Office of Rare Diseases, of which I have very little to say in
terms of programmatic spending. So at the end of the day, you
end up with about $120 million all together that the Office of
the Director directly controls.
But I think the lack of a process of adaptation, and to
speak in terms of policy and long-term future, there is no such
process that would allow a reasoned, learned evaluation of
appropriateness of structure relative to mission.
Chairman Barton. As we move toward reauthorization, would
it be appropriate for the legislation in conjunction--working
with the stakeholders to create a new structure, or would it be
more appropriate to give the Director's office the ability to
do the restructuring, the authority to do the restructuring?
Mr. Zerhouni. I think, in my view, since you have the
reality in this institution, NIH is still a wonderful
institution that performs very well in most aspects. I think
what would be more important is the process, with authority,
obviously, to look at certain structural elements and the need
for them to change. But it should be mandated in some fashion.
Chairman Barton. I want to give the center directors and
institute directors a chance on that last question. You may not
want to change the structure. You may think 27 is great. That
is a fair policy position. But if you think there needs to be a
reorganization, do you three ladies and gentlemen want us to
provide it, or do you want us to in some way give the
institution the authority to do it?
Mr. Fauci. I think it would be fraught with danger, sir, if
you legislate structural changes as opposed to providing the
kinds of flexibility that would allow the NIH to evolve with
the scientific evolution of things, the way Dr. Zerhouni
mentioned. The difficulty with legislating something that is a
structural change is then it is there, and if you want to move
and have the flexibility that virtually all of us alluded to,
that would only create a different model that would be as
inflexible as the concerns we have now with the inflexibility
of the model. So I would be much more in favor of providing the
NIH, through the Office of the Director, the flexibility to do
certain things----
Chairman Barton. If the Congress provides it, it will be
done. If we give the authority to do it, and we let the various
stakeholders interact, it might not be done. We may create a
process that has no end, where obviously if we do it in law,
almost by definition it is going to be imperfect, but at least
something will be done.
Mr. Fauci. But I would submit to you, sir, that the
authority would be in law, and then I believe, at least in my
rather extensive experience in dealing with the Congress, is
that the Congress looks at us carefully, as they should,
because we get what we get from the Congress, and that you have
ample opportunity in the future once you give the authority to
the NIH to be able to be flexible with those changes that if
the kinds of flexibility that are evolving are something that
you are concerned about, you can get us in front of you and
say, well, let's explain that; what are you talking about.
Chairman Barton. Of course, the reason you have 27 centers
and institutes is over time the Congress has dictated that.
Mr. Fauci. Right.
Chairman Barton. We have mandated that this or that be set
up, so we are the ones who created the structural problem we
are trying to address.
Mr. Fauci. So help us to be able to have the flexibility of
fixing it rather than trying to legislate a fix.
Chairman Barton. That is why I asked the question.
We have got two more directors, if you would wish to
comment.
Mr. von Eschenbach. I come from the perspective that the
structure really should be driven by function. I think the
authority to define functions and to involve trans-institute
and center collaborations is something that I think would be
very important for the NIH Director to have. That would allow
flexibility without dismantling the structure that is there.
You could work with that structure. When it is appropriate and
necessary for integration, you would be able to create that.
When it was most appropriate for those institutes and centers
to stay very mission-focused, that would also then be possible.
I think in that regard it perhaps then approaches it not
from making structural changes, but making certain that the
authorities allow functional activities to be able to occur in
a fluid way.
Ms. Volkow. I would agree with my colleagues. I think one
of the things that we have seen over the past 10 years in
science is that the boundaries, the categories, the labels
given to specific fields, are no longer so clearly delineated,
and so we see a tremendous overlap across areas in science.
The same thing is happening across our institutes even
though we are dealing with different diseases. For example, we
are starting to recognize that much of the basic knowledge
pertains to multiple disease processes. To me the important
aspect is how do you ensure an infrastructure that will allow
you to optimize the information and resources required in order
that you do not become redundant to the point that you are
wasting your resources. How to achieve that, though, is not
straightforward.
I think that the element to me is not predefining rigid
structures, and again I bring forth the concept that my
colleagues have voiced of flexibility that will allow us to
drive the organization as the new discoveries and the new
emerging trends develop. What I think is important is to
recognize the need in a scientific organization like the NIH of
having that flexibility. And it will not be automatic, so your
help will be required in order to, in certain instances, allow
it to proceed more easily.
Chairman Barton. I have a number of questions, and I will
submit them for the record.
Mr. Bilirakis. I am gathering before I go to Ms. Capps that
you all agree in your responses to Mr. Barton, because you went
into these very lengthy responses, you apparently feel that
some fixing does need to be done. I don't know when you shake
your head yes or no to that effect. Anyway, that is what I get
out of that.
Mrs. Capps to inquire.
Mrs. Capps. Thank you, Mr. Chairman.
If I am not mistaken, I believe Galileo was either
excommunicated or threatened with excommunication for daring to
posit a fanatical belief that the Earth revolved around the
Sun. My colleague who brought up common sense has had to leave,
but I wonder, if common sense had dictated, if we would have
ever had a man on the moon or if we would have ever undertaken
mapping the DNA. I know that most of us here are very
supportive of the work that you do and the way in which you do
it.
I will start with you, Dr. Zerhouni, but this really
relates to any of the people on the panel to explain to us how
the peer review process works and why it is considered the gold
standard worldwide for determining scientific quality. Some of
us get ahold of the grant applications, and they may sound
inappropriate when it is one paragraph. Some of this supports
science around esoteric projects, but underlying it is the need
to understand the millions of Americans who suffer from HIV/
AIDS, sexually transmitted disease, sexual dysfunction, mental
health consequences of abuse and various hard topics to get
hold of. That is what I would like you to address.
Mr. Zerhouni. Sure. I will summarize.
What I can tell you is one of the most common questions I
get as I travel around the world, how is our peer review
process so effective in identifying areas of science. Over the
years, as you know, we have had over 105 Nobel Prizes that have
come through the peer review process. The process is as
follows: We have two systems that work in succession and
sometimes in parallel. There is a center for scientific review
which is independent of the institutes, so Dr. Fauci or Dr. von
Eschenbach do not directly control the reviews that are done
for grants in NCI or NIAID that go through the center for
scientific review. So when a scientist proposes an idea, it
goes to that center, and that center combines multiple review
sections that are categorized according to fields of science.
In 1999 this was reviewed and restructured, because science
evolves. So we have review sections which are made up of
members which are under the FACA rules, the Federal Advisory
Committee Act rules, and the members have to represent a
diversity of regions, disciplines, and gender. The members
rotate every 4 years.
So those sections are the ones who do what we call the
first review, and they score the grants according to scientific
merits. Our administrators then compare all the scores across
and give a percentile ranking. Those grants then go to the
institutes and our Center for Scientific Review will look at a
grant and will say this is most appropriate for cancer, or this
is most appropriate for NAID or NIDA. It will then go there and
undergo the second level of review, which is the advisory
council of the institute. The advisory council is, again, a
FACA committee made up of usually 18 members, 12 scientific
members and 6 public members, and they have the final say in
what gets funded or doesn't get funded.
They can't not fund things that have received high review.
They can also fund things that are at the borderline of grants.
The second--this is about 60 to 70 percent of our grants
come through this--the second is what we call special
initiatives, where there are special review programs that are
organized by the institute. So Dr. Fauci organized last year a
competition for having universities create biodefense research
centers.
In this context it is such a specialized initiative, that
the NIAID puts together an independent peer review panel
focused on that area.
For example, Mr. Brown was mentioning muscular dystrophy.
If we have a special competition for muscular dystrophy center,
that will be reviewed by a special emphasis panel. So 70
percent is independent of the institutes, done independently by
scientific review, reviewed again at the advisory council.
Thirty percent is done by the institutes, or thereabout, and
then reviewed at the advisory council as well.
Mrs. Capps. And at some level the public has representation
on those committees as well, and all of this--are the names of
people on the screening committees, are those made public?
Mr. Zerhouni. Right. The names of all the study review
panel members are made public. The first degree of review,
which is a scientific review, is not open to the public. The
second is always open to the public. All advisory councils are
open to the public.
Mrs. Capps. And then finally----
Mr. Zerhouni. I am sorry. But the scientific review portion
can be closed to the public; but the members are known, who
participates and how.
Mrs. Capps. The members are well known?
Mr. Zerhouni. They are known.
Mrs. Capps. And the professional community respects this.
Is this an internationally understood process that is accepted
worldwide, or understood at least?
Mr. Zerhouni. I can tell you I just--the latest
communication I had is the Chinese Government wants to create
an NIH in China, and one of the first questions was, tell us
how to organize peer review. We get that all the time so it is
the gold standard of review worldwide.
Mrs. Capps. Thank you. I yield back.
Mr. Bilirakis. Mr. Greenwood to inquire.
Mr. Greenwood. Thank you, Mr. Chairman. Hello to all.
Dr. Zerhouni, this is a very basic question, and perhaps
naive, but something I don't fully understand. I think the
number is something like 80 percent of the dollars that flow to
NIH then continue out to the universities and health centers
and so forth, and something like 20 percent remains inside.
What fundamentally distinguishes the research that is done
inside NIH versus outside?
Mr. Zerhouni. That is an excellent question. The reason why
NIH has created what we call an intramural program was to
address historically issues of public health which could not be
addressed. There was no research capacity. There was no talent
out there to really address it.
Let me give you a specific example: safety of the blood
supply. In the 1960's, you may remember, all the blood
collection agencies and so on, and in the 1960's the rate of
transmission of a disease through transfusion was 30 percent.
We didn't know about hepatitis B and C and all the infections
that could be carried through blood transfusions. It was clear
at the time that you needed a very dedicated government-driven
process to understand all these viruses, and Dr. Harvey Alter
has led this program over 30 years.
You can't do this in the system of extramural granting,
where every 5 years you have to come in and have your grants
reviewed, and if it is a process that takes years, you can't
fund it. So typically that is the----
Mr. Greenwood. I have three questions I am trying to sneak
in here in 5 minutes. Do I not get 8 minutes, Mr. Chairman?
Don't I get 8 minutes instead of making an opening statement?
Mr. Bilirakis. No. You get 5 minutes.
Mr. Greenwood. The investor-initiated applications of
research. On the one hand we pretty much have decided as a
matter of congressional policy that we try to minimize the
micromanagement. We don't want to say, listen, we have had
constituents ask us to have more research done on this rather
than that, and we have left it to the peer review process.
On the other hand, I am not--when the requests for research
are coming from the research community, to some extent that is
a function--what they want to study is a function of what they
want to study, not necessarily a function of what needs to be
studied. So how does your process have an overarching plan and
still respond to the not exactly random but somewhat random
inputs?
Mr. Zerhouni. I will make three short comments. No. 1 is
other governments have tried to more micromanage research
worldwide, and it hasn't worked. The pharmaceutical industry is
a good example of how you do targeted research. They spend
twice as much money than NIH and we hear about the productivity
of that. So whenever you focus energy at a difference from what
the scientists themselves know they can do, you have a loss of
efficiency.
How do we know it is really relevant? Well, look at the
system. We fund primarily academic health centers. Well, you
get promoted because you make a difference in life. You don't
get promoted because you are studying some, you know, disease
of the right toe. You are trying to solve cancer and you are
trying to--that is how the system has a culture that pushes our
investigators toward relevant questions.
At the review panel, the public relevance of the program is
a component of the evaluation. So it is relevant to public
health needs as expressed both by NIH and by the CDC or other
components. That is how the integration gets done.
Mr. Greenwood. One more question----
Mr. Fauci. Can I answer? You asked the question, how does
research that may be important get done if the investigators
don't want to do that? We have been faced with that in the
early years of HIV-AIDS when no one was interested in it and in
the early years which we are in right now with biodefense.
There is a mechanism called a request for application and a
request for proposal, which are program-driven, that we are
interested in this area. We let the investigators come in with
their own creative ideas, but we tell them we have money that
we want to invest in this area, and that is how you get people
involved in things that they may not otherwise spontaneously
get interested in.
Mr. Greenwood. Got it.
Last question, what my oversight investigation had on. How
do you make sure that there are no conflicts of interest
between your reviewers who are deciding which grants get
approved if they may have consulting arrangements?
Mr. Zerhouni. Right. So all appointments to these review
panels are under the Federal Advisory Committee Act rules, so
every appointee is a temporary government employee and subject
to all of the disclosures of conflict of interest.
So the way we do it, if you are a member of those sections,
you have to disclose all of your financial arrangements; not
publicly disclose, but disclose internally. If you have a
conflict, you are recused from--a good example of a conflict is
a case that--a grant gets reviewed from a university at which
you yourself are a faculty member. You get excluded from those.
So it is the regular processes that we use as mandated by the
Federal rules.
Mr. Greenwood. Thank you. Thank you, Mr. Chairman.
Mr. Bilirakis. Mr. Stupak to inquire.
Mr. Stupak. Thank you, Mr. Chairman. Thank you to our
panel, thank you for coming and testifying today. I think NIH
does a remarkable job and look forward to working with you on
the many endeavors you undertake. And I think it is just great
that our country invests in life-saving and life-better work at
NIH.
I am particularly interested, though, in ensuring that the
American public has access to safe and effective drugs. Dr.
Zerhouni, as you know, back in 2001 Congress passed the Best
Pharmaceuticals for Children Act to authorize the 1997 law to
grant patent extensions to drug companies in exchange for doing
pediatric safety and effectiveness studies.
I opposed this legislation, because I think we have got it
backwards. I think it is wrong that we continue to grant these
patent extensions once they do a study. I think two things
should occur, and I am going to ask your opinion on it. Not
only should they do the study; should they not change the
labeling on the medicine, on its effectiveness or in
effectiveness or safety concerns of children before you grant
the extension of a patent?
Mr. Zerhouni. Let me make sure I understand the issue. You
are suggesting that the drug should be labeled not for
pediatric use prior to----
Mr. Stupak. Granting the extension of the patent.
Mr. Zerhouni. I do not know how to answer that question.
This is really an FDA-type of authority, but I will really look
it up and respond to you on the record.
Mr. Stupak. Well, we show on average it takes them 14
months after they get the extension to change the labeling.
That is 14 more months that we put children at risk--health at
risk.
Let me ask you this question, then. The act also gave, and
NIH was mandated to research certain on-patent and off-patent
drugs. How many off-patent drugs need to be studied? Do you
have any idea?
Mr. Zerhouni. The list is made up in coordination--if I----
Mr. Stupak. With the FDA?
Mr. Zerhouni. With the FDA and NIH. The list is primarily
the responsibility of NIH.
Mr. Stupak. Okay.
Mr. Zerhouni. And then this list is used in our BPC
program----
Mr. Stupak. Do you have any idea how many are on this list?
Mr. Zerhouni. No. I don't have the exact number.
Mr. Stupak. How about the on-patent drug----
Mr. Zerhouni. I will provide you that number, sir.
Mr. Stupak. Is it safe to say, given the resources
available to you today, that a lot of these drugs that need to
be studied are not being studied?
Mr. Zerhouni. That is a fair statement.
Mr. Stupak. Didn't NIH just complete a report on the gap on
the on-patent and off-patent studies and what needs to be done?
Did you not just do a report?
Mr. Zerhouni. We actually did review the BPCA
implementations. So there is a report that I think we sent to
Congress, if I am not mistaken, but I will----
Mr. Stupak. We haven't seen that yet. Could you provide
that to this committee, because I would like to see those
numbers and what drugs are and are not----
Mr. Zerhouni. Definitely.
Mr. Stupak. The Best Pharmaceuticals for Children Act also
included a provision to create an independent foundation. It
was called a foundation for pediatric research, to collect
funds and award grants for research on on-patent drugs when the
drug companies do not want to do the studies themselves. And,
again, I think those results have been submitted to you and to
the FDA commissioner.
Could you also provide us a copy of that?
Mr. Zerhouni. I will.
Mr. Stupak. In choosing the studies on on-patent drugs, the
ones that are targeted to be studied, what is your involvement
in it, or NIH's involvement in it?
Mr. Zerhouni. Primarily looking at the importance of the
drug and how utilized is it in the population under--you know,
the pediatric population. You will see that, for example, one
area that NIMH, the National Institute on Mental Health, is
very interested in is the use of antidepressants, and that has
become a major issue; so institutes have a way to weigh in and
give their advice in terms of what they think should be tested.
FDA, however, has the primary role, because if they receive
information about it, adverse events, and they know what post-
market surveillance data is available for----
Mr. Stupak. On the antidepressant, actually, with children
lately, we have been seeing a lot of reports on that. So what
involvement would you have on that? I mean, there has been a
lot of controversy with the British studies and one gentleman
at FDA not being allowed to testify publicly. What involvement
would you as NIH say to the FDA to try to get these studies out
and get them up there?
Mr. Zerhouni. Directly, I don't have a lot of personal
involvement in it. But NICHD, which is our lead institute for
children's research, is the lead, and that is the institute
that really interacts with the FDA.
Mr. Stupak. And who heads that institute?
Mr. Zerhouni. Dr. Duane Alexander.
Mr. Bilirakis. The gentleman's time has expired.
Bart, do you have something you can go through real
quickly?
Mr. Stupak. They are pretty long ones. I will submit the
rest of them in writing. And if you would just submit those
studies to us, I would appreciate it, especially on the on-
patent and off-patent drugs.
Mr. Zerhouni. I will certainly do that.
Mr. Bilirakis. Mr. Pitts to inquire.
Mr. Pitts. Thank you, Mr. Chairman.
First I think I ought to try to make a clarification. Some
of my colleagues on the other side seem to question the intent
of some members who question NIH on the merits of particular
studies, and the position of the other side seems to be that
sexually transmitted diseases affect a lot of people, and that
is why it is an important study. And I want to be clear; no one
ever questioned whether STDs are important to study. I think we
all agree with that.
What some question was how paying people to watch
pornography is related to STDs and how is it related to HIV-
AIDS, and how, as one NIH spokesman claimed, it has anything to
do with abstinence education.
I think we all agree that we need to find a cure for HIV-
AIDS, but there is some question about what is the best way to
do it; is there a better way than paying people to watch
pornography? That said, let me move on to my questions.
Over the years there have been questions raised about some
of the grants that NIH has awarded and whether or not these
grants were appropriate uses of taxpayer dollars. And while
some grants arguably have scientific value that may not be
apparent to a layperson, there are also some grants that are
arguably of questionable priority when reviewing the entire NIH
research portfolio.
And, Dr. Zerhouni, I am pleased to hear that you are
working toward more transparency issues, as you stated. And I
want to be clear; I prefer not to go grant by grant and bring
congressional grants up at every hearing, but I can't help but
mention one. I saw a study of dorm-room wall decorations, Web
pages of college students. It was funded 1 year.
How can we convey to constituents, taxpayers, who have
family members with Parkinson's or leukemia or diabetes, who
are concerned about ensuring the safety of the blood supply,
that studying dorm-room wall decorations was more worthy than
finding a cure for their diseases? When a multiyear grant is
awarded by an institute, what if any authority do you have as
NIH director to make a change if it is determined at a later
date that this project is of less significance, given current
public health needs?
Have any of you ever stopped funding of an awarded grant
based on an unexpected budgetary need? And, Dr. Zerhouni, you
can start.
Mr. Zerhouni. Sure. Obviously, this is an issue. As I said,
we need to work on and make sure that we review these grants.
The dorm-room grant study was funded over 3 years ago, is no
longer active. I looked into that because of the questions that
were raised, and what I am told is that the reason the study
was performed is that you can tell the likelihood of mental
health disorders according to the decoration that student in a
college will put up in their room and what kind of analysis--
personality analysis this will be.
So the way I understand why the research was thought useful
at the time by the committee that reviewed it was that it could
provide you with a diagnostic test of children, students--
college students that may be getting in trouble from the mental
standpoint, and their personality disorders and so on. This is
what I am told was the reason for that.
But the question you are asking is a much more profound
question, and that is relative allocation of resources. And
that is something, as I mentioned, to do that better, I think
you will need to have, A, a better understanding of the
portfolio and the relative importance of the portfolio, and
this is why I suggested that we need to have a portfolio review
mechanism, both within institutes and across institutes,
because each program obviously evolves over time, and has the
budget that they need to spend according to both the primary
and secondary review.
So I want to make sure that we have processes in place that
will give you the assurance that it has been reviewed, that its
scientific merit is established, that it is explained in clear
terms so that there is no--like, if you read this title,
obviously it makes no sense, but if you look into it, you
realize that psychological tests that look at drawings, for
example, that children use on their wall tell us something
about the mental state of an individual. And this is something
you can argue in terms of is it right to spend that dollar on
that thing instead of spending it on something else. This is
why I suggested that we need to have better mechanism.
The authority that the director has to stop a grant is, I
would say, very limited; because if it has passed review, has
been approved by the advisory council of an institute, unless
two things happen, one, that the progress reports indicate that
the research is not making any headway or that, two, there is
an inappropriate use of Federal funds, we will terminate the
studies. That is pretty much the authority of the director of
the NIH, but I will let my colleagues comment as well, because
they have greater authorities within their own portfolio.
Mr. Bilirakis. Very briefly, if you would like to comment.
Mr. von Eschenbach. Just to give you a specific example of
the importance of managing the portfolio and making shifts to
strategic priorities, one of the things we did this very year
was make a decision with regard to a study that had been going
on with regard to looking at mammography and its utilization
and standards for interpretation. That study was coming up for
reissuance and rereview, but the analysis indicated that we had
received significant input and information about the outcomes
of that question that was first raised, and essentially that
grant had reached its fulfillment. And rather than reissue it
and continue it on, we stopped that and redeployed those
dollars to other initiatives where we can look at even more
effective ways of detecting breast cancer earlier. So we do
make those kinds of strategic shifts in decisions.
Mr. Fauci. Another example would be human subjects issues.
We have stopped grants that if you look at the design of a
clinical trial, for example, in a developing nation, that
although the science would be compelling to get the answer to
the question, it could not be done under the proper ethical
circumstances, so we would stop it even though it got a good
scientific score. I have done that.
Mr. Bilirakis. The gentleman's time has expired. . And then
I appreciate the patience the gentleman has shown throughout
the entire hearing.
You know, Mr. Waxman made a--focused on keeping politics
out of science and research, and I will tell you, and I have
said this before--I know Dr. Zerhouni at least has heard me say
it a number of times--I think probably maybe the toughest part
of my job--and I will bet I am speaking for Mr. Brown and
virtually every member of this committee--is when someone comes
wheeling into our office as an ALS patient and tells us that
there isn't enough research on ALS and then knows about some of
these other areas that Mr. Pitts and others have mentioned. I
lost my youngest brother to Parkinson's in his mid-fifties, and
you know where--Mohammed Ali has come in and testified.
I think I am losing something here. I think I need some NIH
right about now.
I guess what I am saying is it is a tough thing for us to
tell them, thank you; to tell them that we believe very
strongly that we should not micromanage; we believe very
strongly that we are not in a position to determine where the
funding should go in terms of what specific disease, things of
that nature. So we are staying out of it. But I would also
strongly suggest there is probably a hell of a lot more
politics being played within NIH and among the institutes than
ever comes out of the Congress in this regard.
Whether you agree with me or not, I don't know, but I think
if you kind of search your experiences, you would find that
that is the case.
So we intend to continue to do this tough thing of telling
these many patients, these people who come in here and testify,
that we don't think it is our role to determine what funding
should go--research funding should go to what disease, but we
also like to think that you are helping us in that regard, too,
through some of the grants.
I mean, the word ``common sense'' was used, and issue was
taken with that by another member and that sort of thing. I
don't know what is right and what is wrong. All I know is, put
yourselves a little bit in our shoes when we have to tell these
people and they know what some of this funding award is going
to.
Your explanation in terms of the painting of the dorm walls
and whatnot, well, I can see where there is something behind
that. But when you have that patient out there who is dying, I
think they would rather see that money, rather than go to
studying the painting of those walls, go into something
involving ALS or whatever.
So that is the position that we are always in, and I
appreciate your coming here today. I think you have worked
awfully hard to answer our questions and our inquiries. I am
not sure Mr. Brown is satisfied as far as Dechenne muscular
dystrophy is concerned. For instance, I am not sure that any of
us are completely satisfied, but transparency I think is the
answer, and we depend upon you for that. And you are just the
magicians in our society, your research and all your work and
whatnot. And all of us are strongly supportive of that,
contrary to some of the statements made in this hearing up
here.
We have a number of questions, as usual, to submit to you.
We would appreciate it--as you heard Mr. Barton say, we haven't
reauthorized NIH for quite a few years. It hasn't affected your
work, because you have gotten the money, but it looks like we
may be advancing toward that. I mean, discussions have taken
place between the minority and the majority on maybe some
issues and things of that nature. So I guess what I am saying
is, no, we shouldn't take the flexibility away from you, in my
opinion, but we also need recommendations from you on what we
should be doing that you would need to see legislative changes
in order to take care of some of the problems that you all know
of much better than we do.
Mr. Brown, do you have any closing statements? Please feel
free.
Mr. Brown. Only to add that--and thank you, Mr. Chairman--I
appreciate this hearing, and I always appreciate so much what
NIH has done. I hope we can get our government to pay the same
attention to the CDC that we do the NIH, but I also hope that
we don't make decisions in the next few years that cripple this
Nation's ability to do research, not just the research that you
do but also research with--we are doing better with math and
engineering and where we go in that direction. And I am afraid,
just as we look at budget questions that come about because of
policies that people in this Congress make, that we think more
of the future than we seem to be thinking right now. And I
applaud all four of you and applaud all your colleagues out of
each institute, and especially the employees of NIH and the
contractors at places like Cleveland--in Cleveland with Case
Western Reserve University and other great institutions around
the country that benefit from the decisions you make, and only
the public benefits from the decisions both you and they make.
So I thank you for that.
Mr. Bilirakis. Thank you very much. Thank you, Doctors. The
hearing is adjourned.
[Whereupon, at 4:30 p.m., the subcommittee was adjourned.]
[Additional material submitted for the record follows:]
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