[House Hearing, 108 Congress]
[From the U.S. Government Printing Office]

                            SETTING PROCESS



                               before the

                         SUBCOMMITTEE ON HEALTH

                                 of the

                        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

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                      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
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
DARRELL E. ISSA, California
C.L. ``BUTCH'' OTTER, Idaho

                      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
JOHN B. SHADEGG, Arizona             DIANA DeGETTE, Colorado
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)
  (Ex Officio)


                            C O N T E N T S


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



                            SETTING PROCESS


                        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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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.


    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 

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 
We are committed to NIH's infectious disease research on problems such 
        as AIDS, SARS, West Nile Virus, influenza, malaria and 
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 
    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 
    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 
    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 

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 
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 
A method of assessing outcomes to enhance accountability.
    Thank you for the opportunity to testify. I will be pleased to 
answer your questions.


    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' 
    Dr. Fauci, please proceed with your statement.


    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.
    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 
    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 
    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.


    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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. 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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 
    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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