<DOC>
[109th Congress House Hearings]
[From the U.S. Government Printing Office via GPO Access]
[DOCID: f:20746.wais]
SETTING THE PATH FOR REAUTHORIZATION: IMPROVING PORTFOLIO MANAGEMENT AT
THE NIH
=======================================================================
HEARING
before the
SUBCOMMITTEE ON HEALTH
of the
COMMITTEE ON ENERGY AND COMMERCE
HOUSE OF REPRESENTATIVES
ONE HUNDRED NINTH CONGRESS
FIRST SESSION
__________
MARCH 17, 2005
__________
Serial No. 109-20
__________
Printed for the use of the Committee on Energy and Commerce
Available via the World Wide Web: http://www.access.gpo.gov/congress/
house
__________
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------------------------------
COMMITTEE ON ENERGY AND COMMERCE
JOE BARTON, Texas, Chairman
RALPH M. HALL, Texas JOHN D. DINGELL, Michigan
MICHAEL BILIRAKIS, Florida Ranking Member
Vice Chairman 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
NATHAN DEAL, Georgia FRANK PALLONE, Jr., New Jersey
ED WHITFIELD, Kentucky SHERROD BROWN, Ohio
CHARLIE NORWOOD, Georgia BART GORDON, Tennessee
BARBARA CUBIN, Wyoming BOBBY L. RUSH, Illinois
JOHN SHIMKUS, Illinois ANNA G. ESHOO, California
HEATHER WILSON, New Mexico BART STUPAK, Michigan
JOHN B. SHADEGG, Arizona ELIOT L. ENGEL, New York
CHARLES W. ``CHIP'' PICKERING, ALBERT R. WYNN, Maryland
Mississippi, Vice Chairman GENE GREEN, Texas
VITO FOSSELLA, New York TED STRICKLAND, Ohio
ROY BLUNT, Missouri DIANA DeGETTE, Colorado
STEVE BUYER, Indiana LOIS CAPPS, California
GEORGE RADANOVICH, California MIKE DOYLE, Pennsylvania
CHARLES F. BASS, New Hampshire TOM ALLEN, Maine
JOSEPH R. PITTS, Pennsylvania JIM DAVIS, Florida
MARY BONO, California JAN SCHAKOWSKY, Illinois
GREG WALDEN, Oregon HILDA L. SOLIS, California
LEE TERRY, Nebraska CHARLES A. GONZALEZ, Texas
MIKE FERGUSON, New Jersey JAY INSLEE, Washington
MIKE ROGERS, Michigan TAMMY BALDWIN, Wisconsin
C.L. ``BUTCH'' OTTER, Idaho MIKE ROSS, Arkansas
SUE MYRICK, North Carolina
JOHN SULLIVAN, Oklahoma
TIM MURPHY, Pennsylvania
MICHAEL C. BURGESS, Texas
MARSHA BLACKBURN, Tennessee
Bud Albright, Staff Director
James D. Barnette, Deputy Staff Director and General Counsel
Reid P.F. Stuntz, Minority Staff Director and Chief Counsel
______
Subcommittee on Health
NATHAN DEAL, Georgia, Chairman
RALPH M. HALL, Texas SHERROD BROWN, Ohio
MICHAEL BILIRAKIS, Florida Ranking Member
FRED UPTON, Michigan HENRY A. WAXMAN, California
PAUL E. GILLMOR, Ohio EDOLPHUS TOWNS, New York
CHARLIE NORWOOD, Georgia FRANK PALLONE, Jr., New Jersey
BARBARA CUBIN, Wyoming BART GORDON, Tennessee
JOHN SHIMKUS, Illinois BOBBY L. RUSH, Illinois
JOHN B. SHADEGG, Arizona ANNA G. ESHOO, California
CHARLES W. ``CHIP'' PICKERING, GENE GREEN, Texas
Mississippi TED STRICKLAND, Ohio
STEVE BUYER, Indiana DIANA DeGETTE, Colorado
JOSEPH R. PITTS, Pennsylvania LOIS CAPPS, California
MARY BONO, California TOM ALLEN, Maine
MIKE FERGUSON, New Jersey JIM DAVIS, Florida
MIKE ROGERS, Michigan TAMMY BALDWIN, Wisconsin
SUE MYRICK, North Carolina JOHN D. DINGELL, Michigan,
MICHAEL C. BURGESS, Texas (Ex Officio)
JOE BARTON, Texas,
(Ex Officio)
(ii)
C O N T E N T S
__________
Page
Testimony of:
Zerhouni, Elias, Director, National Institutes of Health..... 10
Additional material submitted for the record:
Zerhouni, Elias, Director, National Institutes of Health,
response for the record.................................... 54
(iii)
SETTING THE PATH FOR REAUTHORIZATION: IMPROVING PORTFOLIO MANAGEMENT AT
THE NIH
----------
THURSDAY, MARCH 17, 2005
House of Representatives,
Committee on Energy and Commerce,
Subcommittee on Health,
Washington, DC.
The subcommittee met, pursuant to notice, at 9:37 a.m., in
room 2123 of the Rayburn House Office Building, Hon. Nathan
Deal (chairman) presiding.
Members present: Representatives Deal, Hall, Bilirakis,
Upton, Gillmor, Shimkus, Shadegg, Pitts, Ferguson, Rogers,
Myrick, Burgess, Barton (ex officio), Brown, Rush, Eshoo,
Green, DeGette, Capps, and Baldwin.
Asso present: Representative Bass.
Staff Present: Cheryl Jaeger, professional staff; Chuck
Clapton, chief health counsel; Brandon Clark, health policy
coordinator; Eugenia Edwards, legislative clerk; John Ford,
minority counsel; Jessica McNiece, research assistant; and
David Vogel, research assistant.
Mr. Deal. Good morning. I will start off this hearing today
by welcoming Dr. Zerhouni here to testify with regard to the
National Institute of Health. We appreciate your joining us
here today to talk about one of the most important priorities,
I think, of the jurisdiction of our committee.
As we are well aware, and many of you have attended the 10
hearings that have taken place over the last 2\1/2\ years on
NIH. We hope that we can continue in the spirit of working
together to achieve some much-needed reform in the
administrative structure of this vital component of our Federal
Government.
As many of you know, we have been working to reauthorize
NIH longer than some of have been in Congress itself. And I
think it is well past time that we get something done in that
direction. It is time for us to put aside the petty projects or
areas of concern that each of us might have, and work together
to try to reorganize and make some changes to this very
important agency and to modernize its organizational structure
so that we can have the scientific discovery that will benefit
everyone.
This is one of the most important issues I think that we
can address in terms of challenging our ability to put aside
partisanship, not only along party lines, but along particular
issue lines, and simply do what is right for the American
people.
Dr. Zerhouni, I think you truly are one of the heroes in
Washington. You have been trying to fight against what is a
siphoning of bureaucracy, and oftentimes, unjustified mandates
that we have placed upon at you at the Agency and that you have
responsibility for. I hope that all of us, as we listen to you
today, will learn and hopefully avoid some of those mandates
that we have tried to impose in the past.
We do appreciate your attendance. We appreciate your
expertise of the subject matter that you are going to talk to
us about. And we look forward to hearing your testimony.
I will now recognize my ranking member, Mr. Brown.
Mr. Brown. Thank you, Mr. Chairman. Thank you, Dr.
Zerhouni, for your excellent work in the National Institute of
Health.
I want to begin by thanking Chairman Deal and Chairman
Barton for your willingness to tackle NIH reauthorization in a
bipartisan manner. We must support NIH so it can remain the
world's flagship medical research institution, and we must
prepare NIH, as well as our entire public health infrastructure
for the challenges of the 21st century.
These challenges are significant. Emerging and as yet
incurable diseases threaten millions of our citizens, even as
we move forth with research on thousands of conditions, from
asthma to myloma to spinal cord injury, educating the public on
strategies for prevent and treatment and cure continues to
remain a significant challenge.
Millions of Americans find themselves without access to any
care, much less to the cutting edge work enabled by NIH
funding. Around the world, we fail to remedy curable diseases,
such as TB and malaria, that take millions of lives each year.
These challenges require us to keep three things in mind as we
move forward with reauthorization.
First, we must examine and improve the oversight rule of
this Congress and this committee.
Second, we have--we must recognize the need for adequate
funding for NIH, funding that reflects the evolution of
medicine and lets us buildupon our monumental successes. After
the successful doubling of NIH funding between 1998 and 2003,
President Bush's budget suggests that NIH can make progress
with flat funding. While this House and the White House tries
to extend tax cuts for the wealthiest in our society, health
care for the poor becomes the scapegoat, and NIH's innovative
research becomes the target. This year, the President's
requested a level of funding well below the level of inflation.
My question is, is that sufficient to sustain the current
pace of medical progress? What are the consequences of flat
funding? Before we enter the process of reauthorization, we
should have concrete understanding of the impact on the
budget--on this budget of the research and the mission of NIH.
Dr. Zerhouni, flat funding NIH makes things especially
difficult for you as you set your own priorities each year, and
as Congress weighs in with the priorities of the public. I am
interested in hearing more about how you would like this
committee to help you set priorities, accommodate and inform
the public, and build a successful research, given the
challenge of the flat line budget and the kinds of budgets that
constrict research and undermine medical progress.
I look forward to hearing about your plans for the Office
of Portfolio Analysis and Strategic Initiatives, how this
office can improve coordination not only among institutes, but
between NIH and other agencies charged with protecting the
public health. I hope it brings us closer to the transparency
and effective dialog that is expected of us by the American
people. It should be a goal of this reauthorization to
understand not only what money NIH spends and how it is spent,
but what these resources mean to you and to your researchers
and what tangible returns the public should expect on its
investment.
Third, we must examine NIH in the context of how each of
our publicly funded health care agencies moves our society
forward. We can be sure that moving forward as society in terms
of the health care that we provide is a function of research
and resources. Without either, we stop progress dead in its
tracks. With this reauthorization, however, we have the
opportunity to influence whether the public investment in
medical progress is used to the benefit of all of our citizens,
including the sickest and the most vulnerable and the poorest.
The AIDS drug Norvir was developed using inventions
produced by NIH, so the American taxpayer footed the bill for
its development. Norvir's manufacturer, Abbott Labs, decided
late 2003 to increase the price of that critical AIDS drug by
400 percent, and to apply that price increase only to U.S.
sales. I thought it was an unreasonable abuse of the American
taxpayer's research dollars for Abbott Labs to quadruple the
price of the resulting product, especially since that price
hike was applied only to American consumers. NIH concluded it
was neither responsible nor equipped to involve itself in
prescription drug pricing, but at the very least, a lot of us
believe NIH should weigh in in acknowledging investing in
research, but ignoring access is a counterproductive exercise,
and one that runs against the American publics' interest, if
you say your goal truly is to promote the public health.
In its mission statement, NIH is charged with using its
research to ``to extend healthy life and reduce the burdens of
illness and disability.'' That mission is promoted every day in
the offices, the clinical facilities, and the laboratories at
NIH, but oftentimes, the potential and inherent NIH sponsored
research is neither fully nor equitably exploited or
distributed.
As Congress considers the budget and as we in this
committee work to reauthorize NIH, I hope that we remember that
the mission of NIH is not just to fund and promote research,
but equally importantly, maybe more importantly, to bring the
benefits of that research to bear for all Americans.
Thank you, Dr. Zerhouni.
Mr. Deal. Thank you, Mr. Brown.
Now recognize Mr. Hall of Texas.
Mr. Hall. I have no opening statement, Mr. Chairman. Thank
you.
Mr. Deal. Recognize Mr. Bilirakis, Florida.
Mr. Bilirakis. Thank you, Mr. Chairman. I, too, am pleased
that we are here this morning to examine portfolio management
at the NIH. And I want to welcome Dr. Zerhouni. I want to
congratulate him for being appointed again to head the NIH, and
to thank you so you very much for your willingness to continue
to serve.
Dr. Zerhouni testified before us several years ago and said
that ``No outstanding organization can remain great without
regularly reviewing its operating principles and plans, and
subjecting itself to critical reexamination.'' Since becoming
director, he has held NIH to that standard by undertaking an
aggressive and ambitious plan to ensure that the Agency
maintains a diverse portfolio of research founded both on
public health need and scientific opportunities. I am eager to
learn of the progress of these initiatives, as well as the
potential the new portfolio management proposals may have to
profoundly impact the progress of medical research and more
quickly transform research into real treatments to help people.
The Institute has worked to advance knowledge and discover
opportunities to prevent, treat, and cure the diseases and
disabilities which affect so many is daunting, indeed,
especially since more than 80 percent of its budget flows
through the extramural community, which supports the work of
research personnel affiliated with universities, hospitals, and
other research facilities. Only about 10 percent of its budget
supports the basic and clinical research activities conducted
by NIH's world-class physicians and scientists. This challenge
has become more pronounced, I think, in light of the
considerable funding increases NIH has received over the past
decade. Congress has doubled the NIH budget, which has opened
new opportunities to further research and find better cures and
treatments for diseases and disabilities. The increased funding
has brought increased scrutiny, and does call for greater
public accountability and transparency. Our constituents expect
and demand that we hold NIH accountable for its use of scarce
taxpayer resources. They deserve to know that NIH and its
various institutes and centers are using their money as
effectively as possible.
I believe that today's hearing will provide important
information to help us, and them, to understand the factors and
objectives NIH considers in the management of its research
portfolio, and ultimately ensure that our national investment
in biomedical research is doing as much good as it possibly
can.
I know we all look forward to your testimony, sir.
Thank you very much, Mr. Chairman.
Mr. Deal. Thank you.
Now recognize our colleague, Ms. DeGette.
Ms. DeGette. Mr. Chairman, I will waive my opening
statement in favor for more time for questions.
Mr. Deal. Recognize Ms. Capps for an opening statement.
Ms. Capps. I will also defer for more time for questioning.
Mr. Deal. Ms. Baldwin.
Ms. Baldwin. Thank you, Mr. Chairman, and thank you, Dr.
Zerhouni, for being here today.
I represent Wisconsin's second Congressional district, and
I am honored to have the University of Wisconsin, Madison, one
of the Nation's premiere research institutions, as a part of
the district that I represent. I am continually amazed at the
research done at the UW and the depth of expertise that they
house in so many different areas of research.
From the initial discovery of how to grow and sustain stem
cells made by Dr. Jamie Thompson back in 1998 to more recent
discoveries involving skin tests for cholesterol levels, the UW
has been a continuing leader in a number of exciting research
fields. This is made possible largely by NIH funding, so I
welcome this opportunity to talk about the NIH, and I look
forward to engaging in a conversation.
These are such exciting times for scientific research. As
we continue to learn more and more about the world works and
the way that our bodies function, and couple this with advances
in technologies, the research possibilities are truly
exploding. The ability to conquer a variety of different
diseases is within our reach, and I feel strongly that we, as
Members of Congress and as government officials, should do
everything we can to aid and encourage our researchers, not to
discourage them or tie their hands in any way.
I personally continue to strongly oppose the President's
arbitrary limits on embryonic stem cell funding because this
area of research holds so much potential for learning more
about and possibly developing cures for a whole host of
conditions and diseases, from juvenile diabetes to spinal cord
injuries to Parkinson's disease. Countless numbers of people
continue to suffer because of this arbitrary limitation on this
promising research, and I find this unacceptable.
But I would also like to ensure that we protect the time-
honored practice of peer review. I was raised by a research
scientist, an NIH-funded research scientist, and I have family
members who continue to work in this area, conducting
scientific research. So I have firsthand knowledge about the
amount of scrutiny and close study that goes into the peer
review process. This process is not simply a formality, it is a
thorough, thoughtful process that ensures that limited research
dollars are being directed toward research that is best needed
and is best designed.
For the most part, members of this legislative body do not
have backgrounds in technical, scientific matters that are
involved in peer review studies, and I would hope that we, as
Members of Congress, remember this and that we resist
politicizing science. We must allow the peer review process to
continue unfettered without Congressional interference.
Dr. Zerhouni, again, I thank you for coming here, and I
look forward to today's discussion.
Mr. Deal. Thank you. I recognize Mr. Barton, chairman of
the full committee.
Chairman Barton. Thank you, Mr. Chairman. I want to welcome
you, Dr. Zerhouni. I know some of the questions today may be a
little bit biting, but it is our job to serve as the oversight
watchdog for the people, and I can say from personal conviction
that you are doing the same thing at NIH as you try to reform
and revise that agency and bring it into the 21st century.
It is not a part of my prepared statement, but I want to
applaud you on your determination to clean up the consulting
situation. I think your policies that you have implemented are
in the right direction, and at the degree of the reform that we
need to be moving. So I am going to applaud you for that.
According to the NIH annual review of spending,
reprioritization of resources at NIH is critical. According to
the NIH missions statement, deciding how and where to
distribute money is a challenge the NIH faces each year. It
requires fresh assessment of the Nation's health needs, and
renewed evaluation of scientific opportunity. That is from your
own mission statement, and I agree with that.
Expanding biomedical research in the 21st century requires
the NIH to function in the most efficient manner so that each
and every penny that is spent on medical research to prevent,
treat, or cure disease is counted and expended meaningfully. To
do so, the NIH has to be able to justify the scientists and the
public alike why some research projects are advanced ahead of
others.
Unfortunately, NIH has grown like topsy-turvy. In 1960, the
NIH was comprised of a director and seven institutes. Today,
there are 27 institutes and centers. The motivation behind this
explosive growth has certainly been sincere. The individual
organizations were created arbitrarily, usually without benefit
of systemic analysis or review of the efficiency of the
structure. This growth has resulted in an almost random
collection of structure in which largely independent institutes
and centers are tasked to advanced research programs, not in
cooperation with one another, but according to diseases, organ
systems, or stage of life in which they specialize. Thus, we
study diabetes and ages in separate places with separate staffs
and separate directors overseeing the research. Plainly, there
is some collegiality and professional cooperation, but it
defies reasons to believe that they will produce the
efficiencies that can be achieved by logically unified
structure.
Furthermore, this silo system produces thousands of pages
of strategic plans, one for each of the 27 institutes and
centers comprising the NIH. Read separately, each institute and
center produces an impressive list of research goals and
targets. Realistically, however, scientific progress can not be
accurately measured and strategic plans set by evaluating the
research activities of one institute alone, when modern
science, as we all know, transcends the research activities at
several institutes and centers.
Dr. Zerhouni, you have accomplished great feats in your
brief time there. You deserve our admiration for taking on one
of the most difficult and important jobs in government, and I
applaud you for your courage and for your success. NIH is the
premiere research organization of its kind in the world, and
your absolute determination to make it even better, in my
opinion, is already paying off. You are going to present to the
committee today why you believe the creation of the new Office
of Portfolio Analysis and Strategic Initiatives will help to
minimize the problems that we have outlined by providing your
agency with the tools to facilitate planning for trans-NIH
initiatives and provide greater accountability to NIH research
programs. That is an important first step, but it is only a
first step. The difficulties that you face are monumental. In
order to achieve fundamental changes that are needed, it is my
opinion, and I think you share this opinion, that the Congress
must act to restructure and reform the NIH.
This hearing today is an opportunity for us discuss the
reforms and the restructuring that is needed. Based on the
information and the experience that we have gained from the
past 2 years of committee hearings and investigative work,
there are three changes that I believe will help you in your
job better manage resources and increase research investments
at NIH.
First, I believe we need to expand your authority--the
authority of the NIH director. Congress should allow the
director to transfer a greater percentage of funds between
institutes and centers and to increase the working budget of
the office of the director to fund more extensive portfolio
management projects, as well as crosscutting research
initiatives.
Second, we need to better align the budget line items at
the agency. Congress has created over 60 separate research
programs, 60, at NIH with authorizations that no longer exist
or are set to expire. Authorization for the National Cancer
Institute, for example, which is the largest institute at NIH
expired in fiscal year 1996. That, as we all know, is 9 years
ago.
The Appropriation Committee allocates funding for NIH
through 26 separate line items aligned primarily with the
institute and center designations. Responsible budget planning
requires Congress to evaluate whether the current funding
allocations and mechanisms meet the scientific demands of NIH.
I believe this committee should consider new creative
approaches, such as budget clusters for allocating resources
throughout the NIH. This concept would build on several
thoughtful recommendations recently circulated to improve the
NIH.
Finally or third, we need to create a new, more transparent
reporting system. Congress should eliminate unnecessary
reporting requirements, such as reports on specific diseases.
We should instead require reports that comprehensively track
research progress in broad areas of interest.
These are just three ideas, Doctor, but I think they are
three that should be analyzed and worked on together. And I am
sure that you have others. I look forward to working with you
and other members of this committee to reform and restructure
and revitalize the National Institute of Health.
Thank you, Mr. Chairman.
[The prepared statement of Hon. Joe Barton follows:]
Prepared Statement of Hon. Joe Barton, Chairman, Committee on Energy
and Commerce
Thank you, Mr. Chairman, for holding this hearing today. It is with
great enthusiasm that I welcome Dr. Zerhouni, once again, before this
Committee.
According to NIH, the annual review of spending and priorities is
critical. According to the NIH mission statement: ``Deciding how and
where to distribute the NIH's money--is a challenge the NIH faces each
year. It requires fresh assessment of the nation's health needs and
renewed evaluation of scientific opportunity.'' I agree.
Expanding biomedical research in the 21st century requires the NIH
to function in the most efficient manner so that each and every penny
that is spent on medical research--to prevent, treat, and cure
disease--is counted and expended meaningfully. To do so, the NIH has to
be able to justify to scientists and the public alike why some research
projects are advanced ahead of others.
Unfortunately, NIH has grown like Topsy. In 1960, NIH was comprised
of a director and seven institutes. Now there are 27 Institutes and
Centers. While the motivation behind this explosive growth was
certainly sincere, the individual organizations were created
arbitrarily, usually without benefit of systemic analysis or review of
the efficiency of this structure.
This growth has resulted in an almost random collection of
structures in which largely independent institutes and centers are
tasked to advance research programs not in cooperation with one
another, but according to diseases, organ systems, or stage of life in
which they specialize. Thus we study diabetes and aging in separate
places, with separate staffs and separate directors overseeing the
research. Plainly there is collegiality and professional cooperation,
but it defies reason to believe they will produce the efficiencies that
can be achieved by logically unified structure.
Furthermore, this ``silo'' system produces thousands of pages of
strategic plans, one for each of the 27 Institutes and Centers
comprising the NIH. Read separately, each Institute and Center produces
an impressive list of research goals and targets. Realistically,
scientific progress can not be accurately measured and strategic plans
set by evaluating the research activities of one Institute alone when
modern science transcends the research activities at several Institutes
and Centers.
Dr. Zerhouni, has accomplished great feats in his brief time there.
He deserves our admiration for taking on one of the most difficult and
important jobs in government, and our applause for his successes. NIH
is the premier research organization of its kind in the world, and Dr.
Zerhouni's absolute determination to make it even better already is
paying off. Dr. Zerhouni will present to the Committee today why he
believes the creation of the new Office of Portfolio Analysis and
Strategic Initiatives will help to minimize this problem by providing
the Agency with the tools to facilitate planning for trans-NIH
initiatives and provide greater accountability to NIH research
programs. It's an important first step. But the difficulties he faces
are monumental. In order to achieve the fundamental changes that are
needed at NIH, Congress must act.
Based on the information and experience that we have gained from
the past two years of Committee hearings and investigative work, I have
identified three changes that will help Dr. Zerhouni to better manage
resources and increase research investments at the NIH.
First, we need to expand the authority of the NIH Director.
Congress should allow the Director to transfer a greater percentage of
funds between Institutes and Centers and increase the working budget of
the Office of the Director to fund more extensive portfolio management
projects as well as cross-cutting research initiatives.
Second, we need to better align the budget line items at the
Agency. Congress has created over 60 separate research programs at NIH
with authorizations that no longer exist or are set to expire.
Authorization for the National Cancer Institute, the largest institute
at NIH, expired in FY96. The Appropriations Committee allocates funding
through 26 line items, aligned primarily with Institute and Center
designations. Responsible budget planning requires Congress to evaluate
whether the current funding allocations and mechanisms meet the
scientific demands of the NIH.
Personally, I believe that this Committee should consider new,
creative approaches, such as ``budget clusters,'' for allocating
resources throughout the NIH. This is a concept that builds on several
thoughtful recommendations recently circulated to improve the NIH.
Finally, we need to create a new, more transparent reporting
system. Congress should eliminate unnecessary reporting requirements
such as reports on specific diseases. We should instead require reports
that comprehensively track research progress in broad areas of
interest.
I look forward to working with Dr. Zerhouni and the Members of this
Committee to get this project done.
Mr. Deal. Thank you, Mr. Chairman.
Now recognize Mr. Green from Texas.
Mr. Green. Thank you, Mr. Chairman. I would like to welcome
Dr. Zerhouni and reserve my time so I have more time for
questions.
[The prepared statement of Hon. Gene Green follows:]
Prepared Statement of Hon. Gene Green, a Representative in Congress
from the State of Texas
Thank you, Mr. Chairman, for calling this hearing on the NIH's
management of its research portfolio.
As we go about a long-overdue reauthorization of NIH, it is
important that we continue to gain knowledge about the structure and
management of the NIH in order to maximize research efforts.
Without a doubt, the work performed at the NIH is invaluable.
Groundbreaking research has provided a lifeline of hope to
countless Americans living with diabetes, cancer, HIV/AIDS and many
other illnesses.
Dr. Zerhouni, thank you for your stewardship of the Institutes and
Centers that have offered our constituents suffering from illnesses
with hope for the future.
I also commend your efforts to place additional focus on trans-NIH
collaboration to better address many of the problems that plague our
society. These days, Americans live with chronic conditions that cannot
be remedied by studying one particular organ, or one part of the body.
Obesity and diabetes, for example, affect virtually the entire
body, and we will hinder real progress on these pervasive conditions if
we don't fully encourage cooperation among the NIH's Institutes and
Centers.
Dr. Zerhouni's increased focus on inter-disciplinary research teams
is a step in the right direction toward achieving the successful
collaboration that will solve our most pressing health problems.
I am particularly interested in learning more about how and whether
these initiatives should be formally incorporated into NIH
reauthorization legislation and what additional authority--if any--Dr.
Zerhouni and future NIH Directors need to ensure that NIH research can
evolve to meet the health care needs of our nation.
If there are obstacles in our current structure that slow the path
of lifesaving research from reaching the patient, then we must overcome
them.
Thank you, Dr. Zerhouni, for appearing before us again today.
I look forward to hearing your testimony.
With that, Mr. Chairman, I yield back the balance of my time.
Mr. Deal. I recognize Mr. Shimkus.
Mr. Ferguson, the vice-chairman of the committee.
Mr. Ferguson. Thank you, Mr. Chairman. I would like to
submit my statement for the record for additional time for the
questioning.
Thank you.
Mr. Deal. Mr. Rogers.
Mr. Rogers. I will yield, Mr. Chairman, for more questions.
Mr. Deal. Ms. Myrick.
Ms. Myrick. I don't have an opening statement, but I just
want to thank you because you have made great strides so far,
and I support very much what you are doing and hope that we can
make happen a lot of what you want to see done, because I think
you are on the right track.
I agree with the chairman's statement, there are some
things that we do have control over that we could do to help
you, so I look forward to hearing from you.
Mr. Deal. Dr. Burgess.
Mr. Burgess. Thank you, Mr. Chairman. Thank you for calling
this hearing. Dr. Zerhouni, I thank you for being here today,
braving all the press outside our room. Apparently they are
very interested in what your testimony is going to be here
today.
Dr. Zerhouni, when I was in practice, we had two kinds of
doctors. We had doctors like me that we called ``doing
doctors,'' and then we had doctors like Dr. Zerhouni. Dr.
Zerhouni is what we call a ``thinking doctor'' and we are
grateful that we have got a thinking doctor in charge of the
NIH.
I am glad you could join us here today to offer your
insight into the inner workings of the NIH, and how difficult
your task is to administer the 27 institutes and centers that
make up the NIH. And today we have an opportunity to look at
how this country prioritizes our health research and the future
of your institute, the National Institute of Health.
The NIH has its roots in researching medical treatment for
our soldiers in World War II, but has since evolved into a
world-class research institution. I believe that the
institution must continue to evolve with medicine and shouldn't
be stymied by bureaucratic lethargy.
It can not be understated, Dr. Zerhouni, that you have a
very difficult job ahead of you. As NIH director, you are
allocated only 3 percent of the overall NIH budget to
coordinate the 27 institutes and centers at the NIH. And last
year in this chamber, in the House, when we debated the
Nation's--restructuring of the Nation's intelligence, we wanted
to created a national intelligence director, it was broadly
recognized that if we created that director of national
intelligence, we had to give that individual budgetary
authority. And unfortunately in your institute, you lack some
of that budgetary authority.
I appreciate what the administration has done with the
increase in the NIH budget during the Bush Administration.
Perhaps it would have been a little more useful to begin the
restructuring and the reauthorization process before adding the
additional money. So perhaps as additional monies are added in
the future, the work that we are going to do here over the next
several months will embellish that and make that pay greater
dividends.
Mr. Chairman, we shouldn't take the NIH for granted. We
shouldn't let it succumb to bureaucratic drift. I look forward
to working with you, Mr. Chairman, and Mr. Barton and with Dr.
Zerhouni as we seek solutions on improving quality and
direction of our medical research in this country.
And I will yield back.
Mr. Deal. Thank you.
Recognize Mr. Upton.
Mr. Upton. Thank you, Mr. Chairman. I am going to put my
statement in to get my extra 3 minutes and defer.
Mr. Deal. We are pleased to have a member of the full
committee, Mr. Bass, with us. Do you have an opening statement
you would like to make?
Mr. Bass. Mr. Chairman, I appreciate the courtesy. I have
no opening statement.
Mr. Deal. Thank you.
Mr. Brown. Mr. Chairman, I ask unanimous consent to put
into the record Mr. Dingell's statement and the statements of
anybody on our side and yours, too, who is not here today.
Mr. Deal. Without objection.
Mr. Brown. Thank you.
Mr. Deal. Dr. Zerhouni, we are very pleased to have you
here, and thankfully, we have not taken too much of the time
with opening statements, and we look forward to hearing from
you at this time.
STATEMENT OF ELIAS A. ZERHOUNI, DIRECTOR, NATIONAL INSTITUTES
OF HEALTH
Mr. Zerhouni. Thank you, Mr. Chairman, and members of the
committee. I am really pleased to be here. I think there is no
more important discussion to have than the one we are
undertaking today.
I have written testimony that I have submitted. I would
like to place it into the record.
What I would like to do instead of reading my testimony is
to make a presentation about some of the issues we are facing
from the standpoint of public health, science, and how we
manage our science at NIH and where opportunities could be
found to improve on the functioning of NIH.
[Slide.]
I would like to direct your attention to the screens, if I
may, and begin by telling you that if you looked at the past
100 years, what you would observe is that we have been able to
accomplish something that has unprecedented in human history,
and that is that we have increased life expectancy by about 1
year every 5 years. And at the beginning of the century, this
was accomplished primarily through hygienic approaches, better
water supply. There was 1 dip that you can see in the screen in
1918, and that was the pandemic flu. And besides that dip,
everything else has been upward. And you can trace the
improvements to significant discoveries. The discovery of
penicillin and antibiotics, polio vaccine, the discovery of the
DNA structure, chemotherapy for cancer starting in the 1950's,
and then in 1976, the first cholesterol reducing drug, Statens,
which have made a huge impact on cardiovascular disease. And
more recently, the completion of the human genome.
So every 5 years of investment over the past 30 years where
everyone agrees that the improvements that we have seen in
health overall have been due to medical discoveries. Every 5
years of investment at NIH have produced 1 year of increased
life expectancy.
Why is that, and what kind of research outcomes have we
seen tangibly that you can then base a decision on how to go
forward in the 21st century. The first one I will show you is
essentially our impact on AIDS and AIDS research. When you look
at the picture that we had in the late 1980's, early 1990's,
what you were projecting was essentially a death rate that
would dominate cancer, mental health, suicide, in the young
population, young age groups. But because of behavioral
sciences and prevention, as well as discovery of new drugs, we
have reverted that curve in the United States with a rate of
disease and death that is \1/6\ of what it would have been if
we hadn't made those discoveries.
Most impressive in our past 30 years' history has been the
progress we have made in controlling coronary heart disease and
stroke. If you looked at our situation in 1970 and you
projected out what the number of death would been in this
year--in the year 2000 for which this statistic was built, you
would have seen 1.3 million deaths a year from heart disease.
Currently, we are seeing about 514,000 too many, but you can
see that the decrease in mortality and morbidity from heart
disease is leading us to a new age in medical research whereby
acute diseases that tended to be very short-term and lethal are
now being transformed into diseases that last a longer time and
where surviving a particular disease is now the rule rather
than the exception, and whereby we spend 75 percent of our
healthcare dollars on chronic conditions rather than acute
conditions.
So progress in itself brings new challenges. More recently,
I would like to show you what the discoveries that we have made
over the past 10 years have led us to in terms of performance
in terms of public health. Because of our advances in
fundamental understanding of the molecular biology of cells,
viruses, and microbes, and our advances in genetic
technologies, our ability to identify DNA, just like we do in
criminal courts, the DNA signatures of microbes and viruses,
what we have been able to do over the past 2 years, alone, we
have developed the first ever vaccine for Ebola. And it is in
trial today, 2003. This took 2 years. Typically, a vaccine in
historical terms takes 15 years to develop.
Anthrax, the problem we knew about but we didn't really
know how to attack it more effectively, we now understand what
is making anthrax so deadly, and we are developing new drugs
for that.
Another one that was totally unknown to us was SARS. When
it happened, nobody knew what it was. We identified the cause
of SARS in 1 month. It took us 4 years to identify the cause of
HIV AIDS. And we are now, at NIH, starting the first trial of a
new vaccine developed at the vaccine research center and on
controlling SARS if it ever comes back to haunt us.
So we are seeing two things. One is a progressive increase
in life expectancy and health, with better health indicators
for the entire population across the board. Second, a shift
from acute to chronic diseases, and third, an acceleration in
the pace of science. Our discoveries are coming at a much
faster pace than they were coming 15 years ago.
How do we tackle that is the real issue that we have to
face? The public health challenges that can--I think we need to
understand what they are at the strategic level. First and
foremost is what I mentioned. There is a shift from acute to
chronic diseases. Second, because of our success, we now have
an aging population and we need to tackle the issues that come
with that. Third, we still see health disparities. Although I
am showing you great curves going all in the right direction
for all populations, there are still significant differences
between the populations within the United States. Fourth, we
are talking about emerging diseases and reemerging diseases. I
am not talking about just SARS or pandemic flu. I am also
talking about obesity. For us, this is a new disease. This is
something that is emerging as a challenge for our society, and
we need to focus on that. And last is obviously a new mandate
for NIH biodefense, and I just showed you some results that
came from that biodefense research effort, which started in
2002.
When you look at this, you can ask yourself ``where are we
going next? What are we going to do in the 21st century?''
[Slide.]
And this slide, I would like to summarize for you what our
strategic view is. In the 21st century, we have to transform
the way we practice medicine. And the way we practiced medicine
over the past 5,000 years has been the same one, and it is the
one on the left side. What did we do? We basically waited until
someone showed symptoms of a disease or lost some function,
whether it be diabetes or Parkinson's disease or cancer, we did
not intervene before the disease struck us. And the reason for
that was very simple. We just didn't know what the normal
evolution of a disease was. We didn't know that heart disease
started 30 years before you had a heart attack. That diabetes
started 25 years, in molecular terms at the cell biology level
before you became deficient in insulin and diabetic. We just
didn't know that. Today, we do. And the problem with
intervening so late is that it is very expensive. As a
physician myself, I have witnessed the growth in technology and
the growth in sophistication that has allowed us to provide
miracles to patients. We have the best medical care in the
world, but we may not have the best health care strategies in
the world.
So what has happened is that as we increased our knowledge,
a new view has come in front of us. And that would require, as
I mentioned, faster adaptation of a structure like NIH to adapt
to the speed of change in research, the speed of change in
public health and our requirements, and what is the best
strategy? We believe in the 21st century, if we don't change
the way we practice medicine today, the costs of practice
medicine as we know it, without new discoveries and new
strategies will be unsustainable. And the best way for us to
overcome that is to intervene before symptoms occur, to prevent
the disease from forcing someone to become disabled. To
understand a disease years before it strikes, and intervene at
that time. Can we really do this? And my belief is we can. We
have completed the human genome, we have engaged in new
research that makes us understand that, in fact, when you look
at diabetes for example, we discovered a major gene last year
called the HNF-4 gene, and we now know that that gene is a
control gene when mutated, increases the chance of someone
getting diabetes. Intervention at that stage is more likely to
be cost effective and orders a magnitude more rewarding, if you
will, in terms of health and in terms of cost as well.
Why can we predict this? Because we do understand much
better than we ever did the preclinical molecular and cellular
events. We can now increasingly see how we could define which
patients are more likely to develop what disease and intervene
at that time. We have already seen that. I mean, we have
reduced, for example, the damage due to high blood pressure,
the damage due to high cholesterol, the damage due to not
recognizing that a colon polyp that we now can eliminate
through a minor colonoscopy was the source of cancer. We didn't
know that 15 years ago.
So we need to continue that progress, and NIH has been sort
of the agency tasked to do this. So let us talk about, I think
what you are concerned, rightly--concerns rightly are. How are
we doing our job? What is the infrastructure and the strategy--
what are the strategies to make sure that we are doing an
optimal job?
And as Chairman Barton mentioned, I would like to go over
the evolution of the NIH from the standpoint of the structure
that is supposed to do its job. If you looked at 1937, we were
part of the Public Health Service. There was a National
Institute of Health, and in 1937, the National Cancer Institute
as a division of the National Institute of Health was created.
The next even was in 1944, the Public Health Service Act, and
by 1947, 1949, what you saw is a combination of what I would
call policy setting and management structures like the Division
of Research Grant, and then the realization that we did have
public health challenges that were extremely important. We knew
about cancer. Very early, we realized that heart disease was
going to be a major public health problem if we did not
intervene. A National Heart Institute was created. Infectious
diseases were a problem in the 1940's, if you recall. These
were the days when we developed penicillin and streptomycin and
the very first miracle antibiotics. The National
Microbiological Institute was created. We also knew that
without basic knowledge, we would not make progress. There is
no way for us to translate a discovery if there is no discovery
to start with. So the Fundamental Experimental Biology and
Medicine Institute was created.
The next snapshot I can give you is 1968, 1969. So by that
time, specific missions needed to be accomplished. So you see
the National Institute of Neurological Disease and Stroke, a
problem that was rising in the 1960's, arthritis and metabolic
diseases, and so on. I do not want to belabor the point, but
what you can see is that as the depth of the problem was better
understood, you needed to have the structures to fight those
problems in depth. At that time, however, no one really knew
that the fundamental biology of a disease in the neurological
system and how cells signaled each other would be the same than
it was in the heart, or it would be the same fundamental
mechanisms in Parkinson's disease versus diabetes or others.
What has happened over the past 30 years is through our
understanding of DNA, DNA reproduction, genetics, molecular
biology, we understand now that every cell in the body contains
the same DNA, and it is regulated maybe in different ways, but
there is a convergence in our science. The second is progress
in our research technologies, whether it be imaging or
microscopy, or our computer sciences, allowed us to understand
biology to an extent never before possible. In that time, no
one knew that 30 years later we would have the entire human
genome at our disposal. No one could even predict that.
So what has happened is obviously with the optimism and the
application of this research with the results that we were
obtaining, the structure continued to grow. And this is the
current structure, 27 institutes and centers. As Chairman
Barton said, different appropriation lines. And more
interestingly, over the last 10 years, something that wasn't
mentioned which I would like to point out to you, has been the
creation of offices in--under the office of the director. So
the office of the director's budget is about $300 million.
About $80 million is for administration, about $220 million is
for these offices.
What are these offices doing? They are responding to a need
that I think all of us see: the need for coordination, a
strategic coordination, a strategic optimization. So what
happened in the 1990's is creation of the Office of AIDS
Research, which has special authorities to look across
institutes and across silos to maximize the investments in AIDS
research. And you have seen the results in AIDS research. I
mean, we can be proud of the fact that in less than 15 years,
we have developed over 80 drugs that are effective in HIV. And
today, we have five candidate vaccines ready to enter trials.
Whether they will work or not, I can't tell. I can't tell.
But the Office of Women's Health was another creation,
which was also responding to the same underlying theme that I
hear for this hearing, and that is how do we make sure that we
have a cross view that maximizes the taxpayers' dollars? Then
you have the Office of Rare Diseases and the Office of
Behavioral and Social Sciences. We have the Office of Minority
Health and Health Disparities, which then became a center. So
you can see historically over the past 10 years, the same theme
has recurred.
Where do we go from here? There is one thing I think we
need to recognize, and that is that despite the complexity,
there is no doubt that NIH has accomplished extraordinary
results. And as a director, I can tell you that there are
reasons for that. One is in the 1944 Public Health Service Act,
the requirement to have peer review, independent peer review at
two levels. And this is recognized as probably the feature
which has made NIH most successful. Whenever I go around the
world, the first thing I get asked is ``how do you run your
peer review system in such a way that it is, in fact, adapting
to the science?'' And the reason is simple. First and foremost,
we do respect ideas from individual scientists, because that is
where innovation comes from. And we, unsolicited, get about--
\2/3\ of our grants come from ideas from the field. One-third
of our grants, on the other hand, are determined by our
institutes. So for example, we decide that something is
important. We then put out requests for application or
contracts to stimulate one area of research or another. There
is always an independent review of the quality of the science,
and it is recognized worldwide that the peer review system that
we have prevents bad science from being funded, and really
promotes high quality science. These then are going to a second
structure called the Institute Advisory Councils. It is very
important to realize that these advisory councils are, in law,
authorized to supervise independently each institute. So they
are made up of scientists and public members. They assess the
programs. They approve the applications, and they define the
priorities from the standpoint of the public members of that
particular institute. And at that time, they release then
funding authorizations for the particular grants. At the peak
of the doubling, we are able to fund 30 percent of applications
that would come to NIH. Today, we fund about 22 percent of
applications, simply because we have been very successful in
having more ideas come to NIH from different fields of science,
and we have an increased number of applications, particularly
when you look across the spectrum of what we do.
Then the research priorities obviously are made in a way
that I think has been a question, a continuous question for
members of this committee and other committees. How do you do
this? And frankly, I think the one thing that NIH does well, it
organizes a tremendous amount of interfaces between patient
advocacy groups, scientific organizations, professional
societies, industry. We have 21,000 nongovernmental advisors
who come to NIH to review grants, review strategic plans, give
advice to the NIH, and that is what forms, if you will, the
fundamental direction that institutes will then take.
So the institutes, as Chairman Barton said, are the core of
what gets planned within NIH. First, through all of this
analysis of the science, analysis of the disease burden, public
health opportunities, different requests, different mandates.
These are put, as you mentioned, in the plan, and this we have
been better at over the past 10 years. We have now a formal
process that each institute has to follow in different ways,
but a formal process that can explain what the plan is. And
then the funding priorities are then determined at that time,
depending on what the science is, and then the decisions are
made by the Advisory Council. So think of this 26 times. That
is what we do.
As you mentioned, the scope of the challenge is not a small
challenge. When you look at the institutes, they are different
in size and budgets. They are, depending upon their mission
area--but the challenge for me is how do you do this job best?
And the challenge for you us how do you structure, if you will,
an organization that will do this as best as possible?
So the way I look at it is obviously, everybody looks at
the total budget, $28 billion, thereabouts, and they look at it
that way. I look at it differently, and I would like to share
with you the way I look at it. I really see that there are
patients at the end of every dollar that we invest, and there
are individual human beings that will eventually suffer from
one disease or another. How do I then change the paradigm as I
told you between the 20th century and the 21st century? Well,
we need to make those investments that are orders of magnitude
more effective than what we do today.
So the way I look at is basically $28 billion is $96 per
American, per year. And as you can see, we distribute that
differently. For example, the National Cancer Institute has
$16, and I am asking my directors to think in those ways,
because that is how you become very, very good at making sure
that your investment is well used. That you are counting
pennies, not just big dollars. And in fact, if you look at the
Cancer Institute at 16, the Infectious Disease is 15, Heart and
Lung, $10, and so on. And that is the investment that we need
to make over a continuum.
When I said the sciences converging, we need more
disciplines in biomedical research. We need computer scientists
and physicists and so on, and that is the 21st century
challenge for NIH. And I think this is where the opportunity to
have an interaction with your committee is going to be
fruitful.
Obviously, we also are aware that this investment needs to
have an impact on the 5,500 per American, per year, health
costs, and fast-rising. So we are not oblivious to the sense
that we need to have an impact, not just do research that has
no practical applications.
One thing that we have tried to do since I became director
was to try to, in fact, put a glue--the glue together. Not the
budget glue, but the intellectual glue that will then dictate
budgetary decisions. I mean, clearly, as I mentioned in the
public at the main before, what we have is a hand with 27 very
strong fingers, but I am not sure the palm is as strong as it
needs to be to coordinate all those activities. Everything that
you do within the mission area is very strong, but do we have a
perfectly organized orchestra, if you will. And that, I think,
is where we pushed our focus and our efforts.
The first year, I asked the question to all the institute
directors, ``what is it in science that has advanced to a point
where no institute sees that as their responsibility or know
that any one institute can fund, that all of NIH needs to do to
advance and accelerate medical research?'' And this is what led
to the NIH roadmap for medical research, with its detailed
planning to accelerate and introduce the concept of
interdisciplinary research, and the concept of understanding
molecular systems 20 years before the disease strikes.
In 2005, we then analyzed and scanned the public health
horizon, and realized that obesity was something that needed a
strategic response. And I put together a strategic group to
come up with what we call the NIH Strategic Plan for Obesity
Research. And we funded this plan, we increased the budget for
that plan within the limits that we could, and within the
limits that you all know well about how budgetary decisions can
get made.
And in 2006, we decided that the next target would be
neuroscience. And the reason for that is if you combine mental
health issues, degenerative neurologic disorders like
Parkinson's, Alzheimer's disease, substance abuse, and
addiction, the total burden of disease is about $500 billion in
economic costs. It is four times larger than obesity, if you
combine all of these issues. So I asked the 15 institutes to
come together in a coordinated fashion to try to come up with a
neuroscience blueprint that would address, if you will, and
accord more synergistic fashion, more optimized fashion, the
issues that we are facing.
Where do we go from here? I think it is important, at least
within my authority, to explicitly address the problem. And the
way we want to do this is by proposing something that I can do
within my authority, to create and Office for Portfolio
Analysis and Strategic Initiatives. As the portfolio is
expending, we need to have tools. We need to have a vision that
tells us exactly if there are redundancies in the portfolios,
if there are inefficiencies, if there are gaps. In other words,
you need radar. And this office, to me, is the radar that will
help NIH navigate the complicated and complex waters of science
today.
How would it do this? Essentially, what we will do is
introduce the concept of that office and provide this office
with the authority to do strategic analysis using modern
management tools. For example, reporting on public health
burdens, having a uniform way of reporting on disease code,
disease efforts, and how much are we making and how do you
measure whether or not this is effective or not? One of the
policies we proposed this year was the Public Access Policy, in
which we are going to have an archive of all of our scientific
papers so we can combine the investment with the output.
The second is evaluation, and I agree, someone on your
committee said that unless you have an explicit way of knowing
whether you are getting the results you are getting, you can't
define success. There is no planning that is necessary because
you just can't spend the money and spend the resources and
strategize without having an endpoint. That is easy to do. But
it is not so easy to measure and evaluate progress in medical
research. As you know, good research often fails. And this is
the challenge, and that is why I wanted a structure that would
tackle that challenge openly.
And the third is strategic coordination. We need to do a
nationwide planning as a matter of institutional policy, not as
a new director's preference. I think it needs to be
institutionalized. I think it needed to develop common
processes across NIH, and work with the IC's to develop plans
as needed.
I think this is essentially what we can do within our
ability to move the agency. We have shown that it can work
through the roadmap and obesity and neuroscience. It will
enhance the priority setting process and improve the
coordination. And it will also provide sound decision support
systems, something that will be transparent with broad public
and scientific input. And it will definitely, in my view,
ensure you that, in fact, we are making the efforts to try to
be as efficient as we can within our resources, while showing
where resources need to be invested, additional or not, in the
public health domain, when we can see data that indicates
indeed, it needs to be done.
So my goal is to really enter into this discussion whereby
authorities versus mechanisms versus vision get together to
serve, I think, science, the public health, and society in a
more--much more effective balancing, I believe, of all of the
opportunities we have and all the challenges we have.
Thank you very much for your attention. I am sorry I took a
little more time than planned.
[The prepared statement of Elias A. Zerhouni follows:]
Prepared Statement of Elias A. Zerhouni, M.D., Director, National
Institutes of Health, Department of Health and Human Services
Throughout history, the toll of suffering from disease and injury
on individuals and societies has been a constant and unforgiving
reminder of the human condition. We have never stopped trying to
overcome this suffering. Our goal is to help people, regardless of age,
gender, race, or nationality. The torment of those who are afflicted by
illness is the main reason that medical research is one of America's
top priorities.
Since the mid-twentieth century, the National Institutes of Health
(NIH), currently the largest supporter of biomedical research in the
world, has been the pilot and engine of the machine that drives
biomedical research. Congress's investment in NIH has paid quantifiable
human health dividends. We have come far in our quest for discoveries
that lead to improvements in diagnostics, prevention therapies, disease
or condition management, and actual cures. Life expectancy continues to
rise steadily; death from heart disease and stroke has sharply
declined; cancer mortality has fallen while survivability increases;
vaccines and drug therapies have proliferated--we can expect these and
other improvements in human health to accelerate as a result of
advances in genomics, molecular biology, proteomics, and computational
biology.
Yet as far as we have come in our journey, we still have a
seemingly infinite and difficult road to traverse. We understand
perhaps ten percent or less of the human biology necessary for the
prevention, diagnosis, and treatment of disease and injury. Our
understanding of the molecular underpinnings of cells and other aspects
of human biology offer promising theoretical applications for medical
treatment, but years of research efforts lie ahead before theories are
translated to concrete discoveries. The challenge of emerging and
reemerging infectious diseases will not lessen. The difficulty of
treating chronic illnesses is a persistent dilemma. Health disparities
continue to affect segments of our population. The threat of
bioterrorism represents yet another challenge.
These challenges are a reminder that NIH must continue to strive to
improve, to seek innovations, and to constantly subject itself to
review and examination. As I testified before this Subcommittee on June
2, 2004, ``Great organizations can maintain greatness only by
continuous reassessment and adaptation.'' Or as the Institute of
Medicine observed two years ago, ``While NIH's success is to be
celebrated, success alone does not answer fully the question of whether
there is a better way to proceed, particularly as one faces a future
where the world of biomedical science is being rapidly transformed in
virtually all its dimensions.''
To meet these challenges, the NIH works very hard to maintain a
research portfolio that balances public health needs and scientific
opportunities. We seek input through multiple channels, both formal and
informal, and maintain an open door policy for communication with our
stakeholders. Ideas for scientific initiatives in specific areas of
science come from many sources--advocacy groups, the biomedical
research community, Congress, and NIH staff, among others. Ideas for
stimulating a particular field or letting it lie fallow become reality
only after rigorous vetting at a number of levels.
The NIH's two-tiered peer review system, which is world-renowned
and respected, has a major influence in the priorities set by NIH. In
this system, the assessment of the scientific and technical merit of an
application is separate from its consideration for funding. In the
first level, the peers of the applicant assess the application's
scientific and technical merit. Advisory councils and boards, which
consist of senior scientific experts and lay members of the public,
provide the second level of review--advice and recommendations to the
Institutes and Centers on the programmatic relevance of the
applications and areas of science that should be emphasized (or not).
Advisory councils and boards are also, however, the NIH's top vetting
place for ideas for scientific initiatives that will receive set-aside
funds, and they are expected to provide advice on the Institute's or
Center's scientific priorities.
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-initiated
peer-reviewed research, NIH ensures that federal dollars support the
latest and best science. But the ideas generated by the scientific
community represent only one factor in a complex, multifaceted process.
Some of the variables in choosing resource allocations include public
health needs such as the burden of disease, new 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 sometimes
simplified by focusing attention on apparent differences between the
toll of certain diseases and the amount spent on research about those
diseases.
While I believe this process has served the public well--in fact,
there is evidence that NIH priorities match well with existing disease
burdens--we can do better. Currently, many priorities are set by the
planning programs at each of the Institutes and Centers at NIH that
support research grants. In recent years, NIH has facilitated
collaborations and co-funded innovative trans-Agency research. In the
case of HIV/AIDS research, we are shifting resources to fund vaccine
research to respond to urgency as well as opportunity. As scientific
fields and disciplines are increasingly becoming interdependent and
advances in one area often make progress possible in another, NIH needs
a horizon view of our research portfolio--a view that complements and
oversees the views of the individual Institutes and Centers with very
specific mission areas.
It is not only the nature of science that is changing. The
condition of our patients has evolved differently as well. Our success
in prolonging life and treating acute diseases means that more patients
are living with chronic and multiple illnesses. Our treatment methods
must adapt to older patients with multiple symptoms just as our methods
of conducting research must adapt to changes in science.
I testified before this Subcommittee last year that we cannot be
static, that 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. I
noted that the system of funding research by allocating resources
directly to disease, organ, or special population-based Institutes and
Centers has been successful. I also observed that science is changing,
driven by new technologies and discoveries. Modern research is often
best conducted by teams, which may include biologists, 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 or accounted for
according to specific diseases.
I told you that I was thinking about ways to refine the priority
setting process and the management of our portfolio. In particular, I
have been examining new and sustained approaches for evaluating NIH's
crosscutting science. While maintaining the support for existing
Institute and Center research programs, we are now using trans-NIH
resources to address emerging challenges and opportunities. These new
areas of investment involve research that no single Institute can
support alone, but that all of NIH needs to pursue because of the
impact on all diseases and scientific areas of inquiry.
I understand the questions about priority setting at NIH that many
have. There are several factors to be considered as we ponder the
answers.
Our challenges are different. The burden of illness has shifted from
acute to chronic diseases as health care costs rise and the
population ages.
As the Institute of Medicine concluded, ``The frontier of biomedical
science has rarely been as exciting and as full of spectacular
opportunities as it is today. From basic science through
clinical research to health services research, the
opportunities made available through the impressive advances of
recent decades in the biomedical as well as the physical,
computational and behavioral and social sciences have brought
us to a frontier of unprecedented opportunity.''
There is a dearth of reliable, integrated data on which to base
priority setting decisions, including insufficient information
on the human and financial costs of disease.
Numerous areas of science continue to rapidly converge in conducting
research, erasing the disease boundaries that had characterized
such research in the past.
After consulting with scientific leaders within and outside NIH,
and in order to meet these challenges while enhancing the priority
setting process at NIH, I have decided that the Agency needs a new
organization that will complement the existing process for determining
strategic research initiatives. I have requested $2 million in the FY
2006 budget to establish this new entity, the Office of Portfolio
Analysis and Strategic Initiatives, within the Office of the Director.
This office will be charged with evaluating the entire Agency research
portfolio to ensure that urgent public health needs are addressed in a
timely way and that a sound decision support system is established that
is based on rigorous and uniform sources of evidence.
Individual research grants remain the mainstay of NIH, and research
in priority areas will always be awarded competitively. However, NIH
also needs a global view of the totality of what we fund in our overall
research portfolio. This new office will provide--with input from the
Institutes and Centers and from the public, health care providers,
policymakers, and scientists--tools that facilitate trans-NIH planning.
It will drive data collection and sharing of information about research
fields, diseases, and conditions, and collect and analyze data on the
burden of disease. More effective analysis and management of our
portfolio will lead to even better progress against disease.
An expanded approach to portfolio analysis will enable NIH to
enhance the priority setting process while increasing coordination,
identify appropriate cycles of change, maintain proper turnover rates
for grants and provide much more accountability to Congress and the
public. Under such processes, in concert with the Institutes and
Centers, we would identify crosscutting research that requires common
investments from the various NIH Institutes and Centers. This approach
must include a regular overview of all research so that we can have
sufficient information to improve management of the entire NIH research
portfolio.
My intent in creating the office is to have a transparent process
and better decision-support tools characterized by a defined scope of
review with broad input from the scientific community and the public; a
solid, uniform database of information; an institutionalized process of
regular trans-NIH evaluations; better tools for weighing scientific
opportunity against public health urgency; and a process that enhances
accountability to Congress, scientists, patients and the public at
large.
The creation of the Office of Portfolio Analysis and Strategic
Initiatives is an important step in the process I began when I became
the NIH Director to increase collaboration among our 27 Institutes and
Centers and to pool resources, where necessary, to expedite research
and adapt to changes in scientific methods and new discoveries. Soon
after becoming the NIH Director, in May 2002, I convened a series of
scientific meetings to chart a ``Roadmap for Medical Research'' in the
21st century. Our purpose was to identify gaps and obstacles in
biomedical research that no single institute at NIH could fill or
overcome alone, but requires efforts by the entire Agency.
Three themes for the Roadmap were identified: Finding New Pathways
to Discovery; Creating Research Teams of the Future; and Re-engineering
the Clinical Research Enterprise. The Roadmap is currently funding $235
million in this trans-NIH initiative and we have requested an
additional $98 million for FY 2006.
The focus of the initiatives under New Pathways to Discovery is to
build a better "toolbox" for medical researchers in the 21st century.
By FY 2006, a network of Molecular Libraries Screening Centers will
identify novel small molecules with potential as biochemical probes for
investigating cellular pathways, and an Imaging Probe Development
Center will be fully operational and servicing the extramural
community.
Scientists need to move beyond the confines of their own discipline
and explore new organizational models for team science. The initiatives
within the Research Teams of the Future theme provide support to
academic and research institutions that focus on creating
interdisciplinary research training programs, workshops and courses for
development of new scientists, new science teams, and new scientific
inter-disciplines. In addition, specific support for high risk and
innovative research will continue to be supported by the NIH Director's
Pioneer Awards in FY2006.
The Re-engineering the Clinical Research Enterprise theme
initiatives aim to integrate and strengthen clinical research networks
and train multidisciplinary clinical researchers in order to accelerate
clinical studies and trials. Efforts to inventory existing networks and
test approaches to enhance informatics infrastructure will culminate in
the launch of the National Electronic Clinical Trials and Research
(NECTAR) network.
Implementation groups have been established to support each of the
three themes. For example, under the initiative to find New Pathways to
Discovery, there are separate groups for Building Blocks, Pathways and
Networks; Molecular Libraries and Imaging; Structural Biology,
Bioinformatics and Computational Biology; and Nanomedicine. These
groups are funding such initiatives as National Technology Centers for
Networks and Pathways; Molecular Libraries; a database of chemical
structures; a core synthesis facility to produce imaging probes; and
planning for nanomedicine centers.
Under the Research Teams of the Future theme, NIH is funding
planning grants to establish Interdisciplinary Research Centers; an
initiative to remove barriers to interdisciplinary research; and an
initiative to facilitate public-private partnerships in science.
Projects are also underway in support of the third theme, Re-
engineering the Clinical Research Enterprise. Initiatives include
Harmonizing Regulatory requirements; integrating clinical research
networks; and establishing core services for the translation of
research findings.
The Roadmap has been a significant step in shifting the culture of
NIH from single-purpose research funded by individual Institutes and
Centers to research that will benefit all endeavors and is funded by
multiple Institutes and Centers for the benefit of the entire Agency.
NIH has been gradually moving in this direction for the last decade,
but now we are advancing by leaps and bounds.
As an illustration of our responsiveness to emerging public health
threats, NIH launched the Strategic Plan for Obesity, a multi-
disciplinary approach to addressing a burgeoning health crisis. There
are 130 million obese American adults who are at risk of premature
death, chronic illness, and reduction in quality of life. In addition,
the obesity epidemic could cost the Nation $117 billion in medical
costs and lost wages. Obesity is an example of a public health
emergency that cannot be addressed by a single Institute, but must be a
trans-NIH research initiative. We have 18 Institutes and Centers
conducting research on such factors in the epidemic as behavioral,
sociocultural, economic, environmental, genetic and biological causes.
NIH researchers have identified an elaborate network of hormones
and other molecules that connect the brain, gastrointestinal tract, fat
cells, and other parts of the body to achieve energy balance. An
increased level of one of the appetite-induced hormones was found in
obese people following diet-induced weight loss. It may explain why
people have difficulty in maintaining weight loss. These hormones are
now targets for the development of drug therapeutics.
This year, another important example of greater trans-NIH
collaborations and coordination is the Neuroscience Blueprint. NIH has
15 Institutes conducting research on the brain, ranging from the
National Institute of Neurological Disorders and Stroke to the National
Institute of Mental Health to the National Institute on Drug Abuse.
This set of diseases exacts a burden estimated to reach $500 billion in
future years. By pooling funds and expertise, our Institutes will
collaborate on research addressing some of the most prevalent causes of
death and disability, including Parkinson's disease, ALS, Alzheimer's
disease, spinal cord injury, dementia, hearing loss, eye disease, and
muscular dystrophy. The Blueprint will conduct research on economies of
scale and train the next generation of neuroscientists.
These are prominent examples of how NIH is adapting to the need for
new approaches to medical research. Another example includes
collaboration on modeling simple organisms used in pre-clinical
research, such as the mouse, the rat, budding yeast, the fruit fly, and
the zebrafish. Also, NIH supports trans-NIH initiatives on health
disparities research, liver disease, autism, pain research, biodefense,
and imaging.
We will continue to facilitate trans-NIH research and assess
priorities in response to public health urgencies and scientific
requirements. However, the mainstays of NIH--peer review and
investigator-initiated research--are the cornerstones of our success.
This should be enhanced and not weakened. But with this in mind, NIH
will continue to seek the best ways of funding the whole continuum of
medical research with the ultimate goal of diagnosing, preventing,
treating, and curing disease.
Mr. Deal. That is all right. Thank you very much for that
overview of your agency.
Let me just start off with a few questions.
First of all, the Institute of Medicine has made a--noted
to us that Congressional disease-specific research mandates in
the late 1980's and early 1990's actually total more than the
overall NIH proposed budgeting, and required cuts in NIH
desired research projects. Over the past decade, Congress
generally has been reluctant to authorize new disease-specific
mandates. How has this impacted NIH's priority setting process?
Mr. Zerhouni. This is an excellent question, and clearly, I
think in the overall picture, when you look at specific disease
mandates, without a scientifically sound and public health
burden sound plan, then what you are doing is really
essentially allocating resources, not knowing if the capacity
is there, the opportunity is there, and so on. So by and large,
I agree with the IOM statement that disease-specific mandates
with dollars attached to them really distort the portfolio.
On the other hand, I think over the past 10 years, I have
to say that Congress has been extremely generous with us, so
that within years where increases were available, I think we
have been able to match our resources to a larger spectrum of
diseases than we did 10 years ago. So when you look across, as
I said, we have several hundred common conditions, about 400-
plus, and 6,000 rare conditions that we have to tackle within
the NIH budget. I can tell you that we have made great progress
in many areas because of this ability to move money within an
institute without having these rigid mandates.
Mr. Deal. Do you believe it would be helpful if a larger
portion of the budget were--was in these trends, NIH
initiatives, and if it was not agency institute specific? How
would you go about in making the decision as to how to allocate
those trends funds? Would it just be your decision or what
process would you use?
Mr. Zerhouni. Again, I think that we have experimented with
several processes the past 3 years. One is obviously--I don't
think top down direction is the right way to go. What you
really need to do is combine those three components. You need
to have a sense of what the public health challenge is, you
need to bring advice from the external scientific community
with the institutes themselves being involved. That is the
first step.
I think at the end of the day, what you really want is a
set of priorities. This is a priority setting mechanism--with
understanding where the scientific opportunity is, then have
the budgets that go with that, and have that as an open process
that would lead us to be able to make budget allocations in
agreement with all of the relevant institutes, just like in the
neuroscience blueprint. This is something that occurs with 14
institutes.
But I think the process needs to be institutionalized. I
think you need to have better tools to measure what it is you
are trying to accomplish, and this is what I would like this
office to start doing. It is not the end all, be all of how you
would do this, but if you have a very rigid appropriations
structure, then everything is moot. I can talk all I want, but
in tight budgets, it becomes very difficult to move monies for
priority areas.
Mr. Deal. The Office of Portfolio Analysis and Strategic
Initiative that you put on the screen, is that something that
would require legislative action by us to authorize, or is this
something within your authority to put in place?
Mr. Zerhouni. Well, as you know, this is within my
authority to put in place within the mandate that I gave the
office. Clearly, moving money and having any authority over
budgets and so on is not within the authority of that office.
Mr. Deal. So the flexibility on the budget authority would
be almost essential to make this work, I would assume.
Mr. Zerhouni. Right. So if you look at the office of the
director, Mr. Chairman, I would suggest that you compare, for
example, the authorities of the Office of AIDS Research with
the authorities of that office. You will see that this office
doesn't have the same authority of AIDS Research or Women's
Health, they have their own appropriation, their own
administrative structures, and their own ability to move money.
So for example, this year--last year I asked all of the
institutes to give me their 5-year forecast in terms of both
budgets, but also scientific challenge, and the NIID told me
about the 4 or 5 new vaccines that are coming--that are
becoming ready to be tested. It is a very expensive proposition
to test vaccines in a colloquial population. So that we could
see that there would be a shortfall of about $200 million in
2006, a tight budget year.
So what we did within the AIDS, Dr. Whitescarver runs the
Office of AIDS Research, reallocated across the entire
portfolio over $100 million for vaccine trials away from other
areas. That is an authority there that you don't necessarily
have. This is in the authorization legislation for that office
to coordinate that disease.
But I think what I am asking you to look at is instead of
having a disease specific authority and everything every time
you have a problem and a lot of mandates, you should see the
number of reports that I sign for transagency this and
transdisciplinary that and I think maybe this office should
really play that role. It should be the point where, you know,
things change. Priorities change. I mean, are we going to offer
to create an office of obesity research? Isn't that a public
health emergency? Why don't we create another one? Pretty soon,
you will end up with 27 institutes and 27 offices to coordinate
all these 27 players. No. you need one conductor, I think, and
that should be a generic office that is charged and empowered
to do what it needs to do to provide, with the institutes and
centers, a sense of coordination and strategic partnership that
is required.
Mr. Deal. Thank you.
Mr. Brown.
Mr. Brown. Thank you, Mr. Chairman.
Dr. Zerhouni, could you put the first chart up, the one you
started with, the life expectancy? Could you put that up for
us? And also, we would--I think many of us on the committee
would request that we have that whole--that we could see all
the charts, if we could. The first one was life expectancy.
Thank you.
I find that interesting. I think what is left out of that
chart and it is something that we all should celebrate in this
country, the success we have had as a government, as a society,
as a medical community of increasing life expectancy. But
really what is left out of that chart is an awful lot of public
health advances, and I would speak particularly--specifically
about safe drinking water laws, clean air laws, seatbelt/airbag
laws, social--the creation of Social Security, the creation of
Medicare, minimum wage laws, workers' compensation laws,
prohibition on child labor laws, prohibition on child labor 100
years ago. All of those are very much a part of that, and I
think if you took that chart and you overlaid it with the
number of dollars we spend as a society on healthcare, it would
make the chart look different. I think if we overlaid it with
healthcare indocies of other rich countries, everything from
life expectancy to infant mortality to maternal mortality to
inoculation rate to all of those things, I think you would get
a different picture.
My point of all of that is to be fair, we need to analyze
this much--not that I am critical of your chart or of your
presentation. We need to analyze it in a more encompassed way
to show that our increase from 45-year life expectancy 100
years to 3, 3.5 decades longer today is more about--is less
about medical technology and more about public health. And that
is what--I think if you look in that direction and you think
about where we are as a country, where we don't rank well, as
much money as we spend on healthcare, we don't look all that
good on a whole host of indocies except for one. Not our life
expectancy, not our infant mortality rate, not our maternal
mortality rate, we look very good on life expectancy at 65. So
in this country, if you get to be 65, you are going to live
longer than almost any other country in the world, and it just
happens to be that we have a healthcare program that starts at
65 for everybody. That is my one point.
My more important point is--and question is what do we do--
what do you do at NIH--and I know this is partly CDC's
function, but what do we do at NIH--what kind of strategies of
access? What are you thinking about? Your job is not just to
develop incredible things as NIH does, but to find a better
strategy to make them accessible. With the health disparities
that we have in this country, people that wear ties to work
like we do and have incomes like we do, and most of the people
in this room have a very long life expectancy in this country.
Many people don't that don't dress this way. The people that
come into this building and clean it at night, their life
expectancies are much, much less. Partly race, partly income,
partly a whole lot of things.
What does--how does NIH structure itself so it does better
with the whole health disparities issue?
Mr. Zerhouni. Obviously, you have studied the issue.
In terms of the life expectancy, I think if you look over
the 100 years and you then say okay, what created the most
progress in what time period, you will see that early on,
clearly, clean water and the ability to have less taxing
manufacturing jobs due to the industrial revolution, and
having, you know, appropriate hygiene methods was the main
driver of improvements in life expectancy.
Then you come to the midrange of the century. I think you
can clearly see that social policies and others have played a
big role.
If you come to the last third of the century to the 1970's
onward, you can't help but say that the progress that we made
has been primarily related to medical discoveries in prevention
and treatment.
So that is my sense. You need to correlate it to what
happened during that period of history. If you look at just
global life expectancy, the best way to make your life
expectancy look good is reduce infant mortality and
childbearing health issues, and you have it great. What is
important is what is the life expectancy at given ages? And you
pointed out that in America, if you live to be 65, you are
going to be living at a higher percentage than other countries.
The problem that we have in looking at statistics between our
country and other countries is what I mentioned as health
disparities. So if you look at different groups, different--you
find statistics that are actually very good. But if you look at
the, you know, combination, we have pockets in this country
where we have healthcare performance and health indocies that
we need to work on. And that is why I consider health
disparities a top five priority of NIH.
In answering your question about what are we doing for
access and making sure that--I consider that NIH has a
continuum of duties, and I will give you an example. One study
that we funded was called the OHAT study, which was the
hypertensive drug trial where we compared five different drugs
used in communities, 600 primary care communities. And we
followed 40,000, I believe, patients over a period of 8 years.
And two things were needed there. One, how do we improve access
to effective medications? And we found that the old diuretics,
the cheapest alternative, was actually the best alternative to
start with if you wanted maximum compliance.
The second is a follow up. We are now working through all
these community practices, and maintaining, if you will, the
knowledge and finding ways by which you do this. And as part of
the roadmap, one of the things that we have decided to do is to
create what we call a core, a community physician core, that
will be associated with NIH that understands that the
translation to better practices and access to medication needs
to be improved.
However, I would agree with you that you can not change
that unless you have a comprehensive set of policies, because
even though we have the best medical care in appointed areas,
where if you had a significant disease, cancer, heart disease,
this is the country to be treated in, but we don't have as
effective a health care system as we should.
Mr. Deal. Thank you.
Chairman Barton.
Chairman Barton. Thank you, Mr. Chairman.
Dr. Zerhouni, again, I would like to compliment you on the
steps that you have already taken and that you are continuing
to take on revitalizing the agency.
I have a few questions here that some of this I think is
going to be pretty straightforward, I think. Hopefully, you
will agree with me.
I suggested three ideas for restructuring and reforming the
agency in my opening statement. The first was that the center
that your directorship should have expanded authority. Do you
agree with that?
Mr. Zerhouni. I agree with that.
Chairman Barton. Okay. I thought you would.
Mr. Zerhouni. Who is going to turn down authority?
Chairman Barton. The second thing that I suggested was that
we need to better align the budget authority within these
various centers. Would you agree with that?
Mr. Zerhouni. I think we need to fluidify and make the
budget authorities less rigid than intended in the statute,
yes.
Chairman Barton. Okay. Well, could you put up--and first, I
compliment you on doing your own Powerpoint presentation. That
is fairly impressive to be sitting here all by yourself looking
at all these folks up here and doing that by yourself with no
staff assistance.
Can you--let us just see how good you are with that
Powerpoint.
Mr. Zerhouni. Now we are going to see.
Chairman Barton. Yeah. I want you to put up the chart that
showed the various centers and all--the latest organizational
chart.
Mr. Zerhouni. Okay. Let us see if I can pass the test,
here.
Chairman Barton. It is the one that shows all 27 centers
and various offices----
Mr. Zerhouni. Yes, I see it. I just need to--here we go. So
we started with this one, and then we went to this one----
Chairman Barton. Right.
Mr. Zerhouni. [continuing] and this one.
Chairman Barton. There you----
Mr. Zerhouni. NIH today?
Chairman Barton. Yes, sir. That is good.
Mr. Zerhouni. Thank you.
Chairman Barton. I was hoping you would goof up on it so I
wouldn't feel so bad.
Now, do you think that you could restructure that so that
it was a little bit more effective and efficient?
Mr. Zerhouni. I think you have two ways of making it more
efficient. One is having better tools, better management
principles. And then this, as I said, is the picture of a
holding company. The question is, what do you really have to
make them hold together as functional units.
Chairman Barton. Which box spends the most money up there?
Mr. Zerhouni. National Cancer Institute.
Chairman Barton. How much is that?
Mr. Zerhouni. $4.8 billion.
Chairman Barton. Which box spends the least money up there?
Mr. Zerhouni. The Fogarty International Center, $63
million.
Chairman Barton. So we go from $4.5 billion to what, $63
million?
Mr. Zerhouni. Right.
Chairman Barton. Okay. Should there be some order of
magnitude similarity between boxes?
Mr. Zerhouni. You could definitely explore that question,
Mr. Chairman, because for each box you create administrative
structures. And the IUM report actually discussed that issue.
Is it justified to create additional administrative structures
every time you have some sort of a structure mission that you
want?
Chairman Barton. And my understanding is that every one of
those boxes has their own administrative personnel, their own
accounting personnel, their own----
Mr. Zerhouni. Advisory councils.
Chairman Barton. [continuing] human factor personnel. I
mean, you created 27 little corporations. Is that correct?
Mr. Zerhouni. Well, I have changed that over the past 3
years. So for example, personnel offices, there is only 1 now
at NIH. We are going to an administrative restructuring plan,
which I can do within my authority for budget and finance.
However, what can not be changed are the, you know,
statutory structures that are there, such as you need a
director, you need an advisory council, you need a staff
infrastructure, FT and budget allocations. Those are things I
can't touch, as you know. If I need to do that, I need prior
permission for anything more than $1 million.
Chairman Barton. Okay. Now, in the office of the director,
the box that I am looking at that is to my right, you control
inside that box, right?
Mr. Zerhouni. Inside the box that is on----
Chairman Barton. On the right.
Mr. Zerhouni. [continuing] the right?
Chairman Barton. Top right.
Mr. Zerhouni. Correct.
Chairman Barton. Okay. What is the difference between the
Office of Community Liaison and the Office of Communications
and Public Liaison?
Mr. Zerhouni. Okay. Community Liaison is the office that
deals with our immediate neighborhood around the campus. As you
know, we have a large campus, a lot of friction between
communities around NIH, traffic, the fence, biosecurity. That
is what this office is supposed to do.
Chairman Barton. And the other----
Mr. Zerhouni. But I would agree with you that you don't
necessarily need to separate those 2.
Chairman Barton. I mean, just even within a little box that
you control, it seems a little bit convoluting to me. You have
got extramural research and intramural research, for example.
Why don't you just have research?
Mr. Zerhouni. That is a very good question. Actually, you
need that by statute. You need to have--grants management,
within our language, we have the obligation to manage grants to
the extramural community differently than we do for Federal
programs within NIH. So that has a good reason. I agree with
you that this needs streamlining.
Chairman Barton. Well, my time is expired and I don't want
to abuse the privilege.
But I would sure like to get some of your people with some
of my people and to coin a phrase, ``Think outside of the box''
a little bit. You know, if there--I don't want everybody that
is in one of those boxes to be all of a sudden that Congress is
out to get them. If that box needs to stay just like it is,
then that is the judgment of the committee and the folks that
we are going to be working with, so be it. But in this day and
age, when you look at how corporate America and all the various
communities are changing their culture and changing the way
their management is structured, it would sure seem to me that
we could create a lot less boxes and give a lot more
transparency and cross-communication and just be much, much
more effective at how we spend the dollars.
So I have said this before, and I want to repeat it. I
really hope that we can work in a bipartisan basis in the next
2 to 3 months to put an NIH restructure and reform bill on the
floor with your help.
And with that, Mr. Chairman, I yield back.
Mr. Deal. Thank you.
Ms. DeGette.
Ms. DeGette. Thank you, Mr. Chairman.
Dr. Zerhouni, I want to congratulate you for your
leadership at NIH, and in particular, I want to congratulate
you on your flexibility in your leadership role. Recently with
the new ethics rules that you instituted, I found that to be an
extraordinary experience where we sat here, we listened to the
testimony of the witnesses about the conflicts of interest, and
then you really thought about what you needed to do and you
enacted that bright line role. So I want to thank you for doing
that.
And I also want to ask you, I assume you are really
planning to review these rules in 1 year to make sure you are
not having a brain drain at NIH.
Mr. Zerhouni. That is correct. As I said, I think it is
very important to do the right thing, and to me what is the
most important goal of these interim final regulations is to
have a bright line, but we intend fully to evaluate the impact
of these rules. I don't think anyone can come up with rules and
regulations that are perfect day 1.
Ms. DeGette. Thank you.
The main thing I want to talk to you about today--you will
anticipate, I think, is the issue of stem cell research and the
NIH. As you know, in 2001, President Bush enacted a stem cell
research policy which said that the cell lines to be used, no
embryonic cell lines past August of 2001 could be used for
research. And I want to talk to you about what NIH is doing
about that, and what we can expect.
The first thing is, as in our previous discussions, is
there--is NIH funding any embryonic stem cell research right
now?
Mr. Zerhouni. Yes, we are funding embryonic stem cell
research under the President's policy.
Ms. DeGette. What is the level of that funding?
Mr. Zerhouni. About $25 million a year for human embryonic
stem cell, and about--actually, you will see how it works at
NIH here. Here is the dollars----
Ms. DeGette. You were expecting my questions, I see.
Mr. Zerhouni. I guess, or the staff was, anyway.
So here is embryonic stem cell research from 2002 to 2004.
It goes from $10 to $24 million.
Ms. DeGette. So----
Mr. Zerhouni. For human embryonic stem cell research.
Ms. DeGette. So it is still $24 million. Do you have
ethical oversight of that research? What kind of ethical
oversight have you instituted?
Mr. Zerhouni. Well, within the research that we can fund,
we have obviously all of the ethical oversight authorities that
we need to have as in the guidelines.
Ms. DeGette. And do you have ethics rules over that
research that you can fund?
Mr. Zerhouni. Well----
Ms. DeGette. You have written----
Mr. Zerhouni. Human subject ethics rules, yes. Obviously,
ethics rules that relate to the conduct of the trial, a human
trial----
Ms. DeGette. Right, but----
Mr. Zerhouni. [continuing] would be relevant, but at this
point, there is none.
Ms. DeGette. You don't have any discreet stem cell research
ethics--embryonic stem cell research ethics----
Mr. Zerhouni. Guidelines. We have guidelines.
Ms. DeGette. Mr. Chairman, I would like to ask Dr. Zerhouni
if he can supplement his answer with a copy of those
guidelines?
Mr. Deal. Sure. I would appreciate it.
Ms. DeGette. Now, when the President enacted his executive
order in August, 2001, Dr. Zerhouni, as you know, the
administration thought there would be roughly 78 lines of stem
cells. Correct?
Mr. Zerhouni. That is correct.
Ms. DeGette. And as I understand it, researchers at this
point in the United States, NIH funded researchers, have access
to only 22 lines. Is that correct?
Mr. Zerhouni. That is correct.
Ms. DeGette. Now, I am wondering if your researchers have
any thought that there might be additional lines to those 22
lines that they could get access to in the future?
Mr. Zerhouni. So when we started in 2001, we had 87
derivations. In other words, cell lines that had----
Ms. DeGette. Yes, I understand.
Mr. Zerhouni. Right. Not been dropped. And in 2002, we had
1 that was widely available, and we developed 22 that are now
widely available.
Ms. DeGette. Right, but are we going to have more in the
future besides the 22?
Mr. Zerhouni. We don't know, because there are 31 of these
derivations that have not been expended or made available by
their owners.
Ms. DeGette. And most of those owners are abroad, correct?
Mr. Zerhouni. Most of them, yes.
Ms. DeGette. But I mean, certainly right now, you have the
22 lines, right?
Mr. Zerhouni. Um-hum.
Ms. DeGette. Now, do you know--there was an NIH internal
report which said it is unlikely that the Federal Stem Cell
Registry will ever have more than 23 lines available. Is that
true?
Mr. Zerhouni. Well, I don't know if it is stated that way.
When we looked and asked, I said what are the lines? What is
the status of the lines? And we have a mechanism by which we
develop these lines, which are called development grants,
infrastructure grants. So based on that, we knew that we had
institutions that were going to develop up to 23 lines. We had
also attempted to develop others which didn't expand. They
failed. And then there were 31 which are in India, Sweden,
Korea----
Ms. DeGette. Right.
Mr. Zerhouni. [continuing] which we had no contractual
relationship for them to develop.
Ms. DeGette. Right. And are you trying to pursue that
contractual relationship to get those lines?
Mr. Zerhouni. We try, but right now most of them want to
keep those in reserve----
Ms. DeGette. Right.
Mr. Zerhouni. [continuing] and when more is known----
Ms. DeGette. So you don't know if you will have access to
those?
Mr. Zerhouni. I can not tell you that we will.
Ms. DeGette. Now, in May of 2004, you told me and
Representative Mike Cassell, who had legislation pending on
stem cell research ``It is fair to say that from a purely
scientific perspective, more stem cell lines may well speed
human embryonic stem cell research.'' Correct?
Mr. Zerhouni. That is correct.
Ms. DeGette. So it would help you to have more cell lines
to expand the research?
Mr. Zerhouni. It may. In other words, one can not--in the
state of knowledge that we have now, one can not say that
purely scientifically you could not have--make a discovery on
lines that are not grown the way that we grow them for NIH
scientists in the stem cell field as early as it is.
So purely--from a purely scientific standpoint--and many
scientists will say that as well as I, that you can not argue
that not having all cell lines at different ages may not be
helpful. But the policy is predicated on its moral and ethical
basis.
Ms. DeGette. I understand. And you and I have discussed
this before, the President's policy is not based on a
scientific principle, but rather the President's own moral
beliefs, correct?
Mr. Zerhouni. That is my----
Ms. DeGette. Okay, thank you.
Now, are you aware that a recent scientific journal article
reported that all of the 22 federally approved lines are
currently contaminated with mouse feeder cells?
Mr. Zerhouni. All lines that have been developed worldwide
have that problem.
Ms. DeGette. Oh, okay.
Mr. Zerhouni. Not just----
Ms. DeGette. Not just the 22, but all of them have the
mouse feeders.
Mr. Zerhouni. It is not just the Federal lines, it is all
150 lines that we know about. There is 1 that may not have been
exposed.
Ms. DeGette. Now, I thought there were some scientists with
private funding who were developing some cell lines that were
not contaminated with mouse feeder cells?
Mr. Zerhouni. Yes, they are attempting, but there is none
that I know of.
Ms. DeGette. In the future, from a scientific standpoint,
Doctor, don't you believe that it is going to be helpful to
have an advancement of cell lines that is not derived from
mouse feeder cells?
Mr. Zerhouni. Clearly, if you look at clinical indications,
downstream, even though this issue of mouse cell line exposure
is not an absolute no-no, because FDA currently approves things
that are on mouse cell lines and so on. And so it is
manageable, but clearly----
Ms. DeGette. It is manageable right now for the basic
research state----
Mr. Zerhouni. That is right.
Ms. DeGette. [continuing] in its nascent stages. But as we
go down the road, clearly, you are not going to be able to
sustain that over time, correct?
Mr. Zerhouni. It is from the purely scientific standpoint,
it would be desirable sources of lines that have not seen
animal products.
Ms. DeGette. And that is not going to happen under the
President's current policy limiting research to the lines in
existence of August, 2004.
Mr. Zerhouni. The facts as we know them is that the--of the
31 lines that have not been expanded under the President's
policy, 16 are said, to the best of our knowledge, not to have
been exposed, and that the only----
Ms. DeGette. But we don't have access to those, they are
fraught, right?
Mr. Zerhouni. No, but the owners of the lines are saying we
will wait until we know more about how to grow these lines
without mouse feeder cells before we expand them. So the
theoretical possibility is that they could come and say we
would like those lines to be available to be eligible for
Federal funding, if that happens.
Ms. DeGette. Thank you, Mr. Chairman.
Mr. Deal. Thank you.
Mr. Bilirakis.
Mr. Bilirakis. Thank you, Mr. Chairman, and thank you,
Doctor, for your as usual fascinating presentation. There
aren't too many of here--although I think most of the members
of the subcommittee here, and I think they ought to just look
at their faces. They were entranced with your comments.
As sensitive as disease specific is that you and I have
talked about this over the years and it has been something that
has been sort of a real problem for me over the years when I
was chairing this subcommittee. But I am going to go into a
question regarding it. It involves the granddaughter of a
member of this House of Representatives, a member from the
other side of the aisle, but it would help us, I think,
understand. You know that the concern here is we can do all of
this great stuff, but it is ultimately what it does at the
bedside that counts. And so possibly, it can help us maybe get
to that.
I am referring to--well, my understanding is that there has
been a growing interest within the research community about a
disease known as primary pulmonary hypertension. As you know
and most of us don't know up here, this is a serious and often
fatal condition in which blood pressure in the lungs rises to
dangerous levels. It is a devastating disorder that
disproportionately affects young women, and often leaves
patients no treatment options other than a heart or lung
transplant.
I wonder--and I have this great concern, too, about the use
of clinical trials and how available that information is to the
general public and whatnot, and to the loved ones of someone
who has a particular disease. So in that connection, and also
relating to this Office of Portfolio Analysis and Strategic
Initiatives, could you update us on how you feel setting up
this office would further promising research into this disease?
And maybe tie that into the clinical trial availability if you
would, please.
Mr. Zerhouni. Thank you. In terms of trials, one of the
good things we have done, which I think whoever you are talking
to should really go look into, is what we call a
clinicaltrials.gov data base. And this data base is public and
we have over 12,600 trials listed in that data base. Actually,
if you look at it, we have 12,667 trials listed, and 51 percent
of those are sponsored by NIH, 21 percent by pharmaceutical
companies, and 25 percent sponsored by universities. So I would
encourage them to look at what could be in that----
Mr. Bilirakis. Does this contain all of the clinical
trials?
Mr. Zerhouni. Every clinical trial we know about.
Mr. Bilirakis. Every one that you know about?
Mr. Zerhouni. Yes. In addition, I would say that in
pulmonary hypertension, the institute that is charged with that
mission is National Heart and Lung and Blood Institute. There
are about 80 projects on that, based on the information that I
have. But there are--in 2007, there is an initiative called
specialized centers of clinically oriented research in
pulmonary vascular disease that they are going to start.
Because as we look at the total burden of this disease, it
turns out that pulmonary diseases are really an issue that we
need to invest more. Specifically in pulmonary hypertension, we
are trying new drugs. As you know, some of the vascular active
drugs, Sovenifil is used in pulmonary hypertension in adults. I
don't know about the age of the patient that you are referring
to. And we are putting an international scientific conference
on that.
So clearly, a problem. We do realize that it affects
younger individuals, and it is a very malignant disease, even
though it is not a cancer. And we are clearly not focused on
this----
Mr. Bilirakis. We are focused on it.
Mr. Zerhouni. Right. The National Heart, Lung, and Blood
Institute, we have not looked at this as a trans-NIH
initiative, sir, because we did not believe that it really goes
across institutes in terms of missions.
Mr. Bilirakis. Thank you for that, sir.
Let me also ask you very quickly on the potential impact
that the neuroscience blueprint may hold for research into
paralysis and spinal cord injury and dysfunction. And I guess
the question, principally is does the current structure inhibit
research in those areas?
Mr. Zerhouni. That is a more difficult question to answer
because in spinal cord injuries, you know, the National
Institute of Neurological Diseases is primarily related. There
is research now that is going in regenerative medicine, stem
cell research that may have an impact on paralysis,
bioengineering research in the National Institute of Bioimaging
and Bioengineering. So there is more and more interdisciplinary
activities in not just curing paralysis, but managing
paralysis. I can show you the funding for it. This is the
funding between 1997 and 2006, went from $60 to $91 million
just for spinal cord injury alone across NIH. Most of the
increase is related to other institutes coming together. We
have a new clinical trial network that is brought together, and
the reason that we have--NINDS is doing this is because the
time at which you intervene after a spinal cord injury is
critical. It is like heart attacks. You can intervene early,
you can have great results. We have had a tremendously
promising set of research programs last year where early
intervention seemed to lead to faster recovery, deeper
recovery.
Where we are not making progress is long-term paralysis,
where I think we need to search for more answers.
Mr. Bilirakis. Thank you so much, sir. Thank you, Mr.
Chairman.
Mr. Deal. As you see, we have a vote on. I think probably
we need to go vote and come back. There are a series of votes.
I would ask you to return and we will continue with the
questioning at that time.
Mr. Zerhouni. I will go by the camera and see if they will
interview me.
Mr. Deal. Okay, you do that.
Mr. Zerhouni. I don't think so. Thank you, Mr. Chairman.
Mr. Deal. We will stand in recess until after the vote.
[Recess.]
Mr. Deal. Ms. Capps, you are next.
Ms. Capps. Thank you, Mr. Chairman and Dr. Zerhouni. Thank
you very much for your thorough presentation and for being here
today. I want to make a cautionary statement, a concern, and
then ask you a series of questions with, I hope, brief answers
so I can cover several topics.
I was pleased to see that you have put a new policy on
enhancing public access to archived publications resulting from
NIH-funded research. I think this a good step forward. But I
have to say I am not convinced that the voluntary publication
of research results is going to be effective, and I am
concerned that this new policy should be as good as it possibly
can be. Public access to the fruits of taxpayer-funded research
really makes sense, and so my caution and concern is that I
hope you will make sure that the policies uphold that priority.
I want to turn to the topic of NIH reauthorization.
Chairman Barton and others have made it very clear that they
want the committee to reauthorize the NIH this year. My
question that could even have a yes or no answer is, do you
think a reauthorization bill is necessary, emphasis on the word
necessary?
Mr. Zerhouni. I think it is appropriate, given the
description of how NIH needs to be more flexible to look into
the opportunities for improvements in the authorization
process, while we provide you with the information that you
need to make that determination.
Ms. Capps. Maybe a better way to say it is would this be
your No. 1 priority or if you had to rank a series of
priorities, where would this fall?
Mr. Zerhouni. As I said, I think better portfolio
management, the ability to integrate our research across more
than one unit is certainly a priority for me.
Ms. Capps. Okay. So I am taking by that, that that would be
your first priority?
Mr. Zerhouni. Right.
Ms. Capps. And our interest in supporting that is what you
would prefer.
Another topic--I am all over the map, I am sorry. But
another issue of great importance on NIH reauthorization is, of
course, stem cell research that our colleague, Ms. DeGette
asked you a series of questions about this. But I want to focus
on your role. As the director of NIH, you would be involved in
any discussion of reauthorization. So I am wondering and
concerned about the flexibility that you have--the authority
that you have, really, to negotiate changes in the
administration's policy on stem cell research. I mean, you were
handed something from the President and have implemented that.
Now if we reauthorize the NIH, how do you see your role in that
process?
Mr. Zerhouni. As you know, I am a Presidential appointee
with Senate confirmations. I am part of the executive branch
under the--and I report to the Secretary of HHS and the
President. So from that position, I am, you know, bound to
represent obviously, the policy of the administration and
uphold--and the President, and uphold the laws passed by
Congress. On the other hand, with my capacity as a scientific
advisor, if you will, I provide information to the executive
branch as well as to Congress, based on what we know from our
scientific exploration of the field, and we have continued to
do so as openly as possible to inform the authorization
process.
Ms. Capps. I guess----
Mr. Zerhouni. I don't have authority to negotiate, is what
I am saying.
Ms. Capps. You do?
Mr. Zerhouni. No, I do not.
Ms. Capps. You do not have the authority to negotiate----
Mr. Zerhouni. Independently, NIH does not have an
independent authority to negotiate policies related to stem
cells or any other issue.
Ms. Capps. When you give the President information----
Mr. Zerhouni. Right.
Ms. Capps. [continuing] do you have persuasive powers in
that respect? I am trying to pin you a little bit.
Mr. Zerhouni. Most--I believe so. I think I have a great
smile and----
Ms. Capps. All right. I am taking it to be that this policy
comes from the White House, and that you--there is no or very
little flexibility there?
Mr. Zerhouni. Actually, in law, we----
Ms. Capps. You are bound by law.
Mr. Zerhouni. [continuing] implement the policies of the
President and the laws passed by Congress.
Ms. Capps. So you cannot, for example, support further
limits on stem cell research?
Mr. Zerhouni. Support further limits?
Ms. Capps. Yes.
Mr. Zerhouni. I can not set policy, and policy is not set
at the NIH level. We can inform policy, and we do so.
Ms. Capps. You can----
Mr. Zerhouni. Inform policy, and we do so.
Ms. Capps. But you can't support tightening restrictions on
stem cell research, and you also can not--oh, I see. You can
support tightening research on stem cell research? You can
support that?
Mr. Zerhouni. No, I can not----
Ms. Capps. You are supporting it, because that is the
President's policy.
Mr. Zerhouni. I inform policy, obviously, and I can talk
about the state of science and how it is evolving, inform the
President and the Secretary of Health and Human Services on the
evolution----
Ms. Capps. Right.
Mr. Zerhouni. [continuing] of science and policymakers like
yourself.
Ms. Capps. Do you----
Mr. Zerhouni. But we do not determine policies.
Ms. Capps. Right. Do you advise the President about his
changing policy?
Mr. Zerhouni. When asked, yes.
Ms. Capps. If you are asked?
Mr. Zerhouni. Yes.
Ms. Capps. Okay. I have a lot of questions on that topic
still, but I want to turn to a very important topic to me and
to many of us here in Congress. The goal of the National Cancer
Institute, which it has set, I am co-chair of the cancer policy
group here in the Congress. You have set out, NCI has set out
the goal--very impressive goal of eliminating all cancer death
by 2015. It is certainly a laudable goal. And we in Congress
were very proud of our record--track record of doubling the
funding for NIH a few years ago. But--and in the subsequent
years, NIH however, has only received an increase of about 3
percent in the last few budgets. But this President's budget
this year, the NIH is restricted in its budget, really flat
lined. And in fact, the NCI has been budgeted in this year's
budget to receive less than a 1 percent increase in funding.
Which given inflation and all the costs of everything, is
really a cut.
So I want to hold on to that goal with you, but I wondered
if you think you have any chance of meeting it at this current
funding level?
Mr. Zerhouni. Any chance of--I am sorry, I missed the----
Ms. Capps. Of meeting that goal, given this budget. This
budget is about to be voted on in the House, but it isn't
appropriated yet.
Mr. Zerhouni. As you know, this is a very laudable goal
that Dr. Von Eschenbach sort of created as a vision, as a reach
goal for the National Cancer Institute to eliminate pain and
suffering--death and suffering from cancer by 2015. This has
the advantage of galvanizing, if you will, the scientific
community, the patient advocacy community, and you know, the
institutions that we work with.
It is clear that as we look at very difficult budgetary
times and increases that require us to make tough choices, the
NCI is going to have to balance. And I think Dr. Von Eschenbach
said it, in this budgetary environment, you are going to have
make choices between what it is you want to do at the expense
of something you no longer will do.
Ms. Capps. I don't want to interrupt you, but I want to be
able to frame the last few seconds on this topic. If NIH
reauthorization comes up, I am hoping from my role that we can
count on your support to make sure that NCI's special status--I
mean, this is a huge part of the budget, but it has this goal
that will be better achieved, if you will, if there is the
proper support for it.
Are you willing to keep that status?
Mr. Zerhouni. I think the authorities of NCI have been
historically very helpful since they started in 1971. I think
some of these authorities, I mean, we should look at them and
maybe have other institutes gain from that. I mean, from my
experience at this time, the authorities have been a positive
thing that I don't think pose a problem for NCI or for NIH. So
it should continue, I believe, to be a positive development for
NCI.
Ms. Capps. The special status of NCI?
Mr. Zerhouni. Right. And what I am saying is if you look at
the authorities, there are different ones, but there are some
that I think we should look into and maybe expand them to other
institutes. The construction authorities, some of the training
authorities, I think there is a debate to have about maybe
expanding the authorities that are in the cancer field to some
other of these groups, because they have been helpful
historically.
Ms. Capps. I understand. Thank you.
Mr. Deal. Gentlelady's time is expired.
Mr. Ferguson.
Mr. Ferguson. Thank you, Mr. Chairman.
I have some questions for Dr. Zerhouni. I thank you very
much for being here. I know you have appeared before our
committee before and I appreciate the opportunity to talk with
you a little bit.
I want to follow up on some of the questions about stem
cells. Of course, one of my colleagues stated earlier that
policy that the President instituted in 2001 had to do with the
President's own personal ethics. Of course, the question of
when life begins is not a question of someone's personal ethics
or their religion or anything else. It is a question of
science. I think that is important to note for the record.
Dr. Zerhouni, are you familiar with Laura Dominguez? She
was a patient who was paralyzed from the chest down through an
injury, and today walks because she has been treated with an
adult stem cell treatment, and she walks today with the help of
braces. It is an incredible story. She was here in Washington
last year telling her story. I am not sure if you are familiar
with a woman named Patricia Durante, who is a leukemia patient.
She was 6 months into her pregnancy when she was diagnosed with
acute leukemia, and they delivered her baby, her daughter early
because she had to be treated for her leukemia. And she was
treated with some of her daughter's umbilical cord stem cells.
And her leukemia has been cured. It is an incredible story.
Both of these treatments had nothing to do with embryonic stem
cell research. And of course, these are just two of the
thousands and thousands and thousands of people who have
actually been treated with varying degrees of success, some
very successfully with adult stem cells or stem cells taken
from placentas or umbilical cords.
My mom passed away a year and a half ago from multiple
myeloma. She was 59 when she died, young woman. When she was
diagnosed, her physicians told her that she would have a year.
She was stage 4 multiple myeloma, bone marrow cancer. When she
was diagnosed, her doctors told her that she had a year,
possibly 2, to live. She ended up living for 6 years. She got a
bone marrow transplant, which didn't work all that well,
unfortunately. And then she was given an experimental adult
stem cell treatment, which enabled her to live an additional 4
years. Absolutely confounded the physicians and her doctors at
Johns Hopkins Hospital in Baltimore with the success of this
treatment. I tell you that story not because--just because I am
happy that my mom was able to live an additional 4 years, but
when she was diagnosed, she had no grandchildren. And when she
died, she had been able to meet three of her grandchildren.
Three of our children had been born by that time. It is not
just important, of course, because my mom got to meet her
grandkids, but frankly, more importantly, her grandkids got to
meet her, and they will have that for the rest of their lives
because of this incredible treatment, which did not come from
embryonic stem cells. It came from adult stem cells. And to my
knowledge, there are exactly 0, not people today who have been
treated with any degree of success at all with embryonic stem--
technology that has come from embryonic stem cell research.
When, in fact, there are thousands and thousands and thousands
of people, dozens and dozens of clinical trials, incredible
work being done. And you know all this, being done with adult
stem cell treatment.
So I think that is certainly important to point out for the
record that the real promise in this field is in adult stem
cell research. And I have a ton of biotech in my district in
New Jersey. We have some of the leading companies, researchers,
and scientists in our district in New Jersey. And I sit with
them and I meet with them on a regular basis. And I ask them
about this. I say, do you do--you know, a lot of them do work
with stem cell research. And I am a big proponent of stem cell
research. I am a proponent of ethical stem cell research,
research that does not destroy human life, that does not
destroy a human embryo. And I think it is important to point
out, after all the hype and the propaganda sometimes, that the
facts are that there are thousands of people today who have
been treated successfully with adult stem cell technology and
research. There are dozens and dozens of clinical trials and
applications from this type of research, and there are 0, none,
that have come from embryonic stem cell research. The most
optimistic proponents of embryonic stem cell research say that
those types of advances are 15 or 20 years away. And why we
would continue to pour money into this frankly most unpromising
field when we have such a promising and ethical form of
research which is already showing such great promise seems to
me should be an easy call.
I want to continue this and I actually do have a couple of
questions. According to a recent RAND Institute study--and I am
sure you have seen this, and I will quote from it. It says
``Using a conservative estimate between the two conversion
rates from blastocytes to stem cells, noted above 27.5 percent,
the research team that calculated this that about 275 embryonic
stem cell lines could be created from the total number of
embryos that are available for research.'' Of course, we are
talking about frozen embryos in banks today. Even this number
is probably an overestimation, because it assumes that all of
the embryos designated for research in the U.S. would be used
to create stem cell lines, which is highly unlikely. That is a
quote from the RAND Institute.
In your opinion, when the proponents of research as I have
described kills human embryos, say that they can cure more than
100 million people from any number of diseases and conditions
if they can just get their hands on those human embryos in the
IBF clinics. Are they basing these claims on hype, or is that
completely backed up by science?
Mr. Zerhouni. Well, thank you for putting me in an area
where I think what you are seeing in the debate is the cross
section or an intersection between science and society. I think
in the context of what you just described, how can you make a
projection of 100 million this or that? I think we are too
early in our knowledge of this field to be able to predict how
or where and when you will be able to truly implement a cure or
a palliation for degenerative process, whether it be diabetes
or Parkinson's disease.
So I think there is no logical connections, I think,
scientifically to connect one to 100 million. I would say, on
the other hand, that the issues that we are dealing with right
now are a more basic level than clinical applications. And at
this point, no one really knows what direction this whole field
will take. It is impossible to tell if it is going to be 100
million in a revolutionary process. But from the scientific
standpoint, there is no doubt that we need to understand what
goes on in stem cells, adult and embryonic, in terms of their
DNA being programmed and reprogrammed and what is the best
pathway? There is no doubt that all scientists would like to
have their science evolve without being in conflict with any
societal or moral issues. But scientists have to also look at
the question in front of us, and that is that can you program
or reprogram the DNA of the cell so that it can do things that
you have lost the natural ability to do to get the miracles
that you are describing?
So I would say that there is no logical connection that you
can drive, as long as you haven't really understood the
fundamentals of this field.
Mr. Ferguson. And of course, I am sure you would agree--and
my time is up. I am sure that you would agree that unbridled
science research is not a virtue in and of itself. Of course,
as in all of these decisions, our ethics must form and inform
our science.
Mr. Zerhouni. I have to tell you, I agree that science has
to evolve within its social context, and scientists do that. I
mean, frankly, when you look at--I just received an award on
behalf of NIH for the recombinant advisory committee, which was
the committee that looked at genetic engineering, a very
concerning area from the standpoint of morals and ethics in
society, and it was created by scientists. There was a
moratorium for a year and a half on all that study until good
guidelines could be put together.
So I think there is no doubt that the question, as you pose
it, is we need to engage in a balanced dialog and a balanced
mechanism to make sure that science evolves in conjunction with
our moral and ethical principles.
Mr. Ferguson. Thank you. Thank you for being here today.
Mr. Deal. The gentleman's time has expired.
Ms. Baldwin.
Ms. Baldwin. Thank you, Mr. Chairman.
Dr. Zerhouni, you deal very gracefully with issues,
controversy in science. And I have two questions that deal with
controversy in science. One really following up on the last two
questioners regarding embryonic stem cells.
Clearly, embryonic stem cells have a different set of
traits than adult stem cells, including the capacity to be
coaxed, if you will, into becoming other specialized cells, a
capacity that adult stem cells do not have. In answer to a
question posed by Mrs. Capps, you talked about your role as a
scientist advising the administration, advising the Secretary,
advising the President. And so I guess I would ask you at this
juncture, how would you advise the President with regard to
what future policy ought to be on embryonic stem cell research
and the limitations?
Mr. Zerhouni. I think I wrote a letter to Congress about
the issue that was asked.
I think right now what we are dealing with is a very basic
stage of the research. We really need to understand better at
the molecular level what draws the differentiation. I mean, you
are talking about the potential of embryonic stem cells to be
pluripotent versus adult stem cells being multipotent, have
different potentials. But frankly, if you look at the data, if
you look at the signs, right now we are even still working on
how to characterize the particular cell lines that we have, the
22 cell lines. So last year, we advised to create a stem cell
bank, a national stem cell bank so we can have side-by-side
comparisons of these lines. We have also pushed for
experimentation in terms of stem cell specialists working with
disease specialists. Remember, embryonic stem cell is a young
field. It is only been funded for 3 years.
So I think my advice is that we need to continue our
exploration of the fundamentals of this field to be able to
really determine where we need to be in the next few years.
Ms. Baldwin. Okay. On another issue, we know that 6 of the
10 leading causes of death in the United States are based in
part on behavioral factors, such as smoking, violence, diet,
and substance abuse. And that other behavioral factors are also
known to increase an individual's risk for disease, disability,
even early death. This is certainly the case in the obesity
epidemic. In a document entitled ``Fiscal year 2006
Justification of Estimates for Appropriations Committee'' put
out by the Department of Health and Human Services, the
importance of behavioral research is underscored, especially as
it relates to the obesity epidemic.
For example, the document reads ``The obesity epidemic has
been fueled by a complex interplay of behavioral,
sociocultural, economic, and environmental factors acting
against a backdrop of genetic and other biological factors.''
And it continues, ``Continued behavioral research should
greatly enhance the understanding of factors that contribute to
obesity and may assist with future design of both pharmacologic
and lifestyle interventions.'' Now, despite the promise that
behavioral research holds in a number of areas, we have seen it
attacked in recent years. These attacks have sometimes occurred
in the media. Periodically, we have even seen legislative
interventions or threats of legislative interventions. And
given the sometimes hostile climate toward behavioral research,
but given its importance to understanding and combating some of
these epidemics, I am asking you what sort of efforts have you
taken to ensure that behavioral research receives appropriate
resources and does not become skewed, if you will, by political
influence?
Mr. Zerhouni. Well, in the context of--first of all, let me
agree with your overall statement that behavioral factors are a
main driver of disease burden, and we need to understand better
what--why is that.
NIH spends about $2.9 billion, you know, on behavioral
social science research, so it is not something that we ignore.
We have an Office of Behavioral Social Science Research in my
office to stimulate and coordinate these activities. The
neuroscience blueprint program that I announced this year
includes a major component to it that focuses on behavioral
research.
In terms of obesity research, what I would like to show you
is the NIH strategic plan, which is on our website, which was
developed last year. And if you look at that plan, you will see
that a major component, in fact, is behavioral research. One of
the things that we are very concerned about is if you look at
obesity and the relationship of obesity in children, relative
to the body mass index of their mothers, what you can see here
is that if you have a patient who is in the normal range of
weight between 18 and 25, there is a 10 percent chance of
obesity in their children, with the mother being in that
weight. There is a 10 percent chance. If you go to an over 40
index, there is almost three times more chances of obesity. So
what the plan talks about is how to intervene behaviorally, not
just in adults or teenagers, but now intervene in the stage of
intrauterine life, and very early preschool. And the NIDDK, the
institute that is leading this effort, is running, actually,
trials to change the behavior.
Now, one of the things that is clear is that this is not
going to happen without a lot of collaboration across multiple
entities of the Federal Government. And we need to really
underscore that. It is going to require changes on the ground
in terms of the set up of cities, and how much walking, how
much diet, exercise you can fit in into a normal life pattern.
But I think I am very clear that behavioral and social science
research is going to take an important--an increasingly
important role.
Mr. Deal. Gentlelady's time is expired.
Mr. Upton.
Mr. Upton. Thank you, Mr. Chairman. I won't use, I don't
think, all of my time.
Dr. Zerhouni, we welcome your opening statement. It was
terrific and I certainly join with all my colleagues on both
sides in very strong support of the NIH and its budget
requests. I was one of those, many years ago, that helped on
doubling the money, and I am glad to see that it was
successful.
I have two basic questions. The first is I want to get a
better understanding of the new grants submission from start to
finish, in terms of review and funding selection as it works,
and then use that--what it is under present, then look and see
how that process changes under the reforms that you are
advocating. That is my first question.
And why don't you go ahead. And I have one other question I
want to make sure I get in, but go ahead.
Mr. Zerhouni. So new grants. Every year, we fund about a
grant for about 4 years. We fund about 43,000 total grants, so
every year we have about 10,000 new grants to make, on average.
Of those, about \2/3\ come from scientific advances and changes
in scientific fields that are of great importance, whether it
be genetics or genomics and so on.
Those come to NIH, and we get about 40,000 applications for
these 10,000 grants. So we fund about 1 in 4, a little less
than 1 in 4. At the doubling, we funded about 1 in 3, but the
doubling, you know, was 3 years old now, and the post-doubling
has led to more applications for new grants.
When the new grant comes in, we have a center for
scientific review which is independent of the institutes. And
there are peer review sections there. They review all of the
grants and they score them in terms of quality of research,
potential public health impact. One of the things we have done
over the past 2 years is to make sure that the public health
relevance of every grant is explained in plain language. Then
those grants go in and they go to the--they are referred to the
various institutes. If it is a cancer grant, it goes to the
Cancer Institute, and so on.
Then within the budget that you have, you have to make a
priority list. In the past, the top 30 percent were given
grants. Now it is the top 22 percent. And it goes to the
advisory council of the institute. They review that and then
they say, you know, we need to fund that many, but we need to
fund more in this area rather than this area. And what they
could see, also, is that perhaps we are not getting enough
grants in a given area of science. Maybe we don't have enough
grants in leukemia, or not enough grants--so what they would do
then, they will instruct institute staff. We need to have more
presence in leukemia or obesity, for that matter, which is what
we did last year.
Then the institute informs the scientific community that we
are interested in receiving applications that deal with
obesity. So when we did obesity last year, this year we have
had in increase in the number of proposals and applications for
obesity research, including behavioral science research.
So I think it is a cycle, Mr. Upton. It really goes from
scientific field, peer review, assessment of----
Mr. Upton. Does that change at all in terms of the reforms?
How does it change from----
Mr. Zerhouni. So what would change, for example, is what we
did with the road map. So we told all the institutes to put a
pool of dollars in the pot, if you will, about 1 percent of the
total NIH budget, and we decided jointly what were the areas
that were not being developed that we needed to push and
develop. And that is what we did through the road map.
Mr. Upton. My second question is this. You said in your
statement that you wanted to prevent bad science from being
conducted. And remember, I am a supporter of the NIH. For the
last number of Congresses, we have had a vote in the House on
an amendment that would de-fund certain grants. And as I
reflect back, it has often galvanized the entire research
community against the amendment.
But you know, as I listen to my colleagues, and as I have a
survivor, a cancer survivor in my family. I have got--I know
many, many people that have utilized the NIH. Your terrific
examples of raising the age that we all live 5 years because of
the research that you do. It just seems--and when you talk
about all of the requests that come in for assistance--and I
have been to the University of Michigan. I have seen some of
their requests. I was there when the researcher in Ann Arbor
actually located the gene for the breast cancer cell. It seems
that some of the projects that get funded that we sometimes
vote on--I'll use the example of the behavior of prostitutes at
trucks stops, I think was one of the votes that we had in the
last year or 2. I voted to stop that. It just seems that that
doesn't measure up to some of the same things that the NIH
ought to be really doing. And I would be anxious to hear your
comments on that.
Mr. Zerhouni. So I reviewed that issue, because this was
raised--Mr. Pitts raised that. And what I did is I reviewed the
entire portfolio. Asked all my scientists, all my institute
directors to explain to me what is the public health relevance
of this research? Because I don't think we can look at this
issue without seeing what the societal impact is, and why is
it.
For this particular example, you should know that sexually
transmitted diseases and HIV AIDS are a rising problem in our
populations. It is not a problem that is going away. And in
particular, when you look at these diseases, unfortunately,
their main root of transmission is the trucking industry around
the world. And in fact, prostitution at truck stops is one of
the main roots of transmission. Not knowing that--not doing
research on that would have been a decision, I think, that
would not serve public health.
I think, on the other hand, that we need to have a better
understanding of the research that is done so that it does not
come across as being, you know, without a public health
purpose. The balance of that is really what I think we need to
focus on. I think focusing on a single grant, sir, I really--
and I have said that. I don't think it is the right way to look
at it, but look at it as a matter of policy. What is the public
health relevance versus how you--what you do and how you do it.
So I have to tell you that I understand very much your point of
view, but I don't think that the public health burden of that
should be ignored, either.
Mr. Upton. Thank you. Yield back.
Mr. Zerhouni. Thank you.
Mr. Deal. Mr. Green.
Mr. Green. Thank you, Mr. Chairman, and again, welcome and
thank your for your patience because of votes and everything
else.
Dr. Zerhouni, I represent a district in Houston and I had
the opportunity last fall to visit the President's Cancer Panel
when it came to M.D. Anderson in Houston, and I am proud of the
M.D. Anderson Cancer Center, and particularly, the Cancer
Institute with Dr. Eschenbach.
Time and again during the discussions of that cancer panel,
speakers identified the lack of collaboration between
scientists as a hindrance to progress in the field of cancer
research, and I assume, in lots of other fields. This
collaboration was not borne out of structural problems at NCI,
maybe, but rather by the tremendous sense of competition
between our academic researchers and the unwillingness to
cooperate, because that would mean having to share the
accolades and the fame that accompanies a discovery.
I am pleased to see that you stress the research teams of
the future initiative to create interdisciplinary research
programs, yet I would like to hear how the structural changes
you envision would invoke a change in the mindset of
researchers. Because every once in a while, even Members of
Congress get jealous of each other's share in credit. But I
understand that, and if you could just explain to us how we
might institutionally change that mindset.
Mr. Zerhouni. You are absolutely correct. Culture has to
change, and the way we try to do this is to lower the barriers
to the cultural change. One thing we are doing this year is we
are allowing grants to be held by multiple investigators rather
than just 1 master of the tribe, if you will. We want every
type of discipline to be able to have an equal role.
The second is the--through the initiatives that we are
taking, we are stimulating for the creation of
interdisciplinary centers, interdisciplinary teams, and so on.
But at the end, you are correct. The change will have to occur
in the institutions. And when--as you know, getting an NIH
grant right now is the main determinant of promotions in the
academic world, worldwide. It has become the label of quality.
If you can get through it, it is almost as good as getting a
diploma. And that is why it is so competitive. It is the only
thing the Federal Government, I think, gives out there that
leads to a professorship is an NIH grant.
So frankly, I think you are right. If we don't change that
from an individual centered thing to a team centered approach,
then the scale of the problems that you have to tackle is going
to be reduced.
Mr. Green. I would offer whatever we can do to help.
Another concern is the research study gains the most
legitimacy through its publication in a medical or science
journal, and subscriptions to these publications are expensive
for the public to have access to. And since the advent of the
Internet, the public increasingly seeks out information about
the latest medical research to determine treatment options. And
since the NIH funded research is openly funded with tax
dollars, I believe the public should have as wide an access to
those research findings as we can.
Can you explain NIH's rationale behind its recent policy on
open access to NIH funded research findings? I know the
proposal originally was to open it after 6 months, and then it
was changed to 12 months. Can you tell us the----
Mr. Zerhouni. So first of all, there was nothing before
this policy.
Mr. Green. That is true.
Mr. Zerhouni. You have a venue for any scientist to make
anything available to the public. So the value of this policy
is that it creates a precedent and a new pathway for the public
to have access to NIH funded research, number 1.
Number 2, when we proposed the policy, we said, you know,
we need to walk before we run. We believe that the scientists
in their majority want to make their research available to the
public, so we said all right on the voluntary basis. We request
that all our grantees put their papers up at 6 months, or
sooner, if the publisher agrees. When we looked at all the
issues through the comment period, we didn't change the policy.
It was always voluntary at the beginning. And then what we
decided to do, sir, was to instead of asking people to just
wait 6 months, to ask them to basically give us the paper as
soon as possible, not wait 6 months, if they can, but up to 12
months if they need to. Meaning that 12 months is an exception.
Why is that? Because when we looked at the publisher world, it
is clear that it is not just driven by for profit large
companies, but you have small journals that really require--and
societies that require and count on that revenue. So what we
did is we provided--we didn't extend it from 6 to 12, sir. We
really said instead of 6, we will give you a range, and we will
tell you as soon as possible.
Mr. Green. Does that include--who makes the decision? Is it
the publisher or the NIH researcher?
Mr. Zerhouni. No. We changed--this is a major change. In
the policy, we said the researcher, the grantee, has the----
Mr. Green. Has the authority?
Mr. Zerhouni. [continuing] decision power, yes.
Mr. Green. Okay. And I know you have had these questions
from both sides on it, as a scientist, and I understand the
President makes the decisions, kind of like with our staff. You
know, our job is to make the decisions with what they do.
When you are advising the President on stem cell research,
would you advise him to reduce or increase the limitations on
stem cell research?
Mr. Zerhouni. Like I said, I have advised the President,
for example, on increasing the amount of funding. The National
Stem Cell Bank is something that we said was needed. Or we
needed to really work through the IP issues and provide that
national resource. The same is true for increasing funding for
translational activities. I provide all of the latest
information on the field, not just federally funded research,
but all of the federally funded research. But I don't determine
policy, obviously.
Mr. Green. Okay. I understand.
For my last question, approximately half of the institutes
and centers within NIH are created administratively, while
others are mandated by Congressional action. I am particularly
interested in the Center for Minority Health and Health
Disparities, which Congress created in 2000. And since
Congress, not NIH, created this center, do you believe that a
separate Center for Minority Health has been a positive
development, or in retrospect, is this an area of health
research that you would prefer to be addressed through your
plan for interdisciplinary research?
Mr. Zerhouni. Well, again, this is one of those areas where
I think this is a top priority for NIH, health disparity. So
the question is, at what time do you create a structure when
you have a priority or what time do you let it sunset? That is
the usual issue.
I think the National Center has played a very important
role in doing two things. One is having resources of its own to
leverage its ability to influence, but also, it has cross NIH
authorities to look at the strategic plan. And that is the
combination that I am saying we need to have as a matter of
institutional policy, not just for minority health or women's
health or AIDS research, but as a process that will serve all
emerging priorities as they come. Health disparities is here,
in my view, as one of the top five priorities. NCMHD is really
needed. It is an office that really coordinated things before.
I think it is necessary, when you have a phase of challenge
like this to have a focused effort.
But I don't think that maintaining these things forever is
a good policy for any structure that we create, but I don't
think NCMHD is at that phase. I mean, NCMHD needs to really do
a lot of work. They just started. They are doing a good job.
Mr. Green. And I know the district that I have is majority
of Mexican-American. Obesity and diabetes is an issue and it
strikes across all ethnic lines. African-Americans and
Hispanics have a higher percentage than the average population.
But it is a problem with everyone.
Thank you, Mr. Chairman, for holding the hearing.
Mr. Deal. Thank you.
Mr. Rogers.
Mr. Rogers. Thank you, Mr. Chairman. Thank you, Doctor, for
taking the time. It is a little bit sad that millionaire
baseball players who abuse drugs get two dozen cameras, and the
guy who is going to help set the pace and structure for curing
AIDS and cancer, and we couldn't even arrange a Polaroid for
you.
Mr. Zerhouni. And discovering steroids as well.
Mr. Rogers. Yes, that is exactly right. And what they do
wrong, right?
I do appreciate, thanks, Doctor. But please know that for
at least those of us who are here today, we appreciate your
work and your effort. And we know, literally, that it is
lifesaving work that you and your organization and the
scientists that plug into your organization do, and we are
thankful for it. Nowhere else in the world does this exist in
the way that we do it, and it shows. And I might disagree with
one of my former members, it really wasn't, because we passed
laws, and if that was the case, more Congressmen would cure
more disease. But we know that your work and research and
development in actually getting to the heart of this stuff has
made a tremendous difference in the lives of every American.
Thank you for that. I appreciate it, and what you are going
through.
And I am intrigued by this cross cutting idea where we can
try to give you the tools that you need versus by mandating it.
And do you believe that if we can come up with a mechanism of
which I hope that you will recommend to us, a cross cutting
mechanism that we can address something I don't think is being
addressed well, and that is pain. I have a huge interest in
pain care and palliative care. Fifty million Americans will--
are either partially or totally incapacitated because of pain.
And I know less than 1 percent of your budget is on there now.
And with your new Office of Portfolio Analysis, do you believe
that is a way, or there is a way that we can start addressing
real research education and access for physicians and patients
across the country on pain care? And really, think about it.
Cancer, AIDS, people who have been treated for a disease are in
pain, and people who have not been treated for a disease suffer
a different kind of pain.
Mr. Zerhouni. I think you are absolutely right. Not only do
we need to think about curing disease, but making sure that the
quality of life you lead is maintained. And pain is a major
determinant of that.
So we have now a pain--I mean, we had it last year and it
started with Dr. Larry Taybach, who is the head of the National
Institute of Dental and Craniofacial Research, who is now
leading a trans-NIH pain consortium. And is doing really a
review of all of our efforts across all the different fields,
because pain in different fields is a different issue.
We also, as I will show you here, increased funding for
pain significantly. And if you look at when we started, it was
about 104 before the doubling. It is $233 million by 2004. So
we recognize the issue. In addition, in the road map we have
developed tools to measure pain that are objective across
diseases. So it is a tool called ``promise'' which is part of
the road map process where we are going to record subjective
outcomes, not just what the doctor says, you know, when they
say the patient is cured, and the patient says well, I am dying
cured. Because in fact, what needs to happen is the measure of
outcome as seen by the patient. And pain becomes, then, a major
determinant. And in that context, I believe that what you are
asking us to do will be enhanced by having the ability to cross
correlate what these efforts are across--because they are just
minor issues for each one, maybe, each disease, but they are
major issues for all patients.
Mr. Rogers. I mean, the number 1 issue that a patient shows
up anywhere to get treatment is pain.
Mr. Zerhouni. Right.
Mr. Rogers. And when you look at even our major educational
institutions are medical institutions, try to find any
physician graduating that has any understanding of pain cure
for a patient where it is really chronic pain. You can't find
it. And certainly, in my research, we have found that there is
very little access for people who have chronic, disabling pain.
And I hope--I would be interested in sitting down with Dr.
Taybach, but you know, the Dental Institute, in my mind, maybe
is not exactly the right place. Maybe that is what we have
hoisted on you, but I would like to try to find a way that we
can address this from a research position as well.
Mr. Zerhouni. And that would be role of the sort of super
functional integrating structures like OPACE to sort of scan
what is happening across, and then maybe have a response that
is not just a tactical response when there is a pressure, but a
strategic response when you look at it as a growing problem for
the population.
I think we can achieve that with that sort of approach as
you mentioned, functionally gluing together the science of pain
management.
Mr. Rogers. I look forward to working with you on that
issue.
Mr. Zerhouni. Sure.
Mr. Rogers. It is incredibly important to me and I think
millions of Americans.
I have to tell you, when I introduced the bill on chronic
pain cure, we got calls from all over the country on support
groups who had developed all on their own that we didn't know
existed, because they felt that they had no one paying
attention to what is a really disabling problem for literally
millions of Americans.
Mr. Zerhouni. I don't want to steal Dr. Taybach's thunder,
but there is a research project that will be announced soon
where they found a way to specifically disable pain neurons in
patients with chronic intractable pain. And I think that is
going to be a revolution, because it is almost like a magic
bullet that goes through the neurons that indicate pain, and
disables them. And we have had some very, very promising
results there. Hopefully it will become confirmed and
eventually established.
But I agree, and with that focus, you can make progress.
Mr. Rogers. Well, I hope that you have the courage to come
back and actually present--try to void of the political fallout
of each institute. Each institute was created because of a
political push somewhere in the system. $1 that we spend on
administration at NIH that we don't have to is 1 person that
may not get cured or 1 day that we don't get a cure for AIDS or
a cure for cancer. And I think you will find that maybe after a
little of that dust settles, you will have a lot of support
from people that say look, we understand that it is best left
to a strong peer review system. It is best left to one person's
control of an umbrella to get that money to labs where it is
doing the most good. It is a little disheartening to see that
flow chart, because you know the duplication is there. It can't
not be there. And it is disheartening when there are so many
counting on your good work and the scientists' good work as
well.
Mr. Zerhouni. But there is also--I agree with you. But
again, if you look at the record, I think it is the balance
that is key, not one or the other. Because my personal
experience as the dean for research at Johns Hopkins and before
is you do it all top down, you really tend to have a lower set
of rich ideas. If you do it all peripheral, then you have
disintegration. So we need a balance between the 2.
Mr. Rogers. I have confidence that you will find it, and I
have put in for the committee for a Polaroid to make you feel
special.
Mr. Zerhouni. All right, sir.
Mr. Rogers. Thank you, Doctor. I look forward to working
with you.
Mr. Zerhouni. Thank you.
Mr. Rogers. I yield back my time, Mr. Chairman.
Mr. Deal. I thank the gentleman.
Recognize Mr. Rush.
Mr. Rush. Thank you, Dr. Zerhouni. I am very much impressed
with your testimony and with your performance.
One of the ways that I judge about how effective a leader
of a department of an agency is that--particularly after they
come and testify on our way to vote or on the train, I ask
them, what do you think about the guy? And everybody that I
have talked to--and it is not--it is just an incomplete
unscientific assessment says that he is very good. Okay. So we
want to let you know that you have our appreciation for the
work that you do.
You indicated earlier, and I was very pleased to hear this,
that you consider health care disparities one of the top five
issues of NIH. And in light of this, would you--how would you
advocate or what kind of position would you take on the
elevation of the National Center for Minority Health and Health
Disparities to an institute level as opposed to a center level?
And what would be the difference in your mind?
Mr. Zerhouni. Functionally, there is no difference between
an institute and a center, the way the Center for Minority
Health works. It has its budget, has its advisory council, and
it has authorities to look across the entire portfolio. So you
call it a center, an institute, it is, I don't think,
functionally there is a difference, to my knowledge. Right now,
it is almost at the level where it can do its job as we speak.
So calling it an institute or a center will not make a
functional difference in my opinion.
Mr. Rush. In your opinion, is there any room for
improvement in terms of the performance of this office and this
center and particularly in light of your new--the Office of
Portfolio Management and Strategic Initiatives? Is there--do
you see areas of where you might improve the effectiveness?
Mr. Zerhouni. Again, I think what you are seeing in that
institute is exactly what I testified to about the need over
the past 10 years to create what I would call ``glue
mechanisms.'' And this was created because there was a sense
that health disparities did not get the focused attention that
it needed to get. So that institute was created to do that. And
my view is that we need to really have a sense of the strategic
plans across all institutes. There was one strategic plan, the
first one that was developed and submitted to Congress. We need
to improve on that and continue to improve on it. So I think it
is a good start. We need to, you know, continue the effort.
But here is the danger. From my standpoint, and I have said
that to the institute directors and to the advisory council of
the center, because you are not specifically focused on one
disease, what you really need to do, you need to be able to
work across diseases because the health disparity populations
equally, whether it be diabetes or heart disease or cancer and
so on, you need to have that.
So here is the tension. The tension is you create a center,
people say well, there is a center now. Now they take care of
it. And you say well, that is not the intent here. The intent
here is to coordinate better. But then within the institute,
you have an advisory council that is here and says you know
what? We need to focus on this and focus on that. So the
challenge for NCMHD and myself is to balance that crosscutting
investment with the specific investment that NCMHD does. And
that is work in progress. And I have told the NCMHD advisory
board, you really need to have better links to the other
advisory councils. And I told the institute directors that you
can't not look at the health disparity research just within
your own window, but you need to be able to do that across.
Now, is this a good fix? Yes. Is this something that you
would want the institution to basically create a new structure
every time you have a coordination problem? My answer is no. I
think you need to have one competent structure to do that
repeatedly and continuously.
Mr. Rush. Sure. Let me ask you--I am running out of time,
here.
What is the NIH doing to increase the participation of
ethic minorities in clinical trials, and not just as patients
and subjects, but also as researchers and investigators?
Mr. Zerhouni. As you know, this is an issue that we have
been working on for the past 20 years. So in terms of
participation and trials, the numbers look very good. I think
we have more participation from minority populations than we
ever did before.
Where I think we need to make progress is to have members
of those communities become health researchers and medical
researchers themselves. I have a fundamental principle that I
will share with you. ``The diversity of those who serve has to
be a mirror of the diversity of those who are served.'' That is
the philosophy. But it is very had. I don't think it is that
easy to identify scientists in the minority populations, and
then direct them to a science career. Just like across society,
you are seeing a decreased interest in science and technology
fields. If you look at the National Science Foundation reports,
one of the strategic problems that they are seeing is that our
own children grow less and less in science and technology. More
so in minority populations where economic opportunities that
don't require many, many years of training and Ph.D.'s and so
on, are more attractive.
So we have a double problem, if you will. We are working on
that. We are committed to making sure that we have better
representations. In terms of recruitment of our own leaders, I
think we have done pretty well. I think there are many
institutions out there that now have a cadre of scientists, but
not enough.
Mr. Rush. Thank you.
Mr. Deal. The gentleman's time is expired. I would tell you
that we are about to come up on a vote on the floor. Hopefully,
we can finish this before we have to leave for the next vote.
Mr. Pitts.
Mr. Pitts. Thank you, Mr. Chairman.
Dr. Zerhouni, on the topic of stem cell research, how much
is the NIH spending on clinical trials for adult stem cell
research?
Mr. Zerhouni. In adult stem cell research, I can show you
the information that I have here in terms of data. But we spend
about 10 times more on adult stem cell than we do on the
embryonic stem cell research. I will show the information so
you can see. So if you are in the red, this is non-embryonic
stem cell human research at the top left, it is $170 million in
2002. And if you looked at 2003, I think it is $190 million,
for $20 million in embryonic stem cell--human embryonic stem
cell.
Mr. Pitts. Now is that the total number? How much is spent
on clinical trials?
Mr. Zerhouni. I don't have these exact figures, you sir. I
will give you this. But I would say it is about half of it,
just guessing.
Mr. Pitts. If you could provide that.
Mr. Zerhouni. Or more.
Mr. Pitts. How much would go toward adult stem cell
plasticity? You know, turning adult stem cells into other
tissue types.
Mr. Zerhouni. I am stymied, sir. I do not have the
information in front of me, but I would bring it to the record.
Most of the research in adult stem cell has, as you know,
has evolved for over 25, 30 years, and much of it is related to
cancer treatment, and as you heard, some diseases like leukemia
and lymphoma and multiple myeloma. So this is where the bulk of
clinical trials are. In terms of plasticity research in adult
stem cells, I don't know the number.
Mr. Pitts. If you could provide----
Mr. Zerhouni. I will provide that for the record.
Mr. Pitts. Are there still frozen embryo stem cells
derivations on the President's approved list that have not yet
been cultivated into ongoing cell lines yet, and therefore
could be cultured on non-animal feeder cells?
Mr. Zerhouni. There are 31 cell derivations that have not
been expanded. To our knowledge, to the best of our knowledge,
there are 16 which we are told by the investigators at the
University of Gutenberg that have not been exposed to animal
products, we are told.
Mr. Pitts. Have you had to turn away any researcher
requesting embryo stem cell research lines because you did not
have any available?
Mr. Zerhouni. Not to my knowledge.
Mr. Pitts. Are the now-eligible lines, 22 I understand are
getting funding, worthless because of mouse feeder cells?
Mr. Zerhouni. All lines--to my knowledge, every line
available in the world, whether it be eligible for Federal fund
or not, has been exposed to animal cell lines, mouse feeder
cell lines. So they are not useless. They are very useful.
Mr. Pitts. For basic research?
Mr. Zerhouni. For basic research.
Mr. Pitts. Finally, do new breakthroughs in the bone marrow
stem cell or adult stem cell or cord blood cell fields
continue? There was an article in the ``Washington Post'' last
month, it says ``Researchers in Boston have isolated a kind of
cell from human bone marrow that they say has all the medical
potential of human embryonic stem cells.'' And I can submit
that article for the record.
But the question, do these breakthroughs continue?
Mr. Zerhouni. They continue across the board, and clearly,
the fundamental scientific issue, sir, is the issue of
programming, deprogramming or reprogramming DNA in cells. When
you take an adult stem cell, your problem is to deprogram it
and then reprogram it to do something else. When you take an
embryonic stem cell, you have a cell that has not been yet
programmed, and you try to program it. Both are advancing, in
terms of fundamental science, to understand how to do that.
Mr. Pitts. And I think I have time for one more question.
How long has embryo research been going on in animals?
Mr. Zerhouni. In animals, mouse embryonic stem cells,
probably 20, 25 years. We do a lot of mouse embryonic stem cell
research, in terms of creating animal models of disease. We
have what we call ``mouse knockout'' or knock in, gene knock
out, gene knock in where you can introduce a gene or remove a
gene.
So it has been going on for at least 2 decades, to my
knowledge.
Mr. Pitts. Thank you, Mr. Chairman.
Mr. Deal. I thank the gentleman.
Dr. Burgess.
Mr. Burgess. Dr. Zerhouni, on the--one of the slides you
showed us, you talked about how the science is converging, and
yet when we see what is happening with the NIH it still seems
to be expanding.
Are there areas where you think you can get early successes
in capturing this convergence of the science and perhaps making
the flowchart for the NIH look a little less complex?
Mr. Zerhouni. Yes, I think we are doing that, quite
frankly. I think the scientists themselves are realizing how
much opportunity there is between disciplines and at the
interface of disciplines. So we haven't really had a lot of
opposition to the sense that we need to have a balanced
portfolio.
What areas--right now, I think where we need the most
progress is two things. One, understand the complexity of the
genome and how it is functioning, its regulation. We are doing
great progress there. We have now a map of all the variations
between humans, and we are going to use this to identify
disease genes.
Last year, we identified 12 genes, I believe, in mental
health, something that had never happened before. So you are
going to see a lot of progress.
The second area is translate that research more
effectively, it is called translational research. I think we
need to train more physicians who are dedicated to science and
to make sure that there is a bench to bedside, bedside to bench
relationship to accelerate research.
Mr. Burgess. Do you feel like you are getting buyoff from
your scientists----
Mr. Zerhouni. I think----
Mr. Burgess. [continuing] both intramural and extramural?
Mr. Zerhouni. It is, you know, like when you are dealing
with a complex situation with a tight budget, people really are
worried about taking a chance. But my message is that there is
no wrong time to do the right thing.
Mr. Burgess. Correct.
Mr. Zerhouni. And that is what we will try to do. But it is
not without tension, sir.
Mr. Burgess. I understand.
On the slide that you have up there that you put up for Mr.
Pitts on the human stem cell research, is that going to include
both what you would call bone marrow stem cells as well as
umbilical cord derived stem cells?
Mr. Zerhouni. Right. I think so. Umbilical cord, we don't
have as much funding in umbilical cord as we do in adult stem
cells. I don't have the exact number. I can look it up here.
But I don't have it separated that way. But umbilical cord
investments are in the same range as embryonic stem--human
embryonic stem cells, I believe.
Mr. Burgess. Is that something that we, as a Congress, need
to pay attention to, or is that better left in your hands and
the----
Mr. Zerhouni. You know, frankly, I think at this point I
would prefer that we do our job, you know, have the stem cell
task force looking at things and through this process, I think
we can see the relative merits. The National Heart and Lung and
Blood Institute is charged to look at cord blood stem cell
issues, and I am waiting for their advice.
Mr. Burgess. Okay. When do you expect to receive those
reports from the task force of the Heart and Lung Institute?
Mr. Zerhouni. They are working on them. They have already
looked at the issue of stem cell banking for cord blood, so I
am not clear, really. I will get back to you on the record
about when that is.
Mr. Burgess. Okay. Because there is a lot of that that I
know as a private practitioner in obstetrics, there is a lot of
that that goes on where people bank their own cord blood.
Let me just ask you a question. That was an intriguing
figure of $16 per American, per year that is spent in research
from the NIH perspective. Do you have any idea what is spent
covering all levels, private research, university research,
pharmaceutical research? What dollar per year figure?
Mr. Zerhouni. NIH for medical research is the main player,
so typically if you look at a university, 80 percent of their
funding is really NIH funded. Maybe 10, 20 percent is
philanthropy and industry. That is research at universities, so
if it is $96, you could say you could add $20 to that, and that
is what universities will get.
If you look at the chart of investment--I wish I had it
here--between industry R&D and industry from biotech versus
NIH, for the longest part of history, NIH was the dominant
funder. In 1991, pharma R&D went up relative to NIH. So NIH
spends about $28 billion, and the pharma spends about $30, $31
billion in R&D, and biotech, $19 billion.
So if you add it up, it is $96 of Federal dollars, and
probably $120, $130, $140 of private dollars.
Mr. Burgess. Are we seeing that same situation occur in the
stem cell arena? Are the private dollars really pouring in to
stem--embryonic stem cell research?
Mr. Zerhouni. Yes, I think there is a much different
distribution there. As you know, with the initiatives and the
private investments, I think the Federal investments is maybe
$25 million right now. And we had some studies that show that
in a private sector was about $200, $233 million in the biotech
area. You have companies like Geron, for example, that are
investing and proposing new therapies with it. And with State
initiatives, you have another influx. And as you know,
California has a new initiative that counts about $300 million
a year in spending.
Mr. Burgess. What----
Mr. Deal. The gentleman's time is expired.
Mr. Burgess. Okay.
Mr. Deal. We are getting close on vote.
Mr. Bass, do you have one question before we go?
Mr. Bass. Thank you, Mr. Chairman. I appreciate the
courtesy. I will be very fast.
Dr. Zerhouni, I would be most grateful if you would be
willing, in the interest of time, to answer four questions that
I have in writing for the record. I appreciate that. And Mr.
Chairman, I would only say, I wasn't planning on asking
questions, but I think that some of the comments made by some
of the previous members of the subcommittee need to be
addressed. To say that research has not been effective--has
been unfunded and ineffective so therefore, it doesn't work, I
don't think really is logical. If you don't fund research, you
can't say it doesn't work. And the fact that embryonic stem
cell research is receiving less than 10 percent of the total
NIH funding, when you, yourself, said that NIH is the main
player in research of this sort, I think begs the question a
little bit. And to say that adult stem cells are more valuable
to science than embryonic can't possibly be true, because the
scientific community doesn't support that contention.
The fact is that adult stem cells have been studied, as you
mentioned, for 35 years with limited success. I am hopeful that
we can continue this debate. It is an important debate for the
sake of science, for the sake of finding cures for most of
America's intractable diseases, and I appreciate your
leadership at NIH, and I am glad that the subcommittee is
moving forward with reauthorization.
I yield back.
Mr. Deal. I thank the gentleman.
Dr. Zerhouni, I want to thank you and your colleagues for
being here today. A most impressive presentation. We look
forward to working with you as we continue forward on an effort
to reauthorize, and hopefully incorporate some of the
suggestions, at least, that you have presented to us.
Thank you very much.
Mr. Zerhouni. Thank you, Mr. Chairman.
Mr. Deal. This hearing is adjourned.
[Whereupon, at 1:06 p.m., the subcommittee was adjourned.]
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