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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

COMMITTEE ON ENERGY AND COMMERCE

JOE BARTON, Texas, Chairman

RALPH M. HALL, Texas

MICHAEL BILIRAKIS, Florida

Vice Chairman

FRED UPTON, Michigan

CLIFF STEARNS, Florida

PAUL E. GILLMOR, Ohio

NATHAN DEAL, Georgia

ED WHITFIELD, Kentucky

CHARLIE NORWOOD, Georgia

BARBARA CUBIN, Wyoming

JOHN SHIMKUS, Illinois

HEATHER WILSON, New Mexico

JOHN B. SHADEGG, Arizona

CHARLES W. ‘‘CHIP’’ PICKERING,

Mississippi, Vice Chairman

VITO FOSSELLA, New York

ROY BLUNT, Missouri

STEVE BUYER, Indiana

GEORGE RADANOVICH, California

CHARLES F. BASS, New Hampshire

JOSEPH R. PITTS, Pennsylvania

MARY BONO, California

GREG WALDEN, Oregon

LEE TERRY, Nebraska

MIKE FERGUSON, New Jersey

MIKE ROGERS, Michigan

C.L. ‘‘BUTCH’’ OTTER, Idaho

SUE MYRICK, North Carolina

JOHN SULLIVAN, Oklahoma

TIM MURPHY, Pennsylvania

MICHAEL C. BURGESS, Texas

MARSHA BLACKBURN, Tennessee

JOHN D. DINGELL, Michigan

Ranking Member

HENRY A. WAXMAN, California

EDWARD J. MARKEY, Massachusetts

RICK BOUCHER, Virginia

EDOLPHUS TOWNS, New York

FRANK PALLONE, Jr., New Jersey

SHERROD BROWN, Ohio

BART GORDON, Tennessee

BOBBY L. RUSH, Illinois

ANNA G. ESHOO, California

BART STUPAK, Michigan

ELIOT L. ENGEL, New York

ALBERT R. WYNN, Maryland

GENE GREEN, Texas

TED STRICKLAND, Ohio

DIANA DEGETTE, Colorado

LOIS CAPPS, California

MIKE DOYLE, Pennsylvania

TOM ALLEN, Maine

JIM DAVIS, Florida

JAN SCHAKOWSKY, Illinois

HILDA L. SOLIS, California

CHARLES A. GONZALEZ, Texas

JAY INSLEE, Washington

TAMMY BALDWIN, Wisconsin

MIKE ROSS, Arkansas

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

MICHAEL BILIRAKIS, Florida

FRED UPTON, Michigan

PAUL E. GILLMOR, Ohio

CHARLIE NORWOOD, Georgia

BARBARA CUBIN, Wyoming

JOHN SHIMKUS, Illinois

JOHN B. SHADEGG, Arizona

CHARLES W. ‘‘CHIP’’ PICKERING,

Mississippi

STEVE BUYER, Indiana

JOSEPH R. PITTS, Pennsylvania

MARY BONO, California

MIKE FERGUSON, New Jersey

MIKE ROGERS, Michigan

SUE MYRICK, North Carolina

MICHAEL C. BURGESS, Texas

JOE BARTON, Texas,

(Ex Officio)

SHERROD BROWN, Ohio

Ranking Member

HENRY A. WAXMAN, California

EDOLPHUS TOWNS, New York

FRANK PALLONE, Jr., New Jersey

BART GORDON, Tennessee

BOBBY L. RUSH, Illinois

ANNA G. ESHOO, California

GENE GREEN, Texas

TED STRICKLAND, Ohio

DIANA DEGETTE, Colorado

LOIS CAPPS, California

TOM ALLEN, Maine

JIM DAVIS, Florida

TAMMY BALDWIN, Wisconsin

JOHN D. DINGELL, Michigan,

(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)

(1)

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 21⁄2 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.

2

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 successVerDate 11-MAY-2000 10:07 Sep 16, 2005 Jkt 000000 PO 00000 Frm 00006 Fmt 6633 Sfmt 6633 20746.TXT HCOM1 PsN: HCOM1
3

ful 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.

4

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

5

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.

6

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

7

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

8

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.

9

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.

10

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

11

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 miVerDate 11-MAY-2000 10:07 Sep 16, 2005 Jkt 000000 PO 00000 Frm 00015 Fmt 6633 Sfmt 6602 20746.TXT HCOM1 PsN: HCOM1
12

crobes 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 paVerDate 11-MAY-2000 10:07 Sep 16, 2005 Jkt 000000 PO 00000 Frm 00016 Fmt 6633 Sfmt 6602 20746.TXT HCOM1 PsN: HCOM1
13

tients. 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

14

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

15

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.

16

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.

17

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

18

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

19

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.’’

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• 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

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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

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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?

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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’

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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

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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?

26

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?

27

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

28

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——

29

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.

30

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——

31

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 upVerDate 11-MAY-2000 10:07 Sep 16, 2005 Jkt 000000 PO 00000 Frm 00035 Fmt 6633 Sfmt 6602 20746.TXT HCOM1 PsN: HCOM1
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date 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 clinVerDate 11-MAY-2000 10:07 Sep 16, 2005 Jkt 000000 PO 00000 Frm 00036 Fmt 6633 Sfmt 6602 20746.TXT HCOM1 PsN: HCOM1
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ical 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?

34

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——

35

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.

36

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.

37

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

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able 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.

39

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 ocVerDate 11-MAY-2000 10:07 Sep 16, 2005 Jkt 000000 PO 00000 Frm 00043 Fmt 6633 Sfmt 6602 20746.TXT HCOM1 PsN: HCOM1
40

curred 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

41

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

42

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

43

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

44

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

45

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

46

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.

47

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.

48

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

49

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.

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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.

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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

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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

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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.]

[Additional material submitted for the record follows:]

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