A new test can predict both the risk of breast cancer recurrence and may
identify women who will benefit most from chemotherapy. Results suggest
that almost half of the more than 50,000 U.S. women diagnosed every year
with estrogen-dependent, lymph-node negative breast cancer may not need
to go through the discomfort and side effects of chemotherapy because
their risk of recurrence is so low. This is important news for many
women and their families.

In a \"proof-of-principle\" study, scientists found they could measure
elevated levels of four distinct cancer-associated molecules in saliva
and distinguish with 91 percent accuracy between healthy people and
those diagnosed with oral squamous cell carcinoma. This marks the first
report in the scientific literature that distinct patterns of the body's
primary macromolecules, known as messenger RNA, are not only measurable
in saliva\--but can indicate a developing tumor.

A new imaging system is under development that may shorten the length of
surgical procedures and allow surgeons to more precisely pinpoint, and
then remove, damaged brain tissue that causes seizures. A
multi-institutional team of engineers, scientists, and physicians, may
improve the chances of successful surgery for some epilepsy patients.
The same kinds of techniques are bringing new hope to patients with
Parkinson's disease through deep brain stimulation.

We are continuing to make investments in understanding and treating
chronic diseases such as diabetes, Alzheimer's disease, Parkinson's
disease, and hypertension. A long-term study has found that diabetes
mellitus was linked to a 65% increased risk of developing Alzheimer's
disease (AD). Current research underway will tell us whether therapies
for diabetes may play a role in lowering the risk of AD or cognitive
decline. We have developed a comprehensive plan to understand, treat and
hopefully prevent autism, a daunting problem.

The research that we are conducting in molecular biology and molecular
genetics to identify disease early and intervene before irrevocable
damage occurs will provide similar payoffs in this new century. We hope
to be increasingly able to thwart diseases as they are emerging years
before they strike, at potentially greatly reduced costs.

As we find new molecular signposts for risk and early signs of disease,
we can lower the burden of disease in a more cost-efficient way. Our
ability to predict and intervene will be orders of magnitude more
effective and can ultimately reduce long term health care costs. But
moving to this new model of medical research will require vision,
management, and scientific progress.

SCOPE OF THE CHALLENGE

The American taxpayer generously invests \$96 per person, per year in
the NIH for the future health of our country. To give a sense of
perspective about our task, I look at the total \$29 billion for NIH --
nearly half the discretionary funding for all of HHS---and I know I need
to make sure that such a large investment is used in a strategic and
systematic way to maximize the chances that better treatments and cures
are rapidly developed to stem the rising burden of disease, as health
care costs consume over \$5,500 per person per year and are rising. NIH
is currently using this investment for research to attack hundreds of
common diseases and an estimated 6,000 rare diseases or conditions that
affect approximately 25 million people in the United States \-- diseases
and disorders that may strike any of us, our parents, our children or
our friends. In recent years, we have taken on new challenges such as
biodefense.

NIH has strengthened its vision, tightened its management, and unified
its scientific efforts. Over the past three years, I have presented to
you examples of major shifts toward a renewed vision that spans the NIH
Institutes and Centers: *The NIH Roadmap* in FY2004, the *NIH Strategic
Plan for Obesity Research* in FY 2005, and now, the *NIH Neuroscience
Blueprint*.

STRENGTHENING THE NIH VISION

[NIH Roadmap for Medical Research]{.underline}. Two years ago, I
announced the *NIH Roadmap for Medical Research.* The Roadmap's
overarching goal is to accelerate research and translate the scientific
results that are ready for implementation into cures. It is a trans-NIH
effort to drive progress in medical research while transforming the way
science is conducted. Through the Roadmap, we are supporting research
initiatives that no single institute could tackle alone, but that will
tangibly benefit all of NIH.

For example, to speed the translation of scientific discoveries into
health care interventions, Roadmap efforts are underway to improve and
extend clinical research network capabilities and embrace a growing
"community of research." Roadmap initiatives will make more powerful use
of data. We will illuminate approaches to standardized clinical research
data reporting and data-and-sample sharing across diseases and health
care settings. We will elicit "best practices" from existing clinical
research data networks and test their interoperability. For example, The
Patient Reported Outcomes Measurement Information System (PROMIS) will
enhance how we measure and manage health improvements as reported by
patients themselves by standardizing the way we track symptoms that
transcend many chronic diseases and conditions. A network of
biocomputing centers is integrating informatics, physical and biologic
technologies for ready access by researchers to harness the increasing
quantity and complexity of biomedical and behavioral scientific data.

New tools such as biochemical and imaging probes will illuminate the
complex series of events that lead to disease, providing a host of new
therapeutic targets. Through the Roadmap, there also will be an infusion
of trained scientists and clinical researchers, able to apply
interdisciplinary and multidisciplinary approaches to complex biomedical
problems.

[NIH Strategic Plan for Obesity]{.underline}. NIH has taken on one of
the nation's most dramatic health challenges\-\--obesity. The most
recent figures show that 65 percent of U.S. adults --- or 130 million
people --- are either overweight or obese. In addition to decreasing
quality of life and increasing the risk of premature death, obesity and
overweight will cost the Nation an estimated \$117 billion in direct
medical costs and indirect costs such as lost wages.

Obesity is not an easy problem. It requires multiple approaches. The
obesity epidemic represents a complex interplay of behavioral,
sociocultural, economic, and environmental factors against a backdrop of
genetic and other biological factors. The *NIH Strategic Plan for
Obesity Research* exploits the breadth of expertise available from a
wide range of disciplines at the NIH, engaging 19 Institutes and Centers
to set an agenda for this research.

NIH-supported scientists are continuing to make significant progress in
obesity research. For example, our investigators 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 some people may have
difficulty "keeping it off" after dieting. Such hormones are now
"targets" for drug design. New drugs now in development might affect
appetite, food absorption, and/or energy expenditure.

[NIH Neuroscience Blueprint.]{.underline} Major causes of human
suffering and the burden of disease include mental illness, neurological
disorders, and a range of behavioral disorders. Together, these
illnesses exact a cost of more than \$500 billion a year. Focusing on
how NIH does neuroscience research, the Directors of 15 Institutes came
together and drafted the *NIH Neuroscience Blueprint.* This model of
strategic leadership and cooperation addresses several of the most
common causes of death and disability, as well as hundreds of rare
disorders that affect the brain, spinal cord, or nerve cells throughout
the body, including drug abuse, alcoholism, Alzheimer's disease,
Parkinson's disease, ALS or Lou Gehrig's Disease, chronic pain
conditions, developmental disorders, dementias of aging, and a myriad of
problems of hearing, vision and other senses. The Blueprint capitalizes
on economies of scale to create resources, tackle common scientific
problems, and train the next generation of neuroscientists. *The NIH
Roadmap*, the *NIH Strategic Plan for Obesity Research*, and the *NIH
Neuroscience Blueprint* represent three milestones in a new strategic
use of resources.

NIH RESPONDS TO EMERGING HEALTH THREATS

New infections are spreading to new places and new populations across
the world, and NIH research is responding to breaking medical challenges
on all these fronts. In addition, we are living with bioterrorism
threats. During FY 2006, NIH is developing countermeasures to address
microbial, nuclear, radiological, and chemical threats. At the same time
that scientists are investigating these emerging challenges, they are
learning about mechanisms of a wide range of disease processes.

Recently, NIH-supported investigators uncovered the three-dimensional
structure of the anthrax toxin complex, going from gene to structure in
an astonishing three weeks and giving scientists a potential target for
blocking the toxin's deadly effects. In addition to anthrax, we are
deeply concerned about the other "Category A" microbes \-- smallpox,
plague, tularemia, hemorrhagic fevers, and botulinum toxin \-- that can
be deadly and can be deployed as bioweapons.

Because of the dramatic ways that disease-causing viruses, bacteria, and
parasites continue to change over time\--positioning the NIH for rapid
response is also critical. Alarmed, we see new pathogens emerge\--and
familiar ones re-emerge. AIDS, West Nile virus, monkeypox, avian
influenza, and SARS, are examples. In this past month, newspapers have
been filled with reports about avian flu. NIH is vigorously supporting
research to develop responses to these threats. Last year I described
how we responded, within weeks, to SARS. I am pleased to report that one
of the three new NIH-developed candidate vaccines is now in its first
human clinical trial.

Last year, worldwide, 4.9 million people became infected with HIV and
3.1 million died due to AIDS. We have developed treatments that
transformed HIV/AIDS from a death sentence into a debilitating chronic
disease. We must do better---we must prevent HIV infection. We have been
working for years to develop an HIV/AIDS vaccine. Now, we have six
viable candidates and testing them is our highest priority. Accordingly,
we plan to increase the portion of our budget devoted to HIV/AIDS
vaccines by \$100 million or 20% to seize this ripe scientific
opportunity for progress.

I have described several ways that the NIH vision has been transformed.
It has been moved by discovery, shifted by disease burden, and has been
transformed to adapt to both threat and need. A former colleague of mine
has contrasted the roles of physicians who save lives "one patient at a
time" with those who serve in public health and who save "millions of
lives at a time." At NIH, and through our investigators in every state
and through their more than 3,000 institutions that include
universities, medical schools, hospitals and other research facilities,
we are providing the research firepower to do *[both]{.underline}*. We
depend upon the creativity and rigor of more than 200,000 scientific
investigators, supported by the NIH appropriation you continue to
provide.

MANAGING FOR THE 21^ST^ CENTURY

As the 21^st^ century begins, we have re-tooled our management tactics.
I have just met with the 27 IC directors to revisit our operations and
to adjust our policies to make full use of the combined skills of our IC
leaders. We are, simultaneously, streamlining our decision-making
processes across the enterprise.

[PART]{.underline}. NIH is understandably proud that major components of
our largest activity, the Extramural Research Program, which supports
research all across the country and accounts for \$24 billion of our
appropriation, received the highest possible rating of "Effective" in
the FY 2006 Program Assessment Rating Tool (PART).

[Investigator-Initiated Research]{.underline}. Individual research
grants remain the mainstay of NIH investment, and research in priority
areas will always be awarded competitively. We are also committed to
supporting new investigators, who without support, might be lost to the
continuum of discovery.

[Portfolio Analysis.]{.underline} The agency is successfully engaging in
trans-NIH initiatives such as the Roadmap, Obesity, and the Neuroscience
Blueprint. It is, however, time to focus additional attention on
creating better institutional tools to analyze, assess, and manage the
NIH-wide research portfolio and to provide better information to support
priority-setting decisions in areas of common interest to all Institutes
and Centers. We are creating an Office of Portfolio Analysis and
Strategic Initiatives to focus such efforts, which will include an
improved process for collecting data on various diseases, conditions,
and research fields, and improvements in data about burden of disease.
The staff will seek broad public input---from the public, health care
providers, policymakers, and scientists---in addition to soliciting
advice from within the NIH and making more coordinated and effective use
of the NIH-wide evaluation process.

More effective analysis and management of our portfolio will lead to
even better progress against disease. *Ninety-six dollars per person per
year.* This is our challenge\--to make the taxpayer's generous
contribution to NIH, \$96 per person per year, stretch as far as is
feasible and to leverage as much discovery as is possible since each one
of us some day may fall prey to disease. We have also responded to
challenges and opportunities that are integral to public trust.

RESPONDING TO PUBLIC TRUST CHALLENGES AND OPPORTUNITIES

[Conflict of Interest.]{.underline} NIH has addressed conflict of
interest challenges with sweeping reform. Once we began a systematic
investigation of what had initially appeared to be some isolated cases,
we realized that conflict of interest was a serious problem that needed
management and reform. Strict and clear rules needed to be put in place.
Nothing is more important to me than preserving the public's trust in
NIH. I am confident that the new HHS rules that were put in place last
month will prevent the recurrence of past abuses and will go a long way
toward preserving the historic role of NIH as the primary source of
unbiased scientific health information for the country. We must have the
public's trust, and we must continue to earn it. In this, we will
carefully evaluate the impact of these new rules on our ability to
recruit and retain the best scientists and adjust our policies
accordingly while maintaining first and foremost our commitment to
preserve the role of NIH as an unimpeachable source of scientific
advice.

[Public Access.]{.underline} On February 3, NIH announced our historic
public access policy. For the first time, the public will have access to
peer-reviewed research publications that result from studies funded by
NIH, and scientists will have a new pathway to make their NIH funded
research known to the public that funded it. Starting in May of this
year, research results will be more readily accessible to you, your
constituents, your staff, the public at large, health care providers,
educators, and scientists. We are moving to maximize participation while
encouraging scientists to submit their final manuscripts as soon as
possible, within 12 months of final publication. The policy creates a
permanent archive to preserve vital research findings, which will be a
searchable compendium of these research publications. The compendium
will be used to manage NIH more efficiently and to help our scientists
and many other stakeholders better understand the NIH research
portfolios, monitor scientific productivity, and, ultimately, help set
research priorities.

BUDGET

The discretionary FY 2006 request for the NIH appropriation is \$28,590
million, an increase of 0.5% over FY 2005. In addition, a \$50 million
increase is included in the request for the Public Health and Social
Services Emergency Fund (PHSSEF) for NIH research in chemical
countermeasures, bringing the total increase for NIH to \$196 million,
or 0.7%. The request sustains funding for important initiatives put
forth in recent years while addressing the need for spending discipline
to support the Administration's initiative to cut the deficit in half
over four years. This request

also represents our first integrated performance budget.

The NIH Budget for FY 2006 has five high priorities. Research project
grants increase by \$56 million, raising the number of new and competing
research project grants by 247 grants to a total of 9,463 in our effort
to preserve to the greatest extent possible the ability of scientists to
obtain individual support, a critical goal. The Roadmap for Medical
Research adds \$98 million to total \$333 million to keep this
multi-year initiative on track, so we can accelerate research toward
treatments and cures \-- the majority of this funding will also go to
individual as well as teams of researchers. To develop countermeasures
against biological and chemical threats, biodefense research increases
by a net \$56 million to \$1.8 billion. The Neuroscience Blueprint that
supports NIH Institutes collaborating to use new tools to solve some
pressing public health challenges receives \$26 million. The highest
priority in combating HIV/AIDS is the development of a vaccine, for
which funding rises by \$100 million to \$607 million.

TRANSFORMING MEDICAL RESEARCH

We are proud of our progress, but we know how much is left to be done.
Health disparities remain a challenging problem for our country. Part of
the reality is that some things never happen as quickly as we might
hope. We continue to manage for change in rapidly changing times and
amidst unprecedented scientific opportunities. We are focused on shared
resources, clear objectives, and the stronger teams needed to tackle the
daunting complexity of biology. We are creating a strategic framework
for future discovery. As the nation's medical research agency I am
confident that we will continue our record of improving health through
discovery at an accelerated pace in the 21^st^ century.

Thank you for this opportunity to testify. I would be pleased to answer
any questions.

Elias A. Zerhouni, M.D.

Director, National Institutes of Health

+-----------------------------------------------------------------------+
| NIH Director, Elias A. Zerhouni, M.D., leads the nation's medical     |
| research agency and oversees the NIH's 27 Institutes and Centers with |
| more than 17,000 employees and a fiscal year 2004 budget of over \$28 |
| billion.                                                              |
|                                                                       |
| The NIH investigates the causes, treatments, and preventive           |
| strategies for both common and rare diseases helping to lead the way  |
| toward important medical discoveries that improve people\'s health    |
| and save lives. More than 80% of the NIH's funding is awarded through |
| almost 50,000 competitive grants to more than 212,000 researchers at  |
| over 3,000 universities, medical schools, and other research          |
| institutions in every state and around the world. About 10% of the    |
| NIH's budget supports projects conducted by nearly 6,000 scientists   |
| in its own laboratories, most of which are on the NIH campus in       |
| Bethesda, MD.                                                         |
|                                                                       |
| Dr. Zerhouni, a well-respected leader in the field of radiology and   |
| medicine, has spent his career providing clinical, scientific, and    |
| administrative leadership. President George W. Bush nominated him to  |
| serve as the 15th Director of the National Institutes of Health, and  |
| he began in May 2002. Since then, Dr. Zerhouni oversaw the completion |
| of the doubling of the NIH budget; initiated a strategic vision for   |
| the agency called the NIH Roadmap for Medical Research; established   |
| an NIH-wide research initiative to address the obesity; accelerated   |
| efforts in health disparities research; streamlined NIH's executive   |
| decision-making process; and named eight new Institute and Center     |
| Directors, and filled many other senior-level positions.              |
|                                                                       |
| Prior to joining the NIH, Dr. Zerhouni served as executive vice-dean  |
| of Johns Hopkins University School of Medicine, chair of the Russell  |
| H. Morgan department of radiology and radiological science, and       |
| Martin Donner professor of radiology, and professor of biomedical     |
| engineering. Before that, he was vice dean for research at Johns      |
| Hopkins. He became a member of the National Academy of Sciences       |
| Institute of Medicine in 2000.                                        |
|                                                                       |
| Dr. Zerhouni has won several awards for his research including a Gold |
| Medal from the American Roentgen Ray Society for CT research and two  |
| Paul Lauterbur Awards for MRI research. His research in imaging led   |
| to advances in Computerized Axial Tomography (CAT scanning) and       |
| Magnetic Resonance Imaging (MRI) that resulted in 157 peer reviewed   |
| publications and 8 patents.                                           |
+-----------------------------------------------------------------------+