I am Ruth Kirschstein, the Acting Director of the National Institutes of Health. I am honored to appear before the Subcommittee to present the President's budget for NIH for FY 2001. Although this is the first time I have appeared before Congress to testify about the overall NIH budget, it has been my privilege to appear before this subcommittee annually for 19 years as Director of the National Institute of General Medical Sciences and for six as the Deputy Director. I am especially gratified to appear before you, Mr. Chairman, as you consider the NIH budget proposal for the last time as head of this panel. Your support and the support of the members of the Subcommittee, has made a substantial difference in improving the public's health and well-being.

Mr. Chairman, all of us, we at NIH, Members of Congress and the citizens we serve, have similar expectations for medical research. We want better ways of diagnosing and treating, and, in the long run, preventing and curing disease. And we want the federal dollars invested in medical research to result in the fulfillment of these expectations.

In the last century, the scientific community, both public and private, worked in collaboration to cure or prevent once deadly infectious diseases that are now given no more thought than the common cold. I was fortunate enough to be at the forefront of the final development of the polio vaccine, one of the truly monumental achievements of the last century. There is not enough time today to list the astounding medical breakthroughs that followed our increased understanding of medical science. I will mention just a few: the development of antibiotics and organ transplantation, life-extending and life-saving cancer therapies, the identification of the AIDS virus and the drugs to treat AIDS, and discoveries involving the chemicals in the brain that are important in drug addiction and mental illness.

As we begin a new century, medical science stands on the threshold of research advances that were once inconceivable. We have identified the genes responsible for a large number of our normal functions and the genetic abnormalities that cause many diseases, such as Huntington's disease, cystic fibrosis, and certain forms of deafness. You will hear much more from my colleagues.

In his budget plan for FY 2001, the President is requesting $18.8 billion for the NIH, an increase of $1 billion or 5.6 percent more than the FY 2000 appropriation. By any measure, the amounts we received in FY 1999 and 2000, both nearly 15 percent increases, were dramatic and unprecedented. These generous budgets have allowed us to undertake many new and important programs and to improve conditions throughout the medical research enterprise. The funds requested in FY 2001 will permit us to continue our FY 1999 and 2000 initiatives and to begin new undertakings and expand others under our Areas of Research Emphasis. I will say more about these areas later.

We are pleased that the public, the Congress, and the Administration place a high value on good health and understand the role that medical research plays in improving the health of the American public. These improvements result from new diagnostic advances, more effective treatment options, better ways to prevent some diseases, and ways to delay the onset or progression of other diseases and disabilities.

We feel confident of public support for our research enterprise, but are aware of our need to deliver to the public the two things it most wants from the NIH:

• research advances, year after year, that improve the health of all members of society
• assurance that we spend the public's money wisely.

What the Public Wants from the NIH: Research Advances that Contribute to the Health of Everyone

In the past year alone, we have seen dramatic advances that are likely to have a direct, near-term effect on public health. The NIH will continue to emphasize clinical research in FY 2001 because it is critical in improving public health:

• Scientists completed the first sequence of a human chromosome, 22, which has been implicated in immune system function, congenital heart disease, schizophrenia, mental retardation, birth defects, and several cancers, including leukemia. The 33.4 million nucleotides that make up chromosome 22 comprise the longest continuous stretch of DNA ever deciphered. The magnitude of this work is amplified by the insights it will give us into many diseases.
• A clinical trial (carried out in collaboration with scientists and clinicians in Uganda) has demonstrated an affordable and practical strategy for preventing transmission of the HIV virus from mother to infant. A single oral dose of the antiretroviral drug nevirapine given to an HIV-infected woman during labor and another to her baby within three days of birth reduced the transmission of virus by half compared with a similar short course of AZT. This treatment might prevent some 300,000 to 400,000 newborns per year from becoming infected and eventually developing AIDS at a cost which is affordable in developing countries.
• Preeclampsia is a precursor to eclampsia, a potentially fatal complication of pregnancy. It is characterized by high blood pressure, excessive weight gain, and severe headaches. Eclampsia leads to convulsions and causes a variety of birth complications. Months before symptoms appeared, women with preeclampsia were found to have an imbalance of two chemical compounds, prostacyclin and thromboxane, which control blood pressure. The discovery suggests new and early treatments for this condition for which there is currently no cure or treatment.
• An important gene that makes Salmonella a deadly bacterium was identified. Without the gene, which encodes for the enzyme called Dam, Salmonella bacteria not only did not kill the mice into which they were injected, but also serve as a vaccine against future infection by deadly Salmonella. Because Dam is found in many other dangerous bacteria, this discovery opens possibilities for a whole new generation of antibiotics and vaccines.

What the Public Wants from the NIH: Assurance that its Funding is Well Spent

It is clear that the public wants a fuller understanding of the NIH's funding allocations and how it sets priorities — that is, an assurance that the taxpayers' dollars are well spent. We believe, in fact, that the more the public knows about our processes the more it will support both what we do and how we do it. I want to touch on six principles relevant to establishing priorities:

• An obligation to respond to public health needs, judged variously by the incidence, severity, and cost of specific disorders. However, calculations cannot be correlated with research spending in a simple manner.
• A responsibility to capitalize on previous discoveries and to seize the scientific opportunities that offer the best prospects for obtaining new knowledge and better health. Not all problems are equally approachable, regardless of their importance to public health. Some only yield to a new technology or insight. We must, however, create environments that stimulate new ideas about difficult problems.
• A need to maintain a diverse portfolio on a wide range of diseases. We cannot always know in advance which discovery will be applicable to which disease.
• An obligation to insure first-rate scientific workforces and research facilities.
• A need to seek advice from many sources, including the public.
• And last, but truly foremost in our minds, a commitment to support scientific work of the highest caliber.

Peer review is the cornerstone of our efforts to fund the best science. To identify research worthy of funding, about 40,000 grant applications are peer reviewed at the NIH each year. Of these, approximately 75 percent are evaluated within the NIH Center for Scientific Review (CSR). The NIH is ensuring that CSR has sufficient resources so that its review will recognize, and capitalize on, the opportunities created by the diverse successes of the medical research enterprise, will anticipate emerging fields of research, and accommodate to the rapid pace of scientific change.

In 1998, the Congress asked the Institute of Medicine (IOM) to review the NIH's process for setting priorities. While supporting our principles, the IOM made some useful suggestions about ensuring that our programs are responsive to the public. Over the past year, NIH has responded by appointing a Council of Public Representatives (COPR). The COPR improves our accountability by bringing public views to the NIH, by looking at how the NIH carries out different aspects of its mission, and by conferring on trans-NIH issues. The COPR will be involved in many aspects of NIH programs and policies.

Following another suggestion in the IOM report, this past year the NIH Director required each Institute and Center to produce a strategic plan of research needs and opportunities over two to five years. The plans were developed with input from a wide range of NIH constituents, including patient and other health advocates, scientists, health-care providers, the Congress, the Administration, NIH staff, and other representatives of the public. These strategic plans will be available in the near future and should improve public understanding of the challenges all components of the NIH are facing .

This past year, for the first time, the NIH held a Budget Retreat in June to help develop its presentation of priorities and Areas of Research Emphasis for the President's 2001 budget. The meeting involved ten external advisors, five from COPR and five from the Advisory Committee to the Director (ACD), and created enthusiasm for new areas for collaboration across institutes.

In another major effort to bring public views to bear upon the NIH's programs, priorities, and activities, 26 individuals from outside the agency — scientists, physicians, other health-care providers, patients, and representatives of the ACD and the COPR – met in October to evaluate the scientific quality and relevance of the outcomes of NIH research, a requirement of the Government Performance and Results Act. A report of their assessment has been sent to the Congress as part of the President's budget.

Realizing the Potential of the FY 2001 Budget Request

Generous increases in the last two budget cycles have allowed the NIH to begin many new programs. The funds requested for FY 2001 will advance these programs and, with sound management, allow us to begin new ones. To ensure that NIH can support new initiatives that offer the most promise of expanding knowledge and improving health, and to ensure our ability to support a healthy number of new and young investigators, we will limit growth in commitments and in the size of awards to a two percent average increase for new and continuing grant awards. In addition to initiatives on mental health, cancer, and diabetes, new activities include:

Clinical Research. To take full advantage of rapid research advances in the last five years, which have provided abundant new therapies to study, the NIH will begin a series of programs to expand clinical research. Career development programs will continue to improve the number and quality of investigators. We will start new pilot and early-phase clinical trials thereby speeding the testing of new therapies. We will develop new, and expand older, networks for multi-center studies of pediatric cardiovascular disease, diabetes, digestive diseases, and treatment for drug abuse. We will establish new multi-center clinical trials to evaluate complementary and alternative medical practices for insomnia, pain relief, and liver diseases. Moreover, the public will have greater access to new information on an expanded national clinical trials database (ClinicalTrials.gov) to be launched soon . It will carry information on the many clinical trials funded by the NIH, by other federal agencies, and by industry.

Health Disparities. The NIH has a central role to play in eliminating persistent, even increasing, health disparities through medical research, research training, and dissemination of scientifically sound medical information. In FY 2001, the NIH will continue to invest in this area, allocating $20 million to establish a new Coordinating Center for Research on Health Disparities within the Office of the NIH Director. A new trans-NIH Working Group will develop a strategic plan to eliminate or reduce health disparities among different segments of the American population. The plan, will include goals, timetables, and mechanisms for tracking budgets and accomplishments.

Genetic Medicine. Last November, the Human Genome project finished sequencing one billion of the estimated three billion base pairs of human DNA and deposited them in GenBank, NIH's public database, thus putting us on schedule to have a working draft of the full human genome by this spring. Scientists can use this information to find the genes involved, e.g., in heart diseases, cancer, epilepsy, Alzheimer's, and psychiatric disorders. Companion activities, like developing genomic resources for organisms such as mice, rats, and fruit flies, will help speed the arrival of more precise medical interventions. We are rapidly moving to a time when diagnosis, treatment and even prevention will depend on a precise understanding of the genetic makeup of an individual.

Neurosciences. This is a particularly exciting time for expansion of research in fields of neuroscience, such as neurogenetics and imaging. To foster collaboration and sharing of ideas among the many Institutes which support intramural research in this area, we are requesting funds for construction of a facility for the new National Neuroscience Research Center to house outstanding trans-NIH neuroscience research programs. A total of $73 million is requested over two years, with $47 million in FY 2001 and $26 million as an advance for FY 2002.

Other Sciences, Including Biomedical Computing. Many medical advances build on the knowledge and technology of other scientific disciplines. To exploit our new understanding of biological processes, we need new teams of diverse and skilled researchers to overcome complex technological and research problems. In FY 2001, NIH will establish an Office of Bioengineering and Bioimaging to help the Institutes and Centers set priorities in these areas of science and to enhance collaboration with other agencies.

Based on a report by outside experts, NIH has developed the Biomedical Information Science and Technology Initiative to work toward an intellectual fusion of biomedicine and information technology. In FY 2001, the NIH plans to provide the infrastructure to train the next generation of interdisciplinary scientists, to develop new means for storing, managing, and accessing vast data collections, and to enhance basic research in biomedical computing.

Mr. Chairman, that concludes my opening statement. I will be glad to respond to any questions.