DEPARTMENT OF HEALTH AND HUMAN SERVICES
National Institutes of Health

National Heart, Lung, and Blood Institute
Fiscal Year 2006 Budget Request

Witness appearing before the
House Subcommittee on Labor-HHS-Education Appropriations

March 9, 2005

Elizabeth G. Nabel, M.D., Director
National Heart, Lung, and Blood Institute

I am pleased to present the Fiscal Year (FY) 2006 President's Budget request for the National Heart, Lung, and Blood Institute (NHLBI). The FY 2006 budget includes $2,951,270,000, an increase of $10,069,000 over the fiscal year 2005 enacted level of $2,941,201,000 comparable for transfers proposed in the President's request. I come to you with pride on behalf of the NIH component that is responsible for much of the gain in life expectancy that we have enjoyed over the past three decades in the United States, as shown in this chart. At the same time, however, I come with deep concern because the diseases under NHLBI responsibility still comprise three of the four leading causes of death in this country—heart disease, stroke, and chronic obstructive pulmonary disease (COPD). Clearly, we have come a long way, but we have far to go.

As the NHLBI's first new director in 22 years, I would like to take this opportunity to share with the Committee my vision for the Institute. This vision is based upon a fundamental set of values—excellence, integrity, innovation, respect, and compassion—that will permeate all activities in the NHLBI. I believe that scientific discovery provides the basis for progress and that the NHLBI is uniquely positioned to catalyze changes that must be made to transform our new scientific knowledge into tangible benefits for the people of this country. Within this framework, let me articulate four themes that will guide priority setting of our research agenda.

The first theme—stimulating basic discoveries of the causes of diseases—is vital to developing new, critically needed treatments. Basic research provides the foundation of the NHLBI portfolio and has been one of its great strengths. The typical model of investigation—research conducted by single investigators or small groups of investigators on projects of their own inspiration—accounts for most of the unanticipated and major scientific discoveries in this country. I believe strongly that we must protect and nurture investigator-initiated research. The NHLBI will continue to invest in the most talented scientists conducting the highest caliber research. Innovation and creativity using the most advanced biomedical technologies will be our goal.

We have an exciting opportunity to support emerging new scientific fields. Major strides are being made in computer sciences, bioengineering, material sciences, chemistry, and other areas of study that vastly benefit medical research, and the pace of discovery in these disciplines should be accelerated. One approach is to develop funding mechanisms (e.g., for support of high-risk research) that encourage innovative thinkers to turn their attention to the major current challenges in heart, lung, and blood diseases.

Another objective is to generate large, publicly available sets of reagents and data that could function as a “tool kit” for NHLBI investigators. Gene sequences and maps, cell lines, knockouts and knockdowns of genes in selected animals, reference sets of proteins, protein affinity reagents, and libraries of small molecules are examples of resources that will provide our investigators with the technologies required for innovative discoveries.

Our second task is to speed translation to clinical applications so that people can benefit as quickly as possible from the basic research enterprise. Clinical research, and more specifically, translational research (“bench to bedside”) are vital to our mission, so that we can translate basic discoveries into the reality of better health for our country.

The NHLBI must further develop the infrastructure for clinical research so that it serves the evolving field of scientific discovery and provides a foundation for evidence-based clinical decision-making. Clinical research is critical to ensuring that new products and techniques are safe and effective before they are widely applied. However, clinical research is often time-consuming and inefficient, and is increasingly burdened by regulatory hurdles. Our challenge is to expand clinical research to complement the exciting basic science discoveries, while making it more efficient and cost-effective.

We intend to develop a translational research agenda supported by clinical trials, clinical networks, and clinical workforce training. Key components will focus on increasing interactions between basic and clinical investigators and easing the movement of new tools from laboratories to clinics. We will build upon our rich experience with clinical trials and networks to develop new partnerships among organized patient communities, community-based physicians, and academic researchers. We will work on improving bioinformatics and clinical databases, standards for clinical research protocols, measures of clinical outcomes, and quality assessment. Translational research requires the expertise of many fields and should include analysis of health education, outcomes, health-care delivery, and health-care economics. This focus fits well with the Re-engineering the Clinical Research Enterprise of the Roadmap.

The NHLBI must cultivate a cadre of clinical researchers who have skills commensurate with the complexity and needs of our research enterprise. Clinicians must be trained to work in the interdisciplinary, team-oriented environments that characterize today's research efforts. We further anticipate that specific training will be required in an array of disciplines important to clinical research, including genetics, epidemiology, biostatistics, and behavioral medicine.

At the core of this vision is the need to develop new partnerships of research with organized patient communities, community-based health care providers, and academic researchers. We will rely on our partnerships to facilitate the conduct of this clinical research, to train our clinical investigators, and most important, to achieve our common goals of improved health for the public.

The third theme is facilitating communication between scientists and physicians so that new ideas can be generated, shared, and advanced.

Today's science is far more complex than that of yesteryear. Research, whether basic or clinical, is now commonly done by teams of scientists wherein each individual brings specific talents and expertise to the overall effort. We will stimulate and facilitate the conduct of interdisciplinary research, so that advances can be made more quickly. Principal-investigator status will be granted not to just one investigator, as is the norm, but to all key members of the research team. Integrated reviews of grants will take into account the melding of various disciplines to address the problem at hand, and interdisciplinary teams will be encouraged to evolve in both directed and unexpected ways.

An essential component of our efforts in research collaboration will be community-based clinical trials, which enhance the conduct of clinical research at academic medical centers. An outstanding example is our ALLHAT (Antihypertensive and Lipid-Lowering to Prevent Heart Attack Trial), in which physicians from many types of medical settings—a total of 623 sites in 47 states, Puerto Rico, the U.S. Virgin Islands, and Canada—successfully enrolled over 42,000 patients and followed them for 6 years. The physicians participated because they believed in the importance of the scientific questions being addressed with regard to patient care and because of the direct benefits of participation to their patients, including free medications. These community-based physicians conducted the trial at very high standards—follow up was over 97 percent. As part of our plan to disseminate the ALLHAT results, participating community physicians are now working with other doctors in their local communities to treat patients with high blood pressure.

Our fourth task is to effectively communicate our research advances to the public to improve understanding of new, promising science.

The NHLBI has an outstanding history of outreach in the areas of high blood pressure, cholesterol, asthma, heart attack, obesity, sleep disorders, and women's cardiovascular health, and new efforts are under way with respect to COPD and peripheral arterial disease. I wholeheartedly support these programs that serve the mission of our Institute and the Nation. Education of our patients and the public regarding prevention and treatment of heart, lung, blood, and sleep disorders is one of my highest priorities.

We will continue to work collaboratively with our colleagues in the DHHS, including the CDC and the FDA, to support prevention and control programs. We also have an unprecedented opportunity to build upon our partnerships with professional organizations, who have a large stake in developing and implementing practice guidelines and monitoring their effectiveness, and with patient advocacy groups. One of our most gratifying partnership programs has been The Heart Truth, which is successfully raising awareness nationwide that heart disease is the leading cause of death among American women. The “reach” of this campaign continues to expand as we forge additional fruitful partnerships with entities in the public and private sectors.

Disparities in health status constitute a significant global issue. Research is essential to understand the diverse contributions of genetics, health behavior, diet, socioeconomic status, culture, and environmental exposures in the genesis of health disparities in heart, lung, and blood diseases and to formulate, evaluate, and disseminate well-conceived, focused intervention programs. This work will necessarily entail a vigorous effort to increase the representation of minorities in the ranks of NHLBI researchers. We are also cognizant of the need to improve and expand programs to prevent, manage, and treat diseases and conditions that disproportionately affect U.S. minority and underserved populations, such as cardiovascular disease and asthma, and to evaluate the effectiveness of our research, treatment, and education programs. A full resolution of the health disparities problem will occur only through committed and sustained efforts by many in our government, health centers, and society.

The realization of this vision will require the efforts of many. We are engaged in a special form of public service, that is, the promotion of patient and public health. I will work diligently to preserve public trust in the Institute, the NIH, and the biomedical research enterprise, and to ensure that the NHLBI serves the public with the highest level of integrity. This trust is essential for meeting our common goals of making important new scientific discoveries and translating them to improve health in this country.

Thank you, Mr. Chairman. I would be pleased to answer any questions that the Committee may have.