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DEPARTMENT OF HEALTH AND HUMAN SERVICES
National Institutes Of Health

Fiscal Year 2005 Budget Request

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

Barbara Alving, M.D., Acting Director
National Heart, Lung, and Blood Institute
April 1, 2004

I am pleased to present testimony before this Committee on behalf of the National Heart, Lung, and Blood Institute (NHLBI).

The NHLBI leads a national program directed at alleviating the burdens of diseases of the heart, blood vessels, lungs, and blood. The Institute also is responsible for research on the clinical uses of blood and its products and the management of blood resources. For more than a decade, the National Center on Sleep Disorders Research has been part of the NHLBI and, since fiscal year 1998, the NIH Women’s Health Initiative has been administered by the Institute. Our diseases and the burdens associated with them touch the lives of all Americans.

BASIC AND CLINICAL RESEARCH APPROACHES

The ultimate goal of the NHLBI is to improve the public health through discovery of effective methods to prevent and treat disease. Progress toward this goal depends on the existence of a coordinated program that focuses on clinical investigation as the culmination of basic research to unravel the fundamental processes that govern health and disease. The Institute has fostered and sustained a longstanding commitment to laboratory investigations of relevance to its mandate. Moreover, in recent years it has allocated a significant share of the generous budget increases provided to it to aggressive pursuit of promising, cutting-edge opportunities in such disciplines as genomics, proteomics, and nanotechnology. Advances in these areas promise to enable, among other things, more specific approaches to health promotion based on detailed assessment of individual characteristics rather than on general observations about what does or does not foster good health. Our optimism about the probable yield of these new endeavors cannot be overstated.

However, the health-related outcomes of these basic science endeavors depend greatly on the extent to which laboratory discoveries are translated into approaches applicable to “real-life” health problems. And that, in turn, depends on clinical research. Being a disease-oriented agency, the NHLBI has for many years placed strong emphasis on developing and maintaining a robust clinical research portfolio. Particularly with regard to clinical trials, the Institute has worked to design efficient, less costly research approaches to evaluating therapeutic and preventive strategies. As part of this effort, the NHLBI has developed and refined the “clinical research network” concept and successfully applied it to evaluate new therapeutic approaches to conditions such as pediatric cardiovascular disease, asthma, acute respiratory distress syndrome, and Cooley’s anemia. The networks provide an infrastructure that enables rapid and cost-effective testing of new therapies as they come to light.

THE NIH ROADMAP – CLINICAL RESEARCH

It naturally follows that the NHLBI is an enthusiastic participant in the NIH Roadmap initiative titled Re-Engineering the Clinical Research Enterprise: Feasibility of Integrating and Expanding Clinical Research Networks. This new solicitation seeks to identify ways in which clinical research networks can collaborate to conduct clinical trials and other multicenter clinical research studies more efficiently than the current system allows. We at the NHLBI believe that application of lessons learned from this Roadmap initiative will better position the Institute to accelerate the pace of research and to reduce barriers that prevent research advances from becoming incorporated into clinical practice.

POSTMENOPAUSAL HORMONE THERAPY

Major unexpected findings from the NIH Women’s Health Initiative (WHI) illustrate the critical importance of the randomized, controlled clinical trial in determining the risks and benefits of preventive strategies. The study, which assessed the role of estrogen therapy, with or without added progestin, in preventing major causes of death and disability among postmenopausal women, was predicated on strongly suggestive evidence from basic research, observational studies, and smaller clinical trials that often measured so-called surrogate end points (e.g., changes in heart disease risk factors or subclinical manifestations), rather than events such as heart attacks or deaths from coronary disease. Indeed, at the outset of the WHI, much doubt existed regarding the feasibility and ethics of conducting the trial, because “everybody” already “knew” that hormone therapy helped women remain youthful and “feminine forever,” by not only relieving troublesome menopausal symptoms but also improving general health. Much to the surprise of researchers, practicing physicians, and women themselves, the trial of estrogen plus progestin last year was halted when it found increased risks of heart attack, stroke, invasive breast cancer, and blood clots among women assigned to take hormones. And quite recently, the estrogen-alone part of the study was discontinued because the hormone did not appear to have the hoped-for beneficial effect on heart disease (or, on the other hand, the feared unfavorable effect on breast cancer), but it did increase risk of stroke. These findings have major public health significance: the conclusion is that postmenopausal hormones, once ranking among the most-prescribed preparations in the United States, should generally be used only for short-term alleviation of menopausal symptoms.

LUNG-VOLUME-REDUCTION SURGERY (LVRS)

Another trial of great practical importance was a rigorous assessment of LVRS, a procedure that was first used to treat emphysema during the 1950s. Although some patients seemed to benefit from this radical and invasive procedure, high mortality and morbidity discouraged its widespread use until the early 1990s, when some surgeons began performing LVRS again and insurance reimbursement became one of several issues demanding resolution. The National Emphysema Treatment Trial (NETT) clarified the short-and long-term risks and benefits of LVRS and identified the characteristics of patients who may be most likely to benefit from LVRS, as well as those who are at greater risk of death and complications from the procedure. The NETT reflects a unique relationship in which the NIH funded and administered the study and the Centers for Medicare and Medicaid Services (CMS), which sought evidence regarding the advisability of providing Medicare reimbursement for LVRS, supported participants’ care costs. Additionally, the Agency for Healthcare Research and Quality contributed support for analysis of the cost-effectiveness of LVRS. The study results have provided a scientific basis for reassessment of Medicare coverage for LVRS.

TRIALS OF HYPERTENSION CONTROL AND PREVENTION

Last year, we reported results from the ALLHAT (Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial), which found persuasive evidence that traditional diuretics should be the initial treatment of choice for lowering high blood pressure. This is a study that only the NIH would likely have undertaken, as the comparison drugs – a calcium channel blocker and an ACE (angiotensin-converting enzyme) inhibitor – were already established as blood-pressure-lowering agents; it further illustrates the unique role played by the NIH in addressing issues of public health importance. Of additional interest is the observation that blood pressure control rates among ALLHAT participants increased from 25 percent at the beginning of the ALLHAT to 66 percent after five years of followup. These gains were achieved in a variety of clinical practice settings and in subgroups of people known to experience difficulty with blood-pressure control, such as blacks, the elderly, and diabetic patients. These results offer encouragement that blood pressure control is obtainable, and they challenge us to pursue this goal vigorously.

The ALLHAT findings, in combination with evidence from other research studies, prompted issuance of an updated set of guidelines for hypertension management – the so-called JNC 7, or Seventh Report of the Joint National Committee on Prevention, Detection, Evaluation, and Treatment, of High Blood Pressure. An important feature of the guidelines is a reclassification of blood pressure levels that includes the new category “prehypertension” (120 to 139 mm Hg systolic and/or 80 to 89 mm Hg diastolic blood pressure). Individuals with prehypertension are strongly encouraged to pursue lifestyle changes – losing excess weight, eating a heart-healthy diet, increasing physical activity, quitting smoking – to forestall development of overt hypertension. To date, most behavioral interventions have focused on only one or two lifestyle changes at a time. However, findings from a recent clinical trial indicate that an all-in-one approach to lifestyle changes is feasible and effective in lowering blood pressure. Trial participants who addressed many elements of a healthy lifestyle simultaneously also significantly reduced their weight and became more fit – providing even more incentive to undertake such changes.

HYDROXYUREA THERAPY FOR SICKLE CELL DISEASE

A breakthrough for patients occurred in 1995 when the NHLBI announced the results of a major trial of the first treatment for adults with sickle cell disease. The study found that use of the drug hydroxyurea slashed rates of painful crises and acute chest syndrome, and sharply reduced the need for blood transfusions and hospitalizations. A followup study of the trial participants recently reported that hydroxyurea not only protects patients from episodes of severe illness associated with their disease, but also prolongs their lives. Even the sickest patients – those who suffered three or more painful crises a year – benefitted. These results have important implications both for improving patient care and for decreasing health care costs associated with sickle cell disease.

IMPROVING SURVIVAL FOR VICTIMS OF CARDIAC ARREST

Cardiac arrest – in which the heart stops beating effectively, blood does not circulate, no pulse can be felt, and the victim collapses into unconsciousness – is a frequent occurrence in this country. Despite several decades of efforts to train members of the public to perform CPR (cardiopulmonary resuscitation), few victims of out-of-hospital cardiac arrest survive the experience. The NHLBI Public Access Defibrillation trial trained volunteer rescuers to use an automated external defibrillator, a device that shocks the heart back into normal rhythm. It found that use of CPR plus the defibrillator, compared with use of CPR alone, markedly increased survival of people who suffered cardiac arrest in various community settings, and caused no major injuries or serious safety problems. An important next step, currently under way with NHLBI support, is to determine the safety and effectiveness of providing defibrillators to families of heart attack patients for use when a cardiac arrest occurs at home. In addition, the Institute is establishing a research consortium of investigators, hospitals, emergency medical services, and local communities to investigate promising experimental strategies to resuscitate patients who experience out-of-hospital cardiac arrest.

COMBATING THE OBESITY EPIDEMIC

Obesity is a problem of great concern to the NHLBI, as it strongly influences the risk for developing diseases and conditions such as coronary heart disease, hypertension, and diabetes. Thus, the Institute is strongly involved in the overall NIH effort to reverse the U.S obesity epidemic, and I have been especially pleased to serve as cochair of the NIH Obesity Research Task Force.

The NHLBI recently launched a major study that addresses one of the most challenging aspects of weight control – keeping lost pounds off. The Weight Loss Maintenance Trial will initially assist overweight or obese adults participants in making lifestyle changes to reduce their weight and, subsequently, it will test various strategies to help the participants maintain their weight loss over the next several years. The trial focuses on persons who are being treated for high blood pressure or high blood cholesterol and, consequently, have particularly strong reasons to achieve and maintain a healthy weight.

Another new initiative will assess the effectiveness of worksite interventions for preventing or controlling overweight and obesity in adults. Strategies to be considered include implementing environmental and policy changes to increase employees’ physical activity (e.g., flextime or fitness-center discounts), offering healthful food choices in cafeterias and vending machines, providing information about nutrient and calorie content of foods at the point of purchase, and enhancing social support from fellow workers to encourage improved diet and physical activity.

A third NHLBI initiative will explore the potential use of bioengineering approaches to address problems of obesity. For example, new methods for imaging body fat content may enable more specific identification of who needs to lose weight and their success in doing so. Bioengineering techniques may also offer a solution to the difficult technical challenge of obtaining precise measurements of energy intake and expenditure. One can envision development of a wristwatch-like gadget from which the wearer could easily determine whether an energy intake goal has been exceeded or an energy expenditure has been met. New approaches might provide accurate, convenient, easily understood, and inexpensive devices that would foster research, improve clinical management of adults and children, and help the public eat less and exercise more.

CONCLUSION

These examples illustrate the extraordinary potential of clinical research, and particularly clinical trials, to address issues of major importance to the public health. The NHLBI will continue its commitment to stimulate and support clinical research, and to ensure that the knowledge thereby gained is rapidly, efficiently, and fully applied to disease treatment and prevention.

BUDGET STATEMENT

The fiscal year 2005 budget includes $2,963.9 million, an increase of $172.1 million over the fiscal year 2004 enacted level of $2,791.8 million.
I would be pleased to answer any questions that the Committee may have.

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