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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