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Testimony on Setting Research Priorities at NIH by Harold Varmus, M. D.
Director, National Institutes of Health
Accompanied by
Richard Klausner, M.D., Director, National Cancer Institute
Claude Lenfant, M.D., Director, National Heart, Lung and Blood Institute
Phillip Gorden, M.D., Director, National Institute of Diabetes and Digestive and Kidney Diseases
William Paul, M.D., Director, Office of Aids Research
and
Dennis P. Williams, Deputy Assistant Secretary, Budget, DHHS
U.S. Department of Health and Human Services
Before the House Committee on Appropriations, Subcommittee on Labor, HHS, Education
June 10, 1997
Mr. Chairman and Members of the Subcommittee, I am Harold Varmus, Director
of the National Institutes of Health. I am pleased to appear before you to
discuss setting research priorities at the National Institutes of Health. I want
to thank you for the opportunity to discuss this important issue with you and
the Members of this Subcommittee.
Introduction: The issues
Each year, as you know, the Director of the NIH brings to Congress the
President's request for funds to support medical research during the next fiscal
year and reports on promising recent results from NIH-supported investigators.
After Congress and the Administration have agreed upon the level of funding, the
monies are spent by the specific Institutes and Centers (IC) to which they have
been appropriated, in the hope of continued progress in understanding biological
phenomena and combating disease. Over the past half-century, this has proven to
be a highly productive relationship, responsible for many of the discoveries
that have improved health around the world, deepened our knowledge of living
organisms, and placed the United States in the forefront of medical research and
development.
Today we are meeting to discuss how our money is spent. What kinds of
decisions must be made? Who makes those decisions? What factors are considered
in making them? How reliable are the decisions? How do we evaluate them and make
appropriate adjustments?
Underlying these questions are some fundamental and controversial issues:
- How much of the NIH budget should be devoted to plans to conquer
specific diseases? and how much should be invested in fundamental science
without immediate relevance to a specific disease?
- Where should the final authority reside for making these important
decisions about the distribution of NIH's resources? In the hands of the
scientists who lead the NIH and conduct its business? Or in the hands of the
public's representatives --- those in Congress who appropriate funds to the NIH
or those in the Executive Branch who have jurisdiction over the agency?
- How should the NIH --- and the Federal Government --- respond-to the
threats to health posed by specific diseases or injuries? In particular, how
should the government respond to those advocates for research on certain
diseases who argue that inadequate resources are devoted to their cause?
Resource allocation at the NIH
I would like to confront these issues in the context of ten
observations---based on historical facts, operating principles, and personal
experience --- that help explain how the NIH manages its budget.
- Resource allocation is not a single issue, many decisions must be made
during the complex process of deciding how the NIH will spend its money.
- In the appropriations process, the Administration must recommend --- and
the Congress must then ultimately determine, with the President's agreement
--- how much money to provide to each IC. Since each IC is, by law and by name,
heavily committed to certain domains of medical science (e.g., cancer, aging,
nursing, or mental health), the annual budget distribution to ICs sets rough
limits on what can be achieved each year in those domains and provides a
framework for making subsequent decisions.
- Within the framework of the President's budget request, the intent of the
Appropriations bill, and its existing commitments, each IC must determine how to
allocate its funds to different mechanisms for the support of science --- that
is, to investigator-initiated grants, contracts, centers, the intramural
program, or training programs. These decisions must be closely tailored to the
IC's research objectives.
- Each IC must also decide which specific applications for funding to
support and whether to emphasize certain research topics within its authorized
domain.
- The net effect of these multiple processes and decisions will determine
how much of the entire NIH budget is devoted to work in certain scientific
disciplines or on certain diseases.
- The entire budget cannot be subjected to unfettered realignment each year
enduring impact of past decisions and the need to provide stable support for
scientific work restrict the funds that can be redirected.
Some past decisions, especially the creation of an Institute, have effects
of long duration on the outlines of the NIH budget, since history has shown that
each Institute is likely to receive each year at least as much as in the
previous year. Other decisions (such as the development of an extramural
centers program supported by an individual Institute) have effects that can last
decades, because such programs are developed over multiple years and
disassembled only with difficulty. Even decisions to award new grants to
individual investigators have a longer life span (four years on average) than
the annual appropriations. Historical decisions and the substantial base of
research funds already committed to grant recipients leave only a relatively
small fraction of each year's appropriation that can be affected by changes in
funding policies.
- There are legitimate limits to our ability to plan science.
Because science attempts to discover what is unknown, it is inherently
unpredictable; in this sense, it is unlike most industries, which can employ
well-established methods to generate planned amounts of known products. History
has repeatedly shown the benefits of allowing a significant portion of our
research activity to be governed by the imagination and productivity of
individual scientists, not by a regimented plan for alleviating diseases we do
not yet fully understand.
- The development of recombinant DNA methods dramatically illustrates the need
for unplanned, untargeted, high quality fundamental science. Studies in the
1960's and early 1970's of circular DNA found in certain bacterial strains and
of bacterial enzymes that attacked the DNA of bacterial viruses---both kinds of
work then without obvious linkage to human disease --- forever changed the way
all biomedical research is done. These studies provided the tools that
underwrite the biotechnology industry, a lucrative part of the U.S. economy
that already supplies many important therapeutic products. And they set the
stage for the Human Genome Project, which is revolutionizing approaches to
virtually all diseases.
- In the light of this example and many others, a substantial fraction (on
average, just over half) of each IC's budget is devoted to the support of the
best proposals received from extramural grant applicants, regardless of apparent
applicability to prevention or treatment strategies for specific diseases. As a
result, much of the research done by each IC may be difficult or impossible to
explain as a part of a research plan against a specific disease. Nevertheless,
such work --- on, for example, the pathways cells use to interpret chemical
signals, the three- dimensional structures of proteins, or the processes of cell
death---could ultimately form the basis for practical advances against any of
several diseases.
- Sometimes it is possible to design relatively precise research strategies,
even outside the boundaries of specific diseases. For example, current efforts
to map and sequence all the human chromosomes through the Human Genome Project
are proceeding according to a coordinated plan, with scientific and budgetary
milestones --- a plan made possible by earlier, serendipitous discoveries of the
necessary techniques. Similarly, recent discoveries about cancer genes through a
variety of unplanned routes now permit a deliberate attempt to describe the full
range of genetic abnormalities in many human cancers. (The plan for this Cancer
Genome Anatomy Project is an initiative in the NIH Areas of Emphasis for FY 1998,
supporting a proposal for additional funds for the NO in the current budget
request.)
- But overall, the ICs cannot and should not provide precise plans for their
entire research portfolios. Instead, they try to estimate the amounts they are
likely to spend on various disciplines in laboratory and clinical science, based
upon existing commitments, past experience, and knowledge os are equally approachable, regardless of their
importance for public health. Fortunately, opportunities to pursue rare diseases
or exotic phenomena often have unexpected benefits for more common concerns.
- A need to maintain a diverse portfolio that supports work in many
scientific disciplines and on a wide range of diseases. Because we cannot know
when major discoveries will occur and what opportunities they will create, it is
important to support ongoing research along a broad frontier.
- An obligation to insure a strong scientific infrastructure, with a high
quality workforce and excellent research facilities. Productive science cannot
be done without well-trained investigators and modem equipment and laboratories.
For these reasons, the NIH supports training programs, individual fellowships,
purchase of instruments, limited construction projects, and a host institution's
administrative and facilities costs.
- To evaluate these many criteria for making decisions, the NIH requires
and seeks advice from many sources
The factors that influence the planning and spending of budgets are complex,
so opinions about them are solicited and provided from many quarters --- the
extramural scientific community, patient advocacy groups, Congress and the
Administration, and the NIH staff. We gather these opinions through many means,
as appropriate to the decision-making process:
- we use review groups composed of accomplished investigators to evaluate
grant applications s are equally approachable, regardless of their
importance for public health. Fortunately, opportunities to pursue rare diseases
or exotic phenomena often have unexpected benefits for more common concerns.
- A need to maintain a diverse portfolio that supports work in many
scientific disciplines and on a wide range of diseases. Because we cannot know
when major discoveries will occur and what opportunities they will create, it is
important to support ongoing research along a broad frontier.
- An obligation to insure a strong scientific infrastructure, with a high
quality workforce and excellent research facilities. Productive science cannot
be done without well-trained investigators and modem equipment and laboratories.
For these reasons, the NIH supports training programs, individual fellowships,
purchase of instruments, limited construction projects, and a host institution's
administrative and facilities costs.
- To evaluate these many criteria for making decisions, the NIH requires
and seeks advice from many sources
The factors that influence the planning and spending of budgets are complex,
so opinions about them are solicited and provided from many quarters --- the
extramural scientific community, patient advocacy groups, Congress and the
Administration, and the NIH staff. We gather these opinions through many means,
as appropriate to the decision-making process:
- we use review groups composed of accomplished investigators to evaluate
grant applications for scientific merit;
- each IC convenes meetings of national advisory councils, with members from
the public, medical, and scientific communities, to review a broad range of IC
policies;
- many conferences, workshops, and studies are organized or commissioned
each year to gather opinions on specific scientific, health, ethical, and
administrative issues;
- the ICs have highly evolved processes for reviewing scientific progress in
their areas of responsibility, for developing long-range research objectives,
and for formulating annual budgetary plans and research initiatives;
- in the past few years, the NIH has made frequent use of extramural
advisory groups to assess trans-NIH activities (the intramural research program,
the Clinical Center, gene therapy, clinical research, and AIDS research) and to
recommend budgetary and programmatic changes in those areas; and
- the IC Directors and NIH staff consult frequently with members of other
Federal agencies, with the OMB and DHHS, with Congressional members and staff,
and with professional and health advocacy organizations for guidance on a
variety of common concerns.
Despite these many means of gathering opinions and evaluating them,
assembling each IC's research portfolio is a difficult and imperfect process,
for which IC Directors and the NIH Director must assume ultimate
responsibility.
- Assessing or designing a research portfolio from numbers alone is a
hazardous enterprise.
The public and the Congress have a right to know how our money is spent.
Efforts to find out often begin with questions about the amount of money
provided to certain ICs or devoted to specific diseases. No disease is confined
to one Institute, and no Institute is confined to a single disease; hence
distribution of funds by IC is usually an inadequate measure of support for
research on a specific disease. Coding of funds by disease category across the
NIH, though useful for some purposes, is also inherently imprecise. In
particular, it is difficult to assign to specific diseases the large investments
in basic research that are most likely to have profound effects on our ability
to control disease. (Consider, for example, the large difference between the
number of grants that can be assigned specifically to research on Parkinson's
disease and the much bigger number addressed to topics such as nerve cell
biology, dopamine metabolism, and neurodegeneration that have obvious
implications for understanding and treating this disease see Figures .)
As a further complication of coding research by disease, we know from
repeated experience that research aimed in one direction frequently provides
benefits in an unexpected direction. I confront the problem directly when I
attempt to categorize the research in my own laboratory. A gene that we study
because it can cause cancers in animals is essential for maintaining normal bone
density. Another gene we discovered as a cause of breast cancer in mice has a
central role in the development of the brain and other tissues. I don't know how
to classify such projects accurately.
For these reasons, there is no "right" amount of money or number of projects
for any disease. Inevitably all such numbers are approximations. It is therefore
crucial that the system for allocating NIH funds be sufficiently flexible to
accommodate a new proposal with an important, imaginative idea, regardless of
the category to which it might be assigned.
- Scientific work is not simply a commodity that can be purchased, the
effective shifting of priorities requires new ideas and new personnel, as well
as budgetary realignments.
- For understandable reasons, campaigns to expand NIH- supported research on
defined topics or diseases often focus on efforts to increase spending on those
topics or diseases. A mere increase in financial support of a field, however,
without efforts to enlarge its scope, opportunities, and personnel, is likely to
benefit only those investigators already established in the area; this approach,
by itself, is unlikely to make optimal use of scarce resources. To augment
research on specific topics in a more responsible fashion, it is necessary to
show that under-explored opportunities exist and that they can attract
investigators --- either newly trained scientists or scientists from other
fields---who will then propose meritorious projects.
- Several means are used by the NIH to recruit new talent to a scientific
problem:
- advertising an IC's interest in making funds available to pursue a new
scientific opportunity or a public health challenge;
-
- opportunities and needs in an underserved field; and
- training new scientists to work in a designated area.
- A decision to increase support of one area of medical science now usually
constrains the support of something else.
For many years, especially in the 1950's and 1960's, decisions to create new
Institutes or expand programs to study specific diseases were accompanied by
dramatic increases in the total NIH budget, in the numbers of NIH-supported
investigators, and in the size and diversity of our research agenda. No programs
needed to be attenuated by these historic decisions, which have largely shaped
the contemporary NIH.
This is no longer the case --- and has not been so for several years.
Although the NIH continues to fare relatively well in a constrained fiscal
environment, recent percentage increases have been modest by historical
standards. Therefore, directives to spend more, for example, in certain
disease-specific areas constrain spending in other disease areas or in our most
productive, non-targeted areas.
- Existing methods for resource allocation at the NIH are preferable to
excessive Congressional directives.
We are living in a time of great productivity in the biological sciences.
Many fields of medical research deserve increased financial support and could
move faster with more funds. Because resources are limited, pushing funds
vigorously in one direction limits the flow in others. This situation compels us
to consider especially carefully whether proposals to enhance investments in
certain fields are justified by new scientific opportunities, by the urgency of
public health issues, or by the other criteria described above.
I have already indicated numerous ways in which the distribution of our
funds is influenced by many constituencies, many processes, and many factors,
including Congressional concerns. At this time in our history, with fierce
competition among agencies for Federal funds and among grant applicants for NIH
support, it is particularly important to achieve consensus on new initiatives
before assigning scarce Federal dollars to them through legislative actions.
The methods my colleagues and I have used to build the NIH budget emphasizes
evaluations of current scientific opportunities and public health needs, while
maintaining our traditional commitment to a strong component of
investigator-initiated research. When we make requests for increased funding for
specific ICs, we do so based on carefully considered proposals that aim to:
- exploit recent discoveries, such as the isolation of new genes for human
diseases;
- encourage studies of diseases that have been relatively neglected, poorly
controlled, or recently made more accessible to scientific study; or
- strengthen research technologies, such as computer science, imaging
devices, neuroscience, or gene mapping, applicable to a broad range of
disciplines and diseases.
(Many such proposals appear in each year's budget proposal as part of the
NIH's Areas of Emphasis, and they stimulate the budget readjustments I make
during the fiscal year through my one percent transfer authority and
discretionary fund.)
- Many novel and powerful means are available, and should be used,
to-heighten the interest of scientists in the public benefits of their research.
The NIH and the scientists we support have a responsibility to promote the
health of the public through our research efforts. Indeed that responsibility
is part of the mutually beneficial compact the NIH has held with the public over
many years. Nevertheless, the public and the Congress are often frustrated with
the pace of progress for a number of reasons:
- medical science is inherently hard and slow;
- advances do not occur at equal rates against all diseases and resources
are not committed in equal measure to all objectives;
- the long-term relevance of basic science to the diseases that now produce
human misery may be difficult to comprehend; and
- scientists themselves may be deficient in comprehending or communicating
the connections between their work and its potential value for the public.
I have argued here, on several grounds, that the best solutions to such
frustrations do not reside in directives to reroute dollars to specific
diseases. But I do not mean to suggest that advocates for disease-oriented
research cannot or should not do more than is currently done through traditional
NTH processes to advance their causes.
We at the NIH encourage efforts to engage all our scientists, even those
working at the most basic levels, to learn more about human disease. Especially
now that scientific advances have brought basic research so close to clinical
problems, my colleagues and I urge that trainees in all disciplines be exposed
to disorders of human biology and that medically-trained individuals be
encouraged to take up research careers. Advocates for the study of specific
diseases can themselves be effective at the local or national level by visiting
individual scientists or professional societies, thereby stimulating the
interest of working investigators in unappreciated implications of their work;
this strategy has been effectively used by proponents of research on cystic
fibrosis, ataxia telangiectasia, scleroderma, and many other diseases.
The NIH often responds to concerns that research on a specific disorder is
underserved by convening a workshop on the disease; such workshops are intended
to evaluate and publicize research opportunities, bring potentially
collaborative disciplines together, and stimulate the interests of new
investigators. Major workshops have recently been held on the topics of autism,
spinal cord injury, and Parkinson's disease, and another will soon be held on
diabetes mellitus. The workshop on Parkinson's disease was especially notable
for having brought together clinicians and geneticists who then collaborated to
identify a new chromosomal locus that predisposes to a familial form of the
disease. The research opportunity created by this finding will attract new
investigators and could be the basis of major advances against the more common,
non-familial form of Parkinson's disease.
Mr. Chairman, I am grateful to you for providing a forum to present these
views about an important, contentious, and complex issue. I would be pleased to
answer any questions you might have.
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