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Testimony on Research Priorities for NIH by Harold Varmus, M. D.
Director
National Institutes of Health
U.S. Department of Health and Human Services
Before the Senate Committee on Labor and Human Resources, Subcommittee on Public Health and Safety
May 1, 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, 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. Historically, Congress grants the request and often adds money to
it. The funds are then 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
combatting disease. Over the past half-century, this has proven to be a highly productive
arrangement, 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 (including the White House, the OMB, the
Secretary of HHS, and the NIH and IC Directors) 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; the 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 FYI 998, supporting a proposal for
additional funds for the NCI 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 of scientific trends.
- Many criteria guide the development and expenditure of the NIH budget.
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 FYI 998, supporting a proposal for
additional funds for the NCI 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 of scientific trends.
Answers to questions that affect resource allocation at the NIH --- how much to give to a
single IC? how much to devote to a certain discipline, disease, or grant mechanism? or which
applicant to fund? --- are influenced by several factors:
An obligation to respond to public health needs, as judged by the incidence, severity, and cost of
specific disorders. Changes in the nature or burden of disease are especially important, as
illustrated by the increased resources recently devoted to a new disease (AIDS), to diseases
disproportionately affecting a growing segment of the population (the aged), and to a resurgent
disease (tuberculosis). But calculations of public health needs are difficult, and the results cannot
be correlated with research spending in a simple manner.
A commitment to support work of the highest scientific calibre. A basic tenet of our stewardship
is the pledge to maximize the return on the public's investment in research; to do this, we demand
that all requests for support pass stringent review for scientific quality.
A responsibility to seize the scientific opportunities that offer the best prospects for new
knowledge and better health. As administrators of science, we have learned that the most
significant and rapid advances are likely to occur when new findings, often serendipitous, have
expanded experimental possibilities. Not all problems 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 isdity that can be purchased; hence the effective shifting of
priorities requires more than 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 persue a new scientific
opportunity or a public health challenge;
- inviting scientists from allied fields to workshops that highlight 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 budgetary 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 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. 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 NIH 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.
CHART 1
Parkinson's Disease and Related Research Awards, FY 96
>>
Parkinson's Disease (192 Awards) accounts for about 25% of the total Research Awards granted
for Fiscal Year 1996. The Research Awards (755 Awards) include:
-
- Dihydroxyphenylalanine
- Neural Degeneration
- Transneuronal Degeneration
- Basal Ganglia
- Dopamine Transporter
- Parkinson's Disease
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