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November 2, 1992

JOINT STEERING COMMITTEE ON PUBLIC POLICY
AMERICAN SOCIETY FOR CELL BIOLOGY
AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY
BIOPHYSICAL SOCIETY
GENETICS SOCIETY OF AMERICA

HOW TO MAINTAIN U.S. LEADERSHIP IN BIOMEDICAL RESEARCH

INTRODUCTION

Government investment in biomedical research over the past three decades has catalyzed a
scientific revolution. Powerful new methods for studying life forms now offer unprecedented
opportunities to understand the fundamental principles of life, to improve human health, and
to strengthen the nation’s economy through the biotechnology industry, in which the United
States remains in the forefront. Further progress and American leadership are, however,
threatened by an economic crisis, by the application of political “litmus tests" to science policy,
and by the deterioration of the research community’s physical and educational resources. We
need innovative policies to organize and conduct biomedical research to reap the benefits of
recent advances and Keep ahead of foreign competitors.

THE PROMISE OF BIOMEDICAL RESEARCH

The traditional goals of biomedical research have been the prevention and treatment of human
disease. Unprecedented progress in biology during the past few decades has markedly enhanced
the prospects for relief from disease and has inspired a broader view of the possible effects of
biomedical research upon our society. The fundamental components of biological
systems---cells, genes, and molecules---can now be studied at levels of detail that were
unimaginable even ten years ago. Given adequate support, biomedical science now has the tools
to discover the basic mechanisms of living systems. This knowledge can transform medicine by
allowing rational and less expensive approaches to the diagnosis, treatment and prevention of
disease. The growth of the biotechnology industry has proven that new research tools can be
rapidly converted to useful products, including novel means to improve human health, and can
generate jobs and satellite industries that stimulate economic growth. Biomedical research has
the potential to cause wide-ranging effects upon our culture in the following ways.

1) New approaches to treatment of human diseases. The prospects for fulfilling the
traditional objectives of biomedical research have never been greater, as a result of new-found
abilities to understand diseases at a genetic and molecular level. These advances have already
stimulated novel approaches to diagnosis, therapy, and drug discovery for cancer and for
infectious and inherited diseases.
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2) Progress in preventive medicine. New biomedical methods offer many opportunities
to prevent birth defects, to measure genetically-determined predispositions to disease, to make
diagnoses early in the course of an illness, and to produce vaccines against infectious diseases.

These approaches can significantly reduce human suffering by preventing or minimizing the
effects of serious diseases.

3) Containment of health-care costs, It is sometimes claimed that technological advances
increase the cost of health-care. But advances in biotechnology can often reduce costs by
preventing diseases, diagnosing them at early stages, or providing relatively inexpensive
therapies produced in simple biological systems. For example, polio vaccines are much less
expensive than iron lungs; a vaccine against hepatitis B, made from recombinant DNA, is less
costly than care for chronic hepatitis; and genetically engineered blood cell factors reduce
hospitalization for cancer patients undergoing chemotherapy.

4) Preparation against unanticipated diseases. The AIDS epidemic, the emergence of
Legionnaire’s Disease, and the recent resurgence of tuberculosis are grim reminders that we
cannot prejudge the spectrum of maladies we will have to confront in the future. For this reason,
it is important to support a broadly-based research program that will prepare us to respond to
novel diseases. The vigorous funding of research on cancer viruses in the decade preceding the
appearance of AIDS fortuitously provided a ten-year head-start towards an understanding of
the AIDS virus. In contrast, the neglect of bacteriology, especially the study of mycobacteria,

during the past twenty years, has compromised our ability to cope with the current epidemic
of tuberculosis.

5) Economic growth through biotechnology and allied industries, Advances in basic
biomedical research, particularly the development of recombinant DNA methods, stimulated
the birth and growth of the biotechnology industry in the United States and its expansion
around the world. This industry has prospered in our country because of our solid research base,
interactions with a strong pharmaceutical industry, and innovative thinking and financing.
Biotechnology has already generated useful and profitable products and promises to produce
many more; it offers jobs that have attracted workers with a wide range of technical abilities and
educational backgrounds; and it has supported the development of many satellite industries that
make the materials required for molecular research here and abroad.

6) Advances in agriculture. Feeding the rapidly expanding world population will be a
serious problem in the next decades. Genetic engineering, developed in the biomedical sector,
has recently been applied to plants. As a result, new agricultural products will have extended
shelf lives, resistance to conventional insect and vira] infestations, and higher yields. These

advances will have profound effects upon food production and distribution in this country and
world-wide.

7) Environmental protection. Recombinant DNA technology offers multiple strategies for

helping our society to cope with environmental problems. For example, genetically altered
bacteria can help repair environmental contamination (bioremediation); biomaterials can

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replace petroleum-based products and thereby help alleviate the demands for oil; and
insect-resistant plants can reduce the use of chemical pesticides.

8) Public understanding of science and technology. The excitement and measurable
benefits of biomedical research stimulate broad interest in science and technology. A strong
national program for biomedical research can have the important secondary effects of
encouraging people to seek further education and jobs in biology, improving education in the
sciences, and raising the level of scientific literacy in the country.

HALLEN TOT NTI FB ED TH
US.

Enlightened federal programs for promoting basic research in biology and allied fields over the
past few decades have established this country as the world’s leader in biomedical research,
nurtured the remarkable scientific progress that now promises many improvements in the
quality of life, and subsidized the founding of the biotechnology industry with its many
economic and social benefits. Nevertheless, American progress and pre-eminence in biomedical
research are currently threatened by several factors, If swift and appropriate responses are not
made, we will fail to deliver on the promises of biomedical research. Furthermore, our
competitors in Japan and the European Community will capitalize on our earlier investments
in basic research and attempt to take the lead in biotechnology within the next decade. Thus
the biotechnology industry, like the electronics industry, could be transferred overseas, unless
we find ways to counteract the problems listed below.

1) Underfunding of biomedica] research. The productivity of biomedical research is
currently limited by available resources. The annual cost of health care is more than $600
billion, but the nation’s investment in biomedical research is only about $10 billion. This is low
compared to research investments in other areas. (The pharmaceutical industry spends about
10% of its budget on research, the defense industry, about 15%, the health care industry, about
2%.) In the past few years, the growing budget deficit, the recession, competing social
priorities, and rising costs of research have conspired to reduce the effective level of federal
support for biomedical research, despite the remarkable opportunities for progress. Although
funding for the study of some highly visible diseases has been less severely affected, support for
many important programs, particularly fuadamental research in basic biology, has been
inadequate. Budgetary restrictions on biomedical research have caused much promising research
to go unfunded and delayed the new discoveries required for practical applications. Such
budgetary constraints upon biomedical research not only have an immediate and direct impact
on scientific progress, but also discourage students from pursuing science.

2) Deterioration of the physical infrastructure, Limited funding for biomedical research
has postponed the long-overdue renovation of research facilities and the replacement of
outmoded equipment. In many outstanding institutions, the conduct of biomedical research
is constrained and even dangerous as a result of crowded, dilapidated laboratories and
inadequate instruments. Although in recent years Congress has appropriated substantial funds

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for construction at research institutions of their choice, they have done so in a way that
circumvents peer review, serves Jocal needs rather than the advancement of science as a whole,

slights our academic centers of excellence, and, thus, undermines the cost-effective use of federal
funds for research.

3) Poor administrative organization of science. The federal bureaucracy has failed to keep
pace with developments in science and technology. As a result, agencies that should be working
together to promote research in the life sciences are instead isolated in multiple departments
with competing agendas. Furthermore, many agencies that fund life sciences research lack
appropriate mechanisms for initiating, judging, and administering programs or have not adapted
their mechanisms to progress in research. For example, NIH study sections, comprised of
scientific peers who review applications, are organized according to outmoded categories. In

addition, the scientific community is not adequately involved in administrative decisions to
initiate targeted projects.

4) Inappropriate criteria for selection of scientific leadership. In recent years, it has
become commonplace to consider political views on issues such as abortion and the use of fetal
tissue in reseatch in the choice of appointees to high-ranking positions. This tendency has
compromised our ability to select federal leaders of American science based on scientific
accomplishments and the capacity to manage complex programs and make objective decisions.

5) Shortsighted objectives for biomedical research. Constraints on federal support for
science have encouraged calls for increased application of current knowledge to practical
problems. These appeals have special resonance in biomedical science now that so many
opportunities for practical applications are at hand. (For example, the polymerase chain
reaction (PCR), a powerful new method for studying genes, based upon the principles of DNA
synthesis, has already been used for medical diagnosis, criminal law, evolutionary studies,
anthropology, and identification of victims of political oppression.) The improved prospects tor
direct application of biomedical science to societal problems are important, but they should
have their primary effects on the private sector, where such applications are most likely to be
developed efficiently. In particular, they shouid not be used to justify policies thai
support practical applications at the expense ot traditional, broadly conceived explorations or
the basic principles of biology through government-supported research in the academic
community. If that occurs, we will soon fail to make the new discoveries that are required ior
continued advances of practical] value to society, lose our position of leadership in biomedical
research, and discourage the best minds from entering this field.

6) Obstacles to technology transfer. Given the requirement to sustain fundamental
research, there is also the need to improve the means by which we recognize and develop ihe
application of advances in basic science. Ways must be found to foster technology transfer,
preferably in the private sector, because that is where there is the most sensitivity to market
forces. The growing federal] attention to conflict of interest has sent confused messages to
academic scientists and has been a deterrent to technology transfer.
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7) Declining educational programs in science. Both academic biomedical] research and the
biotechnology industry depend upon the nation’s schools to supply a competent workforce by
stimulating interest in scientific thought and by training students in scientific methods, Many
indicators show that we are failing to achieve these goals, especially in the early school years
and when viewed in comparison to other countries.

RECOMMENDATIONS
1. To Build an Improved National Agenda for Biomedical Research

a) Develop an investment strategy for biomedical research. As measured by the percentage
of health care costs reinvested in research, biomedical research is underfunded, although it will
result in improved health, reduced medical costs, and the creation of jobs in the private sesctor.
It is presently difficult to determine the optimal level of federal biomedical research support and
the appropriate allocations to fundamental and applied areas. The government and the
scientific community should work together to formulate an economic strategy based on an
analysis of the following factors: 1) the opportunities made available by the recent revolution
in biology for the prevention and treatment of the major diseases; 2) the potential danger and
costs of new epidemics and diseases of modern society; 3) the potential contribution of new
treatments to reducing health care costs and improving the quality of life; 4} the potential
effects of government sponsored biomedical research on the environment, agriculture and
industry; and 5) investment strategies and competition from other countries.

b) Bring creative science to critical problems. Biomedical science has contributed
effectively to new areas such as AIDS research, biotechnology, rationa) drug design, cancer
research and agriculture. Nevertheless, there are several important problems, such as
tuberculosis and sexually-transmitted diseases, that are critical to the national interest and have
not received sufficient attention. The best strategy for identifying these problems and for
planning to solve them is likely to come from the active participation of the scientific
community, rather than from administrative directives. There should be an ongoing planning
process, presided over by the NIH Director with members of the intramural and extramural
NIH community, to set goals in these areas and to modify NIH policies. Some of the
intellectual rigidity within NIH can be traced to the peer review system, which is indispensable
but in need of reform. In particular, the system should be periodically reevaluated with the
advice of the scientific community so that the organization of the review groups reflect changes
in scientific practice.

c) Develop strategies for improving the application of fundamental discoveries. ‘The need
for expanded research on technology transfer should be met with strategic planning designed
to encourage such research in the private sector, by economic incentives, alliances with
academia, clarifying the issues of conflict of interest, and other means.

da) Develop a common strategy for the NIH and the NSF that supports fundamental
research. In the face of well-intentioned calls for more applied research, the President and his

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advisor should reiterate their commitment to basic research. New missions of the NIH and
NSF in education and in technology should not be allowed to erode the long term investment
in fundamental] research that has been the source of virtually all the recent advances in
biomedical science and biotechnology.

e) Direct the NIH and NSF to support programs on the basis of scientific rather than
political principles. Narrow political considerations have caused the government to ignore
important areas of biomedical concern in reproductive biology, such as research on
contraception and population biology, Work on diabetes and Parkinson’s disease has been
obstructed by preventing scientists from using fetal tissue. The NIH should be given some
latitude to direct a limited amount of funds into areas that are deemed important by the
intramural and extramural scientific communities for national well being, and the management
of the NIH should be insulated from partisan political debates.

2. To Strengthen the Federal Administration of Biological Science

a) Assure a central role for the President’s science and technology advisor. The President's
science and technology advisor should have strong scientific credentials and should play a major
role in policy decisions about industrial development, health, environment, and energy,
reflecting the importance of science and technology in many aspects of our society. The advisor
should coordinate research programs in all relevant government agencies and should be directly
involved in proposing legislation, budget planning, and designing scientific programs. Since
biology will embrace many of the most significant scientific and technological issues for the 21st
century, the advisor should have current knowledge of recent developments in biology and
medicine, maintain close contact with active scientists, and have outstanding biomedical
scientists among the members of the Office of Science and Technology Planning and the
President’s Council of Advisors in Science and Technology.

b) Establish the NIH as an independent federal agency and reconsolidate its authority.
The role of the NIH is too broad to subsume it under the large Department of Health and
Human Services, Administratively it has suffered from a chain of command that requires
approval from secretaries and undersecretaries with little expertise and interest in science, and
it has been damaged by irrelevant political considerations. Its broad role and its nonpolitical
nature would be improved by its establishment as an independent agency like the NSF and
NASA. All aspects of biomedical research now associated with the NIH should be returned to

the full authority of the Director, reversing the autonomy granted to some sectors (such as the
National] Cancer Institute) by Congressional action.

c) Employ appropriate criteria for choosing scientific administrators. Individuals who
direct governmental programs in biology and medicine, such as the NJH and the NSF, should
be chosen on the basis of their scientific accomplishments, their stature in the research
community, and their ability to administer complex scientific organizations. The choices should
not be based on political allegiances or on the basis of other irrelevant political considerations.

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The tenure of scientific appointments should be tied to professional performance and not to
electoral fortunes.

3. To Improve the Practice and Teaching of Science in Academic Institutions

a) Reform the assignment and use of indirect cost money. A more uniform and
comprehensible indirect cost policy should be implemented that provides incentives to
institutions for cost savings. The policy should insure that funds provided through indirect cost
recovery be used only to support the infrastructure necessary for the scientific work. Reforms

of the indirect cost policies should be instituted gradually to avoid disrupting academic
programs.

b) Develop a program for repairing the scientific infrastructure. The President shouid
propose a program for long term investment in laboratories and equipment. The awards should
be based on merit and need and should be peer reviewed. The President should discourage
individual set asides and scientifically unsound initiatives.

c) Increase federal attention to science education, particularly in K-12. Science education
should become a central issue on the biomedical] research agenda, as it directly affects the future
of biomedical science in the U.S. and the industrial and scientific base on which it is founded.
Since biomedical science is arguably the most exciting area of current science, the one most
accessible to the general population, and an area of clear U.S. superiority, biomedical science
should play an important role in education of teachers and students,

We believe that these recommendations are realistic goals for a new administration.
They will help to control disease, contain healthcare costs, promote technology, stimulate
industrial development, advance agriculture, protect the environment, and contribute to the
education of the American people.

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