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5
Enhancing NIH's Ability to
Respond to New Challenges
The highly decentralized organizational structure of the National Institutes of
Health (NIH) has come about through a complex process of evolution over a long
period marked by substantial increases in resources and extraordinary discoveries
on the biomedical frontier. The evolutionary process involved numerous events and
responses to pressures from a wide variety of interested constituencies that resulted
in the creation of many largely independent organizational units. The governance
of NIH has been profoundly influenced by that evolution. For example, Congress
has created most additional units with their own budgets and decision- making
authorities, which constrains the ability of the NIH director to influence the deci-
sions and choices made by individual institutes and centers and makes the scientific
leadership and management of NIH as a whole extremely challenging.
The Committee's view of those complexities was governed by the desire to be of
practical assistance to all those who wish NIH to continue as an effective, indeed
outstanding, organization, and it proceeded on the premise that its task included
assessing the organizational configuration of NIH and the key processes and
authorities that play roles in NIH-wide decision-making. Although the borders
between structure, mission, and priorities are not well defined, the Committee tried
not to take too expansive a view of its responsibilities.
On the one hand, a highly decentralized organization may be generally appro-
priate for a research organization because research and creativity often prosper
through a bottom-up approach that encourages the flow of ideas from the widely
dispersed scientific community and does not impede the role of individual investiga-
tors in choosing productive avenues of research. On the other hand, when there is
a need for NIH to respond to important new health concerns or scientific opportu-
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84
Enhancing the Vitality of the National Institutes of Health
nities especially when inter-institute or "trans-NIH" initiatives are needed the
NIH director's authority to mobilize the needed resources is limited. There is no
formal mandate for NIH to identify, plan, and implement cross-cutting strategic
initiatives. In fact, the Committee has come to believe that NIH's current structure,
governance, and management mechanisms have become barriers to its effectiveness
in using its resources most efficiently to foster progress in large- and small-scale
scientific endeavors that directly affect human health and that a more diverse set of
mixed strategies for supporting research is essential.
As discussed in previous chapters, most of what NIH does should continue to
operate as usual through activities and decision structures of the institutes and
centers and the peer review system. Indeed, the Committee concluded that the
existing NIH structure is fundamentally healthy and should continue to pay large
dividends in scientific progress and meeting the nation's health needs. However,
organizational changes should be made to increase NIH's effectiveness, improve its
ability to respond to new scientific needs and opportunities, and thereby enhance its
vitality. In this chapter, the Committee focuses on: planning and implementation of
trans-NIH initiatives, which require more authority and resources for the director;
development of a new mechanism to address high-risk research; and improvement
in the NIH intramural research program's ability to move quickly and flexibly to
meet urgent new needs and to work more collaboratively with the extramural
research community.
THE AUTHORITIES OF THE DIRECTOR AND TRANS-NIH INITIATIVES
Despite the enormous success of NIH, and in part because of that success, the
changing world of biomedical science and the stewardship of this great enterprise
require increased attention to a number of critical scientific and health issues that no
institute or center can address alone. In particular, as described in Chapter 3, over
the last decade or more there has been growing recognition of the importance of
both large- and small-scale interdisciplinary science, of the importance of strategic
trans-institute initiatives, and of the increasing dependence of biomedical researchers
on a broad array of new infrastructure investments. NIH has responded to those
forces by, for example, sponsoring and successfully carrying out a number of large-
scale interdisciplinary projects, such as cancer research and the Human Genome
Project. Moreover, it has become increasingly clear that there is a high payoff
potential for carefully selected large- and small-scale strategic projects that require
the participation of numerous organizations working in partnership. Well-planned,
broad-based, trans-NIH programs will be necessary to meet most effectively scien-
tific or public health needs or to complete a task, with the assumption that at some
point particular programs will have met their intent and cease to exist in any formal
way. Although NIH has been successful in putting together some initiatives in which
more than one institute co-funds a research program of mutual interest, it has not
been as successful in jointly planned and implemented efforts across institutes. In
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Enhancing NIH!s Ability to Respond to New Challenges
this respect, the decentralized, federated structure and governance patterns of NIH
are a disadvantage. Furthermore, there is no formal mandate for NIH to identify,
plan, and implement such cross-cutting strategic initiatives.
In particular, the Committee believes that the difficulties encountered in initiat-
~ng trans-NIH initiatives have been one reason why in the past some groups have
called for new free-standing organizational units, which in turn has led to the
proliferation witnessed over the past few decades. What might have been perceived
as a lack of responsiveness on the part of NIH in some cases might have been more
related to its inability to mount a sufficient response within the existing organiza-
tional framework.
The Committee suggests changes in the Office of the Director (OD) to improve
the agency's agility and ability to respond to emerging scientific and health needs.
These alterations would provide new mechanisms for selecting and planning strate-
gic initiatives and would also give NIH an additional set of strategies for managing
science an approach the Committee concludes is not only appropriate, but also
desirable.
The Authorities of the Director
The roles of the NIH director are to provide leadership and direction to the
NIH research enterprise and to coordinate and direct important initiatives that cut
across the agency. The OD is responsible for the development and management of
policy for intramural and extramural research and training, the review of program
quality and effectiveness, the coordination of selected NIH-wide program activities,
and the administration of centralized support activities essential to the operations of
the NIH. The director also oversees relationships between NIH and various other
agencies in and outside the Department of Health and Human Services.
However, the NIH director has limited formal authority and OD lacks an
adequate budget for its many roles. Institute and center (IC) directors have their
own budgets, appropriated directly to them by Congress, which for the larger
institutes, such as the National Cancer Institute (NCI) and the National Institute of
Allergy and Infectious Diseases (NIAID), amount to several billion dollars. The
NIH director has only a modest budget (see Table 5.1 in the section on the structure
of the director's office, below) with a small discretionary fund ($10 million) and the
authority to transfer up to 1% of the IC budgets to start new initiatives. An
unanticipated decision to use that transfer authority during a fiscal year can prove
highly problematic. The ICs, having typically committed their entire budgets, must
cut funding for planned activities to accommodate an unexpected transfer. If a
transfer is called for late in a fiscal year, the disruption to ongoing activities can be
serious. Furthermore, even 1% of the budget might not be adequate for high-
priority new initiatives. The reality is that the NIH director cannot mobilize impor-
tant trans-NIH efforts to address new strategic goals because the authority for
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Enhancing the Vitality of the National Institutes of Health
doing so is absent and he or she must rely largely on persuasion and goodwill to
make even relatively modest changes.
The execution of current Director Zerhouni's "Roadmap" initiatives illustrates
the problem well. Zerhouni has won much praise for his ambitious exercise to plan
major new trans-NIH research projects, but their long-term future is by no means
clear. Zerhouni has given notice that he intends to use the director's 1% transfer
authority in FY 2004, and the President's budget request for FY 2004 contains an
extra $35 million (0.1 percent of the NIH budget) for OD to implement the
Roadmap. But no major new initiative is a 1-year effort, so sources for FY 2005
funding and beyond will be needed. Moreover, the committee believes that there
should be, over time, a series of such initiatives. Ideally, FY 2004 initiatives would
be adopted as part of the relevant ICs' regular research programs in FY 2005 and
beyond, but the director has no authority to ensure that this happens.
Strategic Planning for Trans-NIH Initiatives
Although the Committee is not recommending a major structural reorganiza-
tion of the NIH ICs, it concluded that to meet the scientific and health goals of the
nation, NIH needs to mobilize coordinated funding from many institutes for high-
priority time-limited initiatives that cut across individual institutes' purviews. The
Committee believes that the best means to achieve that is through multiyear strate-
gic planning that involves all ICs.
Scientific mechanisms, risk factors, and social and behavioral influences on
health and disease cut across traditional disease categories. Many patients have
multiple chronic conditions, so a patient-centered approach to health care and
health promotion will sometimes require integration and synergy across ICs. For
example, there have been recent calls for the establishment of an institute on obesity,
which is a major public health concern. Because obesity is associated with diabetes,
coronary artery disease, and arthritis, multiple NIH institutes could logically claim
obesity as a critical component of their research portfolio. This is one of many
potential topics that lend themselves to a strategic coordinated trans-NIH response
in which multiple institutes could collaborate on a research plan that cuts across
administrative structures in terms of planning, funding, and sharing and disseminat-
ing results. The Committee believes that a trans-NIH strategic initiative on obesity
is a better mechanism to address this problem than the creation of a new institute.
Proteomics, already cited by NIH Director Zerhouni as a critical enabling technology
for discovery in the Roadmap, is another current example. Multiple institutes are
independently holding workshops and considering or issuing Requests for Applica-
tions at a time when concerted trans-NIH work on the assessment of existing and
emerging technology platforms and database formats utilizing reference specimens,
could help to advance the whole field and guide NIH-supported studies. A trans-
NIH initiative need not involve every IC and need not proceed indefinitely. But it
would require dedicated funds, leadership, and scientific merit or it will not work.
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Enhancing NIH!s Ability to Respond to New Challenges
NIH shared with the Committee evidence that the ICs are co-funding grants
that account for about 20% of new awards, although the research topics of these
awards have not been selected through NIH-wide strategic planning. It appeared to
the Committee that, in many cases, these initiatives really involved only a few lead
institutes that contributed the lion's share of the budgets. NIH managers told the
Committee that these multi-institute programs are difficult to administer: they
require sign-off by each institute involved, with each institute maintaining its own
accounts and oversight. Thus, if five institutes are involved, there are five parallel
administrative and oversight efforts in place.
Other efforts to improve cooperation and collaboration among institutes have
met with limited success. For example, NIH intramural scientists have formed some
70 scientific interest groups across institutional boundaries. These groups are no
doubt important forums for scientific exchange but they do not set priorities, plan
programs, or expend research funds. A relatively new and path-breaking attempt at
trans-NIH science is the consolidation of the intramural programs of the
neuroscience-related institutes in the newly constructed Porter Center on the
Bethesda campus. Other cooperative attempts, such as the NIH Pain Research
Consortium although well intended have started and faltered over many years
because funding generally has not been available and research programs are depen-
dent on the willingness of individual institutes to fund specific projects (IOM,
2003b). The Committee was told in numerous interviews with NIH leadership that
past efforts by the NIH director to "raise funds" from ICs to support trans-NIH
initiatives have been viewed by the ICs as intrusions on their budgets. This is a
direct consequence of the federalist structure of NIH and one this Committee would
like to see reformed.
The Committee expects that many IC directors would see the expansion of such
collaborations through planning and disbursement of research and training funds as
an opportunity for leadership and leverage on topics important to them and their
constituencies. To reiterate, the Committee is convinced that trans-NIH initiatives
are a more direct and effective means to address emerging scientific and health
improvement opportunities than is the creation of new centers or institutes.
The Committee concluded that the NIH director's authorities and resources
must be increased to make it possible to achieve those goals. The Committee
recommends that the director be given the responsibility and authority to develop
and implement, with and through the ICs, a set of time-limited trans-NIH initiatives
that are identified through a broad-based strategic planning process open to partici-
pation by all internal and external stakeholders and transparent to the public. Such
a process should be conducted regularly, for example, every other year. The Com-
mittee envisions the process producing a sufficient breadth and diversity of initia-
tives to make it readily feasible for each institute and center, with the director, to
identify one or more initiatives that are compatible with its own mission and goals
in which to participate. In fact, the Committee is convinced that such a requirement
from Congress is likely to stimulate ICs to propose and even lead trans-NIH initia-
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Enhancing the Vitality of the National Institutes of Health
fives. In any case, each institute and center should be required to reserve a substan-
tial portion of its budget for such participation, starting initially at a few percent,
but increasing over the next 4-5 years to 10% or more if initial efforts prove
successful. The Committee believes that the initiatives will, over time, allow each of
the institutes and centers to pursue its goals and interests more effectively. The
Committee envisions the strategic initiatives selected through the planning process
being temporary in the sense that their status as "new initiatives" will extend only
through one or a few planning cycles, after which other initiatives will take their
place. However, as the work involved in these initiatives is performed, the Commit-
tee expects that at least some elements of the work will spin off into new compo-
nents in the portfolios of many of the ICs that become part of their regular research
agendas. In addition, many activities covered by existing grants and programs are
likely to be relevant to some strategic initiative topics, and could become part of IC
participation in the trans-NIH initiatives if the NIH director's review confirms their
appropriateness for inclusion. That is, an institute or center could include aspects of
existing programs in its trans-NIH obligation with confirmation from the director
that they are relevant and should be counted as part of the IC's participation.
The Committee identified several options for organizing and managing a trans-
NIH budgeting process:
Sufficient funds (for example, 5°/O of the NIH budget would be about $1.5
billion) could be appropriated to OD for the NIH director to make alloca-
tions to the participating ICs through the planning process.
The target proportion of funds appropriated to each institute or center could
be treated as though "in escrow" until the NIH director affirms that the unit
has committed its expenditure for one or more of the identified trans-NIH
. . . . , .
~n~t~at~ves ot re" evance to it.
The use of the target proportion within each IC budget could be left to the
IC and its director, with retrospective review by the NIH director and Con-
gress. The annual performance review of the IC director would include
attention to this element.
In the Committee's view, the second, or "escrow," option is preferred. The
NIH director should have the authority to require the necessary funding commit-
ments from the ICs for their participation in the initiatives chosen, but the committed
funding should not be transferred either to the NIH director or to another IC.
Rather it should be set aside to represent each unit's participation in furthering the
chosen research initiatives. The initiatives should be carried out extramurally
through multi-unit grant or contract programs, or as a combination of multi-unit
extramural and participating unit intramural efforts.
The implementation of each of the initiatives should be overseen by special
temporary task forces formed for this purpose with representation from each of the
participating ICs. The commitment of the ICs should be reflected in the assignment
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Enhancing NIH!s Ability to Respond to New Challenges
of excellent staff to trans-NIH task forces on a full-time basis. As appropriate, NIH
should also periodically sponsor scientific symposia to inform the relevant NIH
constituencies and the director of progress on each trans-NIH strategic initiative.
Such a process would give NIH a capacity to respond to newly identified health
needs in a coherent organization-wide manner. Together, the initiatives would have
the effect of greatly expanding trans-NIH research and cooperation and breaking
down barriers among IC research agendas. It might also make the NIH research
enterprise more efficient and less apt to duplicate effort. Although OD would lead
the process, its consensus-driven nature would incorporate the views of NIH's many
internal and external constituencies and provide the potential to increase under-
standing and satisfaction of the external scientific and health advocacy communities.
Recommendation 4: Enhance and Increase Trans-NIH Strategic Planning and
Funding
a. The director of NIH should be formally charged by Congress to lead a trans-
NIH planning process to identify major cross-cutting issues and their associated
research and training opportunities and to generate a small number of major
multi-year, but time limited, research programs. The process should be con-
ducted periodically perhaps every 2 years and should involve substantial
input from the scientific community and the public.
b. The director of NIH should present the scientific rationale for trans-NIH
budgeting to the relevant committees of Congress, including a proposed target
for investment in trans-NIH initiatives across all institutes. For example, an
average target of 5% of overall NIH funding in the first year, growing to 10%
or more over 4-5 years, may be appropriate.
c. The appropriations committees should annually review budget justifications
and testimony from the NIH director and from individual IC directors about
the participation of each unit in the planned trans-NIH initiatives and the
portion of their budgets so directed. Congress should include budget targets in
the appropriations report language. The Committee recommends beginning
with 5 % of the overall NIH budget.
d. To ensure that each IC uses the target proportion of its budget for trans-NIH
initiatives of its choosing, that proportion of the annual appropriation to each
unit should be treated as "in escrow" until the NIH director affirms that the
unit has committed to its expenditure for the identified trans-NIH initiatives.
e. The President should include in the budget request, and Congress should
include in the NIH appropriation for OD, funds to support an appropriate
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To
Enhancing the Vitality of the National Institutes of Health
number of additional full-time staff to conduct the trans-NIH planning process
and "jump-start" the initiatives that emerge from this process.
Once again, the Committee believes that IC directors should view such planning
as an opportunity for leadership and leverage on topics important to them and their
constituencies and as a means for adapting their missions to new developments.
Advocacy organizations, scientific societies, and NIH advisory bodies, including the
Council of Public Representatives, likewise should see this process as an opportu-
nity to gain synergies across the many interrelationships among diseases. If they do,
the commitment to the trans-NIH task force should be reflected by the assignment
of staff on a full-time basis, a career assignment viewed as a plum. The structure to
accomplish the trans-NIH initiatives identified in the strategic process could take
several forms depending on the size of the initiative, the number of institutes that
need to be involved, and the likely time it will take to see the initiative to fruition.
The Committee recognizes that the prospects for putting new and significant
trans-NIH objectives into practice will be affected by the growth of the NIH budget.
If all existing programs continue to enjoy the highest priority there will likely be
resistance in the early years of the initiative by institutes that claim difficulty in
meeting their commitments while still offering some new grants. As a result the NIH
director will have to exert superb and compelling leadership to withstand requests
to release "escrowed" funds from trans-NIH projects. For these reasons, it is
particularly critical that IC leadership comes to view participation in these initia-
tives as beneficial, and that Congress ask IC directors to report each year on the
extent to which they are participating.
THE STRUCTURE OF THE OFFICE OF THE DIRECTOR
More than 40 unit heads report to the director the directors of 27 ICs, the
heads of 4 program offices and the heads of 12 staff offices in OD. Although the FY
2002 budget of $239 million for the OD may seem ample, the vast majority of this
funding was earmarked for the support of a group of program offices and special
programs, and that has been the case since 1993. (See Table 5.1.) The composition
of the earmarked amount has changed regularly, however, as OD has been used as
an incubator for offices and programs that were established and then spun off as
centers or institutes or absorbed into existing institutes. For example, the Office of
Alternative Medicine became the National Center for Complementary and Alterna-
tive Medicine in 1998, the Office of Research on Minority Health became the
National Center on Minority Health and Health Disparities in 2000, and the Office
of Bioengineering and Bioimaging became the core of the new imaging and bioengi-
neering institute in 2000.
To carry out the responsibilities of managing, planning, and coordinating the
programs of the 27 ICs, the NIH director is assisted by a number of staff units
collectively called OD Operations. A series of staff offices are headed by associate
OCR for page 91
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92
Enhancing the Vitality of the National Institutes of Health
directors. They include the Office of Science Policy, the Office of Budget, the Office
of Communications and Public Liaison, the Office of Legislative Policy and Analysis,
several components of the Office of Management Financial Management, Human
Resource Management, and Research Services and several other units.
The FY 2002 budget for OD Operations was less than $80 million. Although
the OD Operations offices assist the director in managing NIH, they are small and
their budgets have not grown in proportion to NIH's research funding. The OD
Operations budget increased by 88% from 1993 to 2002 compared with 125% for
all of NIH. It amounts to 0.3% of the total NIH budget, down from 0.4°/O in 1993.
Because of the tight budget for OD Operations, when unforeseen needs surface, as
has happened recently with the development of stem cell research policies and
harmonizing the rules for human subjects protection, OD is likely to have to "pass
the hat" to the ICs to gather the additional resources needed. The Committee
believes that the director should be given either a more adequate budget to support
OD's management roles or greater discretionary authority to reprogram funding
from earmarked components of the OD budget when necessary to meet emerging
needs. Funding for OD Operations has not kept pace as NIH has expanded and has
not grown in proportion to NIH's research budget; it is the Committee's view that
it is inadequate for the effective management of the organization.
Recommendation 5: Strengthen the Office of the NIH Director
The Office of the Director should be given a more adequate budget to support
its management roles or greater discretionary authority to reprogram funding
from the earmarked components of its budget when necessary to meet unantici-
pated needs. In particular, if the director is given the responsibility and authority
to conduct NIH-wide planning for trans-NIH initiatives, the director's budget
will need to be amplified to take the costs of such planning into account.
In addition, the earmarking of funds by Congress for the establishment and
continuation of programmatic offices in OD sometimes limits the director's
flexibility and fluidity of resources, as well as his or her ability to effect change
across the organization. It is difficult to ascertain whether the programmatic offices
within OD have achieved their intended goals. Certainly, offices that move up and
out to become centers or institutes reach the level of prominence desired by their
advocates. But when the creation of an office in OD does not accomplish what the
advocacy community desires, it increases the pressure for elevation of that office to
a higher-level unit. The Committee believes that the process recommended in
Chapter 4 for evaluating the merits of proposed additions to or subtractions from
the list of ICs should also be applied to the creation of new offices in OD itself. The
Committee is concerned that the creation of programmatic offices in OD could
defeat the purpose of efforts to draw greater attention to important cross-cutting
concerns because the creation of an issue-oriented office in OD tends to shift the
responsibility for that issue to OD and away from the ICs, thereby reducing the
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Enhancing NIH,s Ability to Respond to New Challenges
attention that it might deserve. The time may be right to assess the effect that the
programmatic offices in OD have had, including their role in the NIH director's
policy and planning processes, whether the programs have clear goals, and whether
there is a need to "sunset" an office once it achieves its goals.
Recommendation 6: Establish ~ Process for Creating New OD Offices and
Programs
The public process recommended in Chapter 4 (Recommendation 2) for evalu-
ating a proposal to create a new institute or center or to consolidate or dissolve
institutes or centers should also be used for a proposal to create, consolidate, or
dissolve offices in OD. The process should be used to evaluate the scientific
needs, opportunities, and consequences of the proposed change, the likelihood
of resources being available to support it, and public support for it.
FOSTERING HIGH-RISK, HIGH POTENTIAL PAYOFF RESEARCH
To increase investment in high-risk, high potential payoff research, the Com-
mittee also believes that there is a need for a "Director's Special Projects Program"
external to the budgets of the ICs and funded as an OD line item. The goal of the
program would be to fund the initiation of high-risk, innovative research projects.
In a broad sense, the Committee imagines the program to be patterned after the
Defense Advanced Research Projects Agency (DARPA), but with important
differences.
The current peer-review mechanism for extramural investigator-initiated
projects has served biomedical science well for many decades and will continue to
serve the interests of science and health in the decades to come. NIH is justifiably
proud of the peer review mechanism it has put in place and improved over the years,
which allows detailed independent consideration of proposal quality and provides
accountability for the use of funds. However, any system that focuses on account-
ability and high success rates in research outcomes may also be open to criticism for
discriminating against novel, high-risk proposals that are not backed up with exten-
sive preliminary data and whose outcomes are highly uncertain. The problem is
that high-risk proposals, which may have the potential to produce quantum leaps in
discovery, do not fare well in a review system that is driven toward conservatism by
a desire to maximize results in the face of limited funding resources, large numbers
of competing investigators, and considerations of accountability and equity. In
addition, conservatism inevitably places a premium on investing in scientists who
are known; thus there can be a bias against young investigators. The current steep
decline in the growth rate of the NIH budget proposed in the President's FY 2004
budget may make it even less likely that high-risk proposals will be funded.
The DARPA approach specifically seeks high-risk research and expects fail-
ures a marked difference from the NIH study sections or the consensus approach
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94
Enhancing the Vitality of the National Institutes of Health
of committees. DARPA's mission is to develop imaginative and innovative ideas
that have the potential for important defense-related technological impact. Such an
impact is, however, by no means guaranteed. DARPA was developed specifically to
foster research focused on high-risk, high potential payoff technology development.
Typically, DARPA research establishes feasibility, and the results are handed off to
other branches of the military services for development. The process has been
successful: DARPA can claim credit for the foundational research that led to many
noted and highly recognizable accomplishments, such as the Saturn rocket (1960s);
the M-16 rifle (1970s); the Stealth fighter, global positioning system, and Arpanet/
internet (1980s); the Predator unmanned aircraft (199Os); and the Global Hawk
aircraft (2000s). Results of DARPA projects were also influential in the develop-
ment of the National Science Foundation's (NSF's) nanotechnology and computer
sciences programs (Betz, personal communication). It must be noted that much of
the research funded by DARPA results in failure, which is the expected price of the
quest for unusual breakthroughs.
Cook-Deegan (1996) provided examples of how real situations in the past
might have been helped by the presence of a DARPA-like entity at NIH. In 1981,
both NIH and NSF turned down a request from Leroy Hood and colleagues at
Caltech for funding to automate DNA sequencing. The Caltech researchers subse-
quently obtained funding from the Weingart Institute instead, and by 1984 had
made sufficient progress in prototype development to win NSF funding. Their
method eventually became the dominant one on the market. In 1989, the National
Center for Human Genome Research held a peer reviewed competition for large-
scale DNA sequencing. It took about a year to develop and announce the competi-
tion and another year to review proposals and make funding decisions, but two
years is a long time in a fast moving field. Ultimately the process rejected proposals
from T. Craig Venter and Leroy Hood to do automated sequencing and selected a
technology that was already a decade old. Hood's and Venter's subsequent suc-
cesses in speeding up various sequencing efforts are well documented.
Cook-Deegan notes that many people assume that DARPA's approach is only
suitable for engineering and technology development, but not pure science. "Expe-
rience suggests otherwise, however. Packet switching for electronic communica-
tion, computer time-sharing, integrated large-scale chip design, and networking
were as conceptually 'basic' when DARPA was funding them as most molecular
biology experiments are today." It is not difficult to identify research areas in
today's biomedical science that might benefit from such an approach, for example,
optics in neuroscience. Miller (2003) reported that in vitro studies of cultured
neurons and brain tissue have built-in limitations for understanding how learning
takes place in the brain. The "wish list" of neuroscientists includes finding a way to
visualize individual neurons and track minute changes in the cells' structure and
electrical activity; using two-photon microscopy to peer about half a millimeter into
the brain to visualize the cortex and see into the unanesthetized brain; and finding a
means to visualize deeper structures, such as the hippocampus. Fulfilling this wish
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list could bring about an optical revolution in neuroscience, but many of the needed
techniques remain far off.
The Committee is aware that a number of alternative pathways might be used
to establish a greater capability to support high risk research at NIH. NSF, for
example, maintains a program of Small Grants for Exploratory Research (SGER)
and allows its program officers to fund a limited number of small-scale, explor-
atory, and high-risk research projects at their own discretion subject only to internal
NSF merit review. Such projects focus on preliminary work on untested and novel
ideas, the application of new expertise or new approaches to "established" research
topics, and work having extreme urgency with regard to availability of or access to
data, facilities, or specialized equipment, including quick-response research on natu-
ral disasters and similar unanticipated events (NSF, 2002b). The SGERs are limited
to $100,000. As Cook-Deegan (1996) points out, this is a good idea, but there is no
reason to think that innovative projects will always be small. The Committee
believes that a mechanism to promote high-risk research at NIH must allow for
larger scale efforts to be effective. Another approach might be to experiment with
the idea of a DARPA-like program with a pilot in only one or a few ICs. The
Committee believes, however, that such an approach is likely to have limited success
for two reasons. First, the establishment of such a program inside one or a few ICs
is bound to limit its scope to the topical areas already in the ICs' portfolios, which
could partly defeat its purpose. Second, locating such a program inside one or a few
ICs would make it overly subject to their prevailing culture, which is already biased
against high-risk research. (It should be noted that DARPA was created to report to
a high-level Department of Defense official outside the research organizations of the
military services to protect it from the hostility of those services, which sought to
eliminate it. Augustine, personal communication, 2002.) The Committee believes
that the proposed Director's Special Projects Program would have its best chance
for success if it were located in OD and had a leader who reports to the NIH
director.
The proposed Director's Special Projects Program at NIH would, like DARPA,
be designed to foster the conduct of innovative, high-risk research. Research ini-
tially funded through the program that generates useful results would be handed off
after 3-5 years for further development and funding through the standard NIH peer-
review mechanisms of the ICs. If positive results were not generated after a reason-
able period of time, as is anticipated for much of this type of research, the projects
would be terminated. The Committee expects that there would be clear missions
and finite life spans for these projects and that multidisciplinary teams of investiga-
tors would perform most of them.
The heart and soul of DARPA are its program managers, the scientists and
engineers who initiate and oversee the research programs. They are responsible for
developing program ideas and choosing contractors to perform the research, usually
at universities or in industry. (DARPA has no intramural research program.) The
program managers are not permitted to spend more than 4 years at the agency.
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During their tenure, they have much autonomy in initiating programs and in choos-
ing the investigators to be funded. DARPA reports to the Department of Defense's
director for Defense Research and Engineering and operates in coordination with
but independently of the military research and development establishment.
A cadre of talented program managers to select and manage the projects under
the NIH Program could be drawn from academia, industry, and the ranks of NIH
intramural scientists. Their most important feature would not be their previous
affiliations, but rather that they are "idea people," capable of developing or recog-
nizing unusual concepts and approaches to scientific problems. As at DARPA, the
program managers would be appointed to strictly limited terms (such as 2-4 years)
that are not renewable. The limitation on terms ensures that the programs are
continually infused with fresh ideas and talent, which is thought to be a key reason
that DARPA has been successful. The Committee believes that the NIH program
managers should be able to accept ideas either through unsolicited proposals or
more directed responses to requests for applications or through peer review when
appropriate from the extramural and intramural scientific communities, as well as
drawing on their own ideas. In addition, to allow for appropriate peer review,
review panels specifically charged with selection of high-risk, high potential return
projects could be constituted outside the standard peer review mechanisms to assist
the program managers in selecting projects for funding.
The Committee believes that such a program will have its best chance to suc-
ceed if Congress provides new funding. The Committee suggests that a budget of
$100 million for FY 2005 would be appropriate to initiate the program with a full-
time program director and four to six program managers. Because it is likely that it
will take 8-10 years for the program to reach full maturity, a commitment to keep it
going at least this long should be made. The Committee envisions the program's
budget increasing over the 8-10 years to as high as $1 billion per year.
Recommendation 7: Create ~ Directors Special Projects Program
A discrete program, the Director's Special Projects Program, shouic! be estab-
lishec! in OD to fund the initiation of high-risk, exceptionally innovative research
projects offering high potential payoff. The program shouic! have its own
leacler, who reports to the director of NIH, and a staff of short-term (2-4 years)
program managers to manage iclentifiec! projects with acivice on program con-
tent from extramural panels. The program shouic! be structured to permit rapid
· · · ~ · ~ · · · ·' · · ~
review ant ~n~t~at~on ot promising projects; ~t peer review Is c eemec appropr~-
ate, the program shouic! use peer review panels created specifically for it ant!
charged with selecting high-risk, high potential return projects. Congress should
be prepared to provide new funding in the amount of $100 million, growing to
as much as $1 billion per year for this endeavor, and commit to support it for at
least S-10 years so that a sufficient number of projects can reach fruition and a
full assessment of program efforts can be made. A program review should be
conducted during the fifth year to provide mid-course guidance.
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Consistent with Recommendation 5 on sufficient funding for OD, this recom-
mendation requires that the NIH director have the resources to hire first-rate scien-
tists to help manage these increased responsibilities for developing programs.
THE INTRAMURAL RESEARCH PROGRAM
The performance of the NIH intramural research program (IRP) has been evalu-
ated several times in the last 25 years by advisory groups in response to administra-
tive and legislative mandates. The evaluations included a review of NIH by the
President's Biomedical Research Pane! (Department of Health, Education, and Wel-
fare, 1976), an Institute of Medicine report (IOM, 1988), a report by the Task Force
on the Intramural Research Program of the National Institutes of Health (NIH,
1992a), and the report of the External Advisory Committee on the Intramural
Research Program (NIH, 19941. That might seem to be an excess of scrutiny. But
one might equally wonder whether the repeated calls for review reflect a continuing
concern about the quality of programs and performance and a lack of response to
criticism and recommendations. The IRP has faced persistent difficulties, including
problems with recruitment and retention of senior scientists, expansion of a
postdoctoral training program of uncertain and uneven quality, cumbersome
administrative requirements, inadequately funded congressional and administrative
mandates, and deteriorating facilities, in particular in the Clinical Center.
Like the extramural program, the IRP has a fragmented federated structure. The
IRP, with its $2.5 billion annual budget, comprises 19 separate intramural pro-
grams associated with the individual ICs.
lust as each institute has a different
legislative history and mandate from Congress, their IRPs vary widely in goals,
scope, and size. Prior reviews have found this administrative structure to hinder
unified or effective management of the IRP by the OD and to contribute to uneven-
ness in quality, quality control, and productivity across NIH.
The IRP's proportion of the total budget has been reduced to only about 9 or
10% of the total NIH budget today and the IRP's budget growth has in recent years
been deliberately slowed. Despite those reductions in the program, the question of
what makes the IRP unique still recurs. In the past, the justification for the program
was that it has distinctive input characteristics, including relatively long-term and
stable funding of research programs, the availability of the Clinical Center's patient
investigational facilities, few or no distractions from research for scientists, and a
primarily retrospective, rather than prospective, review process for maintaining
. . ,. .
scenic qua" sty.
For many years, the NIH campus was an exceptional training ground, espe-
cially for clinical investigators. Indeed, a large fraction of the senior leadership of
the extramural biomedical research community received its training in the NIH IRP
in the 1960s and 1970s. But the rapid growth in the NIH extramural program
enabled biomedical research across the country to expand in size and scope, provid-
ing superb opportunities for training at academic facilities elsewhere.
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The most recent of the IRP evaluations, by the External Advisory Committee
(EAC) of the Director's Advisory Committee, also known as the Marks-Cassell
committee, originated because of concerns expressed by Congress and others regard-
ing the quality, appropriateness, size, and cost of the NIH IRP. In its many recom-
mendations to the NIH director, the EAC concluded that the problems plaguing the
IRP, unless addressed, "may destine it to a mediocre future." The committee identi-
fied many areas of concern:
The review process for tenured scientists and scientific directors,
The review process for appointment to tenure,
Postdoctoral training,
Administrative issues affecting recruitment and retention,
NIH-private sector collaborations,
The process for allocating funds between the extramural and intramural
programs, and
Renewal of the Clinical Center.
The EAC recommended that each institute be subjected to an individual review
along lines proposed by the EAC.
In response to the EAC report, NIH prepared and implemented a plan to
address the review process for tenured scientists, a tenure-track program, and
changes in postdoctoral recruitment and training. In addition, progress has been
made in removing some of the administrative impediments to research and in
enhancing the attractiveness of employment in the IRP through changes in the pay
scale and retirement options for senior investigators. Some ICs implemented the IC-
leve! reviews recommended by the EAC.
The present Committee, given the time and resources available for it to com-
plete its task, did not attempt to evaluate the quality of the IRP systematically. The
Committee is, however, persuaded that the significant efforts of recent years to
reinvigorate the IRP and respond to various advisory committee recommendations
have met with considerable success and that there has been a promising trend
toward improved overall quality in the IRP. The Committee applauds the efforts of
the NIH deputy director for intramural research to improve the program overall.
Nevertheless, the balkanization of the IRP persists because of its multiple institu-
tional budgetary and programmatic lines, which reinforce the "stove-piping" and
continue to make it difficult for the senior management of NIH to ensure that the
IRP supports NIH's overall strategies and plans. The Committee therefore suggests
that it would be useful to consider mechanisms to foster interactions among the
IRPs of the individual ICs, such as large-scale reassignments of space to bring
similar programs in individual institutes together to create synergies. It might also
be useful to explore reducing the balkanization of the IRPs by clustering programs
that share common themes, approaches, and tools, similar to the approach currently
being taken to integrate the neurosciences in one building.
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The Committee is convinced that the IRP should not merely be an internal
extension of the extramural community, but rather should be doing distinctive
research that the extramural research community cannot, or will not, undertake.
The Marks-Cassell committee stated that "quality not necessarily uniqueness,
should be of the highest priority in determining support for the intramural research
program." The present Committee does not fully agree with that statement, espe-
cially with its implementation, which typically has ignored uniqueness. Too little
weight has been placed on the need for distinctive contributions by the IRP. Unique-
ness and quality should both be essential justifications of the IRP, and it is not clear
what distinguishes many of the current activities of the IRP from programs con-
ducted by the extramural community.
Although evaluation of the quality of the clinical research protocols conducted
in the Clinical Center was beyond the scope of the Marks-Cassell committee, that
committee did ask the IC directors to characterize and prioritize their clinical proto-
cols to assess their quality. The criteria used for the assessment included alignment
with the NIH and Clinical Center missions, the extent to which the protocol repre-
sented cutting-edge science, whether the Clinical Center environment was uniquely
appropriate for the study, whether the protocol addressed a national public health
emergency, the importance of the protocol for training, whether the protocol was
crucial to the institute's research program, whether the protocol was likely to con-
tribute to patient care or patient comfort, and whether the protocol attempted to
improve the efficiency or cost effectiveness of patient care. Some of the findings of
the assessment such as that only half of the protocols of NCI's Division of Cancer
Therapy, the largest user of the Clinical Center, received excellent or good
rankings led to the identification of programs that were candidates for being
phased out.
The present Committee believes that a similar process could be devised for the
IRP as a whole to identify programs that represent neither excellent science nor
science that is appropriately distinctive for the IRP. They are likely to constitute
only a small fraction of the IRP's programs. The identified programs should be
considered for phasing out, and the funding associated with them considered for
diversion to other high-priority uses, such as trans-NIH projects selected under the
proposed NIH strategic planning effort. Opportunities for intramural-extramural
collaboration, particularly for clinical research (see Chapter 4) and for research that
is capital intensive and requires substantial investments in costly or specialized
equipment should also be explored. Such collaborations would improve the IRP's
ability to make distinctive contributions to research and NIH should find mecha-
nisms for facilitating and managing them.
The Committee supports the principle that the science conducted by the IRP
should be subject to standards of quality similar to those of the extramural pro-
gram. As noted earlier, the peer review process used to evaluate most extramural
research proposals commands widespread respect for its rigorous standards for
maintaining research quality. At least some ICs are using comparable peer review
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Enhancing the Vitality of the National Institutes of Health
for their IRPs. But the peer review process also has a tendency to enforce conserva-
tism by discriminating against research whose outcome is highly uncertain. To
evaluate research at the "cutting edge" fairly requires a culture, mindset, and process
that views informative failures as the necessary price of strategic innovation. Inves-
tigators who conduct projects based on promising but unproven ideas that fail for
reasons that could not be foreseen must receive credit for their work. Indeed, the
special status of the IRP obligates it to take risks that might not be taken in the
extramural program. Such considerations may require novel mechanisms for review,
whose adoption could facilitate efforts to distinguish the IRP's role from what can
be performed under the current extramural program. It should be reiterated that
the Director's Special Projects Program proposed above should be open to ideas
from IRP scientists.
The Committee agrees that another important aspect of the IRP is that it is
capable of moving quickly and flexibly to meet urgent new needs. There is a lag of
about a year while scientists outside NIH apply for and obtain funds to address new
topics, but scientists in the IRP can shift focus very quickly simply by electing to do
something different. In the middle 1980s, the IRP mounted a major AIDS research
program a year before it was possible to award external grants. The importance of
that history has again been well illustrated recently as NIAID redirected the efforts
of many of its researchers to respond quickly to the threat of bioterrorism and the
need for new vaccines and countermeasures; they are also a logical leader in
addressing the latest viral epidemic, SARS. NIH's Vaccine Research Center is another
example of the IRP filling an important scientific need, for example, by designing a
good manufacturing process pilot plant to develop and manufacture large amounts
of HIV vaccine candidates for Phase I through Phase III trials. Another example is
the high throughput screening program provided by NCI for cancer drug develop-
ment studies, which is used extensively by academic and industrial laboratories.
Finally, the Committee heard repeatedly that there are historic and cultural
factors that have stymied intramural-extramural research collaboration in general.
Although there are some notable exceptions, these appear to be more through
default than by design. NIH would benefit by promoting the exchange of personnel,
space, and resources between the intramural and extramural communities, as appro-
priate, and as dictated by scientific or health needs.
Recommendation 8: Promote Innovation and Risk-Taking in Intramural Research
The intramural research program should consist of research and training pro-
grams that complement and are distinguished from those in the extramural
community and the private sector. The intramural program's special status
obligates it to take risks and be innovative. Regular in-depth review of each
component of the intramural program should occur to ensure continuing
excellence. Allocation of resources to the intramural program should be closely
tied to accomplishments and opportunities. Inter-institute and intramural-
extramural collaborations should be supported and enhanced.
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SUMMARY
Although the Committee is not recommending major changes in the number or
structure of NIH's institutes and centers, it concludes that the organization needs to
be and can be transformed in other ways to meet its and the nation's scientific and
health goals. Most important, the Committee concludes that it is time to begin to
redirect, over the next 4-5 years, a small but significant fraction of the NIH budget
to a series of strategic trans-NIH initiatives that will be carried out by both the
intramural and extramural programs under the auspices of the individual institutes
and centers working in partnership. Redirected funds will in many cases pro-
foundly influence the core missions of the ICs. This will require the formalization of
a careful, open, and consensus-driven planning process under the direction of the
NIH director that should be used to select strategic initiatives, assign responsibilities
for them, and elicit commitments of funds from participating units. The Committee
commends the current NIH director for undertaking what has been referred to as
the Roadmap effort. Congress should formalize the process by charging the director
to lead a regular trans-NIH planning process to identify major crosscutting issues
and opportunities and to generate a small number of high-priority research initia-
tives. The process should be periodic perhaps once every 2 years and should
involve substantial input from the scientific community and the public.
The Committee finds that funding for the operations offices of the NIH director
has not kept pace as NIH has expanded and has not grown in proportion to NIH's
research budget. OD Operations funding is inadequate for the effective manage-
ment of the organization and should be increased. The Office of the Director does
not have the resources to respond to unexpected needs of NIH as a whole without
appealing for support from the ICs. Programmatic offices in OD that were created
with specific functions should be assessed for successes and failures and whether
these entities should be perpetuated indefinitely. The public process for evaluating
proposals to create organizational units described in Chapter 4 should also be
applied to programmatic offices in the OD.
Finally, to enhance the quality and innovative nature of NIH's portfolio, the
Committee proposes a variety of adjustments in intramural research and the cre-
ation of a new program in OD to promote high-risk, high-payoff research.
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Summary
NIH is increasingly called on to undertake research that involves multiple institutes,
multiple disciplines, and complex diseases to be responsive to new challenges, such as public
health emergencies ant! the threat of acts of bioteorrism. A key question posed to the Committee
was whether NIH's decentralizes! structure has become too fragmented! to respond aclequately to
those challenges or whether, on the contrary, it is well suited to respond to changes in
opportunity and need. Related questions included whether, to help equip NIH for the future, the
director's authorities shouIc! be increaser! and in what way or whether managerial mechanisms
should be strengthened or new ones adopted in place of or in conjunction with structural
reorganization.
The Committee's view of those complexities was governed by the desire to be of some
practical assistance to all who wish NIH to continue to be an effective - indeed, outstanding
organization. Thus, the Committee proceeded on the premise that its task included assessing the
organizational configuration of NTH ant! the key processes and authorities that play roles in
trans-NTH decision-making. Although the borders between structure, mission, and priorities are
themselves not well defined, the Committee tried not to take too expansive a view of its
responsibilities. It concluded on the one hand that in many ways NTH is performing
exceptionally well, using decentralization as a strength. On the other hand, it made multiple
recommendations to enhance NTH's vitality and accountability through change, augmentation of
existing structures, modifications of policies and practices, and measures that aim to transcend
clecentralization.
Whether needs and opportunities will be accommodated in existing NTH units or
proliferation or consoliciation will occur in the near future is an issue to be addressed by
administrations, Congress, the scientific community, ant! the public. NIH will continue to be
shaped by the dynamics of many constituencies interacting. Interests will converge or conflict,
depending on the issue. The degree of convergence and divergence will continue to be
influenced by other factors such as annual appropriations. The recommendations made in this
report are intended! to help NIH to continue to be responsive, accountable, ant! effective in its
leading role in the vast international humanitarian enterprise aimed! at a better understanding of
the human condition, the prevention and relief of the burdens of disease, and at the promotion of
good health throughout the stages of life.
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Representative terms from entire chapter:
intramural research
