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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- 83

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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 85

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86 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- 87

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88 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 89

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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

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so so 1 1 of o - en - s~ son o - no an en o - ~ o o no ~ o pa ~ ~ ~ - L ~ _ O o (L) Cal O O O O O O ~ ~ ~ O O O ~ ~ ~ ~ ~ ~) ~ O ~ ~ O ' ~ ~ 00 00 ~ ~ A) ~ no ~ ~ Cal en O O ~ ~ ~ ~ 00 ~ ~ 0 00 ~ 00 ~ ~ ~ ~ 00 ~ O ~ no ~) ~) ~) O ~ ,'` ~ ~ ~ 1 ~ ~ _ 1 ~ ~ ~ ~ O ~ ha ~ 'in O ~ ~ ~ 00 ~ ~ 00 00 ~ ~ ~ ~ ~ ~ ~ ~ ~ ~ O oo ~ ~ ~ ~ O oo ~^ ~ ~^ - ^ ~ - ^ ~ ~ ~ O^ O ~ O ~ O ~ ~ ~ ~ ~ ~ O ~ O ~ ~) O O ~ ~ ~ ~) ~ ~) ~ ~ ~ O O O ~ ~ O~ ~^ W o^ O~ O~ m~ m^ O O ~ ~ ~ ~ ~ ~ O O ~ ~ ~ ~ ~) ~ ~ ~ ~ ~ O O^ ~)^ ~^ m~ O^ '^ m^ m~ m~ ~ - ^ O ~ ~ O O O ~ ~ ~ ~ O O ~ ~ ~) ~ O ~ ~, ~ ~ ~ O ~ ~ ~^ ~ ~ ~ ~ O O m~ O ~ ~^~ '~ ~ ~^ ~^^ '~^ O ~^ ~) ~, O ~ ~ O ~ ~ ~ ~ ~ ~ ~ ~ O O ~ ~ ~ ~ ~ ~ ~) ~ ~ ~ ~ O ^ r~~ ~^^ C%] ~ ^ ,~^ ~ ~ ~ ~ o ~^ O O ~ ~ ~ ~ ~ ~ ~ ~ ~ O ~ ~ ~ ~ ~ ~ =\ ~ ~ ~ - ^ O ^~ - ~ - ~m ~^ - ~^ ~, ~ ~ ~ o ~ ~ ~ ~ ~ o ~ ~ ~ ~ o ~ ~ ~ ~ ~ ) ~' ~ ~' =\ ^ c~ ^ ^ ~ ~ ^ ^ ~ ~ ~ ~ ~ ~w ~ ~ ~ ~ ~ ~ ~ ~ c~ ~ ~w ~ ~ ~ ~ ~ o ~ ~ ~ ~ ~ ~ o ~ ~ ~ ~ o ~ ~ ~ ~ ~ ~w ~ ~w =\ - ~ - ~ w^~ - o - ~^ ~ ~ ~ ~ ~w ~ ~ ~ ~ ~ ~ ~ ~ ~ ~ ~ ~w ~ ~ ~ ~w ~ ~ ~w ~ ~ ~ ~ ~ o ~ ~ ^ o^o^~^~^~^o~o^~^ m^~^ ~^~^ - ~ cd o ~= ~' ~ ~ ~ ~ o ~ ~ ~ - O ~ O ~ V) O ~ .> ~ 0 ~ ~ :- . ~ ~ U, ~ O O O ~ O O ~ ~ ~ ~ o _ U, o - S~ S~ ~ ~;. 91 g s~ ~ ~o s~ ~ bC ,C~ _ ~o ~ C<, ._ ~ ~ .- o o ~ o g bC r, o ~ o ~-= - ~ =. ~ p4 o ;= ~ ~ bC o ~ ~ =) ~ ~ ~ ~ o o ~ ~ s~ o ~ ~ ~o ~ ~= o o ~ .o ' .m - ~ ~b ~ <~N o Q, ~ >^ ~ =, ~ ~^ p4

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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 93

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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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Enhancing NIH!s Ability to Respond to New Challenges 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. 95

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96 Enhancing the Vitality of the National Institutes of Health 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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Enhancing NIH!s Ability to Respond to New Challenges 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. 97

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98 Enhancing the Vitality of the National Institutes of Health 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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Enhancing NIH!s Ability to Respond to New Challenges 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 99

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100 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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Enhancing NIH!s Ability to Respond to New Challenges 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. 101

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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. 102