Summary

During the past half-century, biomedical research has expanded exponentially, yielding many discoveries that offer the promise of improved human health. Translating basic and clinical research findings into clinical and community practice has been slow and cumbersome, however, and many years may pass before the benefits of research reach individual patients and communities. Recognizing the need for a new impetus to spur clinical and translational research, the National Institutes of Health (NIH) established the Clinical and Translational Science Awards (CTSA) Program in 2006.

The CTSA Program was designed to “provide integrated intellectual and physical resources for the conduct of original clinical and translational science,”1 and individual CTSA sites were intended to serve as “catalysts and test beds for policies and practices that can benefit clinical and translational research organizations throughout the country.”2 In its first 7 years, the CTSA Program grew from 12 initial sites to the current 61, which are housed at academic health centers and other institutions across the United States. During this time, the program has made notable strides in accomplishing its initial goal of reshaping clinical and translational research at these institutions and has begun to build a national network that has the potential to catalyze further progress.

In 2012 the NIH contracted with the Institute of Medicine (IOM) to conduct a consensus study to assess and provide recommendations on the

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1 Zerhouni, E. A. 2005. Translational and clinical science—time for a new vision. New England Journal of Medicine 353(15):1621–1623.

2 Zerhouni, E. A. 2006. Clinical and Translational Science Awards: A framework for a national research agenda. Translational Research 148(1):4–5.



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Summary During the past half-century, biomedical research has expanded exponentially, yielding many discoveries that offer the promise of im- proved human health. Translating basic and clinical research findings into clinical and community practice has been slow and cumbersome, however, and many years may pass before the benefits of research reach individual patients and communities. Recognizing the need for a new impetus to spur clinical and translational research, the National Institutes of Health (NIH) established the Clinical and Translational Science Awards (CTSA) Program in 2006. The CTSA Program was designed to “provide integrated intellectual and physical resources for the conduct of original clinical and transla- tional science,” 1 and individual CTSA sites were intended to serve as “catalysts and test beds for policies and practices that can benefit clinical and translational research organizations throughout the country.”2 In its first 7 years, the CTSA Program grew from 12 initial sites to the current 61, which are housed at academic health centers and other institutions across the United States. During this time, the program has made notable strides in accomplishing its initial goal of reshaping clinical and transla- tional research at these institutions and has begun to build a national network that has the potential to catalyze further progress. In 2012 the NIH contracted with the Institute of Medicine (IOM) to conduct a consensus study to assess and provide recommendations on the 1 Zerhouni, E. A. 2005. Translational and clinical science—time for a new vision. New England Journal of Medicine 353(15):1621–1623. 2 Zerhouni, E. A. 2006. Clinical and Translational Science Awards: A framework for a national research agenda. Translational Research 148(1):4–5. 1

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2 THE CTSA PROGRAM AT NIH appropriateness of the CTSA Program’s mission and strategic goals and whether changes were needed. The committee was also tasked with providing an independent appraisal of and advice on the implementation of the program by the National Center for Advancing Translational Sci- ences (NCATS), while exploring the contributions of CTSAs in acceler- ating the development of new therapeutics, facilitating disease-specific and child health research, and enhancing the integration of research funded by NIH institutes and centers. To conduct this study, the IOM con- vened a 13-member committee with expertise in community outreach and engagement, public health and health policy, bioethics, education and training, pharmaceutical research and development, program evaluation, clinical and biomedical research, and child health research, along the full continuum of clinical and translation research. The committee’s overarch- ing conclusion is that the CTSA Program is contributing significantly to the advancement of clinical and translational research and is therefore a worthwhile investment that would benefit from a variety of revisions to make it more efficient and effective. THE CTSA PROGRAM The CTSA Program is a direct outgrowth of the NIH’s General Clin- ical Research Center Program, which for more than 40 years provided clinical research infrastructure funding. Development of the CTSA Pro- gram was an integral part of the implementation of the 2004 NIH Roadmap for Medical Research. From 2006 to 2011, the program was administered through the NIH’s National Center for Research Resources. In fiscal year (FY) 2012, NIH established NCATS, and the CTSA Pro- gram became the largest component of that center. Individual CTSAs are funded through 5-year cooperative agree- ments, and site budgets range from $4 million to $23 million annually, with a total CTSA Program budget of $461 million in FY2012. Building an active and productive CTSA at an institution often involves not only the funds from the CTSA cooperative agreement but also substantial fi- nancial and staff commitments from the institution; although institutional cost sharing is not required. The committee could not identify any data to quantify these institutional contributions but heard testimony from many individuals about the depth of efforts and the commitment to the CTSA Program from top leaders at health research institutions across the nation.

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SUMMARY 3 Currently the 61 CTSAs provide a wide array of training and re- search support to help researchers identify promising therapeutics and interventions and move them forward as rapidly as possible. Research support is provided in areas that include core facilities; biomedical in- formatics; pilot funding; regulatory knowledge and support; biostatistics, epidemiology, research design, and ethics; participant and clinical inter- action resources; and community engagement efforts and resources. From the outset of the program, the NIH charged the CTSAs with developing a national consortium to promote the identification and use of best research practices. This effort has developed into the primarily self- governing CTSA Consortium, which oversees numerous collaborative committees. The CTSA Consortium’s efforts are guided by three leader- ship committees: an Executive Committee, a Steering Committee, and a Child Health Oversight Committee. In addition, CTSA principal investi- gators, researchers, and staff work on 5 strategic goal committees and 14 key function committees (plus a number of interest groups, task forces, and work groups) that were established over time to discuss crosscutting issues, promote collaboration, and identify and implement best practices. In November 2011, the CTSA Consortium Coordinating Center was established at Vanderbilt University through a competitive application process. The coordinating center has taken many steps to standardize and coordinate consortium activities and is working to ensure the availability of best practices, facilitate the uptake of available tools and resources, and promote collaboration, in part through its website, CTSACentral.org. CONTEXT AND VISION FOR THE CTSA PROGRAM The CTSA Program does not exist in isolation; it is part of a larger clinical and translational research ecosystem that plays a vital role in an increasingly complex and dynamic U.S. health care system. Decades of innovation and technological advances have led to progress in biomedi- cal sciences, medicine, and public health, contributing to increased life expectancy and improved individual and population health. At the same time, however, the accelerating pace of scientific discoveries has also been one cause of the increasing complexity of the U.S. health care sys- tem, contributing to inconsistent health care quality and escalating costs. Across the United States, momentum is growing in support of a learning health care system in which researchers and health care provid- ers design and implement care, evaluations, or research based on needs

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4 THE CTSA PROGRAM AT NIH of specific communities and populations. The findings are disseminated to inform clinical practice and research models to improve health. A learning health care system is founded on the concept of continuous im- provement and the imperative to translate “what we know” into “what we do.” Such a system fuels greater value in health care by harnessing the promise of new technological capabilities, market opportunities, and policies. Thus, clinical and translational research is integral to a learning health care system. The CTSA Program has been successful in establishing CTSAs as academic focal points for clinical and translational research. The chal- lenge for the next phase of the program—which NCATS has described as CTSA 2.0—will be to set the goals and create incentives for its 61 sites to function as the core of a national network that initiates and sus- tains collaborations both inside and outside their home institutions; across NIH institutes and centers; and with community, industry, and research network partners. The IOM committee envisions a transfor- mation of the CTSA Program from its current, loosely organized struc- ture into a more tightly integrated network that works collectively to enhance the transit of therapeutics, diagnostics, and preventive interven- tions along the developmental pipeline; disseminate innovative transla- tional research methods and best practices; and provide leadership in informatics standards and policy development to promote shared resources. The committee identified four key opportunities for action:  Adopt and sustain active program leadership—NCATS should increase its leadership presence in the overall program, con- sistent with the cooperative agreement model under which the CTSAs are funded. A centralized leadership model that includes participation by NCATS, leaders of individual CTSAs, communi- ty partners, and other stakeholders will increase overall program efficiency, enable mechanisms for maximizing accountability, and provide the direction needed to develop and nurture substantive partnerships.  Engage in substantive and productive collaborations—The CTSA Program needs to capitalize on the collaborations devel- oped within and among individual CTSAs and continue to initi- ate and forge true partnerships with other NIH institutes and centers and with entities external to the program, including pa- tient groups, communities, health care providers, industry, and regulatory organizations.

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SUMMARY 5  Develop and widely disseminate innovative research resources— Fully developing the role of the CTSA Program as a facilitator and accelerator of clinical and translational research will require en- hanced efforts to engage and support researchers and other stake- holders as they develop, refine, widely disseminate, and implement novel research and health informatics tools, methodologies, poli- cies, and other resources.  Build on initial successes in training and education, community engagement, and child health research—The CTSA Program needs to continue its strong efforts in each of these areas. A ro- bust and diverse workforce that is well trained in team science is critically important. Ensuring an emphasis on community in- volvement across the research spectrum will bring a range of much-needed perspectives and innovations along with increased public support for research. Program efforts can also help over- come the paucity of research specific to child health. LEADERSHIP Today’s CTSA Program has a complex, multilevel structure of or- ganization and oversight involving NCATS, individual CTSAs, the CTSA Consortium with multiple levels and type of committees and working groups, and the CTSA Coordinating Center. An initiative with the scope and structure of the CTSA Program inherently faces challenges in balancing grassroots and top-down leadership approaches. To date, the program has, for the most part, relied on the energy and efforts of indi- vidual CTSAs and their principal investigators. As the program moves forward, the IOM committee sees the need for a more centralized approach to leadership, one in which NCATS plays a much more active role. The IOM committee envisions the primary governance of the pro- gram residing within a new NCATS-CTSA Steering Committee that would be responsible for program oversight and direction; trans-CTSA activities; collaborative efforts with external partners; promotion of col- laborative opportunities within and outside of NIH; identification, dis- semination, and implementation of best practices; and implementation of a proposed new innovations fund to promote collaboration with other NIH institutes and centers and external partners. A strategic planning process is needed as NCATS leads the program into its next phase. The program’s mission statement should be updated

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6 THE CTSA PROGRAM AT NIH to clarify its overall purpose and to align it with the mission of NCATS. Identifying and disseminating a set of clearly defined, measurable strate- gic goals is the starting point for shaping the program’s future. These measurable goals should serve as a foundation for developing high-level common metrics and measures that could be applied and publicly report- ed on consistently to demonstrate progress. At this point, NCATS’s plans for evaluating individual CTSA sites and the CTSA Program as a whole are unclear. Progress is being made at the individual CTSA level in terms of self-evaluation, but the current lack of transparency in reporting and lack of high-level common metrics are barriers to overall program ac- countability. Streamlining the current consortium structure is an urgent need. However, the structure and governance should evolve during the next year or two as a component of the recommended strategic planning pro- cess. Only those consortium committees that are most relevant to the program’s revised goals and priorities should be retained. Recommendation 1: Strengthen NCATS Leadership of the CTSA Program NCATS should strengthen its leadership of the CTSA Program to advance innovative and transformative efforts in clinical and translational research. As it implements CTSA 2.0, NCATS should  increase active involvement in the CTSA cooperative agree- ments and the CTSA Consortium;  conduct a strategic planning process to set measurable goals and objectives for the program that address the full spec- trum of clinical and translational research;  ensure that the CTSA Program as a whole actively supports the full spectrum of clinical and translational research while encouraging flexibility for each institution to build on its unique strengths;  form strategic partnerships with NIH institutes and centers and with other research networks and industry;  establish an innovations fund through a set-aside mechanism that would be used for collaborative pilot studies and other initiatives involving CTSA institutions, other NIH institutes, and/or other public and private entities (e.g., industry, other

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SUMMARY 7 government agencies, private foundations, community advo- cates and organizations);  evaluate the program as a whole to identify gaps, weaknesses, and opportunities and create mechanisms to address them; and  distill and widely disseminate best practices and lessons learned by the CTSA Program and work to communicate its value and accomplishments and seek opportunities for fur- ther efforts and collaborations. Recommendation 2: Reconfigure and Streamline the CTSA Consortium NCATS should reconfigure and streamline the structure of the CTSA Program by establishing a new multistakeholder NCATS- CTSA Steering Committee that would  be chaired by a member of NCATS leadership team and have a CTSA principal investigator as vice-chair, and  provide direction to the CTSA Coordinating Center in devel- oping and promoting the use of available shared resources. Recommendation 3: Build on the Strengths of Individual CTSAs Across the Spectrum of Clinical and Translational Research Individual CTSAs, with the leadership of NCATS, should em- phasize their particular strengths in advancing the program’s broad mission and goals. In doing so, CTSAs should  drive innovation and collaboration in methodologies, pro- cesses, tools, and resources across the spectrum of clinical and translational research;  emphasize interdisciplinary team-based approaches in train- ing, education, and research;  involve patients, family members, health care providers, and other community partners in all phases of the work of the CTSA;  strengthen collaborations across the schools and disciplines in their home institutions;

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8 THE CTSA PROGRAM AT NIH  build partnerships with industry, other research networks, community groups, and other stakeholders; and  communicate the resources available through the CTSA Program. Recommendation 4: Formalize and Standardize Evaluation Processes for Individual CTSAs and the CTSA Program NCATS should formalize and standardize its evaluation processes for individual CTSAs and the CTSA Program. The evaluations should use clear, consistent, and innovative metrics that align with the program’s mission and goals and that go beyond stand- ard academic benchmarks of publications and number of grant awards to assess the CTSA Program and the individual CTSAs. CROSSCUTTING TOPICS The CTSA Program has demonstrated progress in three crosscutting domains that the IOM committee believes are integral to advancing clini- cal and translational science effectively: training and education, commu- nity engagement, and child health research. These efforts, along with the program’s contributions in building infrastructure and providing a range of research resources, make the CTSA Program a unique national re- source within the clinical and translational research landscape. Each of these functions can be strengthened, as discussed below. Training and Education Sustaining a vibrant clinical and translational research enterprise in the future depends on building and retaining a diverse research work- force. Education and training in clinical and translational research are priorities for the CTSA Program. All CTSA institutions are expected to provide robust postgraduate training, and many have extensive training programs that include undergraduate and predoctoral student training as well as training for research staff and community collaborators. The KL2

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SUMMARY 9 and TL1 training awards3 have been an integral part of CTSA training programs. In FY2011, 501 scholars participated in the KL2 program, and 469 trainees participated in the TL1 program through the CTSA Pro- gram. Moving forward, the committee urges increased flexibility in train- ing and education programs with options available to personalize the training experience to meet the needs and goals of individual partici- pants. This flexibility will be valuable in attracting and retaining scholars and trainees and may be particularly pertinent to the clinician-scientists who are essential in clinical and translational research. NCATS and indi- vidual CTSAs have the opportunity to lead changes in the following: emphasizing the team-based skills that are required in clinical and trans- lational research, developing metrics to assess clinical and translational training and education programs, and instituting incentives for recogni- tion and promotion of those involved. New benchmarks that value team- based efforts and collaborative approaches are needed to complement the traditional benchmarks for academic success that focus on individual accomplishments and products (e.g., publications, new grants). Recommendation 5: Advance Innovation in Education and Train- ing Programs The CTSA Program should provide training, mentoring, and education as essential core elements. To better prepare the next generation of a diverse clinical and translational science work- force, the CTSA Program should  emphasize innovative education and training models and methodologies, which include a focus on team science, lead- ership, community engagement, and entrepreneurship;  disseminate high-quality online offerings for essential core courses for use in CTSA and other institutions; 3 The KL2 Mentored Clinical Research Scholar Program is a career development award that provides individuals who have a doctoral degree with formal research experience and funding support to help them become independent investigators in clinical and transla- tional research. The TL1 Clinical Research Training Program provides an introduction to clinical and translational research to pre- and postdoctoral candidates or others who want to learn more about these types of research.

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10 THE CTSA PROGRAM AT NIH  champion the reshaping of career development pathways for researchers involved in the conduct of clinical and transla- tional science; and  ensure flexible and personalized training experiences that of- fer optional advanced degrees. Community Engagement The ultimate goal of translational research—to improve human health—requires meaningful community engagement across the entire spectrum of research from basic science to community and population health research. Communities can contribute to the full range of clinical and translational research in important ways that are not always recog- nized. For example, partnerships with community representatives can identify community health needs and priorities, provide critical input and data on clinically relevant questions, develop culturally appropriate clini- cal research protocols, promote successful enrollment and retention of research participants, and, ultimately, disseminate and implement re- search results more effectively. The initial commitment to community engagement within the CTSA Program should be commended. However, NCATS’s vision for how community engagement will be a part of the CTSA Program moving forward remains unclear. Although indications point to community en- gagement remaining an important feature of the program, there are seri- ous concerns that if it is not an explicit requirement for all CTSAs, it may fade in importance. The IOM committee fully supports community engagement and involvement throughout the entire research process and believes that this program component is essential and needs to be pre- served, nurtured, and expanded. Because involving the community in the continuum of research is a new experience for many researchers, the CTSA Program and NCATS must provide clear guidance and leadership that effectively define and communicate goals and expectations. Recommendation 6: Ensure Community Engagement in All Phases of Research NCATS and the CTSA Program should ensure that patients, family members, health care providers, clinical researchers, and

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SUMMARY 11 other community stakeholders are involved across the continu- um of clinical and translational research. NCATS and the CTSA Program should  define community engagement broadly and use this defini- tion consistently in requests for applications and communica- tions about the CTSA Program;  ensure active and substantive community stakeholder partic- ipation in priority setting and decision making across all phases of clinical and translational research and in the lead- ership and governance of the CTSA Program;  define and clearly communicate goals and expectations for community engagement at the individual CTSA level and across the program and ensure the broad dissemination of best practices in community engagement; and  explore opportunities and incentives to engage a more di- verse community. Child Health Research For too long, research examining the safety and efficacy of medica- tions and other health interventions has focused on adults, and little has been known about health- and development-related impacts of medica- tions, devices, and preventive measures on children. Thus, clinical and translational research is urgently needed in the area of child health. The IOM committee believes that the CTSA Program has placed an appropri- ate emphasis on accelerating clinical and translational research to im- prove child health, and the CTSA Program, through the CTSA Consortium Child Health Oversight Committee (CC-CHOC), has made important steps toward streamlining and accelerating this type of research. To strengthen these efforts, the IOM committee believes that the NCATS-CTSA Steering Committee should identify a relatively small number of CTSAs with established expertise that provide outstanding efforts in child health research as leaders in this arena. This would not preclude other CTSAs from being involved in child health research. In- stead, the IOM committee hopes that such focused efforts would encour- age and promote collaborations among CTSAs for multisite studies and other efforts. The committee also believes that CTSAs should be en-

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12 THE CTSA PROGRAM AT NIH gaged in a life-span approach that includes research on the transition from adolescence into adulthood. As part of a learning health care system, those involved in child health research need to be sure that this area of investigation is well posi- tioned to fully embrace the use of electronic health records for research purposes and to actively partner with practice-based research networks. Implementing these types of strategies will allow researchers to under- stand what is occurring in clinical practice and will allow pediatric health care providers, patients, and families to learn about new medications, therapeutics, and preventive measures. Recommendation 7: Strengthen Clinical and Translational Research Relevant to Child Health NCATS should collaborate with the CTSA Consortium Child Health Oversight Committee to strengthen clinical and transla- tional research relevant to child health through efforts to  identify and designate CTSAs with expertise in child health research as leaders in advancing clinical and translational research relevant to child health and as coordinators for CTSA programwide efforts and other collaborative efforts in this research; and  promote and increase community engagement specific to child health by o raising awareness of the opportunities for children and families to participate in research efforts with clear in- formation conveyed on the risks and potential benefits; and o involving parents, patients, and family members more fully at all stages of the research process, including iden- tifying priorities and setting research agendas. CONCLUSION: OPPORTUNITIES FOR ACTION With the ultimate goal of improving human health, the CTSA Pro- gram now has the opportunity to propel clinical and translational re- search efforts forward rapidly. To move to CTSA 2.0, the CTSA

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SUMMARY 13 Program can build on its foundation; draw on the creativity and dedica- tion of CTSA principal investigators, researchers, and staff; use the ever- expanding capabilities of informatics and other technologies; share data and research support tools as openly and freely as possible; and fully en- gage new cadres of researchers focused on team-based science. The IOM committee believes that the CTSA Program should be the national leader for advancing innovative and transformative clinical and translational research to improve human health. To achieve this, the CTSA Program should reshape its goals to reflect its new location within NCATS; build on the work of individual CTSAs to provide institutional leadership; focus on team-based education and training; and establish a national network that will accelerate the development of new diagnostics, therapeutics, and preventive interventions and, at the same time, will drive innovation in clinical and translational research methods, process- es, tools, and resources. The committee’s recommendations are summa- rized in Box S-1. Because the CTSA Program is not disease specific in its orientation, strong collaborations must be forged across disciplinary units within in- dividual CTSA institutions and with other NIH institutes and centers, as well as with other government funders, industry, philanthropies, and community organizations. The CTSA Program should continue to lead efforts in expanding and diversifying the research workforce and to co- ordinate and advance child health research by streamlining and building on the expertise of individual CTSAs. In all these efforts, community engagement is essential. In short, the contributions of the individual CTSAs and the CTSA Program are vital to the clinical and translational research enterprise, and the nation’s health can benefit greatly from strengthening their efforts.

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14 THE CTSA PROGRAM AT NIH BOX S-1 Overview of Recommendationsa The next steps for the Clinical and Translational Science Awards (CTSA) Program and opportunities for advancing clinical and translational research are as follows:  Strengthen leadership of the CTSA Program by the National Center for Advancing Translational Sciences (NCATS).  Reconfigure and streamline the CTSA Consortium.  Build on the strengths of individual CTSAs across the spectrum of clinical and translational research.  Formalize and standardize evaluation processes for individual CTSAs and the CTSA Program.  Advance innovation in education and training programs.  Ensure community engagement in all phases of research.  Strengthen clinical and translational research relevant to child health. _______________________________ a The full text of the recommendations appears throughout the summary and in Chapters 3 and 4 of the report.