1

Introduction

During the past half-century, biomedical research has expanded exponentially, becoming increasingly complex (IOM, 2013). As a result of improved scientific knowledge, the biomedical research enterprise in the United States has witnessed many successes that offer the promise of improved human health. For example, advances in the fields of genomics and proteomics have led to new targeted diagnostic tools and therapies for diseases as diverse as lung cancer, schizophrenia, and cystic fibrosis (IOM, 2012).

Despite the production of new data and numerous publications disseminating these research findings, translating the results of basic and clinical research into clinical and community practice has been slow and cumbersome, and many years may pass before the benefits of basic science discoveries and clinical investigations reach individual patients and communities. Barriers to translation include long research timelines; the large number of clinical trials that must be abandoned because of limited enrollment; data-sharing challenges; a lack of available resources (including investigators, study participants, and financial support for clinical trials); and increasing costs, complexity, and regulatory burdens (Collins, 2011; Kitterman et al., 2011; NCATS, 2013d; Zerhouni, 2005). These persistent challenges required a new approach to accelerating the translation of research to clinical applications and maximizing improvements in individual and public health.

Recognizing the need for a new research paradigm, the National Institutes of Health (NIH) developed a Roadmap for Medical Research in 2004 to focus efforts on the challenges facing medical research (NIH, 2006, 2011, 2013a; Zerhouni, 2005). The NIH Roadmap sought to facilitate new pathways to discovery; promote interdisciplinary and collabora-



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1 Introduction During the past half-century, biomedical research has expanded ex- ponentially, becoming increasingly complex (IOM, 2013). As a result of improved scientific knowledge, the biomedical research enterprise in the United States has witnessed many successes that offer the promise of improved human health. For example, advances in the fields of genomics and proteomics have led to new targeted diagnostic tools and therapies for diseases as diverse as lung cancer, schizophrenia, and cystic fibrosis (IOM, 2012). Despite the production of new data and numerous publications dissem- inating these research findings, translating the results of basic and clinical research into clinical and community practice has been slow and cumber- some, and many years may pass before the benefits of basic science discoveries and clinical investigations reach individual patients and com- munities. Barriers to translation include long research timelines; the large number of clinical trials that must be abandoned because of limited enrollment; data-sharing challenges; a lack of available resources (including investigators, study participants, and financial support for clinical trials); and increasing costs, complexity, and regulatory burdens (Collins, 2011; Kitterman et al., 2011; NCATS, 2013d; Zerhouni, 2005). These persistent challenges required a new approach to accelerating the translation of research to clinical applications and maximizing improve- ments in individual and public health. Recognizing the need for a new research paradigm, the National In- stitutes of Health (NIH) developed a Roadmap for Medical Research in 2004 to focus efforts on the challenges facing medical research (NIH, 2006, 2011, 2013a; Zerhouni, 2005). The NIH Roadmap sought to facili- tate new pathways to discovery; promote interdisciplinary and collabora- 15

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16 THE CTSA PROGRAM AT NIH tive research teams; and “re-engineer the clinical research enterprise” by harmonizing regulatory policies, encouraging multidisciplinary training, and facilitating the establishment of academic homes for clinical and translational research (NIH, 2006; Zerhouni, 2003). As part of its effort to implement its Roadmap and spur clinical and translational research, the NIH established the Clinical and Translational Science Awards (CTSA) Program. The CTSA Program was designed to “provide integrated intellectual and physical resources for the conduct of original clinical and translational science” (Zerhouni, 2005, p. 1622), and individual CTSA sites were intended to serve as “catalysts and test beds for policies and practices that can benefit clinical and translational re- search organizations throughout the country” (Zerhouni, 2006, p. 4). The CTSA Program originally focused on “re-engineering existing capabili- ties at medical research institutions and developing new resources in the areas of clinical and translational research training, community outreach and informatics” (NCATS, 2013a). Although the CTSA Program does not directly fund or conduct large-scale clinical and translational re- search, it supports the development and application of shared resources and innovative technologies for clinical and translational studies across the full spectrum of research (NIH, 2012b). Consistent with NIH’s Roadmap, the CTSA Program’s initial goals were to  “create academic homes for clinical and translational research;  provide investigators and research teams with research cores, tools and a local environment that encourages and facilitates the conduct of clinical and translational research, including with community and industry partners; and  train the scientific workforce needed for the translational sciences” (NCATS, 2013a). Diverse groups of stakeholders (researchers, funders, the public, and congressional representatives) increasingly seek evidence that the enor- mous U.S. investment in biomedical research, including the CTSA Pro- gram, is bearing tangible fruit in the form of new and better preventive and treatment options. A 2011 congressional conference report high- lighted the success and additional promise of the CTSA Program and “urge[d] NIH to support a study by the Institute of Medicine to evaluate the CTSA program and to recommend whether changes to the current mission are needed” (U.S. Congress, 2011). The report specified the charge as follows:

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INTRODUCTION 17 CTSAs now represent an investment of half a decade of innovation in translational research. To ensure the bene- fits of this investment are maintained, the conferees urge NIH to support a study by the IOM that would evaluate the CTSA program and recommend whether changes to the current mission are needed. The review should in- clude stakeholders’ input and be available no later than 18 months after the enactment of this bill. (U.S. Congress, 2011, p. 1137) SCOPE OF WORK AND STUDY PROCESS In 2012 the NIH contracted with the Institute of Medicine (IOM) to conduct a consensus study to review the CTSA Program. 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 evalua- tion, clinical and biomedical research, and child health research, along the continuum of clinical and translation research (Appendix B). The committee’s statement of task (see Box 1-1) directed it to assess the CTSA Program and its mission and strategic goals and to offer advice on the implementation of the program by the National Center for Ad- vancing Translational Sciences (NCATS), while exploring the contribu- tions of CTSAs in accelerating the development of new therapeutics, facilitating disease-specific research and child health research, and en- hancing the integration of research funded by NIH institutes and centers. When presenting the charge to the committee at its first meeting in Octo- ber 2012, NCATS leaders offered the following questions to help clarify the statement of task:  “Is the breadth of the program supporting T1 through T4 re- search appropriate?  Should the goal of creating an academic home for clinical and translational sciences continue to be a major focus of the CTSA program?  Are CTSAs effectively providing innovative education, training, and career development support to meet the needs of the biomed- ical research workforce? How could this aspect of the program be strengthened?

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18 THE CTSA PROGRAM AT NIH BOX 1-1 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translation Sciences Statement of Task In response to a request from the National Institutes of Health (NIH), the Institute of Medicine (IOM) will assemble an ad hoc expert committee to provide an independent appraisal of and advice on the NIH Clinical and Translational Science Awards (CTSA) Program as it will be implemented by the newly formed National Center for Advancing Translational Sciences (NCATS). The current mission of the CTSA program includes services and infrastructure support for the full continuum of clinical and translational re- search. The services provided by CTSAs have supported T1 through T4 research, with primary emphasis on support for human subjects research extending from first-in-man and proof-of-concept studies through efficacy and effectiveness studies, and including research on how to achieve com- munity and patient engagement, implementation and dissemination sciences, and behavioral research. The IOM committee will review existing evaluations and available stakeholder input on the program, and will seek additional stakeholder in- put as needed. Based on this assessment, the committee will provide rec- ommendations on the appropriateness of the program’s current mission and overarching goals and whether changes are needed. This study will explore the contributions of the CTSAs in accelerating the development of new therapeutics with consideration given to the role of the CTSA program in facilitating disease-specific research and pediatric research and in en- hancing the integration of programs funded by the categorical NIH Institutes and Centers.  Are the CTSAs configured effectively to accelerate new thera- peutics, and if not, what changes should be implemented?” (Briggs and Austin, 2012). Throughout this study, the committee considered these questions and used a forward-looking approach to respond to its statement of task. This report builds on previous program evaluations and assessments, although it was not designed to provide a comprehensive and in-depth evaluation of the operations, administration, or achievements of individual CTSA sites or the program as a whole. The committee’s advice and recommen- dations are intended to help NCATS implement the program effectively and enable the full realization of its potential. During the course of its work, the committee held four meetings, two public workshops, and four open-session conference calls to solicit input about the successes, challenges, and future directions of the CTSA Pro-

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INTRODUCTION 19 gram (Appendix A). Throughout the study, the committee heard from a number of CTSA principal investigators (PIs) and researchers, members of the NIH and NCATS leadership, community and patient advocacy organizations, industry partners and representatives, and thought leaders and researchers in the clinical and translational sciences arena who were not connected to the CTSA Program. As part of its assessment, the com- mittee reviewed the scientific literature, previous CTSA Program evalua- tions, available progress reports, responses to formal NIH requests for information (RFIs) related to the CTSA Program, information submitted by a range of CTSA Consortium committees and stakeholder groups, and data and recommendations from other relevant working groups and stakeholder meetings. The committee’s work was also informed by re- sponses to a series of public input questions that focused on the CTSA Program’s mission and strategic goals and its role in advancing research along the continuum of clinical and translational science.1 CLINICAL AND TRANSLATIONAL RESEARCH Translational research means different things to different people but it seems important to almost everyone. —Steven Woolf (Woolf, 2008) The CTSA Program’s focus, by definition, is on clinical and transla- tional research. Clinical research involves human participants and in- cludes epidemiological and behavioral studies; outcome and health services research; and patient-oriented research, such as the study of dis- ease pathology and mechanisms, development and testing of therapeutic interventions or technologies, and clinical trials (NIH, 2013b). The NIH’s definition of translational research includes two broad areas: the translation of basic science and preclinical discoveries into human sub- ject research and the subsequent translation of clinical trial results, re- search findings, and knowledge into practice in clinical and community settings (NIH, 2013b). For the purposes of this report, however, the committee has adopted a conceptual model of translational research that exists along a dynamic continuum that connects basic research findings to decisions made within clinical settings and interventions that are applied 1 Public testimony and other materials submitted to the committee are available by re- quest through the National Academies’ Public Access Records Office.

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20 THE CTSA PROGRAM AT NIH in community and public health settings to improve health broadly. Figure 1-1 defines and illustrates the conceptual progression across the five phases of translational research, from the initial stages of research (such as preclinical and animal models) to large-scale research in communities and populations. The translational phases along this continuum are some- times referred to as “bench-to-bedside” and “bedside-to-community” (Blumberg et al., 2012; ITHS, 2013; Khoury et al., 2007). Despite efforts to raise the profile and improve the accessibility of translational research, misconceptions persist about its scope, and many people conflate the concepts of clinical and translational research. As illustrated below, the continuum of translational research (T0–T4) is broader than clinical research (T1–T3). Although the depiction of the separate phases of translational research above suggests a linear model with a finite beginning and end, in reality the operational phases of trans- lational research include many feedback loops. Its process is more circu- lar and interdependent across research phases, requiring continuous data gathering, analysis, dissemination, and interaction (see Figure 1-2). In formation sharing at each stage ensures that researchers are meeting pa- tient and community health needs and that progress in the clinic and T0 T1 T2 T3 T4 Basic science Translation to Translation to Translation to Translation to research humans patients practice community Phase 2 clinical Phase 4 clinical Proof of concept Population-level Preclinical and trials trials and clinical Phase 1 clinical outcomes animal studies Phase 3 clinical outcomes trials research trials research Defining mechanisms, New methods of Controlled studies Delivery of targets, and lead diagnosis, treatment, leading to effective recommended and True benefit to molecules and prevention care timely care to the society right patient Translation of new data into the clinic Translation from basic science to human studies and health decision making FIGURE 1-1 Operational phases of translational research (T0–T4). SOURCE: Adapted with permission from Macmillan Publishers Ltd.: Nature Medicine (Blumberg et al., 2012), copyright 2012.

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INTRODUCTION 21 Basic Laboratory Patient-Oriented Research Clinical Research Improved Health Community- and Population-Based Clinical Trials Research FIGURE 1-2 An integrated model of clinical and translational research. SOURCE: Adapted from Austin, 2013. community, in turn, informs the work in the laboratory. As a result, the impact of translational research on health improvements hinges on an integrated and responsive research infrastructure, similar to models of a “learning health care system” (discussed in Chapter 2). The value of continuous feedback from communities to researchers is illustrated by three of the many advances in clinical and translational research the CTSA Program has achieved (see Box 1-2), which are being used by individuals and communities to improve health and quality of life. BOX 1-2 Examples of Advances Accomplished Through Successful Clinical and Translational Research Cystic fibrosis. On January 31, 2012, the Food and Drug Administration (FDA) approved the drug Kalydeco for people with a rare form of cystic fi- brosis. Kalydeco is the first drug to target the underlying cause of this type of cystic fibrosis and is the result of a unique collaboration between the Cystic Fibrosis Foundation, Vertex Pharmaceuticals, and 10 CTSA institutions. This collaboration facilitated clinical trials that garnered FDA approval (FDA, 2012; NCATS, 2012, 2013b).

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22 THE CTSA PROGRAM AT NIH Diabetes. To improve the health of individuals with diabetes or those who are at risk of developing diabetes, investigators at CTSAs in California, Connecticut, and South Carolina are working with local communities to ex- plore models of diabetes prevention and interventions tailored to the needs of those communities. For example, researchers at Yale University are cur- rently testing and implementing a 12-week intervention at a local community health center that features intensive lifestyle changes for women with pre- diabetes (NIH, 2012b; Tamborlane, 2009; Yale School of Medicine, 2012a,b). OVERVIEW OF THE CTSA PROGRAM History The CTSA Program is a direct outgrowth of the NIH’s General Clini- cal Research Center (GCRC) Program, which for more than 40 years pro- vided clinical research infrastructure funding.2 The GCRCs provided clinical researchers with dedicated inpatient beds, outpatient units, core laboratory support, and staffing support (e.g., research nurses, laboratory technicians, biostatisticians) (Robertson and Tung, 2001). Beginning in 2005, with the implementation of the NIH Roadmap and NIH’s efforts to revitalize its work in clinical and translational research, the GCRC Pro- gram was phased out. Funding from that program was redirected and consolidated with other existing resources (e.g., T- and K-training and career development awards), along with additional support from the NIH Common Fund, to launch the CTSA Program (NIH, 2005; Shurin, 2008). In 2006, 12 CTSA sites were funded through 5-year cooperative agreements as a first step toward establishing academic homes for clini- cal and translational research (Briggs and Austin, 2012; NIH, 2005; OIG, 2011). Institutional CTSA funding levels were based on the applicant’s previous GCRC funding and other NIH training awards (Briggs and Austin, 2012). A number of GCRC institutions applied for and received CTSA Program funding, and their GCRC clinical research units and as- sociated resources were folded into the new program (NIH, 2012a). Fol- lowing the first round of awards, the NIH added 5 to 14 new CTSA sites annually until, by the end of 2012, the program reached capacity at 61 2 The GCRCs began in the early 1960s and focused on metabolic and nutritional studies (Briggs and Austin, 2012; Robertson and Tung, 2001). Researchers applied to the GCRC in a specific institution to use its clinical research units for their institutional review board (IRB)-approved research. In 2005 there were 78 GCRC sites with a total budget of ap- proximately $288 million (Briggs and Austin, 2012).

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INTRODUCTION 23 sites nationwide (see Figure 1-3) (Briggs and Austin, 2012; Reis et al., 2010). The annual budgets for these sites range from $4 million to $23 million (Briggs and Austin, 2012; CTSA Central, 2013a), and the total annual budget for the program for fiscal year (FY) 2012 was $461 million. The CTSA Program was administered by the National Center for Re- search Resources (NCRR) through 2011, when the Consolidated Appro- priations Act of 2012 (Public Law 112-74) established NCATS and dissolved the NCRR (Collins, 2011; Reed et al., 2012). The NCATS mission focuses on catalyzing innovative methods and technologies re- lated to the development of diagnostics and therapeutics, and it became home to the CTSA Program and several smaller, related NIH programs. The CTSA Program accounted for approximately 80 percent of the NCATS budget in FY2012 (Briggs and Austin, 2012). WA ME MT ND VT OR MN NH ID SD WI NY MA MI CT RI WY PA NJ IA NV NE OH MD IN DE CA UT IL WV CO VA KS MO KY NC TN OK AR AZ SC NM MS GA AL TX LA AK FL HI = CTSA-funded institutions = States with CTSAs FIGURE 1-3 CTSA-funded institutions and participating states. SOURCE: CTSA Central, 2013a. Reprinted with permission from the National Institutes of Health, U.S. Department of Health and Human Services.

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24 THE CTSA PROGRAM AT NIH During the initial transition period and under interim leadership, NCATS sought input from a wide range of stakeholders through a varie- ty of mechanisms (e.g., RFIs, internal NIH working groups) on the im- plementation of the CTSA Program and strategies for enhancing it. As NCATS begins to lead the program forward, it is taking incremental steps to reshape its work. For example, its first request for applications (RFA) signaled more flexibility in focus for individual CTSAs (see Chap- ter 3) and a new funding structure in which support for individual CTSAs will be based on their institution’s total NIH research funding base (NIH, 2012c). Structure of the CTSA Program Today’s CTSA Program facilitates the training and education of in- vestigators and fosters a collaborative environment to promote improve- ments in the quality, safety, efficiency, and cost-effectiveness of clinical and translational research. It does not focus on any one disease or disor- der, although individual CTSA sites and projects may do so (NIH, 2012c). The program has a complex, multilevel organizational and over- sight structure involving individual institutions, the CTSA Consortium, the CTSA Coordinating Center, and NCATS. NCATS’s Division of Clinical Innovation oversees the CTSA Program and provides funding and other resource support for individual CTSA sites. Individual CTSAs At the heart of the CTSA Program are the 61 individual CTSAs es- tablished in academic health centers and other institutions across the United States and the commitment and ingenuity of the researchers and partners who work with them. These institutions have some flexibility in how to structure their individual programs. Many CTSAs are housed in medical schools and collaborate with other departments and schools in their universities, as well as with other universities, hospitals, and health care systems (CTSA Central, 2013h). Each CTSA must set up an exter- nal advisory committee that meets annually to provide advice on struc- ture, progress, and challenges of the program (NIH, 2012c). Many CTSAs also have established internal advisory committees and other ad- visory and governance committees (e.g., executive committees, commu- nity advisory boards).

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INTRODUCTION 25 Because CTSA funding levels vary widely across sites, the scope of their activities also varies (see also Chapter 3). In general, CTSA sites provide an array of training and research resources and support tools de- signed to help investigators conduct promising research, including  core facilities (e.g., translational technologies, core laboratory services, and novel methodologies, such as specific embryonic stem cell lines, nanotechnology, and epigenomics);  biomedical informatics (e.g., behavioral data analysis, geograph- ic coding, proteomics, registration for trials through tools such as ResearchMatch [see Chapter 3], customized software);  pilot funding (e.g., through CTSA pilots, institutional pilots, trainee and scholar pilot programs, and partnerships with indus- try and not-for-profit organizations);  regulatory knowledge and support (e.g., auditing and compliance measures, HIPAA compliance, conflicts-of-interest management, protocol development and preparation, and IRB agreements);  biostatistics, epidemiology, research design, and ethics (e.g., ethics consultations, adaptive trial design, randomization and blinding, statistical modeling and analysis, multicenter coordina- tion, grant application support);  participant and clinical interaction resources (e.g., cost recovery planning, case report form development and reporting compli- ance, research nurse support, research subject advocacy); and  community engagement efforts and resources (e.g., adult literacy assessment, cultural competency training, public databases, pro- motion of research participation) (Rosenblum, 2012). CTSA Consortium Committees and Coordinating Center CTSA Consortium committees From the outset of the program, the NIH charged the CTSAs with developing a national consortium to pro- mote the identification and use of best research practices (Berglund and Tarantal, 2009). The original funding announcement called for a consor- tium steering committee comprised of CTSA PIs. In addition, it directed that subcommittees be formed around NIH-identified key functions (e.g., education, informatics, regulation) (NIH, 2005). Further details on the governance and structure of the CTSA Consortium and its committees were formalized in 2008 as part of the PI-directed strategic planning pro- cess. At that time, the CTSA Program consisted of two dozen individual

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26 THE CTSA PROGRAM AT NIH CTSAs (Reis et al., 2010). The initial NIH guidance and the strategic planning effort formed the basis for the complex, multi-tiered CTSA Consortium committee structure that exists today, which includes hun- dreds of participants. The CTSAs’ collaborative efforts are overseen by three leadership committees:  The Consortium Executive Committee is the main governing body and has 31 members (more than 20 of whom are voting members), including leadership of the Consortium Steering Committee (described below), 5 CTSA PIs, NCATS staff, and other members who serve 1-year terms. Its purpose, in part, is to facilitate interactions among the PIs, NCATS staff, and the vari- ous consortium committees (CTSA Central, 2013e).  The Consortium Steering Committee has more than 175 mem- bers (more than 85 of whom at voting members), including PIs from each CTSA institution and representatives from NCATS and a number of other NIH institutes and centers. It provides leadership and management of the consortium and is responsible for setting strategic goals and priorities (CTSA Central, 2013g).  The CTSA Consortium Child Health Oversight Committee fo- cuses on overcoming barriers and promoting opportunities for child health research and has more than 230 members, almost 60 of whom are voting members (also discussed in Chapter 4). In addition to the leadership committees, CTSA PIs, researchers, and staff coordinate collaborative efforts and work to improve program func- tioning through numerous CTSA Consortium committees, interest groups, working groups, and task forces that have evolved with the growth of the program (see Box 1-3). Five strategic goal committees consist of 20 to 30 members each. These committees identify and priori- tize efforts related to achieving the strategic goals defined in 2008 (CTSA Central, 2013b; Reis et al., 2010). Fourteen key function committees discuss crosscutting issues, pro- mote collaboration, and identify and implement best practices. The key function committees reflect areas deemed essential to the program’s mis- sion, and some of them have been required in NIH funding announce- ments. The number and focus of the key function committees have fluctuated over the life of the CTSA program as priorities shifted (Evanoff, 2012). Many of these committees include more than 100 mem-

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INTRODUCTION 27 bers and have subcommittees, working groups, and task forces (CTSA Central, 2013c). In addition, numerous informal groups of CTSA re- searchers have developed around topics of mutual interest. After the development of the strategic goals in 2008, an effort was made to map the key functions to correspond to one or more of program strategic goals (Reis et al., 2010). Table 1-1 indicates that, although re- lated, strategic goals and key function groups do not consistently align. A commitment to participate in CTSA Consortium efforts, including the committees, has been a condition of receiving a CTSA award (NIH, 2010). All the committees described above convene regular conference calls to discuss progress and share best practices. The committees com- prise PIs and researchers who take on these extra responsibilities in addi- tion to their work at their institution’s CTSA. BOX 1-3 CTSA Consortium Committees and Working Groups Consortium Leadership Committee  Consortium Executive Committee  Consortium Steering Committee  CTSA Consortium Child Health Oversight Committee Consortium Strategic Goal Committees  Strategic Goal Committee 1—National Clinical and Translational Re- search Capability  Strategic Goal Committee 2—Training and Career Development of Clinical/Translational Scientists  Strategic Goal Committee 3—Enhancing Consortium-Wide Collaborations  Strategic Goal Committee 4—Enhancing the Health of Our Communi- ties and the Nation  Strategic Goal Committee 5—T1 Translational Research Key Function Committees  Administration Key Function Committee  Biostatistics/Epidemiology/Research Design Key Function Committee  Clinical Research Ethics Key Function Committee  Clinical Research Management Key Function Committee  Clinical Services Core Key Function Committee  Communications Key Function Committee  Community Engagement Key Function Committee

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28 THE CTSA PROGRAM AT NIH  Comparative Effectiveness Research Key Function Committee  Education and Career Development Key Function Committee  Evaluation Key Function Committee  Informatics Key Function Committee  Public−Private Partnerships Key Function Committee  Regulatory Knowledge Key Function Committee  Translational Key Function Committee CTSA Thematic Special Interest Groups  CTSA Nurse Scientist  CTSA Pain Research Interest Group  CTSA TEAM (TElemed, teleheAlth, Mhealth)  CTSA-USCIITG Critical Care Interest Group  Dentistry and Oral Health  Emergency Care Researchers  Neuroscience Researchers  Sleep Research Network  VA Research Collaboration  Women in Clinical and Translational Research Interest Group SOURCES: CTSA Central, 2013c,f,i. TABLE 1-1 Alignment of the CTSA Key Function Committees and Strategic Goal Committees Strategic Goal Committees 1 2 3 4 5 Key Function Committees That Support Strategic Goal Committees Clinical Research Management X Clinical Services Core X  Regulatory Knowledge X Education and Career Development X   Community Engagement X Comparative Effectiveness Research X Public–Private Partnerships   X Translational X Communications  X   Crosscutting Key Function Committees Informatics      Evaluation      Biostatistics/Epidemiology/Design      Clinical Research Ethics     

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INTRODUCTION 29 Strategic Goal Committees 1 2 3 4 5 General Grant/Consortium Operations Administration      NOTE: ● = alignment for related agenda topics and deliverable support; X = alignment for reporting purposes and on-going management of deliverables. SOURCE: https://www.ctsacentral.org/committees (accessed May 6, 2013). CTSA Consortium Coordinating Center In November 2011, the CTSA Consortium Coordinating Center was established through a com- petitive application process, which awarded Vanderbilt University a budget of $20 million over 5 years to  establish a high visibility and accessible “national home” for the CTSA Program;  manage meetings, projects, and communications of the CTSA Consortium;  compile and organize the CTSA Consortium’s networking re- sources; and  develop and disseminate research tools and resources that sup- port translational research (CTSA Central, 2013d; Snyder, 2011). Since its inception, the coordinating center has taken many steps to standardize and coordinate consortium activities (e.g., project and meet- ing support, listservs). In its efforts, the coordinating center also attempts to ensure the dissemination of best practices, facilitate the uptake of available tools and resources, and promote collaboration, in part, through its website, CTSACentral.org. The coordinating center facilitated PI ef- forts to produce a joint publication on the program’s transition to NCATS and a joint response to an NCATS RFI regarding opportunities to enhance the CTSA Program (Bernard, 2012; CTSA PIs, 2012; Pulley, 2013). The coordinating center is also striving to improve connections between the CTSA Program and NIH institutes and centers through a new liaison effort. Under that initiative, 40 CTSA PIs are working with 18 NIH institutes and centers to increase communication, awareness of available CTSA resources, and the integration of trans-NIH resources (Bernard, 2012).

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30 THE CTSA PROGRAM AT NIH ORGANIZATION OF THE REPORT This report provides the IOM committee’s findings and recommen- dations regarding the progress and potential of NIH’s CTSA Program. The report covers the breadth of the statement of task and highlights op- portunities to bolster the program and ensure its continued success and sustainability in supporting clinical and translational researchers and serving the needs of the communities CTSAs are linked to and in which they reside. Chapter 2 discusses the ecosystem in which NCATS and the CTSA Program operate and provides the committee’s vision for the next phase of the CTSA Program. Chapter 3 emphasizes the need for strong and active leadership by NCATS in establishing a clear vision and mis- sion for the program along with measurable goals; supporting individual CTSAs; partnering and collaborating within the NIH and with external partners; and evaluating and communicating the program’s value. Chap- ter 4 highlights specific opportunities and priorities in the areas of train- ing and education, community engagement, and research related to child health. On the basis of discussions, conclusions, and recommendations outlined in the preceding chapters, in Chapter 5 the report concludes with next steps and potential future directions. REFERENCES Austin, C. P. 2013. National Center for Advancing Translational Sciences: Catalyzing translational innovation. PowerPoint presented at Meeting 3: IOM Committee to Review the CTSA Program at NCATS, Washington, DC, January 24. http://www.iom.edu/~/media/Files/Activity%20Files/ Research/CTSAReview/2013-JAN-24/Chris%20Austin.pdf (accessed February 13, 2013). Berglund, L., and A. Tarantal. 2009. Strategies for innovation and interdisciplinary translational research: Removal of barriers through the CTSA mechanism. Journal of Investigative Medicine 57(2):474–476. Bernard, G. 2012. CTSA Consortium Coordinating Center (C4). PowerPoint presented at Meeting 2: IOM Committee to Review the CTSA Program at NCATS, Washington, DC, December 12. http://www.iom.edu/~/media/Files/ Activity%20Files/Research/CTSAReview/2012-DEC-12/3-1%20Gordon%20 Bernard.pdf (accessed March 28, 2013). Blumberg, R. S., B. Dittel, D. Hafler, M. von Herrath, and F. O. Nestle. 2012. Unraveling the autoimmune translational research process layer by layer. Nature Medicine 18(1):35–41.

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INTRODUCTION 33 ———. 2012c. RFA-TR-12-006: Institutional Clinical and Translational Science Award (U54). http://grants.nih.gov/grants/guide/rfa-files/rfa-tr-12- 006.html (accessed February 13, 2013). ———. 2013a. About NIH. http://www.nih.gov/about (accessed February 13, 2013). ———. 2013b. Glossary and acronym list. http://grants.nih.gov/grants/glossary. htm (accessed February 13. 2013). OIG (Office of the Inspector General). 2011. NIH administration of the Clinical and Translational Science Awards Program. https://oig.hhs.gov/oei/reports/ oei-07-09-00300.pdf (accessed April 8, 2013). Pulley, J. 2013. CTSA PI response to RFI NOT-TR-12-003. Submitted to the IOM Committee on January 6. Available by request through the National Academies’ Public Access Records Office. Reed, J. C., E. L. White, J. Aube, C. Lindsley, M. Li, L. Sklar, and S. Schreiber. 2012. The NIH’s role in accelerating translational sciences. Nature Biotechnology 30(1):16–19. Reis, S. E., L. Berglund, G. R. Bernard, R. M. Califf, G. A. FitzGerald, and P. C. Johnson. 2010. Reengineering the national clinical and translational research enterprise: The strategic plan of the National Clinical and Translational Science Awards Consortium. Academic Medicine 85(3):463– 469. Robertson, D., and C.-S. Tung. 2001. Linking molecular and bedside research: Designing a clinical research infrastructure. Journal of Molecular Medicine 79(12):686–694. Rosenblum, D. 2012. Access to core facilities and other research resources provided by the Clinical and Translational Science Awards. Clinical and Translational Science 5(1):78–82. Shurin, S. B. 2008. Clinical Translational Science Awards: Opportunities and challenges. Clinical and Translational Science 1(1):4. Snyder, B. 2011. VUMC to lead national CTSA consortium. Reporter: Vanderbilt University Medical Center’s Weekly Newspaper. http://www.mc. vanderbilt.edu/reporter/index.html?ID=10883 (accessed February 13, 2013). Tamborlane, W. 2009. Changing lifestyles for better health: Diabetes mellitus behavioral intensive lifestyle intervention, NCT00848757. http://clinicaltrials. gov/show/nct00848757 (accessed February 13, 2013). U.S. Congress, House of Representatives. 2011. Military Constructions and Veterans Affairs and Related Agencies Appropriations Act: Conference report to accompany HR 2055, 112th Cong., 1st sess. http://www.gpo. gov/fdsys/pkg/CRPT-112hrpt331/pdf/CRPT-112hrpt331.pdf (accessed May 6, 2013). Woolf, S. H. 2008. The meaning of translational research and why it matters. JAMA 299(2):211–213.

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34 THE CTSA PROGRAM AT NIH Yale School of Medicine. 2012a. Diabetes Endocrinology Research Center: Clinical trials. http://derc.yale.edu/cores/translational/clinicaltrials/index. aspx (accessed February 13, 2013). ———. 2012b. Diabetes Endocrinology Research Center: Diabetes translational core. http://derc.yale.edu/cores/translational/index.aspx (accessed February 13, 2013). Zerhouni, E. A. 2003. The NIH Roadmap. Science 302(5642):63–72. ———. 2005. Translational and clinical science—time for a new vision. New England Journal of Medicine 353(15):1621–1623. ———. 2006. Clinical and Translational Science Awards: A framework for a national research agenda. Translational Research 148(1):4–5.