A

Data Sources and Methods

The Institute of Medicine (IOM) Committee to Review the Clinical and Translational Science Awards (CTSA) Program at the National Center for Advancing Translational Sciences (NCATS) was tasked with providing an independent appraisal of the CTSA Program. The specific goals of this congressionally requested review were to assess the CTSA Program’s mission and goals and to explore the contributions of the CTSA Program in accelerating the development of new therapeutics, in facilitating disease-specific research and children’s health research, and in enhancing the integration of programs funded by the National Institutes of Health (NIH) institutes and centers. In conducting its work and responding to the statement of task, the IOM committee reviewed information that was collected from a variety of sources, including scientific literature, previous evaluations and progress reports, open-session meetings and conference calls, public testimony and input, and other publicly available resources.

COMMITTEE EXPERTISE

The study committee comprised 13 individuals 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. Appendix B provides biographical sketches of each of the committee members. The committee’s expertise was supplemented by the



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A Data Sources and Methods The Institute of Medicine (IOM) Committee to Review the Clinical and Translational Science Awards (CTSA) Program at the National Cen- ter for Advancing Translational Sciences (NCATS) was tasked with providing an independent appraisal of the CTSA Program. The specific goals of this congressionally requested review were to assess the CTSA Program’s mission and goals and to explore the contributions of the CTSA Program in accelerating the development of new therapeutics, in facilitating disease-specific research and children’s health research, and in enhancing the integration of programs funded by the National Insti- tutes of Health (NIH) institutes and centers. In conducting its work and responding to the statement of task, the IOM committee reviewed infor- mation that was collected from a variety of sources, including scientific literature, previous evaluations and progress reports, open-session meet- ings and conference calls, public testimony and input, and other publicly available resources. COMMITTEE EXPERTISE The study committee comprised 13 individuals with expertise in community outreach and engagement, public health and health policy, bioethics, education and training, pharmaceutical research and develop- ment, program evaluation, clinical and biomedical research, and child health research, along the full continuum of clinical and translation re- search. Appendix B provides biographical sketches of each of the com- mittee members. The committee’s expertise was supplemented by the 147

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148 THE CTSA PROGRAM AT NIH knowledge and insights of a number of experts who presented research during open-session meetings and conference calls. OPEN-SESSION MEETINGS, CONFERENCE CALLS, AND PUBLIC INPUT Between October 2012 and February 2013, the committee convened three open-session meetings and four open-session conference calls (Boxes A-1 to A-7). The committee’s first meeting in October was held remotely via conference call because of weather conditions associated with Hurricane Sandy. Over the course of the study, the committee also held a number of closed-session conference calls and a closed-session meeting in March 2013 to conclude its deliberations. The open-session meetings and calls allowed the committee to hear from a wide range of stakeholders, including a number of CTSA principal investigators and researchers, members of the NCATS and NIH leadership, community and patient advocacy organizations, industry partners and representa- tives, and thought leaders and researchers in the clinical and translational sciences arena who were not connected to the CTSA Program. Each of the open-session meetings included a public comment period that allowed the committee to hear from other researchers, stakeholders, and members of the public. Because of budget constraints, all of the open-session meetings were held in Washington, DC. To provide addi- tional opportunities for public input by individuals who were unable to travel to the meetings or participate by conference call, the committee used an online public input tool with questions to guide further testimony and input. A link to the public input tool was made available on the IOM’s website from December 2012 through March 2013. The NCATS and IOM study listservs were used to notify stakeholders and the public about the opportunity to provide additional input to the committee’s work and the availability of the online tool. The list of questions included in the public input tool can be found in Box A-8. During the 3 months that the tool was available, 27 individuals submitted responses to the questions. This input was catalogued in the study’s public access file and is available by request through the National Academies’ Public Access Records Office. The committee also reviewed input submitted through its e-mail address, CTSAReview@nas.edu, throughout the duration of the study.

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APPENDIX A 149 INFORMATION GATHERING AND DOCUMENT REVIEW In addition to information that was gathered during the open-session meetings and conference calls, the committee conducted a review of the available scientific literature with a focus on areas related to the CTSA Program and its work in clinical and translational sciences, training and education, community engagement, and child health research. The com- mittee also reviewed previous evaluations of the CTSA Program, includ- ing the reports from the 3-year Westat evaluation and the evaluation of the administration of the CTSA Program under the NIH’s National Cen- ter for Research Resources that was conducted by the Department of Health and Human Services’ Office of the Inspector General; progress reports developed by the CTSA Consortium committees and the NIH; responses to the NIH’s requests for information related to the CTSA Program; a wealth of information provided by NCATS, a range of CTSA Consortium committees, and stakeholder groups; and results and recom- mendations from other working groups and stakeholder meetings that have considered the future directions of the CTSA Program. Any infor- mation that was provided to the committee from outside sources was cat- alogued in the study’s public access file and is available by request through the National Academies’ Public Access Records Office. BOX A-1 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translational Sciences 500 Fifth Street, NW Washington, DC Monday, October 29, 2012 Agenda 9:30 – 9:45 Welcome and Introductions Alan Leshner, Committee Chair Sharon Terry, Committee Vice-Chair 9:45 – 11:30 Charge to the Committee and Discussion of Statement of Task Chris Austin, Director, NCATS, NIH Josie Briggs, Acting Director, Division of Clinical Innovation, NCATS, NIH

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150 THE CTSA PROGRAM AT NIH 11:30 – 12:30 Lunch Break 12:30 – 2:30 Overview of CTSA Committees and Strategic Goals CTSA Consortium Steering and Executive Committees Bradley Evanoff, Washington University Goal 1: National Clinical and Translational Research Capability Clay Johnston, University of California, San Francisco Goal 2: Training and Career Development of Clinical/Translational Scientists Robert Toto, University of Texas Southwestern Medical Center Goal 3: Consortium-Wide Collaborations Anantha Shekhar, Indiana University Goal 4: Health of Our Communities and the Nation Kathleen Brady, Medical University of South Carolina Goal 5: Advance T1 Translational Research Nora Disis, University of Washington Q&A with Panelists and Committee Discussion 2:30 – 2:45 Break 2:45 – 4:45 Enhancing Integration: CTSAs and NIH Institutes and Centers National Cancer Institute Linda Weiss, Director, Office of Cancer Centers, NCI, NIH National Heart, Lung, and Blood Institute Susan Shurin, Deputy Director, NHLBI, NIH National Institute of Allergy and Infectious Diseases Hugh Auchincloss, Deputy Director, NIAID, NIH National Institute of Diabetes and Digestive and Kidney Diseases Gregory G. Germino, Deputy Director, NIDDK, NIH National Institute of Neurological Disorders and Stroke Walter J. Koroshetz, Deputy Director, NINDS, NIH Q&A with Panelists and Committee Discussion 4:45 Public Comment Period 5:00 Adjourn

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APPENDIX A 151 BOX A-2 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translational Sciences Conference Call – November 19, 2012 11 a.m. to Noon (Eastern) Agenda 11:00 – 12:00 Open Session  Nora Volkow, NIDA  Steve Katz, NIAMS BOX A-3 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translational Sciences Conference Call – November 30, 2012 10:30 a.m. to Noon (Eastern) Agenda 10:30 – 12:00 Open Session  Steve Hirschfield, NICHD  Dianne Murphy, FDA  Charles Thompson, Pfizer Inc.  Ron Portman, Bristol-Meyers Squibb  Phil Pizzo, Stanford University BOX A-4 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translational Sciences National Academy of Sciences Building 2101 Constitution Avenue, NW Washington, DC Wednesday, December 12, 2012 Agenda 8:00 – 8:05 Welcome and Opening Remarks Alan Leshner, Chair

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152 THE CTSA PROGRAM AT NIH 8:05 – 8:20 CTSA Overview – Goals of the Program Tom Insel, National Institute of Mental Health, NIH 8:20 – 9:50 Panel 1: Translation of Basic Science to Human Studies: Advancing T1 and T2 Research Facilitator: Cliff Rosen 8:20 – 8:25 Panel Introductions 8:25 – 8:35 CTSA Perspective Garret FitzGerald, University of Pennsylvania 8:35 – 8:45 CTSA Perspective Sundeep Khosla, Mayo Clinic 8:45 – 8:55 Community Perspective Bernard Ewigman, University of Chicago 8:55 – 9:05 Industry Perspective Jacqueline B. Fine, Merck Research Laboratories 9:05 – 9:50 Discussion with the Committee 9:50 – 10:00 Break 10:00 – 11:30 Panel 2: Children’s Health Research: Role of the CTSA Program Facilitators: Meg McCabe and Phyllis Dennery 10:00 – 10:05 Panel Introductions 10:05 – 10:15 CTSA Consortium Child Health Oversight Committee Jonathan Davis, Tufts University 10:15 – 10:25 CTSA Perspective Margaret Grey, Yale School of Nursing 10:25 – 10:35 CTSA Perspective Terence Flotte, University of Massachusetts 10:35 – 10:45 Community Perspective Susan Weiner, Children’s Cause for Cancer Advocacy 10:45 – 11:30 Discussion with the Committee 11:45 – 12:30 Lunch 12:30 – 1:45 Panel 3: Collaborations Across CTSAs: Current Status and Future Plans Facilitator: Sue Curry 12:30 – 12:35 Panel Introductions

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APPENDIX A 153 12:35 – 12:45 CTSA Coordinating Center (C4) Overview Gordon Bernard, Vanderbilt University 12:45 – 12:55 Collaborations on Informatics Paul Harris, Vanderbilt University 12:55 – 1:05 CTSA Perspective Marc Drezner, University of Wisconsin 1:05 – 1:15 Community Perspective Mickey Eder, Access Community Health Network, Chicago 1:15 – 1:45 Discussion with the Committee 1:45 – 3:00 Panel 4: Evaluating the CTSA Program Facilitator: Robin Kelley 1:45 – 1:50 Panel Introductions 1:50 – 2:00 Westat Evaluations Joy Frechtling, Westat 2:00 – 2:10 NCATS Request for Information Josie Briggs, NCATS, NIH 2:10 – 2:20 NCATS/CTSA Integration Working Group Steve Katz, NIAMS, NIH 2:20 – 2:30 Evaluating Large Scale Programs— Frameworks and Considerations David Chavis, Community Science 2:30 – 3:00 Discussion with the Committee 3:00 – 3:15 Break 3:15 – 4:45 Panel 5: Roundtable Discussion: Future Directions for the Mission and Goals of the CTSA Program 3:15 – 3:20 Roundtable Introductions 3:20 – 4:00 Opening Comments on the CTSA Program Tachi Yamada, Takeda Pharmaceuticals (via conference call) Wylie Burke, University of Washington (via conference call) John Adams, University of California, Los Angeles 4:00 – 4:45 Roundtable and Committee Discussion 4:45 – 5:30 Public Comment Period 5:30 Adjourn

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154 THE CTSA PROGRAM AT NIH BOX A-5 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translational Sciences 20F Conference Center 20 F Street, NW Washington, DC Thursday, January 24, 2013 Agenda 8:00 – 8:05 Welcome and Opening Remarks Alan Leshner, Chair 8:05 – 8:45 Opening Speaker Chris Austin, NCATS 8:45 – 10:10 Panel 1: Training and Education Facilitator: Cliff Rosen 8:45 – 8:50 Panel Introductions 8:50 – 9:30 Panel Presentations David Shackelford, University of California Los Angeles Lisa Ceglia, Tufts University Kelly Edwards, University of Washington Cynthia Morris, Oregon Health and Science University 9:30 – 10:10 Discussion with the Committee 10:10 – 10:20 Break 10:20 – 11:45 Panel 2: Engaging Community Organizations and Patient Advocacy Groups Facilitators: Sharon Terry and Susan Axelrod 10:20 – 10:25 Panel Introductions 10:25 – 11:05 Panel Presentations Donna Appell, Hermansky-Pudlak Syndrome Network Inc. Bray Patrick-Lake, Clinical Trials Transformation Initiative

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APPENDIX A 155 Sarena Seifer, Community-Campus Partnerships for Health Joan Reede, Harvard University 11:05 – 11:45 Discussion with the Committee 11:45 – 12:30 Lunch Break 12:30 – 2:00 Panel 3 Advancing Research on Clinical Practice and Population Health: T3 and T4 Research Facilitator: Ann Bonham 12:30 – 12:35 Panel Introductions 12:35 – 1:15 Panel Presentations Lloyd Michener, Duke University Joe Selby, PCORI (Patient-Centered Outcomes Research Institute) Leonard Sacks, FDA John Steiner, Kaiser Permanente Colorado 1:15 – 2:00 Discussion with the Committee 2:00 – 3:15 Panel 4: Future Directions for Using CTSA Programs and Resources Facilitator: Ralph Horwitz 2:00 – 2:05 Panel Introductions 2:05 – 2:35 Panel Presentations Robert Califf, Duke University Martha Curley, University of Pennsylvania Edith Parker, University of Iowa 2:35 – 3:15 Discussion with the Committee 3:15 – 3:30 Break 3:30 – 5:00 Panel 5: Roundtable Discussion: Future Directions for the Mission and Goals of the CTSA Program Facilitator: Edith Perez 3:30 – 3:35 Roundtable Introductions 3:35 – 4:05 Opening Comments on the CTSA Program Morrie Schambelan, University of California, San Francisco Stephen Thomas, University of Maryland Gigi Hirsch, Massachusetts Institute of Technology

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156 THE CTSA PROGRAM AT NIH 4:05 – 5:00 Roundtable and Committee Discussion 5:00 – 5:30 Public Comment Period 5:30 Adjourn BOX A-6 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translational Sciences Conference Call – January 30, 2013 12:30 to 2:00 p.m. (Eastern) Agenda 12:30 – 2:00 Open Session 12:35 – 12:40 Welcoming Remarks Alan Leshner, Committee Chair 12:40 – 12:50 Petra Kaufmann, NINDS, NIH 12:50 – 1:00 Committee Q&A 1:00 – 1:20 Discussion with Francis Collins, NIH 1:20 – 1:30 Eric Topol, Scripps Research Institute 1:30 – 2:00 Committee Q&A BOX A-7 Committee to Review the Clinical and Translational Science Awards Program at the National Center for Advancing Translational Sciences Conference Call – February 27, 2013 3:30 to 4:30 p.m. (Eastern) Agenda 3:30 – 4:30 Open Session Discussion with Chris Austin, Director of NCATS

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APPENDIX A 157 BOX A-8 Public Input Questions Mission  Is the mission of the CTSA Program clear and appropriate for defining the success of the program and for supporting the mission of NCATS?  Is the scope of the mission realistic given the available resources, sup- port, and infrastructure?  Is the mission being disseminated adequately? Are potential stakehold- ers aware of the resources available through the CTSA Program, and are there barriers to the use of those resources? Strategic Goals  Are the strategic goals of the CTSA Program clear and appropriate? Do they clarify the purpose and mission of the CTSA Program?  Are the strategic goals realistic given the available resources, support, and infrastructure?  Are the strategic goals being disseminated adequately?  Do you have suggestions for refocusing and revising the strategic goals of the CTSA Program? Role of the CTSA  Since the inception of the CTSA Program, have the CTSA institutions, individually and collectively, played an appropriate and adequate role in: (please check the boxes where you believe the CTSA Program has played an adequate and appropriate role) o accelerating the development of new therapeutics o facilitating disease-specific research o facilitating children’s health and pediatric research o enhancing the integration of research funded by the NIH institutes and centers o involving and interacting with community organizations and patient advocacy groups?  Could the mission and strategic goals be improved to address these is- sues? If so, how should they be improved? Continuum of Research  Please comment on the balance of CTSA Program efforts across the continuum of research from first-phase studies in humans to clinical tri- als to population-based research on health outcomes and comparative effectiveness.  Does the balance need to shift? Why or why not? Successes, Challenges, and Future Directions  What do you see as successes, challenges, and future directions of the CTSA Program? Additional Comments

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