Appendix A

Workshop Agenda

Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020

November 7-8, 2011

20 F Street NW Conference Center 20 F Street NW Washington, DC 20001

Background:

There is increasing recognition that the clinical trials enterprise in the United States faces substantial challenges impeding the efficient and effective conduct of clinical and translational research needed to support the development of breakthrough medicines. A gap exists between the desired state where medical care in the United States is provided solely based on high-quality evidence and the reality of our limited ability to generate timely and practicable evidence. Eighty-five percent of clinical decisions in the United States are not supported by high-quality evidence. At the same time, U.S. clinical trials that generate medical evidence are becoming increasingly costly while experiencing greater setbacks. In addition, the shifting “footprint” of clinical trials toward sites outside the United States prompts questions about the generalizability and applicability of the results of those clinical trials to the U.S. population and represents a competitive challenge for the United States.



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Appendix A Workshop Agenda Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020 November 7-8, 2011 20 F Street NW Conference Center 20 F Street NW Washington, DC 20001 Background: There is increasing recognition that the clinical trials enterprise in the United States faces substantial challenges impeding the efficient and effec- tive conduct of clinical and translational research needed to support the development of breakthrough medicines. A gap exists between the desired state where medical care in the United States is provided solely based on high-quality evidence and the reality of our limited ability to generate timely and practicable evidence. Eighty-five percent of clinical decisions in the United States are not supported by high-quality evidence. At the same time, U.S. clinical trials that generate medical evidence are becom - ing increasingly costly while experiencing greater setbacks. In addition, the shifting “footprint” of clinical trials toward sites outside the United States prompts questions about the generalizability and applicability of the results of those clinical trials to the U.S. population and represents a competitive challenge for the United States. 85

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86 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE The limited ability of the nation’s clinical trials system to support drug development and evaluation exists within a broader context of a need for a “learning health system,” where “knowledge generation is so embedded into the core of the practice of medicine that it is a natural outgrowth and product of the health care delivery process and leads to continual improvement in care.” An essential component of such a learning health system is a robust and well-working clinical trials enterprise to support drug development; inform quality improvement; and support surveil- lance, international, and comparative effectiveness research. The IOM’s Forum on Drug Discovery, Development, and Translation has established an initiative to address challenges facing the U.S. clinical trials enterprise and to engage stakeholders in an open discussion of potentially transformative strategies to improve the efficiency and effectiveness of clinical trials. Workshop Objectives: • Frame the problem and discuss a vision for a clinical trials enter- prise that is efficient and effective and fully integrated into the health delivery system of 2020. Define how the envisioned clini - cal trials enterprise differs from the current system and suggest approaches to transform our current system into a learning system. • Consider the following core themes in framing an agenda to effect transformation of the U.S. clinical trials enterprise: — Providing a vision for a clinical trials enterprise in the health care system of 2020. — Developing a robust clinical trials workforce. — Aligning cultural and financial incentives. — Building an infrastructure to support a transformed clinical trials enterprise. • Workshop presentations and panel discussions will be supported and supplemented by discussion papers prepared by participants in Forum activities. Each of the four workshop sessions will be prefaced by presentations from discussion paper authors. NOVEMBER 7, 2011 8:30 a.m. Breakfast available 9:00 a.m. Welcome and Introductions Jeffrey Drazen, Workshop Chair Editor-in-Chief New England Journal of Medicine

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87 APPENDIX A SESSION I: FRAMING THE NEED FOR CHANGE: ENVISIONING A CLINICAL TRIALS ENTERPRISE IN THE HEALTH CARE SYSTEM OF 2020 Session Objectives: • Set a framework within which to establish a vision for 2020 that addresses the reinvention of the clinical trials enterprise around partnership with the institutional health care delivery system of 2020. • Given the anticipated changes in the structure of the U.S. health care system, consider how an efficient, effective clinical trials enter- prise can be integrated into the health care delivery system of 2020. alastair J.J. WooD, Session Chair Partner & Managing Director Symphony Capital 9:10 a.m. Presentation of Discussion Paper: The Clinical Trials Enterprise in the United States: A Call for Disruptive Innovation robert Califf Director, Duke Translational Medicine Institute Professor of Medicine Vice Chancellor for Clinical and Translational Research Duke University Medical Center Gary filerman President Atlas Health Foundation riCharD murray Vice President, Global Center for Scientific Affairs Office of the Chief Medical Officer Merck & Co., Inc. 10:00 a.m. Panel Discussion with Speakers: A Framework for the Clinical Trials Enterprise in the Health Care System of 2020 Objectives: • uggest and discuss the key components of a transformed S clinical trials enterprise incorporated into the integrated health delivery system of 2020.

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88 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE • iscuss the research outputs needed from the clinical D trials enterprise to drive improved patient care and feed into a learning health system. • onsider broad transformational changes including the C changing shape/structure of the pharmaceutical industry and the increasing globalization in clinical trials and drug development. alastair J.J. WooD, Panel Moderator Partner & Managing Director Symphony Capital neil Weissman President MedStar Health Research Institute ihor rak Vice President, Clinical Neuroscience AstraZeneca 10:45 a.m. BREAK 11:00 a.m. Keynote Address: The Learning Health System riCharD Platt Professor, Harvard Medical School Co-Chair, Clinical Effectiveness Research Innovation Collaboration IOM Roundtable on Value and Science-Driven Health Care 11:30 a.m. Discussion with Keynote Speaker, Panelists, and Workshop Participants: • ow can the national clinical trials enterprise fit into the H envisioned learning health system? • hat infrastructure is needed to embed randomized con- W trolled trials in a learning health system? • hat are the key barriers impeding integration of clini- W cal trials into a learning health system (e.g., financial, cultural, regulatory)? • here are the opportunities to address these barriers W and build the needed infrastructure? What stakeholders/ organizations/sectors should take the lead and how can their efforts best be coordinated?

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89 APPENDIX A Peter yu, Moderator Oncologist, Palo Alto Medical Foundation Chair, Electronic Health Record Working Group American Society of Clinical Oncology bryan luCe Senior Vice President, Science Policy United BioSource Corporation 12:15 p.m. LUNCH SESSION II: DEVELOPING A ROBUST CLINICAL TRIALS WORKFORCE Session Objectives: • efine a clinical trials “workforce” and identify workforce needs D that will be necessary to support clinical trials. • iven the defined workforce, discuss the needs for career paths G and career development opportunities. sherine Gabriel, Session Chair William J. and Charles H. Mayo Professor of Medicine & Epidemiology Mayo Medical School Co-Principal Investigator and Director of Education, Mayo Clinical Center for Translational Scientific Activities (CTSA) 1:15 p.m. Presentation of Discussion Paper: Developing a Robust Clinical Trials Workforce robert Califf Director, Duke Translational Medicine Institute Professor of Medicine Vice Chancellor for Clinical and Translational Research Duke University Medical Center elaine Gallin Principal QE Philanthropic Advisors miChael lauer Director, Division of Cardiovascular Sciences National Heart, Lung, and Blood Institute National Institutes of Health

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90 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE 2:15 p.m. Panel Discussion with Speakers: Developing and Sustaining a Clinical Trials Workforce Objectives: • ist key skills, techniques, and areas of expertise needed L by the workforce. • hat are the benchmarks and metrics of success for an W effective clinical trials workforce? • ropose and discuss the core competencies of a clinical P trials workforce. sherine Gabriel, Panel Moderator William J. and Charles H. Mayo Professor of Medicine & Epidemiology Mayo Medical School Co-Principal Investigator and Director of Education, Mayo Clinical Center for Translational Scientific Activities (CTSA) briGGs W. morrison Senior Vice President, Worldwide Medical Excellence Pfizer Inc. rebeCCa JaCkson Professor and Associate Dean for Clinical Research Director and Principal Investigator, OSU Center for Clinical and Translational Science The Ohio State University 3:00 p.m. BREAK 3:15 p.m. Keynote Address: Sustaining Institutional Support and Patient Engagement in Clinical Trials: Models and Mes- sages from the NIH Clinical Center John Gallin Director National Institutes of Health Clinical Center 3:45 p.m. Discussion with Keynote Speaker, Panelists, and Workshop Participants: • hat are the needs for patient and broader public engage- W ment in clinical trials?

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91 APPENDIX A • iscuss broad as well as disease-specific efforts to part- D ner with patients in the development and conduct of clinical trials. How might these partnerships help create a national culture of clinical research in the United States? • ow can models and best practices from the NIH Clinical H Center be adapted to the realities of other institutions and community systems to advance and promote the conduct of clinical trials in those systems? Janet tobias, Moderator Co-founder and Partner, Ikana Health Adjunct Assistant Professor, Mount Sinai School of Medicine annetine GeliJns Professor of Health Policy Co-Chair, Department of Health Evidence and Policy Mount Sinai School of Medicine heather snyDer Senior Associate Director, Scientific Grants Medical and Scientific Relations Alzheimer’s Association 4:30 p.m. ADJOURN Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing an Agenda for 2020 NOVEMBER 8, 2011 8:30 a.m. Breakfast available 9:00 a.m. Welcome and Introductions Jeffrey Drazen, Workshop Chair Editor-in-Chief New England Journal of Medicine

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92 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE SESSION III: ALIGNING CULTURAL AND FINANCIAL INCENTIVES Session Objective: • onsider institutional needs to support clinical trials including C addressing cultural issues and aligning financial incentives to sup- port and sustain an efficient clinical trials enterprise. arthur h. rubenstein, Session Chair Professor, Department of Medicine, Division of Endocrinology Raymond and Ruth Perelman School of Medicine University of Pennsylvania 9:10 a.m. Presentation of Discussion Paper: Transforming the Economics of Clinical Trials JuDith kramer Associate Professor of Medicine, Duke University Medical Center Executive Director, Clinical Trials Transformation Initiative (CTTI) kevin sChulman Professor of Medicine and Business Administration Duke University School of Medicine and The Fuqua School of Business 9:50 a.m. Panel Discussion with Speakers: Aligning Cultural and Financial Incentives to Support Clinical Trials Objectives: • iscuss incentives, reward structures, and business D models impacting the conduct of clinical trials, and the key needs and challenges for advancing and improving upon these models. • onsider regulatory, financial, and cultural changes that C are needed to improve the environment for clinical trials. arthur h. rubenstein, Panel Moderator Professor, Department of Medicine, Division of Endocrinology Raymond and Ruth Perelman School of Medicine University of Pennsylvania

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93 APPENDIX A riCharD ruDiCk Hazel Prior Hostetler Chair of Neurology, Cleveland Clinic Professor of Medicine, Cleveland Clinic Lerner College of Medicine Case Western Reserve University ChristoPher bearDmore Chief Executive Officer Translational Research Management sCott steele Director, Research Alliances Adjunct Associate Professor, Community and Preventive Medicine University of Rochester 10:35 a.m. BREAK SESSION IV: BUILDING AN INFRASTRUCTURE TO SUPPORT A TRANSFORMED CLINICAL TRIALS ENTERPRISE Session Objectives: • et out an organizational framework for an infrastructure to con- S duct clinical trials. • iscuss: D — organizational frameworks for the infrastructure (i.e., network models or other approaches); — informatics needs; — regulatory reform or harmonization both within the United States and internationally. ClyDe yanCy, Session Chair Magerstadt Professor of Medicine Chief, Division of Cardiology Feinberg School of Medicine Northwestern University

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94 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE 10:55 a.m. Presentation of Discussion Paper: Developing a Clinical Trials Infrastructure Petra kaufmann Director, Office of Clinical Research National Institute of Neurological Disorders and Stroke National Institutes of Health Paul eisenberG Senior Vice President Global Regulatory Affairs and Safety Amgen Inc. Janet WooDCoCk Director, Center for Drug Evaluation and Research U.S. Food and Drug Administration 11:45 a.m. Panel Discussion with Speakers: Establishing an Infrastructure to Support a Transformed Clinical Trials Enterprise Objectives: • onsider the types of trials that should be done in the C United States and what the site/locus for the conduct of these trials should be within the infrastructure model proposed. • hat are the roles and responsibilities of the various par- W ticipants and stakeholders in the proposed infrastructure of the clinical trials enterprise? ClyDe yanCy, Panel Moderator Magerstadt Professor of Medicine Chief, Division of Cardiology Feinberg School of Medicine Northwestern University louis fiore Principal Investigator, VA Cooperative Studies Program Director, VA Cooperative Studies Program Coordinating Center, Boston Department of Veterans Affairs miChael kinG Jolly Senior Vice President Quintiles Innovation

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95 APPENDIX A karen marGolis Professor of Medicine, University of Minnesota Medical School Director of Clinical Research HealthPartners Research Foundation 12:30 p.m. LUNCH SESSION V: DEVELOPING AN AGENDA FOR THE CREATION OF A TRANSFORMED CLINICAL TRIALS ENTERPRISE Session Objectives: • efine the key steps and responsible parties needed to achieve the D elements of a transformed clinical trials enterprise discussed dur- ing the workshop. • onsider the prioritization of transforming different elements of C the clinical trials enterprise—what is feasible and most desirable? • onsider any unintended consequences of transforming the clini- C cal trials enterprise. • uggest a transformative path forward for decision makers and S those implementing the work of clinical trials across the enterprise. 1:30 p.m. Jeffrey Drazen, Session Chair Editor-in-Chief New England Journal of Medicine Presentation of Key Themes/Suggested Paths from Session Chairs alastair J.J. WooD, Session I Chair Partner & Managing Director Symphony Capital sherine Gabriel, Session II Chair William J. and Charles H. Mayo Professor of Medicine & Epidemiology Mayo Medical School Co-Principal Investigator and Director of Education, Mayo Clinical Center for Translational Scientific Activities (CTSA)

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96 ENVISIONING A TRANSFORMED CLINICAL TRIALS ENTERPRISE arthur h. rubenstein, Session III Chair Professor, Department of Medicine, Division of Endocrinology Raymond and Ruth Perelman School of Medicine University of Pennsylvania ClyDe yanCy, Session IV Chair Magerstadt Professor of Medicine Chief, Division of Cardiology Feinberg School of Medicine Northwestern University 2:30 p.m. Reflecting on Potential Paths Forward: Setting an Agenda for a Transformed Clinical Trials Enterprise Jean rouleau Scientific Director Institute for Circulatory and Respiratory Health Canadian Institutes of Health Research (CIHR) Jeffrey Drazen, Panel Moderator Editor-in-Chief New England Journal of Medicine DouGlas CroPPer President and Chief Executive Officer Genesis Health System lynn ethereDGe Co-Founder Health Insurance Reform Project and Rapid Learning Project George Washington University ronalD krall Associate Fellow University of Pennsylvania, Center for Bioethics James DoroshoW Director, Division of Cancer Treatment and Diagnosis National Cancer Institute, National Institutes of Health 4:30 p.m. ADJOURN