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Institute of Medicine (US). Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing An Agenda for 2020: Workshop Summary. Washington (DC): National Academies Press (US); 2012.

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Envisioning a Transformed Clinical Trials Enterprise in the United States: Establishing An Agenda for 2020: Workshop Summary.

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Appendix AWorkshop 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.

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 surveillance, 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 enterprise that is efficient and effective and fully integrated into the health delivery system of 2020. Define how the envisioned clinical 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

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 enterprise 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:
  • Suggest and discuss the key components of a transformed clinical trials enterprise incorporated into the integrated health delivery system of 2020.
  • Discuss the research outputs needed from the clinical trials enterprise to drive improved patient care and feed into a learning health system.
  • Consider broad transformational changes including the 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:
  • How can the national clinical trials enterprise fit into the envisioned learning health system?
  • What infrastructure is needed to embed randomized controlled trials in a learning health system?
  • What are the key barriers impeding integration of clinical trials into a learning health system (e.g., financial, cultural, regulatory)?
  • Where are the opportunities to address these barriers and build the needed infrastructure? What stakeholders/organizations/sectors should take the lead and how can their efforts best be coordinated?
 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:
  • Define a clinical trials “workforce” and identify workforce needs that will be necessary to support clinical trials.
  • Given the defined workforce, discuss the needs for career paths 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
2:15 p.m.Panel Discussion with Speakers: Developing and Sustaining a Clinical Trials Workforce
Objectives:
  • List key skills, techniques, and areas of expertise needed by the workforce.
  • What are the benchmarks and metrics of success for an effective clinical trials workforce?
  • Propose and discuss the core competencies of a clinical 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 Messages 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:
  • What are the needs for patient and broader public engagement in clinical trials?
  • Discuss broad as well as disease-specific efforts to partner 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?
  • How can models and best practices from the NIH Clinical 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

SESSION III. ALIGNING CULTURAL AND FINANCIAL INCENTIVES

Session Objective:
  • Consider institutional needs to support clinical trials including addressing cultural issues and aligning financial incentives to support 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:
  • Discuss incentives, reward structures, and business models impacting the conduct of clinical trials, and the key needs and challenges for advancing and improving upon these models.
  • Consider regulatory, financial, and cultural changes that 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
 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:
  • Set out an organizational framework for an infrastructure to conduct clinical trials.
  • Discuss:

    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
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:
  • Consider the types of trials that should be done in the United States and what the site/locus for the conduct of these trials should be within the infrastructure model proposed.
  • What are the roles and responsibilities of the various participants 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
 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:
  • Define the key steps and responsible parties needed to achieve the elements of a transformed clinical trials enterprise discussed during the workshop.
  • Consider the prioritization of transforming different elements of the clinical trials enterprise—what is feasible and most desirable?
  • Consider any unintended consequences of transforming the clinical trials enterprise.
  • Suggest a transformative path forward for decision makers and 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)
 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
Copyright © 2012, National Academy of Sciences.
Bookshelf ID: NBK114662

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