Excerpt

Recognition is growing that the clinical trials enterprise (CTE) in the United States faces substantial challenges impeding the efficient and effective conduct of clinical research to support the development of new medicines and evaluate existing therapies. A gap has been identified between the desired state where medical care in the United States is provided solely based on high-quality evidence—and the reality—where we have limited ability to generate timely and practical evidence. There have increasingly been calls for transformation of the CTE in the United States to support the efficient development of breakthrough medicines and interventions and the evidence needed for health care decision making. Leaders in research and health care convened to discuss this visionary quest at a 2-day workshop held in November 2011 by the Institute of Medicine (IOM) Forum on Drug Discovery, Development, and Translation.

The workshop focused primarily on one type of clinical investigation, randomized controlled trials (RCTs)

Contents

• THE NATIONAL ACADEMIES
• PLANNING COMMITTEE FOR THE WORKSHOP SERIES ON IMPROVING THE CLINICAL TRIAL PROCESS IN THE UNITED STATES
• FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION
• Reviewers
• Acronyms
• 1. Introduction
  • DEFINING THE PROBLEM
  • LINKING THE TRANSFORMATION OF THE CLINICAL TRIALS ENTERPRISE AND IMPROVING QUALITY OF CARE
  • PURPOSE AND STRUCTURE OF THE WORKSHOP
  • ORGANIZATION OF THE REPORT
• 2. Integrating Community Practice and Clinical Trials
  • A CALL FOR DISRUPTIVE INNOVATION IN THE CLINICAL TRIALS ENTERPRISE
  • A FRAMEWORK FOR THE CLINICAL TRIALS ENTERPRISE IN THE HEALTH CARE SYSTEM OF 2020
  • THE LEARNING HEALTH SYSTEM
• 3. Improving Public Participation in Clinical Trials
  • DEVELOPING A ROBUST CLINICAL TRIALS WORKFORCE
  • OPPORTUNITIES TO CREATE A SUSTAINABLE WORKFORCE FOR THE CLINICAL TRIALS ENTERPRISE
  • SUSTAINING INSTITUTIONAL SUPPORT AND PATIENT ENGAGEMENT IN CLINICAL TRIALS
• 4. Creating a New Business Model for Clinical Trials
  • TRANSFORMING THE ECONOMICS OF CLINICAL TRIALS
  • IDENTIFYING OPPORTUNITIES TO IMPROVE REGULATORY FRAMEWORKS FOR CLINICAL TRIALS
  • OPPORTUNITIES FOR ALIGNING CULTURAL AND FINANCIAL INCENTIVES IN CLINICAL TRIALS
• 5. Building an Infrastructure to Support Clinical Trials
  • GAPS AND CHALLENGES IN THE CURRENT CLINICAL TRIALS INFRASTRUCTURE
  • ESTABLISHING AN INFRASTRUCTURE TO SUPPORT A TRANSFORMED CLINICAL TRIALS ENTERPRISE
• 6. Suggesting an Agenda for Transforming Elements of the Clinical Trials Enterprise
  • REFLECTING ON POTENTIAL PATHS FORWARD
  • BUILDING A FRAMEWORK AND SUGGESTING AN AGENDA
• References
• APPENDIXES
  • Appendix A Workshop Agenda
  • Appendix B Participant Biographies
  • Appendix C Registered Workshop Attendees
  • Appendix D Discussion Paper: The Clinical Trials Enterprise in the United States: A Call for Disruptive Innovation
  • Appendix E Discussion Paper: Developing a Robust Clinical Trials Workforce
  • Appendix F Discussion Paper: Transforming the Economics of Clinical Trials
  • Appendix G Discussion Paper: Developing a Clinical Trials Infrastructure
  • Appendix H Discussion Paper: Canadian Strategy on Patient-Oriented Research
  • Appendix I Discussion Paper: Health Research as a Public Good
  • Appendix J Discussion Paper: Novel Ways to Get Good Trial Data: The UK Experience
  • Appendix K IOM Staff Paper: Context and Glossary of Select Terms Associated with the Clinical Trials Enterprise

Rapporteurs: Neil Weisfeld, Rebecca A. English, Anne B. Claiborne

This study was supported by contracts between the National Academy of Sciences and Department of Health and Human Services (Contract Nos. N01-OD-4-2139 TO #158 and HHSF223001003T), American Society for Microbiology, Amgen Inc., Association of American Medical Colleges, Bristol-Myers Squibb, Burroughs Wellcome Fund, Celtic Therapeutics, LLLP, Critical Path Institute, Doris Duke Charitable Foundation, Eli Lilly & Co., FasterCures, Foundation for the NIH, Friends of Cancer Research, GlaxoSmithKline, Janssen Research & Development, LLC, Merck & Co., Inc., Novartis Pharmaceuticals Corporation, and Pfizer Inc.

Suggested citation:

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

The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the project.

NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine.