The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients' lives through measurement of lifestyle changes.

In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.

Contents

• The National Academies of SCIENCES • ENGINEERING • MEDICINE
• PLANNING COMMITTEE ON REAL-WORLD EVIDENCE GENERATION AND EVALUATION OF THERAPEUTICS: A WORKSHOP
• FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION
• Reviewers
• Acronyms and Abbreviations
• 1. Introduction
  • ORGANIZATION OF THE PROCEEDINGS OF A WORKSHOP
• 2. Improving Evidence Generation for Decision Making on Approval and Use of New Treatments: Some Stakeholder Priorities
  • THE CURRENT LANDSCAPE FOR EVIDENCE GENERATION PROCESSES
  • STAKEHOLDER PERSPECTIVES ON PRIORITIES FOR IMPROVING REAL-WORLD DECISION MAKING
  • POTENTIAL CROSSCUTTING PRIORITIES FOR IMPROVING REAL-WORLD DECISION MAKING
• 3. Opportunities for Real-World Data
  • LEVERAGING ELECTRONIC HEALTH RECORDS
  • THE POWER OF LINKING AND MINING DISPARATE DATA SOURCES
  • COLLECTING REAL-WORLD DATA OUTSIDE THE CLINICAL SETTING USING DIGITAL HEALTH TOOLS
  • CONSIDERATIONS FOR REALIZING THE POTENTIAL OF REAL-WORLD DATA
• 4. Generating and Incorporating Real-World Evidence into Medical Product Development and Evaluation: Building from Successful Case Studies
  • RANDOMIZATION IN THE CLINICAL SETTING
  • RESEARCH EMBEDDED IN REGISTRIES
  • POPULATION-BASED SURVEILLANCE USING HEALTH SYSTEM DATA
• 5. Potential Strategies for a Way Forward
  • DATA AVAILABILITY
  • STUDY DESIGN, DATA METHODS, AND DATA QUALITY
  • INCENTIVES
  • SOME PRACTICAL NEXT STEPS
• APPENDIXES
  • Appendix A. Bibliography
  • Appendix B. Workshop Agenda
  • Appendix C. Participant Biographies
  • Appendix D. Discussion Paper: Real-World Evidence to Guide the Approval and Use of New Treatments
    Steven Galson and Gregory Simon.

Rapporteurs: Anne B. Claiborne, Amanda Wagner Gee, and Autumn Downey.

Suggested citation:

National Academies of Sciences, Engineering, and Medicine. 2017. Real-world evidence generation and evaluation of therapeutics: Proceedings of a workshop. Washington, DC: The National Academies Press. doi: https://doi.org/10.17226/24685.