Real-World Evidence Generation and Evaluation of Therapeutics

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. Table of Contents Front Matter 1 Introduction 2 Improving Evidence Generation for Decision Making on Approval and Use of New Treatments: Some Stakeholder Priorities 3 Opportunities for Real-World Data 4 Generating and Incorporating Real-World Evidence into Medical Product Development and Evaluation: Building from Successful Case Studies 5 Potential Strategies for a Way Forward Appendix A: Bibliography Appendix B: Workshop Agenda Appendix C: Participant Biographies Appendix D: Discussion Paper: Real-World Evidence to Guide theApproval and Use of New Treatments