Examining the Impact of Real-World Evidence on Medical Product Development

Randomized controlled trials (RCTs) have traditionally served as the gold standard for generating evidence about medical interventions. However, RCTs have inherent limitations and may not reflect the use of medical products in the real world. Additionally, RCTs are expensive, time consuming, and cannot answer all questions about a product or intervention. Evidence generated from real-world use, such as real-world evidence (RWE) may provide valuable information, alongside RCTs, to inform medical product decision making.

To explore the potential for using RWE in medical product decision making, the National Academies of Sciences, Engineering, and Medicine planned a three-part workshop series. The series was designed to examine the current system of evidence generation and its limitations, to identify when and why RWE may be an appropriate type of evidence on which to base decisions, to learn from successful initiatives that have incorporated RWE, and to describe barriers that prevent RWE from being used to its full potential. This publication summarizes the discussions from the entire workshop series.

Table of Contents

• Front Matter
• 1 Introduction
• 2 Perspectives on Real-World Evidence
• 3 Learning from Success
• 4 Barriers and Disincentives to the Use of Real-World Evidence and Real-World Data
• 5 Getting Unstuck: Mythbusting the Current System
• 6 When Is a Real-World Data Element Fit for Assessment of Eligibility, Treatment Exposure, or Outcomes?
• 7 How Tightly Should Investigators Attempt to Control or Restrict Treatment Quality in a Pragmatic or Real-World Trial?
• 8 Obscuring Intervention Allocation in Trials to Generate Real-World Evidence: Why, Who, and How?
• 9 Gaining Confidence in Observational Comparisons
• 10 Looking Ahead
• References
• Appendix A: Related Resources
• Appendix B: Workshop One Agenda
• Appendix C: Workshop Two Agenda
• Appendix D: Workshop Three Agenda
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