Skip to Main Content

Evaluating the Promises and Perils of Real-World Evidence in Regulatory Decision-Making

Feasibility of Using Real-world Data to Emulate Postapproval Confirmatory Clinical Trials of Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval (Published in JAMA Network Open, Nov 2021) - Joshua D. Wallach, Audrey D. Zhang, MD, Joshua J. Skydel, MD, Victoria L. Bartlett, BA, Sanket S. Dhruva, MD, MHS, Nilay D. Shah, PhD, Joseph S. Ross, MD, MHS

  • The authors examined the feasibility of using real-world data (RWD) to emulate FDA-required postapproval confirmatory trials for all new drugs that received accelerated approval between 2009 and 2018. They found that none of the 50 FDA-required postapproval confirmatory trials granted accelerated approval during this time period could have been feasibly emulated during the real-world data sources of claims or structured electronic health record data.