Real-World Evidence: Efficacy Assessments Await FDA Clarity, Pilot Projects

Greater development and use of real-world evidence, such as pragmatic clinical trials and observational studies, to support regulatory decisions on drug efficacy are limited by regulatory, technical and practical hurdles, stakeholders say.

Demonstration projects and clarity around FDA expectations are needed before "real-world evidence" on drug effectiveness can be reliably and routinely used for regulatory decision-making, industry, government, academic and other stakeholders said at a March 3-4 meeting convened by the Duke-Margolis Center for Health Policy.

Real-world evidence, such a pragmatic clinical trials and observational studies, could offer biopharma sponsors a quicker, less expensive means for obtaining new

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