Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes

There are emerging opportunities to leverage real world data (RWD) and resultant real-world evidence (RWE) in support of supplemental approval or labeling actions based on substantial evidence of effectiveness as envisioned in 21st Century Cures and PDUFA VI. As part of implementation efforts for this legislation, the U.S. Food and Drug Administration (FDA) published a strategic framework to guide the development of a new program for regulatory uses of RWD and RWE. The Framework suggests the potential integration of clinical trials into the healthcare system by using randomized designs to generate RWE for regulatory submissions. 

On July 11, 2019 and July 12, 2019, under a cooperative agreement with the FDA, the Robert J. Margolis, MD, Center for Health Policy at Duke University will convene a public workshop to bring the stakeholder community together to explore key considerations for utilizing randomized designs, such as large simple trials or those that incorporate pragmatic elements, and real-world data (RWD) to generate real-world evidence RWE. Discussion will focus on key components of trial design including intervention selection, outcome measurement, blinding, and study population characteristics as well as important regulatory considerations. The public workshop is a part of the ongoing efforts at the Agency to explore the utility of RWE and will directly inform the development of the strategic framework.