Regulatory Submissions with Real-World Evidence: Successes, Challenges, and Lessons Learned

The 21st Centuries Cure Act of 2016 focused on accelerating medical product development to deliver innovations faster and more efficiently to patients. Real-world data (RWD) and real-world evidence (RWE) are integral parts of this effort.  The current version of both the Prescription Drug User Fee Act (PDUFA VII) and the Medical Device User Fee Act (MDUFA V) contain several mandates for the U.S. Food and Drug Administration (FDA) surrounding RWD/E. The PDUFA VII mandates include convening a public workshop or meeting to discuss RWE examples, and the MDUFA V mandates include updating stakeholders on the RWE program activities at two or more open public meetings. 

On September 23, 2025, the FDA, under a cooperative agreement with Duke-Margolis Institute for Health Policy, hosted a public meeting to advance dialogue on the use of RWD/E in regulatory submissions for medical products. The convening fulfilled commitments under PDUFA VII and MDUFA V and featured FDA leaders, industry sponsors, and researchers who shared insights from recent RWE-supported approvals, highlighted ongoing Agency initiatives, and discussed best practices for generating robust, real-world evidence. Case studies across drugs, biologics, and devices underscored how rigorously designed RWD studies can provide substantial evidence of effectiveness, particularly for rare diseases. Panelists also examined common challenges in RWE generation, including study design considerations, bias mitigation, and data access. A forward-looking fireside chat explored opportunities to expand RWE use across federal agencies, improve data interoperability, and integrate emerging AI and machine learning tools. Overall, the meeting emphasized the evolving role of RWE in FDA decision-making and the importance of transparency, collaboration, and continued public engagement in advancing the RWE ecosystem.

This event is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award U01FD008451 totaling $1,399,999 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.