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 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. 

During this meeting, experts will discuss lessons learned from case studies in which RWE was used for regulatory decision-making for medical products. Speakers will highlight FDA’s efforts with RWD/E to date and approaches to generate RWE to meet regulatory requirements. This convening fulfills both a PDUFA VII and a MDUFA V requirement.

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.