FDA Eliminates Major Barrier to Using Real-World Evidence in Drug and Device Application Reviews

The U.S. Food and Drug Administration (FDA) today removed a key limitation on the use of real-world evidence (RWE) used in drug and device applications reviews.  In new guidance for certain types of medical device submissions, the agency states it will accept RWE without requiring that identifiable individual patient data collected from real-world data sources always be submitted in a marketing submission.  The FDA similarly intends to consider updating its guidance for drugs and biologics.  While RWE has been promoted as an advance in regulatory policy, past agency expectations have meant that most RWE could not be considered in product applications.  Since 2016, 35 drugs, biologics, or vaccines have included RWE in their applications.  The integration of RWE into device approvals has been more extensive, with over 250 premarket authorizations including RWE for the same period, although even for devices the rate of RWE-based authorizations has plateaued in recent years.  Historically, the FDA has insisted that any RWE submitted to the agency include private, confidential information at the individual patient level.  This approach makes it impractical to use most large databases with valuable macro-level data.  The FDA is responding to the position of many sponsors and data scientists that meaningful information can be extracted from some big data sources without private, individual information.  FDA reviewers will now consider the strength of submitted RWE on an application-by-application basis.

"We're removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster," said FDA Commissioner Marty Makary, M.D., M.P.H.  "This common-sense reform will unlock access to vast databases like cancer and cystic fibrosis registries that contain critical insights about how treatments work in the real world."

This policy change opens the door to using de-identified databases containing millions of patient records — including national cancer registries like the National Cancer Institute’s Surveillance, Epidemiology, and End Results, hospital systems databases, insurance claims databases, and electronic health record networks — resources that have grown exponentially but remained limited for use under previous FDA policy.  These comprehensive datasets track patient outcomes across diverse populations and real-world treatment settings, offering insights that traditional clinical trials cannot capture.