Restrictions on Real-World Evidence Regulatory Reviews Eased

FDA says it is removing a long-standing barrier to the use of real-world evidence (RWE) in regulatory reviews, signaling a significant shift in how the agency evaluates data submitted in drug and medical device applications. “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,” FDA says in a notice. “The FDA similarly intends to consider updating its guidance for drugs and biologics.”

The change addresses a core concern raised for years by drugmakers, device manufacturers, and data scientists: that the FDA’s insistence on access to private, patient-level data has effectively excluded many large, high-value datasets from regulatory use. Those requirements made it impractical to leverage national registries, insurance claims databases, and other large-scale sources that contain aggregated or de-identified information, FDA says.

Since Congress formally encouraged the use of RWE in 21st Century Cures Act legislation in 2016, uptake has been uneven. According to the agency, only 35 drug, biologic, or vaccine applications have incorporated RWE over that period. Device submissions have relied on RWE more frequently, with more than 250 premarket authorizations including such data, though the agency noted that growth in RWE-based device approvals has stalled in recent years.

“We’re removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster,” FDA Commissioner Marty Makary, M.D., M.P.H., said in a statement. “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.”

The policy shift could significantly broaden the range of data sources available to regulators. The FDA pointed to de-identified datasets encompassing millions of patients, including the National Cancer Institute’s Surveillance, Epidemiology, and End Results registry, hospital system databases, insurance claims data, and electronic health record networks. These sources track outcomes across diverse populations and real-world clinical settings, offering perspectives that traditional randomized trials often cannot provide.