FDA eliminates real-world evidence barrier in medical device reviews

The FDA has removed a limitation on the use of real-world evidence (RWE) in drug and device application reviews.

The agency says it will accept RWE without requiring that identifiable individual patient data collected from real-world data sources always be submitted in a marketing submission, according to new guidance.

The FDA said it intends to consider updating its guidance for drugs and biologics.

The FDA previously promoted RWE as an advance in regulatory policy. However, prior expectations mean that most RWE can’t be considered in product applications, the agency said. Since 2016, 35 drugs, biologics or vaccines have included RWE in their applications. More than 250 premarket authorizations in the same period included RWE, but the rate of RWE-based authorizations has “plateaued” in recent years, according to the FDA.

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The FDA says it historically insisted that RWE submitted to the agency include private, confidential information at the individual patient level. However, this renders most large databases with valuable macro-level data impractical, the agency said.

It says the updated guidance comes in response to sponsors and scientists’ position that meaningful information can come from big data sources without private, individual information. As a result, the FDA reviewers now intend to consider the strength of submitted RWE on an application-by-application basis.

The FDA says the policy change could enable the utilization of de-identified databases containing millions of patient records. That includes 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.

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“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 Dr. Marty Makary said in a news release. “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 change in guidance comes shortly after a government watchdog agency said the FDA needs more staffing for recall oversight.