New guidance permits the use of de-identified data without requiring individual patient information in marketing submissions.
The US Food and Drug Administration (FDA) issued new guidance for certain types of medical device submissions, stating it will accept real-world evidence 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.
The policy change addresses what the agency says is significant barrier that has limited real-world evidence use in product applications. Since 2016, only 35 drugs, biologics, or vaccines have included real-world evidence in their applications. Device approvals have seen more extensive real-world evidence integration, with over 250 premarket authorizations including real-world evidence during the same period, though the rate has plateaued in recent years.
Previously, the FDA required any submitted real-world evidence to include private, confidential information at the individual patient level, making it impractical to use most large databases with valuable macro-level data.
“We’re removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster,” says FDA commissioner Marty Makary, MD, MPH, in a 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.”
Access to Large-Scale Databases
The policy opens access to de-identified databases containing millions of patient records, including national cancer registries like the National Cancer Institute’s Surveillance, Epidemiology, and End Results program, hospital systems databases, insurance claims databases, and electronic health record networks.
These datasets track patient outcomes across diverse populations and real-world treatment settings, offering insights that traditional clinical trials cannot capture. The resources have grown exponentially but remained limited for use under previous FDA policy.
FDA reviewers will now consider the strength of submitted real-world evidence on an application-by-application basis, responding to positions from sponsors and data scientists that meaningful information can be extracted from big data sources without private individual information.
The change represents a shift in the FDA’s approach to evaluating evidence from real-world sources, potentially accelerating the review process for products with supporting real-world evidence data while maintaining regulatory standards for safety and efficacy.
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