FDA issues new framework to advance use of real-world data

The Food and Drug Administration is expanding its use of real-world data—such as electronic health records, medical claims and registries—to improve regulatory decisions regarding drugs and biologics.

“Because they include data covering the experience of physicians and patients with the actual use of new treatments in practice—and not just in research studies—the collective evaluation of these data sources has the potential to inform clinical decision making by patients and providers, develop new hypotheses for further testing of new products to drive continued innovation and inform us about the performance of medical products,” said FDA Commissioner Scott Gottlieb, MD, in a written statement.

The agency on Thursday released a new strategic framework to serve as a roadmap for more fully incorporating real-world data (RWD) and real-world evidence (RWE) into its regulatory activities.

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“This framework is intended to advance the collection of data that are appropriate, consistent and provide information and knowledge that can better inform regulatory decision-making,” added Gottlieb. “For example, currently used EHRs and medical claims data may not capture all of the data elements needed to answer significant questions of interest. That’s why part of our new framework is to explore strategies for filling the gaps other sources of RWD, which may include the use of mobile technologies, electronic patient reported outcome tools, wearables and biosensors.”

According to the FDA, patient registries are another source of RWD that could be used to generate RWE, as well as—potentially—electronic healthcare data from other countries.

The agency plans to issue guidance on how to assess the reliability and relevance of RWD from EHRs and medical claims used to generate RWE regarding drug product effectiveness. In addition, the FDA intends to examine how to assess the reliability and relevance of registry data and international electronic healthcare data.

“The healthcare system is integrating more effective ways to leverage electronic tools to gather and use vast amounts of health-related data,” noted Gottlieb. “There’s more data available to inform medical decisions than ever before. But we need to provide clear guidance on the appropriate collection and evaluation of this information.”