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Expanding the Use of Real-World Evidence in Regulatory and Value-Based Payment Decision-Making for Drugs and Biologics

The amount of electronic health care data is increasing at unprecedented rates, due to the now widespread adoption of electronic health records (EHRs) and other technology in health care, as well as the significant increase in the number of patients that are now using technologies—such as wearables, biosensors, apps, and other digital health tools—to track their activities and health status and connect with the health system.

These real-world data sources provide the opportunity to improve the evidence base for the development of new drugs and biologics, regulatory evaluation by the Food and Drug Administration (FDA), and new value-based payment arrangements, which align pricing or payments with outcomes.

Real-world data and evidence have significant bipartisan support. Several provisions related to real-world evidence were contained in the 21st Century Cures Act passed and signed into law in December 2016, as well as the FDA Reauthorization Act of 2017 and other user fee legislation passed in 2017. The FDA has taken several steps to advance the generation and use of real-world evidence for regulatory decision-making, including the publication of the Framework for FDA’s Real-World Evidence Program (FDA Framework) in December 2018.

While there are many opportunities to leverage these new data sources as real-world evidence to support regulatory efforts and new value-based payment arrangements for drugs and biologics, there are also barriers to their use. The recommendations below are expected to advance the generation and use of real-world evidence for regulatory evaluation and value-based payment decision-making.

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