FDA In Brief: FDA issues draft guidance to industry on submitting real-world evidence in new drug and biologic applications

May 8, 2019

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“We’re at a critical point in our health care system in terms of the potential to leverage vast amounts of health-related data to advance medical care, including data collected during the routine care of patients, such as electronic health records and patient-generated data. Understanding how this kind of real-world data (RWD) can add to the body of evidence to inform regulatory decisions continues to be an area of immense interest in the research and medical community, as well as a key strategic priority for the FDA. At the end of the day, care of an individual patient should be informed by reliable evidence derived from studies reflective of their personal and clinical story. Given that RWD captures the use of treatments in practice, and not just in research studies, analyzing and evaluating these data have the potential to develop new hypotheses for further testing of new products to drive continued innovation, and to inform us about the performance of medical products,” said FDA Principal Deputy Commissioner Amy Abernethy, M.D., Ph.D. “Today’s draft guidance is a part of an expanding body at work at FDA focused on broadening clinical evidence generation so that study results are maximally applicable to real-world populations.”

Today, the U.S. Food and Drug Administration published the draft guidance, Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics. The draft guidance is intended to get input on FDA’s proposal to encourage sponsors and applicants who are using RWD to generate real-world evidence (RWE) as part of a regulatory submission to identify in the cover letter that the submission contains RWE in a simple, uniform format. When implemented, FDA anticipates that this approach will allow FDA to identify and track RWE submissions, monitor outcomes and ensure appropriate relevant review expertise.

In December, the FDA issued a strategic framework for its RWE program, and ongoing work at the agency involving RWE includes foundational research to understand potential applications of RWE in regulatory decision-making. The agency plans to continue to engage the public and stakeholders around its work in this area, including a webinar held in March, as well as a workshop planned for later this year.

Related Information

FDA Draft Guidance: Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics
FDA: Real-World Evidence
FDA: Information Exchange and Data Transformation (INFORMED)

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.