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Mandatory Registration and Results Reporting of Real-World Evidence Studies of FDA-Regulated Medical Products

  • Sanket S. Dhruva
    Correspondence: Address to Sanket S. Dhruva, MD, MHS, San Francisco VA Medical Center, 4150 Clement St, 2A-18, San Francisco, CA 94121.
    Department of Medicine, San Francisco Veterans Affairs Health Care System, and University of California, San Francisco, School of Medicine
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  • Nilay D. Shah
    Division of Health Care Policy and Research and Kern Center for the Science of Health Care Delivery, Mayo Clinic, Rochester, MN
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  • Joseph S. Ross
    Section of General Internal Medicine and National Clinician Scholars Program, Yale School of Medicine, New Haven, CT

    Department of Health Policy and Management, Yale School of Public Health, New Haven, CT

    Center for Outcomes Research and Evaluation, Yale-New Haven Hospital, New Haven, CT
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Published:October 21, 2020DOI:
      In June 2017, the Food and Drug Administration (FDA) approved a new indication for use of the Edwards Sapien 3 transcatheter heart valve (Edwards Lifesciences, Irvine, California), 2 years after the initial approval of the device. This approval was unique because the underlying evidence was generated from a postmarket registry of patients receiving transcatheter aortic valve replacement, not from premarket clinical trials. Registries, along with electronic health records (EHRs) and claims data, are among the many data sources generated outside of traditional clinical research settings being used for real-world evidence (RWE).
      • Sherman R.E.
      • Anderson S.A.
      • Dal Pan G.J.
      • et al.
      Real-world evidence: what is it and what can it tell us?.
      Such RWE-based approvals represent not only the future of generating evidence of the benefits and risks of medical products to inform regulatory approvals, but also decisions by payers, patients, and clinicians. Accordingly, to ensure that this evidence is rigorous and reliable, steps must be taken to require registration and results reporting of all RWE studies of FDA-regulated products.

      Abbreviations and Acronyms:

      CDRH (Center for Devices and Radiologic Health), EHR (electronic health records), FDA (Food and Drug Administration), ICMJE (International Committee of Medical Journal Editors), OHDSI (Observational Health Data Science Initiative), RWE (real-world evidence)
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