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Real-world evidence and regulatory drug approval

Nature Reviews Clinical Oncology volume 17, pages 271–272 (2020)Cite this article

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The FDA has demonstrated a willingness to expedite access to new cancer medicines by using real-world evidence to support regulatory drug approval. In this article, we explore three recent examples of such approvals and the lessons that can be learned from this collective experience.

Real-world data (RWD) offers important insights into cancer care that are complementary to information obtained from clinical trials: whereas clinical trials generate precise measures of treatment efficacy and short-term toxicity in highly controlled settings, RWD provide information on treatment access, uptake, long-term safety and effectiveness in routine clinical practice. Accordingly, evidence from clinical trials has traditionally been used to support regulatory drug approval, whereas real-world evidence (RWE) has been used for post-marketing surveillance studies. In an effort to expedite access to new therapies, the FDA has demonstrated a willingness to alter the traditional regulatory paradigm and allow the use of RWE in place of clinical trial evidence to support drug approval. Here, we explore three recent examples in which RWE has supported FDA approvals and highlight lessons that can be learned from this collective experience.

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Acknowledgements

The work of C.M.B. is supported as the Canada Research Chair in population Cancer Care.

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Authors and Affiliations

  1. Division of Cancer Care and Epidemiology, Queen’s University Cancer Research Institute, Kingston, Canada

    Michael J. Raphael, Bishal Gyawali & Christopher M. Booth

  2. Department of Oncology, Queen’s University, Kingston, Canada

    Michael J. Raphael, Bishal Gyawali & Christopher M. Booth

  3. Department of Public Health Sciences, Queen’s University, Kingston, Canada

    Michael J. Raphael, Bishal Gyawali & Christopher M. Booth

  4. Program on Regulation, Therapeutics, and Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA

    Bishal Gyawali

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  1. Michael J. Raphael
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Correspondence to Christopher M. Booth.

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Raphael, M.J., Gyawali, B. & Booth, C.M. Real-world evidence and regulatory drug approval. Nat Rev Clin Oncol 17, 271–272 (2020). https://doi.org/10.1038/s41571-020-0345-7

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