Regulatory guidance to support submission of high validity and relevance of real-world evidence (RWE) in marketing authorization applications include recommendations on the format and content of submissions, check-lists and definitions. Optimal use of RWE for regulatory decision-making raises other questions: for which use cases is RWE most useful (e.g. to provide comparator groups, assess the generalizability or validity of clinical trial data, understand the clinical context, validate endpoints), what criteria are used to assess RWE, and how to estimate the impact of RWE on benefit-risk evaluations. To address these questions, regulators and research organizations have initiated studies to analyze the use of RWE in marketing applications and draw lessons from experience. Based on examples and findings, the session will discuss how the emerging evidence may help shape the future for the optimal use of RWE in the regulatory decision-making.
Professor of Medicine and Epidemiology at Harvard Medical School and Chief of the Division of Pharmacoepidemiology and Pharmacoeconomics in the Department of Medicine, Brigham and Women’s Hospital
Harvard Medical School & Brigham & Women's Hospital