Real‐World Evidence and the Regulation of Medicines

Abstract

This issue of Clinical Pharmacology & Therapeutics features several important articles on realworld evidence (RWE) and attendant considerations in its ability to support regulatory decision making. RWE and observational methods are not new, and their role in drug regulation has been largely limited to assessing and quantifying safety signals that arise once a drug is marketed. There is significant interest in and gradual acceptance of a potentially broader role for RWE in regulatory decision making. Stewart et al., in a recently published paper in this journal, catalogued several drug development innovations used during the coronavirus disease 2019 (COVID19) pandemic that could catalyze durable and scalable global regulatory transformation. One such innovation is the regulatory acceptance of novel approaches to clinical evidence generation, including the use of RWE, particularly for serious and lifethreatening diseases.