Real‐World Evidence in New Drug and Biologics License Application Approvals During Fiscal Years 2020–2022

Abstract Improvements in the relevance and reliability of routinely collected clinical data and statistical methods to analyze the available data have enhanced the adoption of real‐world data (RWD) to generate real‐world evidence (RWE) for regulatory decision making of medical products. As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA VII), the US Food and Drug Administration (FDA) committed to issuing annual reports describing such uses for drugs and biological products. The first report covered fiscal year (FY) 2023 and described two approvals based, at least in part, on RWE: tocilizumab (trade name Actemra) and lacosamide (trade name Vimpat). This article describes New Drug Applications and Biologics Licensing Applications approved by the Center for Drug Evaluation and Research (CDER) in FYs 2020–2022 with RWE that (1) contributed to substantial evidence of effectiveness or (2) provided safety data necessary for approval. RWE contributed to substantial evidence of effectiveness for the approval of applications for fosdenopterin (trade name Nulibry) and tacrolimus (trade name Prograf) in FY 2021 and abatacept (trade name Orencia), vosoritide (trade name Voxzogo), and alpelisib (trade name Vijoice) in FY 2022. No studies provided only safety data necessary for approval. The five approvals included six total studies that provided RWE pivotal for the applications approval. Four studies leveraged registry data, and two leveraged medical record data. In parallel with annual RWE public reporting under PDUFA VII, this report can inform interested parties regarding how RWD are used to generate RWE that can support regulatory decision making for medical products.