Application of Real-World Evidence to Support FDA Regulatory Decision Making

Real-world data (RWD) and real-world evidence (RWE) are valuable resources for drug development strategies. Historically, it has been used for safety evaluation during post-marketing surveillance. RWD and RWE have been utilized in the regulatory decision-making process for drug effectiveness, especially for rare diseases and cancers, where conducting randomized controlled trials is challenging. The Food and Drug Administration (FDA) is actively working on providing trustworthy information derived from RWD and RWE to supplement the data from clinical trials. This review discusses the potential use of RWE to make regulatory decisions on drug effectiveness for certain therapeutic areas as well as the challenges in drawing inferences on drug effectiveness from RWE. A review of FDA-approved new drug applications and biologics license applications suggests that several methodological considerations should be deliberated when designing a study using RWE to demonstrate product effectiveness. The acceptance of RWE, while promising, is dependent on the relevance and reliability of the data. The insight and engagement of all stakeholders contribute to the successful use of RWE for clinical evaluations.