Leveraging Real-World Data to Extend Adult Drug Approvals to Pediatric Populations: A Pharmacometric Approach

This presentation explores the innovative use of real-world data (RWD) to facilitate the extension of adult drug approvals to pediatric populations, a critical area in pharmacometrics. Despite the challenges in conducting randomized controlled trials in children, there is an urgent need for properly evaluated drugs in pediatric medicine. We discuss how RWD can complement traditional clinical trials and support regulatory decision-making for pediatric drug approvals. This presentation will examine case studies where RWD has been successfully utilized to characterize drug safety and effectiveness in children, particularly for treatments initially approved for adults. Through these examples, the speaker will highlight the potential of RWD to provide evidence on long-term outcomes, rare adverse events, and effectiveness in heterogeneous populations that may not be captured in conventional trials.