Contribution of Modeling and Simulation in the Regulatory Review and Decision-Making: U.S. FDA Perspective

The Division of Pharmacometrics at the U.S. FDA engages in regulatory reviews, research and policy development. During 2000–2008, over 50% of pharmacometric reviews of 198 NDA and BLA applications influenced approval and safety decisions. During this time, pharmacometric analyses were used in pediatric dose selection, and approval of doses not directly studied in effectiveness trials. Additionally, pharmacometrics has been used in FDA advice on protocol design to optimize dosing regimens based on benefit-risk for clinical testing, and to provide confirmatory evidence of effectiveness. Current research projects aim to solve drug development challenges and develop policies grounded in pharmacometric principles and methodologies.