Modeling and Simulation to Optimize the Design and Analysis of Confirmatory Trials, Characterize Risk–Benefit, and Support Label Claims

The role of modeling and simulation (M&S) in the design and interpretation of phase III studies, from break out session 4 of the European Medicines Agency (EMA)/European Federation of Pharmaceutical Industries and Associations (EFPIA) M&S workshop, was divided into themes illustrated with case studies (Table 1): (1) M&S being conducted to support the design of confirmatory trials; (2) longitudinal model‐based test as primary inferential analysis (biosimilarity and disease progression trials); (3) assessment of benefit–risk ratio, approval and labeling of an unstudied dose or dosing regimen, and development of future regulatory guidance. CPT: Pharmacometrics & Systems Pharmacology (2013) 2, e27; doi:10.1038/psp.2013.4; advance online publication 27 February 2013