Modeling of Phase 2 and literature data enabled the sponsor to provide the FDA with a convincing efficacy profile and avoided a costly Phase 3 trial, saving $60 Million and 24 Months. The FDA approved the drug in target indication.
A global pharmaceutical company was seeking to optimize the design of pivotal Phase 3 studies for its central nervous system (CNS) drug candidate. Data from more than 10 Phase 2 studies was available on the drug candidate, along with data from an initial Phase 3 confirmatory trial.
As the sponsor prepared for meetings with FDA to finalize the requirements of its confirmatory studies, it become critical to establish a comprehensive picture of the drug candidate’s efficacy profile, given the varied nature of primary and secondary endpoints for the indication and the many different designs of existing clinical studies that would support a submission.