Master protocols: New directions in drug discovery

Abstract A master protocol is a unifying study construct that includes multiple subgroups and substudies, with patients having same or different diseases and that employ one or multiple drugs to treat it. Initially designed for oncology, master protocol trials are intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure. The ability to use a single infrastructure, trial design, and protocol to simultaneously evaluate multiple drugs and/or disease populations in multiple substudies, speeds up drug development and makes it more efficient. Thus, it is important for the clinical trial professionals to understand both the basic principles of master protocol trials and the way innovative trial designs are starting to change the landscape of clinical research.