Keywords: platform design, master protocol
A platform master protocol design refers to those study design in which multiple treatments or treatment regimens are tested simultaneously. The platform trial offers flexible features such as discontinuing treatments earlier for futility or superiority, graduation to next phase of study, or adding new treatment regime to be tested under one overarching master protocol. This presentation will mainly discuss design features of the platform design with application in a Phase 2 NSCLC using immune-oncology agents. The primary endpoint is OS and interim decision as well as graduation decisions are based on predictive probability of Phase 3 success. Main components of clinical design under master protocol as well as developmental challenges will be highlighted including analysis consideration of primary endpoint under non-proportional hazard, development of stopping and graduation criteria, treatment agnostic protocol element and drug specific amendments, and challenge in randomization and drug supply, etc. Design challenges such as statistical considerations in handling contemporary and non- contemporary control as well as regulatory interactions will also be discussed. Due to its complexity, cross functions or cross industry collaborative efforts are critical in the development and execution of the platform design.