PS05: Master Protocol Clinical Trials: Regulatory Considerations and Applications

In recent years, there has been a rise of novel designs, which provides more flexible ways to conduct clinical trials than traditional randomized controlled trials. Master protocol was proposed to allow the investigation of multiple sub-studies with potentially different objectives, interventions, and subgroups conducted within an overall trial structure. A master protocol allows the use of a unified infrastructure, trial design, and protocol to accelerate drug development and achieve greater operational and statistical efficiencies as compared to typical randomized controlled trials. Therefore, it is important for the clinical trial professionals to understand both the foundational principles as well as the key considerations for the design of master protocol trials. In December 2023, the FDA released a draft guidance titled "Master Protocols for Drug and Biological Product Development." In this guidance, the regulatory offers recommendations, as well as considerations for master protocols, with a particular focus on randomized umbrella and platform trials, which evaluate multiple therapies for a single disease to demonstrate safety and substantial evidence of effectiveness. In this session, speakers from the FDA, industry and academia will come together and share their insights and experiences in designing and implementing master protocols. Their presentations will delve into both the benefits and challenges associated with using master protocols.