Keywords: Master protocol, Innovation, Clinical trial
Master protocols are designed to study multiple therapies, multiple diseases, or both. The idea is to provide answers to multiple research questions that have certain parameters or design aspects in common. These types of coordinated research efforts can be ideal settings for innovative trial designs that leverage information across commonalities to yield efficiencies. Innovation can play a role in establishing the trial infrastructure as well as in determining statistical aspects of the trial design. Master protocols may have an important role to play in the FDA’s initiatives relating to complex innovative designs under the PDUFA VI and 21st Century Cures Acts, both passed in 2016. They also have utility in the NIH research environment, where trial networks are used to investigate and/or evaluate different intervention strategies to inform medical practice and policy. Examples of master protocols conducted in both settings will be discussed, and the advantages and disadvantages of pursuing a master protocol will be described, based on experience to date.