Master Protocols: Your Focus May Need More Focus

Music City Center 

A master protocol is a comprehensive document that outlines the plan for clinical research within multiple sub-studies. As with any protocol, it includes detailed information on the objectives, design, methodology, statistical considerations, and organization. Regardless of the protocol type, clinical trials must begin with the fundamental design question "What is the question we want to answer?" followed by the question "What do we need to inform decision making?". For master protocols these questions are considered not only for a single asset or patient population but also for an indication or a therapeutic area. When developing a master protocol, study teams tend to focus on their single asset rather than planning holistically across an indication or therapeutic area resulting in a stitched together "Frankenstein's monster" protocol. By way of examples, this paper will speak to questions, decisions, and design elements to establish a framework that informs the trial design described by the master protocol and its appendices/addendums.

master protocol

clinical trial design 

Biopharmaceutical Section