This guidance provides recommendations on the design and analysis of trials conducted under a master protocol. It focuses on randomized umbrella and platform trials (see definitions below) intended to contribute to evidence of safety and effectiveness of drugs. The document also provides guidance on the submission of master protocol documents for FDA review.
What Is a Master Protocol?
A protocol designed with multiple substudies:
- These substudies may have different objectives.
- These substudies are coordinated within an overall study structure to evaluate:
° One or more medical products
° One or more diseases or conditions
Why Is This Guidance Important?
Compared with traditional standalone trials under separate protocols, trials conducted under master protocols can share control arms, protocol elements, infrastructure, and oversight, thereby maximizing the amount of information obtained from a research effort. This document provides guidance to support well-designed, well conducted trials using master protocols to accelerate drug development, particularly in certain settings, such as where subject recruitment is challenging.
Summary:
In May 2021, FDA published the guidance for industry COVID-19: Master Protocols Evaluating Drugs and Biological Products for Treatment or Prevention, which focused on master protocols evaluating drugs for the treatment or prevention of COVID-19. That guidance was intended to remain in effect only for the duration of the public health emergency related to Coronavirus Disease 2019 declared by the Secretary of Health and Human Services under section 319 of the Public Health Service Act, which has now expired. FDA is issuing this draft guidance because many of the recommendations set forth in the 2021 guidance are applicable outside the context of the section 319 public health emergency and are applicable to other therapeutic areas, not just COVID-19
