Master protocol studies offer innovative opportunities in the design and conduct of clinical trials. The term master protocol is generally used to describe clinical trials that use one overarching protocol to guide multiple, simultaneously occurring sub-studies. Master protocol studies can be used to study multiple diseases, multiple therapies, or both.

While enthusiasm for master protocol studies is growing, the number of organizations that have sufficient experience designing and implementing master protocol studies remains critically low. Several organizations have provided opportunities for stakeholders to learn general information about master protocol studies. Efforts are needed to build off this important work to create practical, detailed resources to support the design and conduct of master protocol studies.

Master Protocol Studies: Opportunities, Challenges, and Emerging Best Practices (“Master Protocol Studies”) (2019 - 2020)

• Compare and contrast the distinct design and operational features of basket, umbrella, and platform trials
• Identify emerging best practices for the design and conduct of master protocol studies

CTTI’s work will increase the appropriate use of master protocol studies.

• A landscape review – Expert interviews and a literature scan were used to develop a high-level roadmap that details strategies to address common roadblocks that inhibit the development of master protocol studies at the pre-planning, planning, or execution stage.

• An Expert Meeting series – Meetings in October 2019 and April 2020 helped identify emerging best practices and prioritize the creation of tools to support stakeholders’ efforts to address common challenges related to the design and implementation of master protocol studies.

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.