Aiding the Adoption of Master Protocols by Optimizing Patient Engagement

Area of Science:

• Clinical Trial Design
• Drug Development
• Patient-Centered Research

Background:

• Master protocols (MPs) are a flexible clinical trial design allowing multiple sub-studies for evaluating therapies.
• Regulatory bodies like the FDA and EMA emphasize the importance of patient engagement (PE).
• Current MP frameworks can be improved by incorporating patient perspectives.

Purpose of the Study:

• To explore the benefits of integrating patient engagement principles into master protocol development and conduct.
• To provide stakeholders, including drug developers and patient advocacy groups, with insights into creating more efficient clinical trials.
• To highlight how patient voices enhance the success of master protocols for all involved parties.

Main Methods:

• Review of existing literature and regulatory guidance on master protocols and patient engagement.
• Analysis of potential benefits of PE within the MP framework for patients, sponsors, and providers.
• Expert perspective from the American Statistical Association (ASA)/DahShu Master Protocol Working Group (MPWG) People and Patient Engagement (PE) Subteam.

Main Results:

• Incorporating PE into MPs can lead to treatments better suited to patient needs.
• MPs with PE can increase treatment availability and expedite patient access to novel therapies.
• PE in MPs can facilitate the development of innovative treatments for rare diseases and pediatric populations.
• Shared control arms in MPs can reduce the likelihood of patients receiving a placebo.

Conclusions:

• Integrating patient engagement into master protocols is a scientifically sound approach to improve clinical trial efficiency and patient outcomes.
• This integration offers significant advantages for drug developers, healthcare providers, and patients, particularly in areas like rare disease research.
• Further development and implementation of PE within MPs are recommended to maximize their potential in advancing therapeutic development.