Harnessing the Potential of Real-World Evidence Master Protocols

Advanced considerations of real-world data and evidence (RWD/E) within the clinical trial ecosystem have increased demand to create more efficient, systematic, and flexible ways to address multiple research questions using a single, master protocol (MP). Despite being a well-established method of testing multiple hypotheses within a single clinical trial, MPs are not fully conceptualized to support RWE studies. In this white paper, we describe key characteristics of RWE MPs and how they can support the development of critical evidentiary needs for medical products. First, we provide an overview on how innovative MP templates are emerging to support more consistent structures and processes for designing and conducting multiple RWE studies under a single research protocol, and how these templates are improving RWE study governance, transparency, and reporting. Next, we highlight four key areas in which RWE policy stakeholders could align to create and capture the value of RWE MPs:

• Shared terminology, definitions, and concepts
• Source data considerations
• Factors toward developing and leveraging RWE MPs
• Stakeholder engagement and collaboration

These proposed next steps could advance stakeholder understanding of RWE MPs and inspire opportunities to further their development and use.

Read the full paper.