Pulling The Thread On FDA's Master Protocol Draft Guidance

By Laura Gilliam, Josh Wooten, and Jennifer Murphy

The U.S. Food and Drug Administration (FDA) has issued draft guidance on the design and analysis of clinical trials conducted under a master protocol, a system that allows multiple substudies to evaluate one or more medical products in one or more diseases. This guidance expands on previous recommendations and encourages application across a broad range of therapeutic areas. Master protocols enhance collaborative sharing of clinical trial elements, which can accelerate drug development and approval.

However, Halloran Consulting Group has pinpointed five areas where the FDA should provide further guidance, including operationalizing a master protocol, submitting master protocols as a new Investigational New Drug application, complex statistical analysis requirements, changes to the informed consent form, and the criticality of oversight.

This article provides an analysis of the draft guidance and its potential implications, as well as recommendations for companies planning to use master protocols in their clinical trials. Master protocols require complex statistical analyses, careful management of informed consent form changes, and rigorous oversight. Consulting firms like Halloran can provide expert guidance throughout the process, from strategic planning to regulatory support and operationalization of master protocols.