Master Protocols for Drug and Biological Product Development (from Catapult’s regulatory round-up – December 2023)

The primary focus of this draft guidance is on randomized umbrella and platform trials that are intended to contribute to a demonstration of safety and substantial evidence of effectiveness of a drug. The concepts discussed may also be useful to consider for early-phase or exploratory umbrella and platform trials as well as those conducted to satisfy post-marketing commitments or requirements. The recommendations and considerations in this guidance do not apply to master protocols evaluating first-in-human drugs given the unique attributes from both a trial design and regulatory perspective that must be considered. This draft guidance provides recommendations on the design and analysis of trials conducted under a master protocol as well as guidance on the submission of documentation to support regulatory review. This should be taken into consideration with FDA guidance for Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial. Comments can be submitted until 22 February 2024.

Published on the 5th of January 2024. Read the entire regulatory round-up for December 2023 here.