This guidance document 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
The primary focus of this 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.
The considerations in this guidance apply to a range of therapeutic areas.
Sponsors considering master protocols in oncology should also consult Master Protocols: Efficient Clinical Trial Design Strategies To Expedite Development of Oncology Drugs and Biologics (March 2022).
Sponsors evaluating cellular and gene therapy products in early-phase development should consult the guidance for industry Studying Multiple Versions of Cellular or Gene Therapy Product in Early-Phase Clinical Trials (November 2022)