Drugs

Agency Updates Draft Guidance on Master Protocols

FDA has released a revised draft guidance aimed at helping sponsors design and conduct clinical trials using master protocols, a trial framework increasingly used to evaluate multiple therapies, diseases, or patient populations within a single overarching study. The draft guidance, Master Protocols for Drug and Biological Product Development, replaces a 12/2023 draft and incorporates feedback received during the public comment period. FDA says the revision is intended to clarify the agency's current thinking on the use of master protocols in drug and biologic development and to provide recommendations on trial design, statistical analysis, and regulatory submissions.

Master protocols have gained attention as a potential way to accelerate drug development by allowing sponsors to evaluate multiple therapies or disease subtypes under a common infrastructure. FDA notes that such approaches can reduce duplication by sharing protocol elements, clinical sites, data management systems, oversight committees, and, in some cases, control groups.

According to the agency, master protocols may improve development efficiency by enabling the evaluation of multiple investigational products within a single trial framework or by leveraging information across related diseases, conditions, or disease subtypes. However, FDA cautions that these designs can introduce operational and statistical complexities, including challenges related to blinding, trial startup, and coordination among multiple sponsors and stakeholders.

The guidance primarily focuses on randomized trials conducted under master protocols that are intended to provide evidence of safety and effectiveness for regulatory decision-making. FDA says the recommendations may also be relevant for early-stage exploratory studies and postmarketing studies conducted to fulfill regulatory commitments or requirements.

Among the topics addressed are randomization, selection of control groups, informed consent, blinding, adaptive trial designs, comparisons between investigational products, evaluation of treatments across multiple diseases or disease subtypes, multiplicity adjustments, and safety monitoring. The document also discusses trial governance, data sharing, dissemination of information, and considerations for regulatory submissions.

A key change in the revised draft is the addition of more detailed recommendations for basket trials, which evaluate a single therapy across multiple diseases or disease subtypes that share a common characteristic. FDA says the revisions were prompted by public comments requesting additional guidance in this area. The agency also made clarifications regarding randomization, choice of control groups, and informed consent.

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