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USFDA Guidance: Considerations, Master Protocols for the Development of Drugs and Biological Products

The U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research released multiple guidance earlier this week (26 December 2023) ie, "Rare Diseases: Considerations for the Development of Drugs and Biological Products" and "Master Protocols for Drug and Biological Product Development".



A rare disease or condition, in part, as a disease or condition that “affects less than 200,000 persons in the United States.

The guidance discusses selected issues commonly encountered in the development of rare disease drugs in order to assist sponsors in conducting efficient and successful drug development programs.


The statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs. Rare diseases present multiple challenges, including limited medical and scientific understanding, poorly understood natural history data, sample size constraints, and a lack of experience in drug development.



Master protocol: a protocol designed with multiple substudies, which may have different objectives and involve coordinated efforts to evaluate one or more medical products in one or more diseases or conditions within the overall study structure.


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.


Also recently Center for Drug Evaluation and Research released another guidance "Development of Monoclonal Antibody Products Targeting SARS-CoV-2 for Emergency Use Authorization" that provides recommendations to sponsors on the development of monoclonal antibody products targeting SARS-CoV-2 intended for the prevention or treatment of COVID-19, including addressing the impact of emerging variants.

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