08/23/2024 | News release | Distributed by Public on 08/24/2024 19:45
Post contributed by guest bloggerMiguel Kitamura of Kitamura Law.Not affiliated with Covington & Burling LLP.
Earlier this summer, the Pharmaceutical Evaluation Division of the Pharmaceutical Safety Bureau within the Japan Ministry of Health, Labor and Welfare ("MHLW") published "Points to Note" summarizing key considerations for sponsors when planning, conducting, analyzing, and evaluating master protocol clinical trials ("MPT Points").[1]
A master protocol is a comprehensive protocol designed to evaluate the multiple objectives of several drugs and diseases in a single trial. A master protocol includes execution plans for multiple sub-studies conducted concurrently. The studies under the master protocol are sometimes referred to as cohorts or sub-studies. This design has the potential to accelerate drug development, but it also raises some complexities. Over the past few years, various drug regulatory agencies throughout the world have issued documents addressing such challenges. For example, the U.S. Food and Drug Administration ("FDA") published guidance on master protocols for development of oncology drugs in March 2022,[2] and a draft guidance document on master protocol trials in the broader, non-oncology-specific context in December 2023.[3] Similarly, the European Medicines Agency ("EMA") addressed master protocol trials in version 6 of the Guideline on the Clinical Evaluation of Anticancer Medicinal Products finalized in November 2023.[4]
The MPT Points discuss the following three types of master protocols:
Among other content, the MPT Points offer the following additional recommendations to sponsors.
Notably, the MPT Points explicitly envisage that master protocol trials could be pursued with participating foreign institutions, or with Japanese institutions participating in foreign master protocol trials. However, the MPT Points do not address the impact of population differences on the acceptability of foreign clinical data (ICH E5), and if a local bridging study is needed, it may complicate the planning of a master protocol.
[1] MHLW, Considerations for the Utilization of Master Protocol Trials in Drug Development (June 2024), https://www.pmda.go.jp/files/000269475.pdf. The original Japanese version can be accessed at https://www.pmda.go.jp/files/000269222.pdf.
[2] FDA, Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics, Guidance for Industry (Mar. 2022), https://www.fda.gov/media/120721/download.
[3] FDA, Master Protocols for Drug and Biological Product Development, Draft Guidance for Industry (Dec. 2023), https://www.fda.gov/media/174976/download.
[4] EMA, Guideline on the Clinical Evaluation of Anticancer Medicinal Products Rev.6 (Nov. 2023), https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-evaluation-anticancer-medicinal-products-revision-6_en.pdf.
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