主方案是針對多個可能具有不同目標(biāo)的子研究而設(shè)計的一個整體性的臨床試驗方案，并涉及在整個試驗框架下對一種或多種疾病或疾病亞型的一種或多種試驗藥物的協(xié)調(diào)評價。使用主方案進(jìn)行良好設(shè)計和實施的試驗，可以通過最大限度地增加從試驗中獲得的信息量來加速藥物開發(fā)。美國FDA在2023年12月發(fā)布了“藥品和生物制品開發(fā)主方案供企業(yè)用的指導(dǎo)原則（草案）”，旨在說明采用主方案設(shè)計的臨床試驗的設(shè)計、實施和分析的要求。詳細(xì)介紹了該篇指導(dǎo)原則，重點圍繞為安全性和有效性提供實質(zhì)性證據(jù)的隨機傘式和平臺試驗，討論了主方案設(shè)計和分析的重要考慮因素，并結(jié)合我國現(xiàn)行的指導(dǎo)原則要求，從監(jiān)管角度對采用主方案設(shè)計展開討論，以期促進(jìn)主方案設(shè)計在我國的研發(fā)應(yīng)用，并指導(dǎo)監(jiān)管實踐。

Master protocol is 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 disease subtypes within the overall study structure. A well-designed and conducted trials using master protocols can accelerate drug development by maximizing the amount of information obtained from the research effort. In December 2023, FDA issued the "Master Protocols for Drug and Biological Product Development Guidance for Industry (Draft)" to describe the requirements for the design, implementation and analysis of clinical trials using the master protocol. This paper introduces the guidance in detail, focusing on the random umbrella and platform design that provide substantial evidence for safety and effectiveness, and discusses the important considerations for the design and analysis of the master protocol. Combined with the requirements of the current guidance in China, the adoption of the master protocol design is discussed from the perspective of regulation, in order to promote the application of the master protocol in China and guide the regulatory practice.

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