FDA“药品和生物制品开发主方案供企业用的指导原则（草案）”介绍

主方案是针对多个可能具有不同目标的子研究而设计的一个整体性的临床试验方案,并涉及在整个试验框架下对一种或多种疾病或疾病亚型的一种或多种试验药物的协调评价。使用主方案进行良好设计和实施的试验,可以通过最大限度地增加从试验中获得的信息量来加速药物开发。美国FDA在2023年12月发布了“药品和生物制品开发主方案供企业用的指导原则（草案）”,旨在说明采用主方案设计的临床试验的设计、实施和分析的要求。详细介绍了该篇指导原则,重点围绕为安全性和有效性提供实质性证据的随机伞式和平台试验,讨论了主方案设计和分析的重要考虑因素,并结合我国现行的指导原则要求,从监管角度对采用主方案设计展开讨论,以期促进主方案设计在我国的研发应用,并指导监管实践。

Introduction to FDA''s "Master Protocols for Drug and Biological Product Development Guidance for Industry (Draft)"

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.