母方案平台试验设计方法及其在相关领域中的应用

Design of the master protocol platform trial and its application in related fields

母方案平台试验是指在针对一种疾病不同亚型的多个子研究中,为长期持久评估多种治疗方法或治疗方法间不同组合,允许多种治疗方案根据一定的决策算法进入或退出其试验平台。相比传统的临床试验设计,母方案平台试验作为一种科学的临床试验创新模式,可加快药物评估速度,以应对众多待评估药物与疾病暂无有效疗法间的矛盾问题。本文以2020年英国牛津大学牵头开展的针对新型冠状病毒感染肺炎住院患者的大规模随机对照临床试验(RECOVERY)为例,介绍母方案平台试验设计的概念、原理和基本流程,以期为药物临床研究提供方法学参考。

The master protocol platform trial is a trial in which multiple treatments or different combinations of treatments are consistently evaluated in the context of a single disease,possibly within several sub-studies for different disease subtypes,allowing multiple treatments to enter or exit the trial platform based on a certain decision algorithm.Compared with the traditional clinical trial design,the master protocol platform trial as a scientific innovative model of clinical trial can accelerate drug evaluation to deal with the contradiction between the awaited evaluation of numerous drugs and lack of effective therapy for diseases.By using the large-scale randomized controlled clinical trial for COVID-19 inpatients(RECOVERY)led by the University of Oxford in the UK in 2020 as an illustration,this paper introduced the concept,design principle,and basic trial process of the master protocol platform trial to provide methodological references for clinical research of drugs.