Tang L, Zhou M, Xia L, Hao R M, Tong X, Chen D M, Song Y Y, Zhao X, Zhang H, Hu W J, Zou L M, Du Y, Qi Y L, Chen X M, Yang Z M
Center for Drug Evaluation, National Medical Products Administration of China, Beijing 100022, China.
Zhonghua Zhong Liu Za Zhi. 2022 Jun 23;44(6):587-592. doi: 10.3760/cma.j.cn112152-20210513-00376.
Single-arm trial refers to a clinical trial design that does not set up parallel control group, adopts open design, and does not involve randomization and blind method. These features, on the one hand, speed up the process of clinical trials, significantly shorten the time to market and meet the needs of patients with advanced malignancies, but also lead to the uncertainty of single-arm clinical trials themselves. Recently, the US Food and Drug Administration held a meeting of the oncologic drug advisory committee to discuss six tumor indications that have been accelerated approved, which once again triggered the discussion of single-arm trials. The basis of accelerated approval by single-arm trial is actually a compromise on the level of evidence-based medical evidence requirements after assessing the benefit risk. Therefore, the sponsor should strictly grasp the applicable conditions of single-arm trial in anti-tumor drugs and conduct single-arm trial scientifically. Post-marketing clinical trial should be implement as early as possible to ensure the benefit of patients. Based on the characteristics of single-arm trial, combined with two guidance relevant to single-arm trial issued by National Medical Products Administration recently, this article is supposed to propose and summarize the strategy of single-arm trial supporting the marketing of anti-tumor drugs.
单臂试验是指一种不设立平行对照组、采用开放设计且不涉及随机化和盲法的临床试验设计。这些特点一方面加快了临床试验进程，显著缩短上市时间并满足晚期恶性肿瘤患者的需求，但也导致了单臂临床试验本身的不确定性。最近，美国食品药品监督管理局召开了肿瘤药物咨询委员会会议，讨论六项已加速批准的肿瘤适应症，这再次引发了对单臂试验的讨论。单臂试验加速批准的依据实际上是在评估获益风险后对循证医学证据要求水平的一种妥协。因此，申办者应严格把握抗肿瘤药物单臂试验的适用条件，科学开展单臂试验。应尽早实施上市后临床试验以确保患者获益。基于单臂试验的特点，结合国家药品监督管理局近期发布的两项与单臂试验相关的指导原则，本文旨在提出并总结支持抗肿瘤药物上市的单臂试验策略。
