单臂试验（SAT）有时可用于支持在欧盟批准抗癌药物的营销授权。产品的抗肿瘤活性水平和持久性以及背景是确定试验结果相关性的重要因素。本研究旨在提供试验结果的背景信息，并评估基于SAT的药物获批后的效益大小。我们的研究重点在于2012年至2021年基于SAT结果获得批准的固体肿瘤抗癌药物。数据来自欧洲公共评估报告和/或已发表的文献。这些药物的效益是通过欧洲医学肿瘤学会（ESMO）临床效益量表（MCBS）进行评估。共有18种药物基于21个SAT获得批准，其中只有少数药物得到了>1个SAT的支持。在大多数临床试验中，已（预）指定了临床相关的治疗效果（71.4%），并且通常提供了相应的样本大小计算。在进行每项测试不同药物的10项研究中，都能找到一个对临床相关治疗效果门槛的合理的解释。至少有12个申请中包括信息以便于试验结果的背景认知，其中有六项支持性研究。在分析的关键性SAT试验（n=21）中，有三个被分配了ESMO-MCBS评分为4，这对应于“重大”效益。对于在SAT试验中测试的固体肿瘤药物所显示的治疗效果的临床相关性取决于效应大小和背景。更好地促进监管决策，预先确定和推动一个临床相关的效应，并将样本大小与该效应保持一致，这是很重要的。外部对照可能有助于背景认知过程，但相关限制必须得到解决。版权所有©2023作者。由Elsevier Ltd.出版。保留所有权利。

Single-arm trials (SATs) can sometimes be used to support marketing authorization of anticancer medicinal products in the European Union. The level and durability of antitumor activity of the product as well as context are important aspects to determine the relevance of trial results. The aim of this study is to provide details on the contextualization of trial results and to evaluate the magnitude of benefit of medicinal products approved based on SATs.We focused on anticancer medicinal products for solid tumors approved on the basis of SAT results (2012-2021). Data were retrieved from European public assessment reports and/or published literature. The benefit of these medicinal products was evaluated via the European Society for Medical Oncology (ESMO)-Magnitude of Clinical Benefit Scale (MCBS).Eighteen medicinal products were approved based on 21 SATs-few medicinal products were supported by >1 SAT. For the majority of clinical trials, a clinically relevant treatment effect was (pre)specified (71.4%) and most often an accompanying sample size calculation was provided. For 10 studies, each testing a different medicinal product, a justification for the threshold for a clinically relevant treatment effect could be identified. At least 12 out of 18 applications included information to facilitate the contextualization of trial results, including six supportive studies. Of the pivotal SATs analyzed (n = 21), three were assigned an ESMO-MCBS score of 4, which corresponds to 'substantial' benefit.The clinical relevance of the treatment effects shown by medicinal products for solid tumors tested in SATs is dependent on the effect size and context. To better facilitate regulatory decision making, prespecifying and motivating a clinically relevant effect and aligning the sample size to that effect is important. External controls may facilitate in the contextualization process, but the associated limitations must be addressed.Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.