摘要

Master protocols combine several sub-trials, each with their own research objectives, which is usually presented as one single clinical trial application. Master protocols have become increasingly popular in oncology and haematology, as either basket, umbrella, or platform trials. Although master protocols are intended to accelerate drug development and to reduce futility, their use poses challenges to ethics committees, patients, study investigators, and competent authorities during the review and authorisation process of a clinical trial application. In this Personal View, we review the experiences of clinical trial applications from two European medical regulators-the Danish Medicines Agency and the German Federal Institute for Drugs and Medical Devices. We view master protocols as a good opportunity to identify new treatment options more quickly, particularly for patients with cancer. However, the complexity of trial documentation, the amount of information resulting from sub-trials, and the volume of changes and amendments made to clinical trial applications can cause issues during trial supervision, and during the analysis and review of a corresponding application for marketing authorisation. We draw attention to the potential issues arising from these trial concepts and propose possible solutions to avoid problems during clinical trial authorisation and trial conduct.

译文

主协议结合了几个子试验,每个试验都有自己的研究目标,通常作为一个单一的临床试验申请提出。主要方案在肿瘤学和血液学中越来越受欢迎,无论是篮子,伞形还是平台试验。尽管主要方案旨在加速药物开发并减少无效,但在临床试验申请的审查和授权过程中,它们的使用对伦理委员会,患者,研究调查员和主管当局提出了挑战。在这个个人观点中,我们回顾了两家欧洲医疗监管机构 - 丹麦药品管理局和德国联邦药品和医疗器械研究所的临床试验申请经验。我们将主协议视为更快地识别新治疗方案的好机会,特别是对于癌症患者。然而,试验文件的复杂性,子试验产生的信息量,以及对临床试验申请的变更和修改量可能会在试验监督期间以及在分析和审查相应的上市许可申请期间引起问题。 。我们提请注意这些试验概念产生的潜在问题,并提出可能的解决方案,以避免在临床试验授权和试验过程中出现问题。

Basket trial

肿瘤 基础研究 临床研究术语
概述  :  

随着二代测序的应用,越来越多低频突变被发现。因基因变异频率较低,如果采用传统的临床试验方式进行研究,很难达到统计学要求的样本量,为此,创新性的临床试验诞生——篮子试验和雨伞试验。美国癌症研究协会(American Association for Cancer Research,AACR)在2014年的癌症进展里面特别指出,针对精准癌医学的创新性临床试验可分成两大类,一类称为Basket Trial,即篮子试验。大致说把表面看来不同,但其实内在特征(主要是基因突变)类似的癌症患者都放在一起,尝

Basket   英 /'bɑːskɪt/   美 /'bæskɪt/

       n. 篮子;(篮球比赛的)得分;一篮之量;篮筐;vt. 装入篮

同根词   basketry n. 篮子编织技艺;篮筐

       Boy, take my basket! I want you to do something for me. 小伙子,拎着我的篮子!我要你为我做点儿事情。

 

Trial   英 /'traɪəl/   美 /'traɪəl/

       n. 试验;审讯;努力;磨炼;adj. 试验的;审讯的

同根词   tried adj. 可靠的;试验过的;trying adj. 难受的;难堪的;费劲的;令人厌烦的;try n. 尝试;努力;试验;tried v. 尝试(try的过去分词);

       Not only do we have one clinical trial, we have two. 我们不是只做了一组临床试验,我们有两组以供对比。

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