集群随机试验 (CRT-estimands) 估计框架的共识扩展的开发:来自国际Delphi研究的结果
Brennan C Kahan1, Melanie Bahti2, Dongquan Bi3
1MRC Clinical Trials Unit at UCL, London, UK. b.kahan@ucl.ac.uk.
Trials
|December 19, 2025
概括
专家们就在集群随机试验中定义估计值的关键项目达成共识,扩大了目前的指导方针. 这项研究阐明了强大的集群随机试验 (CRT) 设计和分析的基本属性.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 估计对于在随机试验中澄清研究目标至关重要,ICH E9 ((R1) 提供了必要的属性.
- 现有的ICH E9 (R1) 准则主要是为个人随机试验制定.
- 集群随机试验 (CRT),其中组是随机的,需要额外考虑估计和规范.
研究的目的:
- 评估利益相关者对ICH E9的共识扩展R1的额外项目的意见.
- 通过专家共识识别集群随机试验中描述估计值的关键项目.
主要方法:
- 一个经过修改的Delphi过程,涉及评估和集群随机试验的专家.
- 三轮的 மதிப்பீடு (评分),项目建议,以及由小组成员提供的理由.
- 共识定义为≥70%的评价属性为"必不可少",<15%的评价属性为"不重要".
主要成果:
- 在三个Delphi轮的高参与率 (第1轮为52%;第2轮为85%;第3轮为95%).
- 专家组主要由统计学家 (85%) 和临床试验参与者 (25%) 组成.
- 达成共识的五个关键项目是:对个体/集群的权重,集群人口,暴露时间,边际与集群特异性影响,以及处理集群级间流动事件.
结论:
- 在集群随机试验中定义估计数的几个关键项目上达成了专家共识.
- 这些发现将为CRT中估计和属性的共识指南的开发提供信息.
- 这项工作提高了复杂的试验设计中估计和定义的清晰度和严格性.
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