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 Bi1
1MRC Clinical Trials Unit at UCL, London, UK.
medRxiv : the preprint server for health sciences
|July 9, 2025
概括
这项研究确定了在集群随机试验中定义估值的重要项目. 专家们就提高试验设计和报告的清晰度的关键属性达成共识.
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) 附录提供了估计和描述的属性,主要用于单独随机试验.
- 集群随机试验需要额外考虑估计和规范.
研究的目的:
- 评估利益相关者对描述集群随机试验中估值的额外项目的看法.
- 为集群随机试验制定ICH E9 (R1) 指南的共识扩展.
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) 和理由,以确定关键项目.
- 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:
- 五个关键项目达成共识,用于描述集群随机试验中的估计值.
- 这些包括对个体和集群的权重,集群人口,暴露时间,边际和集群特定效应,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.
- 这些发现将为在本试验设计中进行估计和描述的共识指南的制定提供信息.
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