在集群随机试验中使用估计值:一篇综述
Dongquan Bi1, Andrew Copas1, Brennan C Kahan1
1MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, UCL, London, UK.
Clinical trials (London, England)
|February 17, 2026
概括
在集群随机试验中,估计很少被描述,这阻碍了研究问题和统计方法的清晰度. 需要指导方针来定义特定于集群随机试验的估计值.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
背景情况:
- 估计定义了研究量化的治疗效果,ICH E9(R1) 概述了关键属性.
- 集群随机试验 (CRT) 提供了独特的估计和考虑,现有指南未完全解决这些问题.
- 目前使用和CRT中estimands的具体考虑仍然不清楚.
研究的目的:
- 评估目前公布的集群随机试验中估计的使用情况.
- 评估估计和属性的描述,包括CRT特定考虑.
- 在CRT中识别研究问题和统计方法中的模两可.
主要方法:
- 在2023年10月至2024年1月期间发表的73个CRT的系统审查.
- 对初级结果的估计,描述和推断能力的评估.
- 评估CRT特定的估计和考虑,分析方法和假设.
主要成果:
- 没有任何试验明确描述了他们对主要结果的估计.
- 在49%的试验中,ICH E9 (R1) 属性是可推断的;在21%的试验中,CRT 特定属性是可推断的.
- 关于个人与集群平均效应 (63%) 和集群级事件 (21%) 的含糊性存在.
结论:
- 在CRT中估计的吸收率很低,这阻碍了对研究问题的理解和估计器的适当性.
- 迫切需要针对CRT的独特方面制定定制的评估指导方针.
- 改进的估计和定义对于CRT研究的清晰度至关重要.
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