解释平行组设计中估计的治疗效果的意义:对Trigg等人发表评论
1Department of Population Health Sciences, Duke University School of Medicine, Durham, NC, USA. kevin.weinfurt@duke.edu.
概括
解释治疗效应需要指定推断水平 (人群或个人). 对于个人与人口估计的基准点和变量有所不同,影响了临床结果评估的解释.
科学领域:
- 临床试验方法论 临床试验方法论
- 医学中的统计推理.
背景情况:
- 美国食品和药物管理局 (FDA) 为基于临床结果评估的终点提供了关于解释治疗效应的指南草案.
- 解释这些效应往往需要分析群体平均值之间的差异.
- 最近的工作由Trigg等人. 研究了为这些集团间差异设定门的方法.
研究的目的:
- 推进关于解释治疗效应对基于临床结果评估的终点的讨论.
- 提供关于为集团间差异推导有意义的值的观察.
- 为了澄清人口层面和个人层面的治疗效应推断之间的区别.
主要方法:
- 对解释治疗效应的现有方法的评论和批判性分析.
- 讨论美国食品和药物管理局的指导草案.
- 检查参考点和变量在影响解释中的作用.
主要成果:
- 需要在人口层面和个人层面的治疗效果推断之间做出明确的区别.
- 根据推断水平,解释的参考点和定变量可能会有所不同.
- 患者内部有意义的变化对于解释平行组设计中的治疗效果并不直接相关.
结论:
- 指定推理水平 (人群与个人) 对于准确解释治疗效应至关重要.
- 建立解释值的方法必须考虑到推断的目标水平.
- 需要进一步的研究来完善在临床试验中解释治疗效果的方法.
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