临床试验中的假设估计:因果推理和缺失数据方法的统一
Camila Olarte Parra1, Rhian M Daniel2, Jonathan W Bartlett1
1Department of Mathematical Sciences, University of Bath, Bath, UK.
Statistics in biopharmaceutical research
|June 1, 2023
概括
在ICH E9附录中,介绍了用于估算的处理间流事件. 这项研究将假设策略的因果推断和缺失数据方法联系起来,使得使用通常未使用的事件后数据可以更好地估计.
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
- 药学研究 药学研究
背景情况:
- 国际协调理事会 (ICH) E9附录定义了间流事件为治疗开始后影响结果观察或解释的事件.
- 提出了处理间流事件以形成估计的五种策略,但没有详细说明具体的统计估计方法.
研究的目的:
- 探索统计方法来估计处理间流事件的假设策略.
- 建立因果推理和缺失数据之间的联系,用于估计和估计的方法.
主要方法:
- 专注于假设策略,在预防间流事件的场景下定义治疗效果.
- 使用因果推理和缺失数据方法来进行估计.
- 使用潜在结果标记来澄清缺失数据方法的假设.
主要成果:
- 确定了特定因果推断估计器和缺失数据估计器之间的等价性.
- 证明了假设的估计值可以通过在间流事件后收集的数据来估计,这通常被忽视.
结论:
- 因果推理和缺失数据方法之间的已确定的联系可以帮助熟悉只有一种方法的研究人员.
- 潜在结果注释澄清了假设,有助于评估估计假设估计的合理性和指导数据选择.
- 利用间接事件后的数据提供了一个新的方法来估计临床试验中的假设估计值.
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