arXiv 2502.033292025-02-05stat.ME

同时使用假想策略与治疗政策策略处理多个并发事件

Dealing with multiple intercurrent events using hypothetical and treatment policy strategies simultaneously

Camila Olarte Parra, Rhian M. Daniel, Jonathan W. Bartlett

AI总结：

本文研究临床试验中同时用治疗政策策略和假想策略处理两个并发事件的 estimand，通过潜在结果与因果图定义不同因果假设，并用模拟和糖尿病试验示例说明其对估计的影响。

摘要：

为了在临床试验中精确定义所关注的治疗效应，ICH E9 estimand 增补文件指出，应识别相关的所谓并发事件，并指定处理这些事件的策略。以不同策略处理并发事件会导致不同的 estimand。本文聚焦于同时涉及用一种治疗政策策略处理一个并发事件、并用假想策略处理另一个并发事件的 estimand。我们利用潜在结果和因果图定义这些 estimand，并考虑两个并发事件与其他变量之间可能存在的因果关系。我们表明，可以采纳不同的因果 estimand 定义和假设，每一种对估计都有不同的影响，这一点通过模拟研究加以证明。文中以一项糖尿病试验为例，从概念上说明了这些不同的考量。

英文摘要：

To precisely define the treatment effect of interest in a clinical trial, the ICH E9 estimand addendum describes that relevant so-called intercurrent events should be identified and strategies specified to deal with them. Handling intercurrent events with different strategies leads to different estimands. In this paper, we focus on estimands that involve addressing one intercurrent event with the treatment policy strategy and another with the hypothetical strategy. We define these estimands using potential outcomes and causal diagrams, considering the possible causal relationships between the two intercurrent events and other variables. We show that there are different causal estimand definitions and assumptions one could adopt, each having different implications for estimation, which is demonstrated in a simulation study. The different considerations are illustrated conceptually using a diabetes trial as an example.

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