Hypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods.
Hypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods.
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DOI：
10.1080/19466315.2022.2081599
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发表时间：
2023
影响因子：
1.8
通讯作者：
Bartlett, Jonathan W.
中科院分区：
文献类型：
--
作者：
Parra, Camila Olarte;Daniel, Rhian M.;Bartlett, Jonathan W.
ICHE9增编引入了并发事件这一术语，指的是在治疗开始后发生的事件，这些事件既可以排除对感兴趣结果的观察，也可以影响其解释。它提出了处理并发事件的五种策略来形成估计，但没有提出估计的统计方法。在本文中，我们将重点放在假设策略上，其中治疗效果是在防止并发事件的假设情景下定义的。对于它的估计，我们考虑了因果推论和缺失数据方法。我们确定某些“因果推断估计量”等同于某些“缺失数据估计量”。这些链接可能会帮助那些熟悉一套方法而不是另一套方法的人。此外，使用潜在结果符号使我们能够更清楚地陈述缺失数据方法所依赖的假设，以估计假设估计。这有助于表明估计假设估计是否合理，以及在分析中应使用哪些数据。我们表明，假设估计可以通过利用并发事件发生后的数据来估计，这通常是不使用的。这篇文章的补充材料可以在网上找到。
The ICH E9 addendum introduces the term intercurrent event to refer to events that happen after treatment initiation and that can either preclude observation of the outcome of interest or affect its interpretation. It proposes five strategies for handling intercurrent events to form an estimand but does not suggest statistical methods for estimation. In this article we focus on the hypothetical strategy, where the treatment effect is defined under the hypothetical scenario in which the intercurrent event is prevented. For its estimation, we consider causal inference and missing data methods. We establish that certain “causal inference estimators” are identical to certain “missing data estimators.” These links may help those familiar with one set of methods but not the other. Moreover, using potential outcome notation allows us to state more clearly the assumptions on which missing data methods rely to estimate hypothetical estimands. This helps to indicate whether estimating a hypothetical estimand is reasonable, and what data should be used in the analysis. We show that hypothetical estimands can be estimated by exploiting data after intercurrent event occurrence, which is typically not used. Supplementary materials for this article are available online.
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