Am J Epidemiol. 2023 Jun 2;192(6):987-994. doi: 10.1093/aje/kwad036.
Most reported treatment effects in medical research studies are ambiguously defined, which can lead to misinterpretation of study results. This is because most authors do not attempt to describe what the treatment effect represents, and instead require readers to deduce this based on the reported statistical methods. However, this approach is challenging, because many methods provide counterintuitive results. For example, some methods include data from all patients, yet the resulting treatment effect applies only to a subset of patients, whereas other methods will exclude certain patients while results will apply to everyone. Additionally, some analyses provide estimates pertaining to hypothetical settings in which patients never die or discontinue treatment. Herein we introduce estimands as a solution to the aforementioned problem. An estimand is a clear description of what the treatment effect represents, thus saving readers the necessity of trying to infer this from study methods and potentially getting it wrong. We provide examples of how estimands can remove ambiguity from reported treatment effects and describe their current use in practice. The crux of our argument is that readers should not have to infer what investigators are estimating; they should be told explicitly.
大多数医学研究报告中的治疗效果定义不明确,这可能导致对研究结果的误解。这是因为大多数作者并没有试图描述治疗效果代表什么,而是要求读者根据报告的统计方法来推断。然而,这种方法具有挑战性,因为许多方法提供了违反直觉的结果。例如,一些方法包括所有患者的数据,但得出的治疗效果仅适用于患者的一部分子集,而其他方法则会排除某些患者,而结果将适用于所有人。此外,一些分析提供了与患者永不死亡或停止治疗的假设情况相关的估计。在这里,我们引入估计目标作为解决上述问题的一种方法。估计目标是对治疗效果代表什么的清晰描述,从而避免读者试图从研究方法中推断出这一点,并且可能会推断错误。我们提供了一些示例,说明估计目标如何消除报告的治疗效果中的模糊性,并描述了它们在实践中的当前使用情况。我们论点的关键是,读者不应该推断研究人员正在估计什么;他们应该被明确告知。
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