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整合试验与非实验数据以检验治疗效果异质性的方法。

Methods for Integrating Trials and Non-experimental Data to Examine Treatment Effect Heterogeneity.

作者信息

Brantner Carly Lupton, Chang Ting-Hsuan, Nguyen Trang Quynh, Hong Hwanhee, Stefano Leon Di, Stuart Elizabeth A

机构信息

Department of Biostatistics, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland 21205, USA.

Department of Biostatistics, Columbia Mailman School of Public Health, New York, New York 10032, USA.

出版信息

Stat Sci. 2023 Nov;38(4):640-654. doi: 10.1214/23-sts890. Epub 2023 Nov 6.

Abstract

Estimating treatment effects conditional on observed covariates can improve the ability to tailor treatments to particular individuals. Doing so effectively requires dealing with potential confounding, and also enough data to adequately estimate effect moderation. A recent influx of work has looked into estimating treatment effect heterogeneity using data from multiple randomized controlled trials and/or observational datasets. With many new methods available for assessing treatment effect heterogeneity using multiple studies, it is important to understand which methods are best used in which setting, how the methods compare to one another, and what needs to be done to continue progress in this field. This paper reviews these methods broken down by data setting: aggregate-level data, federated learning, and individual participant-level data. We define the conditional average treatment effect and discuss differences between parametric and nonparametric estimators, and we list key assumptions, both those that are required within a single study and those that are necessary for data combination. After describing existing approaches, we compare and contrast them and reveal open areas for future research. This review demonstrates that there are many possible approaches for estimating treatment effect heterogeneity through the combination of datasets, but that there is substantial work to be done to compare these methods through case studies and simulations, extend them to different settings, and refine them to account for various challenges present in real data.

摘要

根据观察到的协变量估计治疗效果可以提高为特定个体量身定制治疗方案的能力。要有效地做到这一点,需要处理潜在的混杂因素,并且要有足够的数据来充分估计效应调节。最近大量的研究工作致力于使用来自多个随机对照试验和/或观察数据集的数据来估计治疗效果异质性。有许多新方法可用于通过多项研究评估治疗效果异质性,了解哪些方法最适用于哪种情况、这些方法之间如何相互比较,以及为在该领域继续取得进展需要做些什么非常重要。本文按数据设置对这些方法进行了综述:汇总级数据、联邦学习和个体参与者级数据。我们定义了条件平均治疗效果,并讨论了参数估计器和非参数估计器之间的差异,我们列出了关键假设,包括单个研究中所需的假设以及数据合并所需的假设。在描述现有方法之后,我们对它们进行比较和对比,并揭示未来研究的开放领域。这篇综述表明,通过数据集组合估计治疗效果异质性有许多可能的方法,但要通过案例研究和模拟来比较这些方法、将它们扩展到不同的情况,并对它们进行改进以应对实际数据中存在的各种挑战,还有大量工作要做。

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