研究动态
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医学应用中回归不连续设计指南。

A guide to regression discontinuity designs in medical applications.

发表日期:2023 Aug 01
作者: Matias D Cattaneo, Luke Keele, Rocío Titiunik
来源: STATISTICS IN MEDICINE

摘要:

我们提供了一份针对生物医学背景下回归不连续设计(RD)分析的实用指南。我们首先介绍了连续性框架和局部随机化框架中的关键概念、假设和估计目标。然后我们讨论了这两个框架内的现代估计和推断方法,包括带宽或局部邻域选择方法、最优治疗效果点估计和鲁棒偏差校正推断方法用于不确定性量化。我们还概述了可以用于支持关键假设的经验证伪测试。我们的讨论重点关注生物医学研究中的两个特点:(i)模糊RD设计,这在治疗方法基于临床指南的情况下经常出现,但是得分接近临界值的患者与分配规则相悖而受治疗;(ii)具有离散得分的RD设计,在生物医学应用中普遍存在。我们通过三个实证应用示例来说明我们的讨论:南非HIV患者在使用CD4指南抗逆转录病毒疗法对其保持的影响,美国乳腺癌复发治疗中遗传指南对其复发情况的影响,以及台湾年龄基础的患者分担费用对医疗利用的影响。我们提供了完整的复制材料,包括使用公开可得的数据和Python、R和Stata统计软件,为研究人员提供进行RD分析所需的所有工具。© 2023 John Wiley & Sons Ltd.
We present a practical guide for the analysis of regression discontinuity (RD) designs in biomedical contexts. We begin by introducing key concepts, assumptions, and estimands within both the continuity-based framework and the local randomization framework. We then discuss modern estimation and inference methods within both frameworks, including approaches for bandwidth or local neighborhood selection, optimal treatment effect point estimation, and robust bias-corrected inference methods for uncertainty quantification. We also overview empirical falsification tests that can be used to support key assumptions. Our discussion focuses on two particular features that are relevant in biomedical research: (i) fuzzy RD designs, which often arise when therapeutic treatments are based on clinical guidelines, but patients with scores near the cutoff are treated contrary to the assignment rule; and (ii) RD designs with discrete scores, which are ubiquitous in biomedical applications. We illustrate our discussion with three empirical applications: the effect CD4 guidelines for anti-retroviral therapy on retention of HIV patients in South Africa, the effect of genetic guidelines for chemotherapy on breast cancer recurrence in the United States, and the effects of age-based patient cost-sharing on healthcare utilization in Taiwan. Complete replication materials employing publicly available data and statistical software in Python, R and Stata are provided, offering researchers all necessary tools to conduct an RD analysis.© 2023 John Wiley & Sons Ltd.