Instrumental variable methods in comparative safety and effectiveness research.

Instrumental variable methods in comparative safety and effectiveness research.
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DOI:
10.1002/pds.1908
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发表时间:
2010-06
影响因子:
2.6
通讯作者:
Schneeweiss, Sebastian
Schneeweiss, Sebastian
中科院分区:
医学4区
文献类型:
--
作者:
Brookhart, M. Alan;Rassen, Jeremy A.;Schneeweiss, Sebastian

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工具变量(IV)方法已被提出作为一种潜在的方法,在医疗干预措施的比较研究中的共同问题,不受控制的混杂,但IV方法是不熟悉的许多研究人员。本文的目的是为比较安全性和有效性研究的IV方法提供非技术性的实用介绍。我们概述了有效的IV估计所需的原则和基本假设,讨论了如何解释IV研究的结果,提供了一个审查的工具,已被用于比较有效性研究,并提出了一些最低限度的报告标准IV分析。最后,我们提供了我们的观点的作用,IV估计与更传统的方法的基础上的暴露或结果的统计建模。我们预计,静脉注射方法对于结果非常罕见的药物安全性研究来说往往不够有力,但在不受控制的混淆可能很大的预期效果研究中可能有潜在用途。
Instrumental variable (IV) methods have been proposed as a potential approach to the common problem of uncontrolled confounding in comparative studies of medical interventions, but IV methods are unfamiliar to many researchers. The goal of this article is to provide a non-technical, practical introduction to IV methods for comparative safety and effectiveness research. We outline the principles and basic assumptions necessary for valid IV estimation, discuss how to interpret the results of an IV study, provide a review of instruments that have been used in comparative effectiveness research, and suggest some minimal reporting standards for an IV analysis. Finally, we offer our perspective of the role of IV estimation vis-à-vis more traditional approaches based on statistical modeling of the exposure or outcome. We anticipate that IV methods will be often underpowered for drug safety studies of very rare outcomes, but may be potentially useful in studies of intended effects where uncontrolled confounding may be substantial.
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