Opportunities and challenges in using studies without a control group in comparative effectiveness reviews
Opportunities and challenges in using studies without a control group in comparative effectiveness reviews
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DOI：
10.1002/jrsm.1101
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发表时间：
2014-06-01
影响因子：
9.8
通讯作者：
Ip, Stanley
中科院分区：
文献类型：
--
作者：
Paulus, Jessica K.;Dahabreh, Issa J.;Ip, Stanley
在检查治疗干预措施的证据以回答比较有效性研究问题时，应考虑所有提供干预措施因果效应信息的研究。 “单组研究”纵向评估采用单一治疗策略管理的受试者队列的结果。由于这些研究“缺少”直接的、同时进行的对照组，因此它们通常被认为无法提供比较有效性的信息。然而，原则上，单组研究可以通过推断“缺失”的未治疗组的预期结果来提供有关因果治疗效果的信息。单组研究依赖前后比较、隐式比较或历史比较作为理想比较组的代理。必须根据具体情况仔细检查这些比较的有效性。尽管在许多情况下，研究人员对于这种推断是否合理存在分歧。存在这样的研究被普遍接受作为证据来源的情况。本文概述了与单组研究解释相关的问题，重点是支持其在比较有效性审查中考虑所需的假设。我们讨论了采用单组研究的各种环境、系统评价者需要解释的常见研究设计，以及使用这些设计来告知比较有效性问题相关的挑战。版权所有 (C) 2013 约翰·威利父子有限公司
When examining the evidence on therapeutic interventions to answer a comparative effectiveness research question, one should consider all studies that are informative on the interventions' causal effects. "Single group studies" evaluate outcomes longitudinally in cohorts of subjects who are managed with a single treatment strategy. Because these studies are "missing" a direct, concurrent comparison group, they are typically deemed non-informative on comparative effectiveness. However, in principle, single group studies can provide information on causal treatment effects by extrapolating expected outcomes in the "missing" untreated arm. Single group studies rely on before-after, implicit, or historical comparisons as a proxy for an ideal comparison group. The validity of these comparisons must be carefully examined on a case-by-case basis. While in many cases, researchers will disagree on whether such extrapolations are reasonable; circumstances exist where such studies are generally acceptable as a source of evidence. This article provides an overview of issues related to the interpretation of single group studies with a focus on the assumptions required to support their consideration in comparative effectiveness reviews. We discuss the various settings in which single group studies are employed, common research designs that systematic reviewers need to interpret, and challenges associated with using these designs to inform comparative effectiveness questions. Copyright (C) 2013 John Wiley & Sons, Ltd.