A new "Mechanistic-Practical" Framework for designing and interpreting randomized trials
A new "Mechanistic-Practical" Framework for designing and interpreting randomized trials
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DOI：
10.1016/j.jclinepi.2008.02.009
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发表时间：
2009-05-01
影响因子：
7.2
通讯作者：
Guyatt, Gordon H.
中科院分区：
文献类型：
--
作者：
Karanicolas, Paul J.;Montori, Victor M.;Guyatt, Gordon H.
方法学家传统上将随机对照试验（RCT）分为解释性（代表理想环境）和实用性（代表现实世界环境）。虽然这一框架极大地推进了RCT的设计和解释，但目前对解释-语用框架的解释存在两个主要局限性。首先，他们混淆了目的和结构。其次，他们忽视了那些在真实的世界中使用随机对照试验结果来做出临床和政策决定的人的不同观点。试验的目的应该决定研究者对试验结构的选择，而试验的结构决定了决策者发现结果有用的程度。在这篇文章中，我们介绍了两个术语，明确地指的是一个试验的目的：一个试验是机械的，它解决了生物学的关系。相比之下，试验是实用的，因为它提供了全面的信息，直接关系到具体的卫生保健决策。修订后的框架有助于研究者选择最佳试验设计，以及临床医生对RCT结果的最佳解释。如果我们的目标是与个体患者决策最相关的临床试验，我们将避免使用招募不太可能受益的患者的试验（例如，诊断不确定的患者）;可能不依从的患者;由其不同专业知识可能导致不同结果的从业者治疗的患者;以及允许可能影响治疗效果的共同干预-即，传统的实用主义审判相反，我们将设计，实施，并应用于门诊病人的实际试验的结果。(C)2009 Elsevier Inc. All rights reserved.
Methodologists have traditionally categorized randomized controlled trials (RCTs) as explanatory (representing the ideal setting) and pragmatic (representing the real-world setting). Although this framework has greatly advanced the design and interpretation of RCTs, current interpretations of the explanatory-pragmatic framework suffer from two major limitations. First, they confound purpose with structure. Second, they ignore the varying perspective of those using RCT results to make clinical and policy decisions in the real world. The purpose of a trial should determine researchers' choices regarding the trial's structure and the structure of a trial determines the extent to which a decision maker will find the results useful. In this article, we introduce two terms that refer explicitly to the purpose of a trial: A trial is mechanistic to the extent that it addresses a biological relationship. In contrast, a trial is practical to the extent that it provides comprehensive, information that bears directly on specific health care decisions. This revised framework facilitates investigators' choice of optimal trial design, and clinicians' optimal interpretation of RCT results. If our goal is clinical trials most relevant to individual patient decision making, we will eschew the use of trials that enroll patients unlikely to benefit (e.g., those with uncertain diagnosis); those likely to be noncompliant; treated by practitioners whose differing expertise is likely to result in differing outcomes; and permitting cointerventions that are likely to influence treatment effectiveness-i.e., the conventional pragmatic trial. Instead we will design, implement, and apply the results of practical trials to out-patients. (C) 2009 Elsevier Inc. All rights reserved.