Statistical issues in the design and analysis of expertise-based randomized clinical trials
Statistical issues in the design and analysis of expertise-based randomized clinical trials
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DOI：
10.1002/sim.3448
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发表时间：
2008-12-30
影响因子：
2
通讯作者：
Devereaux, P. J.
中科院分区：
文献类型：
--
作者：
Walter, S. D.;Ismaila, A. S.;Devereaux, P. J.
为了避免常规随机临床试验设计的某些困难。基于专业知识的设计已被提议作为一种替代方案。在基于专业知识的设计中，患者被随机分配到临床医生（例如外科医生），然后临床医生用他们喜欢的干预措施治疗所有患者。这种设计可以识别个体的临床偏好，因此与传统设计相比，可以减少手术交叉的比率。这也可能促进临床医生的招募，因为他们总是被允许提供他们选择的治疗，这一特点也可能对患者有吸引力。基于专业知识的设计避免了所谓的差异专业知识偏差的可能性。如果临床医生通常比新的实验治疗更熟悉标准治疗，那么在常规设计中，与随机分配到实验治疗的患者相比，更多随机分配到标准治疗的患者将拥有专家临床医生。如果专业知识影响研究结果，则会出现治疗组的偏倚比较。我们检验了在基于专家和传统设计中估计治疗效果的相对效率。我们认识到，在以专家为基础的设计中，预期的患者结果可能更好，这反过来可能会修改估计的治疗效果。在特定的。在以专家为基础的设计中，较大的治疗效果有时可以抵消因临床医生效果与治疗混淆而产生的较高标准误差。这些概念是用两种胫骨骨折手术技术的随机试验数据来说明的。版权所有(C) 2008约翰威利父子有限公司
In order to avoid certain difficulties with the conventional randomized clinical trial design. the expertise-based design has been proposed as an alternative. In the expertise-based design, patients are randomized to clinicians (e.g. surgeons), who then treat all their patients with their preferred intervention. This design recognizes individual clinical preferences and so may reduce the rates of procedural crossovers compared with the conventional design. It may also facilitate recruitment of clinicians, because they are always allowed to deliver their therapy of choice, a feature that may also be attractive to patients.The expertise-based design avoids the possibility of so-called differential expertise bias. If a standard treatment is generally more familiar to clinicians than a new experimental treatment, then in the conventional design, more patients randomized to the standard treatment will have an expert clinician, compared with patients randomized to the experimental treatment. If expertise affects the study outcome, then a biased comparison of the treatment groups will occur.We examined the relative efficiency of estimating the treatment effect in the expertise-based and conventional designs. We recognize that expected patient outcomes may be better in the expertise-based design, which in turn may modify the estimated treatment effect. In particular. a larger treatment effect in the expertise-based design can sometimes offset a higher standard error arising from the confounding of clinician effects with treatments.These concepts are illustrated with data taken from a randomized trial of two alternative surgical techniques for tibial fractures. Copyright (C) 2008 John Wiley & Sons, Ltd.