Bayesian adaptive clinical trials: a dream for statisticians only?

Bayesian adaptive clinical trials: a dream for statisticians only?
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DOI:
10.1002/sim.4363
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发表时间:
2012-05-20
影响因子:
2
通讯作者:
Chevret, Sylvie
Chevret, Sylvie
中科院分区:
医学3区
文献类型:
--
作者:
Chevret, Sylvie

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自适应或灵活的设计已经出现,主要是在频域框架内,作为加快治疗评估过程的有效方法。由于其灵活性,贝叶斯方法也被提出用于第一阶段到第三阶段的适应性试验;然而,据报道,它们在实践中使用得很少。我们的目标是通过调查贝叶斯自适应方法在临床试验设置中的实际开发和使用来描述贝叶斯临床试验的国际科学成果。使用PubMed和科学引文索引扩展数据库进行了一项文献计量学研究。发现的大多数参考文献都是来自世界各地不同团队的生物统计学论文。大多数作者来自美国,其中很大一部分来自MD Anderson癌症中心(德克萨斯大学休斯顿分校)。这些文章的传播和使用严重依赖于它们的主题,3.1%的生物统计学文章在发表后5年内至少被引用25篇,相比之下,15%的综述文章和32%的临床文章被引用。我们还研究了在临床试验中限制使用贝叶斯自适应设计方法的原因,以及当前和未来应对这些挑战的研究领域。在统计学家和临床医生中推广贝叶斯方法的努力似乎是必要的。版权所有(C)2011 John Wiley&Sons,Ltd.
Adaptive or flexible designs have emerged, mostly within frequentist frameworks, as an effective way to speed up the therapeutic evaluation process. Because of their flexibility, Bayesian methods have also been proposed for Phase I through Phase III adaptive trials; however, it has been reported that they are poorly used in practice. We aim to describe the international scientific production of Bayesian clinical trials by investigating the actual development and use of Bayesian adaptive methods in the setting of clinical trials. A bibliometric study was conducted using the PubMed and Science Citation Index-Expanded databases. Most of the references found were biostatistical papers from various teams around the world. Most of the authors were from the US, and a large proportion was from the MD Anderson Cancer Center (University of Texas, Houston, TX). The spread and use of these articles depended heavily on their topic, with 3.1% of the biostatistical articles accumulating at least 25 citations within 5 years of their publication compared with 15% of the reviews and 32% of the clinical articles. We also examined the reasons for the limited use of Bayesian adaptive design methods in clinical trials and the areas of current and future research to address these challenges. Efforts to promote Bayesian approaches among statisticians and clinicians appear necessary. Copyright (C) 2011 John Wiley & Sons, Ltd.