Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review.
Biomarker-Guided Non-Adaptive Trial Designs in Phase II and Phase III: A Methodological Review.
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DOI：
10.3390/jpm7010001
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发表时间：
2017-01-25
影响因子：
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通讯作者：
Jorgensen AL
中科院分区：
文献类型：
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作者：
Antoniou M;Kolamunnage-Dona R;Jorgensen AL
生物标志物引导的治疗是医学的一个快速发展的领域，其中治疗选择根据个体的一个或多个生物标志物测量进行个性化。在过去的十年中，已经提出了许多生物标志物指导的试验设计，包括适应性和非适应性试验设计，这些试验设计测试生物标志物指导的治疗方法的有效性，目的是改善患者健康。由于试验设计和分析方面都存在挑战，因此需要更好地了解它们。我们已经进行了全面的文献综述的基础上，在深入的搜索策略，以提供研究界的各种生物标志物指导的试验设计（适应性和非适应性设计）的定义，方法和术语的清晰度，从共211纳入的论文。在本文中，我们专注于非自适应生物标志物引导的试验设计，我们已经确定了100篇论文中提到的五种不同的主要类型。我们以图形方式展示了每种非适应性试验设计，并对其关键特征进行了深入概述。在如何描述试验设计方面，特别是在不同作者使用的术语方面，观察到了很大的差异。我们的全面综述为设计生物标志物指导试验提供了指导。
Biomarker-guided treatment is a rapidly developing area of medicine, where treatment choice is personalised according to one or more of an individual’s biomarker measurements. A number of biomarker-guided trial designs have been proposed in the past decade, including both adaptive and non-adaptive trial designs which test the effectiveness of a biomarker-guided approach to treatment with the aim of improving patient health. A better understanding of them is needed as challenges occur both in terms of trial design and analysis. We have undertaken a comprehensive literature review based on an in-depth search strategy with a view to providing the research community with clarity in definition, methodology and terminology of the various biomarker-guided trial designs (both adaptive and non-adaptive designs) from a total of 211 included papers. In the present paper, we focus on non-adaptive biomarker-guided trial designs for which we have identified five distinct main types mentioned in 100 papers. We have graphically displayed each non-adaptive trial design and provided an in-depth overview of their key characteristics. Substantial variability has been observed in terms of how trial designs are described and particularly in the terminology used by different authors. Our comprehensive review provides guidance for those designing biomarker-guided trials.