Adaptive trial designs in diagnostic accuracy research
Adaptive trial designs in diagnostic accuracy research
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DOI：
10.1002/sim.8430
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发表时间：
2019-11-27
影响因子：
2
通讯作者：
Friede，Tim
中科院分区：
文献类型：
--
作者：
Zapf，Antonia;Stark，Maria;Friede，Tim
诊断准确性研究的目的是评估诊断试验区分患病个体和非患病个体的准确性。根据研究问题，不同的研究设计和准确性措施是适当的。由于计划阶段的先验知识通常非常有限，因此在正在进行的试验期间修改设计方面（例如样本量）可以提高诊断试验的效率。在干预研究中，组序贯设计和适应性设计已得到很好的建立。此类设计的特征在于预先计划的中期分析，从而提供了因有效性或无效性而提前停止或修改研究设计要素的机会。相比之下，在诊断准确性研究中，这种灵活的设计不太常见，即使它们与干预研究一样重要。然而，诊断准确性研究具有特定的特征，这可能需要调整统计方法，或者可能导致序贯和自适应设计的特定优势或局限性。本文就柔性设计在诊断准确性研究中的方法学研究和应用现状进行综述。此外，我们指出，并提倡未来的发展，自适应设计方法和它们的使用，从跨学科的角度诊断准确性试验。术语“跨学科观点”描述了学术和非学术研究专家的合作。
The aim of diagnostic accuracy studies is to evaluate how accurately a diagnostic test can distinguish diseased from nondiseased individuals. Depending on the research question, different study designs and accuracy measures are appropriate. As the prior knowledge in the planning phase is often very limited, modifications of design aspects such as the sample size during the ongoing trial could increase the efficiency of diagnostic trials. In intervention studies, group sequential and adaptive designs are well established. Such designs are characterized by preplanned interim analyses, giving the opportunity to stop early for efficacy or futility or to modify elements of the study design. In contrast, in diagnostic accuracy studies, such flexible designs are less common, even if they are as important as for intervention studies. However, diagnostic accuracy studies have specific features, which may require adaptations of the statistical methods or may lead to specific advantages or limitations of sequential and adaptive designs. In this article, we summarize the current status of methodological research and applications of flexible designs in diagnostic accuracy research. Furthermore, we indicate and advocate future development of adaptive design methodology and their use in diagnostic accuracy trials from an interdisciplinary viewpoint. The term “interdisciplinary viewpoint” describes the collaboration of experts of the academic and nonacademic research.