A Seamless Design for the Combination of a Case-Control and a Cohort Diagnostic Accuracy Study

病例对照研究与队列研究诊断准确性研究的无缝结合设计

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Abstract

In determining the accuracy of a new diagnostic test, often two steps are performed. In the first step, a case-control study is performed as an efficient but potentially biased design. In a second step, a population-based cohort study is performed as an unbiased but less efficient design. In order to accelerate diagnostic research, it has recently been suggested to combine the two designs in one seamless design. In this article, we present a more in-depth description of this idea. The seamless diagnostic accuracy study design is formally introduced by comparison with the traditional pathway, and the basic design decisions are discussed: A stopping rule and a stopping time. An appealing feature of the design is the possibility to ignore the seamless design in the final analysis, although part of the data is used already in an interim analysis. The justification for this strategy is provided by a large-scale simulation study. The simulation study suggests also that the risk of a loss of power due to using a seamless design can be limited by a reasonable choice of the futility boundaries, defining the stopping rule. We conclude that the seamless diagnostic accuracy study design seems to be ready to use. It promises to accelerate diagnostic research, in particular if population-based cohort studies can be started without great efforts and if the reference standard can be evaluated with little delay.

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