Gerke Oke, Høilund-Carlsen Poul Flemming, Vach Werner
Department of Nuclear Medicine, Odense University Hospital, Sdr. Boulevard 29, 5000, Odense C, Denmark; Department of Business and Economics, Centre of Health Economics Research, University of Southern Denmark, Campusvej 55, 5230, Odense M, Denmark.
Biom J. 2015 May;57(3):395-409. doi: 10.1002/bimj.201400020. Epub 2015 Mar 24.
When the efficacy of a new medical drug is compared against that of an established competitor in a randomized controlled trial, the difference in patient-relevant outcomes, such as mortality, is usually measured directly. In diagnostic research, however, the impact of diagnostic procedures is of an indirect nature as test results do influence downstream clinical decisions, but test performance (as characterized by sensitivity, specificity, and the predictive values of a procedure) is, at best, only a surrogate endpoint for patient outcome and does not necessarily translate into it. Not many randomized controlled trials have been conducted so far in diagnostic research, and, hence, we need alternative approaches to close the gap between test characteristics and patient outcomes. Several informal approaches have been suggested in order to close this gap, and decision modeling has been advocated as a means of obtaining formal approaches. Recently, the expected benefit has been proposed as a quantity that allows a simple formal approach, and we take up this suggestion in this paper. We regard the expected benefit as an estimation problem and consider two approaches to statistical inference. Moreover, using data from a previously published study, we illustrate the possible insights to be gained from the application of formal inference techniques to determine the expected benefit.
在随机对照试验中，当将一种新型药物的疗效与已确立的竞争药物进行比较时，通常会直接测量与患者相关的结局差异，例如死亡率。然而，在诊断研究中，诊断程序的影响具有间接性，因为检测结果确实会影响下游临床决策，但检测性能（以灵敏度、特异性和程序的预测值为特征）充其量只是患者结局的替代终点，并不一定会转化为实际的患者结局。到目前为止，诊断研究中尚未进行许多随机对照试验，因此，我们需要替代方法来弥合检测特征与患者结局之间的差距。为了弥合这一差距，已经提出了几种非正式方法，并且有人主张将决策建模作为获得正式方法的一种手段。最近，预期获益被提议作为一种能实现简单正式方法的量，我们在本文中采纳了这一建议。我们将预期获益视为一个估计问题，并考虑两种统计推断方法。此外，利用先前发表的一项研究中的数据，我们说明了应用正式推断技术来确定预期获益可能获得的见解。
