ABSTRACT

Summary: In clinical trials, surrogate outcomes are early measures of treatment effect that are used to predict treatment effect on a later primary outcome of interest: the primary outcome therefore does not need to be observed and trials can be shortened. Evaluating surrogates is a complex area as a given treatment can act through multiple pathways, some of which may circumvent the surrogate. One of the best established and practically sound approaches to surrogacy evaluation is based on information theory. We have extended this approach to the case of ordinal outcomes, which are used as primary outcomes in many medical areas. This extension provides researchers with the means of evaluating surrogates in this setting, which expands the usefulness of the information theory approach while also demonstrating its versatility.

摘要

简介：在临床试验中，替代结局是治疗效果的早期指标，可用于预测对感兴趣的较晚主要结局的治疗效果：因此，无需观察主要结局，可以缩短试验时间。评估替代物是一个复杂的领域，因为给定的治疗可以通过多种途径起作用，其中某些途径可能会绕过替代物。代孕评估的最完善，最实用的方法之一是基于信息论。我们已将这种方法扩展到顺序结果的情况，在许多医学领域中，这些结果被用作主要结果。此扩展为研究人员提供了在这种情况下评估替代指标的方法，这扩展了信息论方法的实用性，同时也证明了它的多功能性。