An evaluation of composite endpoints using data from the EXCITE trial

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In many clinical trials that assess the effect of an intervention, a single outcome does not adequately capture the effect of the intervention. In these cases, a composite outcome that combines information from several individual outcomes may be preferable. Although there have been a number of statistical tests proposed for composite outcomes, these methods tend to focus on the technical issues of maintain the type I error rate and the effects on statistical power. These are important considerations for designing trials, but do not address other key aspects, such as the characteristics of individual outcomes that would make up a clinical relevant composite measure. This proposal is in response to a Notice of Special Interest for using NICHD data resources in secondary data analyses. The proposal will do secondary analyses of the data from the EXCITE trial, available on the NICHD Data and Specimen Hub (DASH), to investigate the proposed statistical methods with actual clinical trial data, and to gain insight into the characteristics of individual outcomes that combine into a relevant composite outcome.