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arXiv · 2001.03396

Decision tool and Sample Size Calculator for Composite Endpoints

Marta Bofill Roig·Jordi Cortés Martínez·Guadalupe Gómez Melis
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Abstract

Summary points: - This article considers the combination of two binary or two time-to-event endpoints to form the primary composite endpoint for leading a trial. - It discusses the relative efficiency of choosing a composite endpoint over one of its components in terms of: the frequencies of observing each component; the relative treatment effect of the tested therapy; and the association between both components. - We highlight the very important role of the association between components in choosing the most efficient endpoint to use as primary. - For better grounded future trials, we recommend trialists to always reporting the association between components of the composite endpoint. - Common fallacies to note when using composite endpoints: i) composite endpoints always imply higher power; ii) treatment effect on the composite endpoint is similar to the average effects of its components; and iii) the probability of observing the primary endpoint increases significantly.

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Marta Bofill Roig, Jordi Cortés Martínez, Guadalupe Gómez Melis. 2020-01-10. Decision tool and Sample Size Calculator for Composite Endpoints. https://arxiv.org/abs/2001.03396

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