Decision tool and Sample Size Calculator for Composite Endpoints
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.
Cite
@article{arxiv.2001.03396,
title = {Decision tool and Sample Size Calculator for Composite Endpoints},
author = {Marta Bofill Roig and Jordi Cortés Martínez and Guadalupe Gómez Melis},
journal= {arXiv preprint arXiv:2001.03396},
year = {2020}
}