Time to Revisit Endpoint Selection in Clinical Trials |Biomedgrid

In clinical trials, power calculation is often performed based on a single primary endpoint to determine sample size required for achieving study objective with a desired power at a pre-specified level of significance. In practice, power calculation based on a single primary endpoint has been criticized. First, how to select the single primary endpoint among a group of primary endpoints? Second, a single primary endpoint may not be sufficient to adequately inform complex cohorts, the disease status and/or treatment effect of the test treatment under investigation. Third, different study endpoints with different data types (e.g., continuous versus binary response) may result in different sample sizes. In addition, with a given sample size, some (single) endpoints may achieve the study objective while others fail to do so. In this opinion article, a conceptual innovation is the development of a therapeutic index that fully utilizes information from all relevant study endpoints proposed.