24: Advancing Study Design: Insights into Study Power Optimization

Music City Center 

Composite endpoint is widely used in clinical trials to evaluate the efficacy of a new treatment or intervention, as it combines multiple clinically relevant outcomes into a single measure. Despite its advantage, a fully quantitative evaluation of factors influencing study power remains underexplored, including correlation between endpoints, effect size, randomization ratio and sample size. These elements can significantly impact the reliability of trial conclusion, particularly when composite endpoint is used as the primary objective. This study systematically investigates how these factors impact study power when composite endpoint, defined by two continuous outcomes, are used as the primary endpoint. By examining different scenarios of study design features, we explore how variations in these factors influence study power. The findings emphasize the critical role of endpoint correlation and effect size, along with the importance of balancing sample size and randomization ratio. This work provides a foundation for extending the methodology to more complex scenarios, including composite endpoint with more than two continuous variables and alternative endpoint types.

Composite Endpoint

Study Power

Simulation

Clinical Trial 

Biopharmaceutical Section