Evaluating Clinical Utility for Composite Hypothesis with Efficacy and Safety in Trial Design

Music City Center 

In clinical trials, new treatment options may present a favorable safety profile but demonstrate efficacy comparable to existing treatments. Traditional trial designs often prioritize efficacy as the primary endpoint, requiring large sample sizes for adequate power and often disregarding the safety benefits of new treatments. To address this, we propose a novel composite endpoint that integrates efficacy and safety for clinical utility, employing a weighted approach in clinical trial design. This study explores efficacy and safety endpoints as binary variables in a randomized clinical trial with two arms (control and test). We evaluate the operating characteristics of a combined hypothesis on clinical utility using this weighted method. Our proposed design shows superior operating characteristics for treatments with satisfactory efficacy and enhanced safety compared to designs focusing solely on efficacy. This approach not only leverages existing therapeutic insights related to both efficacy and safety but also considers the overall efficiency of the drug development from key stakeholders. The importance of balancing efficacy and safety is emphasized for optimal implementation.

Composite endpoint

Clinical Trial Design

Binary Endpoint 

Biopharmaceutical Section