A Win Odds Approach to Advancing Dose Optimization in Drug development

Music City Center 

Choosing the optimal dose is critical for drug development. The FDA's Project Optimus emphasizes that dose optimization should be based on the totality of safety, efficacy, PK, and PD. Current methods, such as the MTD approach, focus solely on safety, while others, like the Clinical Utility Score (CUS), combine weighted endpoints with obscure weight assignments. In contrast, the win odds method offers a comprehensive benefit-risk assessment, which integrates different efficacy, safety, and other endpoints into one composite endpoint. Specifically, the win odds can determine a winner by comparing the overall outcomes of pairs of patients on two different doses, from the most to the least important outcome. Additionally, it can assess benefit and risk within multiple candidate doses. Therefore, the win odds test can identify the optimal dose that has significantly more winners than the other doses. Extensive simulations are conducted to explore the robustness of win odds method across various scenarios. It is also compared with the CUS method and applied to a clinical trial. Overall, the win odds method provides an effective and straightforward approach to dose optimization.

Dose Optimization

Benefit-Risk Analysis

Win Odds 

Isolated Statisticians