JAMA Network Open | 2021
Comparison of Duration of Response vs Conventional Response Rates and Progression-Free Survival as Efficacy End Points in Simulated Immuno-oncology Clinical Trials
Abstract
Key Points Question Does duration of response (DOR), a rigorous metric combining both tumor responses and duration, better inform decisions about continuing or discontinuing before starting phase 3 immuno-oncology clinical trials? Findings This simulated modeling study, which was based on simulated randomized phase 2 trials that resampled patients from completed randomized phase 3 trials of immune checkpoint inhibitors, found that restricted mean DOR consistently outperformed progression-free survival and objective response rate in correctly estimating positive overall survival benefits without inflating type I errors. Meaning These findings suggest that restricted mean DOR may be a sensitive and informative early efficacy end point in randomized phase 2 trials in immuno-oncology.