Evaluating Treatment Effects with Patient-Response–Related Outcomes in Comparative Clinical Trials

AbstractIn comparative clinical investigations for nearly all disease types, patient responses - signifying clinically meaningful improvements in health or reductions in disease burden - are routinely employed as key end points for assessing treatment activity or efficacy. Trials typically use the response rate to summarize treatment effects, a metric with limitations for investigating the temporal profile of the effect. In this Clinical Trials Workshop, the authors present a novel approach for analyzing time to response and duration of response in clinical trials using, as an example, data from the JAVELIN Renal-101 trial of avelumab and axitinib versus sunitinib for treating renal cell carcinoma.