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

Mao, Lu; Tian, Lu; Huang, Bo; Richardson, Paul G; Ludmir, Ethan B; Hughes, Michael D; Wei, Lee-Jen · NEJM Evid · 2025

review · Level V

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Abstract

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.

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