Kehl, V.; Ulm, Kurt (2003): Responder Identification in Clinical Trials with Censored Data. Sonderforschungsbereich 386, Discussion Paper 311




We present a newly developed technique for identification of positive and negative responders to a new treatment which was compared to a classical treatment (or placebo) in a randomized clinical trial. This bump-hunting-based method was developed for trials in which the two treatment arms do not differ in survival overall. It checks in a systematic manner if certain subgroups, described by predictive factors do show difference in survival due to the new treatment. Several versions of the method were discussed and compared in a simulation study. The best version of the responder identification method employs martingale residuals to a prognostic model as response in a stabilized through bootstrapping bump hunting procedure. On average it recognizes 90% of the time the correct positive responder group and 99% of the time the correct negative responder group.