title: On responder analyses in the framework of within subject comparisons - considerations and two case studies.
abstract: A responder analysis is a common tool when clinical data are reported. In this paper, we extend the definition of responders to within subject comparisons and present a rigorous definition of the corresponding statistical functional. Via simulation studies, we get further insights under which conditions these analyses can even result in a higher power compared with an analysis based on the arithmetic mean. We report two case studies where these analyses contributed to a better understanding of the clinical data especially as some large observations were present that had a notable impact on the observed standard deviation.
authors.affiliation: Bayer Pharma AG, Global Clinical Statistics, Berlin, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Kunz
mesh_terms: D001921:Brain / Q000473:pathology; D002986:Clinical Trials as Topic / Q000379:methods*; D003198:Computer Simulation; D003287:Contrast Media / Q000494:pharmacology; D003627:Data Interpretation, Statistical; D006801:Humans; D008279:Magnetic Resonance Imaging / Q000379:methods; D016896:Treatment Outcome
chemical_list: D003287:Contrast Media
keywords: clinical trials; responder analysis; statistical functional; within subject comparison