Title: Assessing Responder Analyses for Patient-Reported Outcomes: Maximum Responder Threshold and Effect

Speaker: Joseph C. Cappelleri, executive director of biostatistics at Pfizer Inc and adjunct professor of statistics at the University of Connecticut.

Time: Wednesday, October 21, 3:30pm,  Austin 313.

Abstract: Continuous outcomes are frequently dichotomized to classify subjects as “responders” or “nonresponders” with the difference in proportion of response between treatment and control groups defined as the “responder effect,” which has been used in regulatory and other settings. Despite the potential benefit of responder analyses in simplifying analysis and lending interpretation of clinically meaningful change between intervention groups, the dichotomization of a quantitative patient-reported outcome for this purpose has come under scrutiny for multiple reasons. The current research complements and supplements previous methodological evidence and conclusions by quantifying and depicting responder analyses for the broader and practical situations of varying group standard deviations. In doing so, new quantitative learnings and findings, with visual insights and enriched understanding, emerge on what threshold yields the maximum responder effect and what is the maximum responder effect. As such, this presentation is intended to provide advanced understanding and additional insights into responder analyses to aid in prudent interpretation of treatment effects.

Bio of Joseph C. Cappelleri: Dr. Joseph C. Cappelleri is an executive director of biostatistics at Pfizer Inc and adjunct professor of statistics at the University of Connecticut. Among the most published authors and most prolific medical researchers in the history of Pfizer and the pharmaceutical industry, Dr. Cappelleri has co-authored hundreds of publications (and external presentations) on clinical and methodological topics, including regression-discontinuity designs, meta-analysis, and health measurement scales, along with six books. He has been prominently involved in approximately 25 medical breakthroughs that have improved and extended quality of life for millions of patients worldwide. Dr. Cappelleri earned M.S. in statistics from the City University of New York (Baruch College), Ph.D. in psychometrics from Cornell University, and M.P.H. in epidemiology from Harvard University.