American Statistical Association
New York City
Metropolitan Area Chapter
May 23, 2012
The New York Metro Area Chapter of the American Statistical Association
In Collaboration With
The School of Health Sciences at Touro College
Are Pleased to Invite You to a Seminar
RESPONDER ANALYSES - A PhRMA POSITION PAPER
Joseph C. Cappelleri, Ph.D., M.P.H.
Ideally, a clinical study should be able to demonstrate not only a statistically significant improvement in a key endpoint, but also that the magnitude of the effect is clinically relevant. One approach to address the question, often proposed by regulatory guidances, is a responder analysis, whereby a continuous efficacy measure is dichotomized into "responders" and "nonresponders." This presentation is taken directly from a recently published paper on responder analyses (Uryniak et al. Responder Analyses-A PhRMA Position Paper. Statistics in Biopharmaceutical Research 2011; 3:476-487) and represents a Pharmaceutical Research and Manufacturers of America (PhRMA) position on responder analyses. After providing a literature review, this presentation covers the results of simulations and the logic of conceptual underpinnings to highlight the consequences of dichotomization on statistical power and the implication of responder analyses in the assessment of a clinically relevant treatment effect. Any real or perceived benefit from a responder analysis is found to be outweighed by its loss of statistical power on the existence of a treatment effect. However, between-group comparisons on the percentages of "responders" can play a role in the assessment and reporting of the clinical meaningfulness of the treatment effect.
About the Speaker
Joseph C. Cappelleri earned his M.S. in statistics from the City University of New York, Ph.D. in psychometrics from Cornell University, and M.P.H. in epidemiology from Harvard University. In June 1996, Dr. Cappelleri joined Pfizer Inc. as a statistical scientist collaborating with Outcomes Research and is a senior director in biostatistics at Pfizer. He is also an adjunct professor of medicine at Tufts Medical Center and an adjunct professor of statistics at the University of Connecticut. He has delivered numerous conference presentations and has published extensively on clinical and methodological topics, including regression-discontinuity designs, meta-analyses, and health measurement scales. Dr. Cappelleri has been instrumental in developing and validating a number of patient-reported outcomes for different diseases and conditions. He is a Fellow of the American Statistical Association.
Joseph C. Cappelleri, Ph.D., M.P.H.
Responder Analyses - A PhRMA Position Paper
Tom Uryniak, Ivan S.F. Chan, Valerii V. Fedorov, Qi Jiang, Leonard Oppenheimer,
Steven M. Snapinn, Chi-Hse Teng and John Zhang (2011):
Statistics in Biopharmaceutical Research, 3:3, 476-487.
Consequences of Dichotomization
Valerii Fedorov1, Frank Mannino1, and Rongmei Zhang, (2009):
Pharmaceutical Statistics, 8: 50-61.
Wednesday, May 23, 2012
4:00 to 6:00 P.M.
27 West 23rd Street
(between 5th and 6th Avenues)
New York, New York
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