TITLE: Advances in Interpretation of Patient-Reported Outcomes
INSTRUCTOR: Joseph C. Cappelleri, Pfizer
MODERATOR:Wenjin Wang
Abstract:
This presentation provides a review mainly on two broad approaches – anchor-based and distributed-based – aimed at enriching the understanding and meaning of patient-reported outcome scores. Anchor-based approaches use a measure (external to the targeted patient-reported outcome of interest) that is well interpretable and correlated with the targeted patient-reported outcome. Examples include percentages based on thresholds, cutoff scores based on severity, criterion-group interpretation, statistical significance and clinical significance, content-based interpretation, and clinically meaningful change and difference. Distributed-based approaches rely strictly on the distribution of the data. Examples include effect size, probability of relative benefit, and cumulative distribution functions. A third approach on interpretation – mediation analysis – is also highlighted and illustrated to assess the effect of treatment on an outcome variable (which may or may not be a patient-reported outcome) indirectly through the effect of a mediator variable (which may or may not be a patient-reported outcome) and otherwise directly through other effects. Applications are based on real-life and simulated examples.
References
Bushmakin AG, Cappelleri JC. A Practical Approach to Quantitative Validation of Patient-Reported Outcomes: A Simulation-Based Guide Using SAS. Hoboken, New Jersey: John Wiley & Sons. 2022.
Cappelleri JC, Zou KH, Bushmakin AG, Alvir JMJ, Alemayehu D, Symonds T. Patient-Reported Outcomes: Measurement, Implementation and Interpretation. Boca Raton, Florida: Chapman & Hall/CRC Press. 2013.
Cappelleri JC, Bushmakin AG. Interpretation of patient-reported outcomes. Statistical Methods in Medical Research. 2014; 23:460-483.
Instructors’ Biography:
Joseph C. Cappelleri earned his MS in statistics from the City University of New York (Baruch College), PhD in psychometrics from Cornell University, and MPH in epidemiology from Harvard University. Dr. Cappelleri is an executive director of biostatistics and the head of Health Economics & Outcomes Research Statistics in the Statistical Research and Data Science Center at Pfizer Inc, where he is the recipient of the Craig A. Saxton Clinical Development Excellence Award (Pfizer’s highest accolade). As an adjunct professor, he has served on the faculties at Brown University (biostatistics), Tufts Medical Center (medicine), and the University of Connecticut (statistics). 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 and hundreds of publications (and external presentations) on clinical and methodological topics, including regression-discontinuity designs, meta-analysis, and health measurement scales. He is the lead author of the book “Patient-Reported Outcomes: Measurement, Implementation and Interpretation” and has co-authored or co-edited five other books. Dr. Cappelleri is an elected Fellow of the American Statistical Association (ASA), elected recipient of the Long-Term Excellence Award from the Health Policy Statistics Section of the ASA, and elected recipient of the Avedis Donabedian Outcomes Research Lifetime Achievement Award from ISPOR — The Professional Society for Health Economics and Outcomes Research.