A Practical Approach to Quantitative Validation of Patient-Reported Outcomes: A Simulation-based Guide Using SAS (Statistics in Practice) - Tapa dura

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ANDREW G. BUSHMAKIN earned his M.S. in applied mathematics and physics from the National Research Nuclear University (former Moscow Engineering Physics Institute, Moscow, Russia). He has more than 20 years of experience in mathematical modeling and data analysis. He is a director of biostatistics in the Statistical Research and Data Science Center at Pfizer Inc. He has co-authored numerous articles and presentations on topics ranging from mathematical modeling of neutron physics processes to patient-reported outcomes, as well as several monographs.

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. He is an Executive Director of biostatistics in the Statistical Research and Data Science Center at Pfizer Inc. As an adjunct professor, he has served on the faculties of Brown University, University of Connecticut, and Tufts Medical Center. He has delivered numerous conference presentations and has published extensively on clinical and methodological topics. He is a fellow of the American Statistical Association and recipient of the ISPOR Avedis Donabedian Outcomes Research Lifetime Achievement Award.

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A Practical Approach to Quantitative Validation of Patient-Reported Outcomes

A Simulation-Based Guide Using SAS

In A Practical Approach to Quantitative Validation of Patient-Reported Outcomes, two distinguished researchers, with 50 years of collective research experience and hundreds of publications on patient-centered research, deliver a detailed and comprehensive exposition on the critical steps required for quantitative validation of patient-reported outcomes (PROs). The book provides an incisive and instructional explanation and discussion on major aspects of psychometric validation methodology on PROs, especially relevant for medical applications sponsored by the pharmaceutical industry, where SAS is the primary software, and evaluated in regulatory and other healthcare environments.

Central topics include test-retest reliability, exploratory and confirmatory factor analyses, construct and criterion validity, responsiveness and sensitivity, interpretation of PRO scores and findings, and meaningful within-patient change and clinical important difference. The authors provide step-by-step guidance while walking readers through how to structure data prior to a PRO analysis and demonstrate how to implement analyses with simulated examples grounded in real-life scenarios.

Readers will also find:

• A thorough introduction to patient-reported outcomes, including their definition, development, and psychometric validation
• Comprehensive explorations of the validation workflow, including discussions of clinical trials as a data source for validation and the validation workflow for single and multi-item scales
• In-depth discussions of key concepts related to a validation of a measurement scale
• Special attention is given to the US Food and Drug Administration (FDA) guidance on development and validation of the PROs, which lay the foundation and inspiration for the analytic methods executed

A Practical Approach to Quantitative Validation of Patient-Reported Outcomes is a required reference that will benefit psychometricians, statisticians, biostatisticians, epidemiologists, health service and public health researchers, outcome research scientists, regulators, and payers.

STATISTICS IN PRACTICE

A series of practical books outlining the use of statistical techniques in a wide range of applications areas:

• HUMAN AND BIOLOGICAL SCIENCES
• EARTH AND ENVIRONMENTAL SCIENCES
• INDUSTRY, COMMERCE AND FINANCE