Cappelleri / Zou / Bushmakin Patient-Reported Outcomes

Measurement, Implementation and Interpretation
Erscheinungsjahr 2013
ISBN: 978-1-4398-7370-0
Verlag: CRC Press
Format: PDF
Kopierschutz: Adobe DRM (»Systemvoraussetzungen)

E-Book, Englisch, 351 Seiten

Reihe: Chapman & Hall/CRC Biostatistics Series

Advancing the development, validation, and use of patient-reported outcome (PRO) measures, Patient-Reported Outcomes: Measurement, Implementation and Interpretation helps readers develop and enrich their understanding of PRO methodology, particularly from a quantitative perspective. Designed for biopharmaceutical researchers and others in the health sciences community, it provides an up-to-date volume on conceptual and analytical issues of PRO measures.

The book discusses key concepts relating to the measurement, implementation, and interpretation of PRO measures. It covers both introductory and advanced psychometric and biostatistical methods for constructing and analyzing PRO measures. The authors include many relevant real-life applications based on their extensive first-hand experiences in the pharmaceutical industry. They implement a wealth of simulated datasets to illustrate concepts and heighten understanding based on practical scenarios. For readers interested in conducting statistical analyses of PRO measures and delving more deeply into the analytic details, most chapters contain SAS code and output that illustrate the methodology. Along with providing numerous references, the book highlights current regulatory guidelines.

Zielgruppe

Researchers and graduate students in healthcare, biostatistics, and the pharmaceutical industry.

Autoren/Hrsg.

Cappelleri, Joseph C.

Zou, Kelly H.

Bushmakin, Andrew G.

Alvir, Jose Ma. J.

Alemayehu, Demissie

Symonds, Tara

Fachgebiete

• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Medizin, Gesundheitswesen Epidemiologie, Medizinische Statistik
• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Medizinische Fachgebiete Pharmakologie, Toxikologie
• Mathematik | Informatik Mathematik Stochastik

Inhaltsverzeichnis

Introduction
Patient-Reported Outcomes in Perspective
Patient-Reported Outcomes in Clinical Research
Terms and Definitions
Measurement Scales
Psychometrics vs Clinimetrics
Selection of a PRO Questionnaire

Development of a Patient-Reported Outcome
Population
Item Generation
Item Wording
Cognitive Interviews

Validity
Content Validity
Construct Validity
Simulated Example Using SAS: Convergent and Divergent Validity
Factors Affecting Response

Reliability
Intraclass Correlation Coefficient for Continuous Variables
ICC Example
ICC Simulated Example
ICC in Context
Bland and Altman Plot for Continuous Variables
Simple Kappa and Weighted Kappa Coefficients for Categorical Variables
Internal Consistency Reliability: Cronbach’s Alpha Coefficient
Simulated Example of Cronbach’s Alpha

Exploratory and Confirmatory Factor Analyses
Exploratory Factor Analysis
Confirmatory Factor Analysis
Causal Indicators vs Effect Indicators
Simulated Examples Using SAS: Exploratory Factor Analysis
Simulated Examples Using SAS: Confirmatory Factor Analysis
Real-Life Examples

Item Response Theory
Classical Test Theory Revisited
Assumptions of IRT
Item Characteristic Curves
Item Information
Item Fit and Person Fit
Differential Item Functioning
Sample Size
Example
Example: Rasch Model Implementation

Cross-Sectional Analysis
Types of PRO Data and Exploratory Methods
Comparing Two or More Samples
Regression Analysis

Longitudinal Analysis
Analytic Considerations
Repeated Measures Model
Random Coefficient Model
Real-Life Examples

Mediation Models
Single Mediator Model
Model Invariance
Advanced Example
Bootstrapping Methodology Implementation

Missing Data
Study Design to Minimize Missing Data
Missing Data Patterns and Mechanisms
Approaches for Missing Items within Domains or Measures
Approaches for Missing Entire Domains or Entire Questionnaires
Sensitivity Analyses
Simulated Example Using SAS: Pattern Mixture Models

Enriching Interpretation
Anchor-Based Approaches
Distribution-Based Approaches
Multiple Testing

Index

A Summary and References appear at the end of each chapter.