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Patient-Reported Outcomes

Measurement, Implementation and Interpretation

By Joseph C. Cappelleri, Kelly H. Zou, Andrew G. Bushmakin, Jose Ma. J. Alvir, Demissie Alemayehu, Tara Symonds

Chapman and Hall/CRC – 2013 – 351 pages

Series: Chapman & Hall/CRC Biostatistics Series

Purchasing Options:

  • Add to CartHardback: $99.95
    978-1-43-987367-0
    December 20th 2013

Description

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.

Contents

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.

Name: Patient-Reported Outcomes: Measurement, Implementation and Interpretation (Hardback)Chapman and Hall/CRC 
Description: By Joseph C. Cappelleri, Kelly H. Zou, Andrew G. Bushmakin, Jose Ma. J. Alvir, Demissie Alemayehu, Tara Symonds. 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...
Categories: Statistics for the Biological Sciences, Clinical Implementation