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

© 2013 – Chapman and Hall/CRC

351 pages | 177 B/W Illus.

Purchasing Options:
Hardback: 9781439873670
pub: 2013-12-19
US Dollars$104.95
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e–Inspection Copy

About the Book

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.

Reviews

"The book is clearly set-out and easy to follow and each chapter ends with a summary and detailed list of references. … an excellent general text covering the whole spectrum of working with PROs from design of measures through to the analysis of PRO data."

ISCB News, 59, June 2015

"The book is well written and well organized. Good examples contribute to a better understanding of statistics. The focus is quantitative and includes psychometric and statistical methods relevant to the vast majority of patient-reported outcome measures, those based on multi questions scales or summed assessment scales. The content is also relevant for individualized and preference-based instruments. … The book is recommended for health care professionals and health researchers who wish to further their understanding of psychometric and statistical methods necessary for the development, testing and use of patient-reported outcome measures."

—A Garratt, Journal of the Norwegian Medical Association, April 2015

"This book includes 11 chapters on 330 pages. The first six chapters cover topics in psychometrics, and the remaining five cover topics in statistics. … the book is a quick, easy, and understandable introduction to qualitative and quantitative psychometric methods for the inexperienced reader."

—John Brodersen, Statistics in Medicine, 2015

"The recent book, Patient-Reported Outcomes, Measurement, Implementation and Interpretation, by Cappelleri and his colleagues is a timely synthesis of the current thinking and practice of psychometrics applied to patient reported outcomes (PROs), and PRO data analysis. Written primarily by statisticians drawn from the same large multinational pharmaceutical company, the book is squarely aimed at health economists, outcomes researchers and statisticians in the industry who may require a foundation in psychometrics and the analysis of PRO data. That said it may also be of benefit and utility to advanced researchers in academia and consultancy wanting to increase their knowledge in this field. … In summary, this book attempts to cover a lot of complex material and does so successfully. It effectively teaches core concepts related to the measurement, analysis and interpretation of PROs. There is something for readers of all levels of familiarity with PROs, from the novice to the more experienced researcher."

Qual Life Res, 2014

"This is a timely publication that provides comprehensive information on an important subject. … there are many who can benefit from reading this book, including health care professionals, health services researchers, policy makers, regulators, patients, caregivers, and other health-related stakeholders. … This book includes many excellent simulated and real-life examples. … For many practitioners, the sample codes provided in the book provide an excellent template for them to carry out their own analyses. This book is well organized and clearly written. Accessible language and articulate writing make this a very readable book. Ample examples with step-by-step illustrations of the analysis of data and interpretation of the results will be particularly helpful to readers who are new to PROs. It can also serve as a valuable reference for those who have experience working with PROs."

Journal of Biopharmaceutical Statistics, 2014

Table of 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.

About the Series

Chapman & Hall/CRC Biostatistics Series

Learn more…

Subject Categories

BISAC Subject Codes/Headings:
MAT029000
MATHEMATICS / Probability & Statistics / General
MED071000
MEDICAL / Pharmacology
MED090000
MEDICAL / Biostatistics