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Design and Analysis of Quality of Life Studies in Clinical Trials

By Diane L. Fairclough

Chapman and Hall/CRC – 2002 – 328 pages

Series: Chapman & Hall/CRC Interdisciplinary Statistics

Purchasing Options:

  • Hardback:
    978-1-58488-263-3
    March 28th 2002
    Out-of-print

Description

More and more frequently, clinical trials include the evaluation of Health-Related Quality of Life (HRQoL), yet many investigators remain unaware of the unique measurement and analysis issues associated with the assessment of HRQoL. At the end of a study, clinicians and statisticians often face challenging and sometimes insurmountable analytic problems.

Design and Analysis of Quality of Life Studies in Clinical Trials details these issues and presents a range of solutions. Written from the author's extensive experience in the field, it focuses on the very specific features of QoL data: its longitudinal nature, multidimensionality, and the problem of missing data. The author uses three real clinical trials throughout her discussions to illustrate practical implementation of the strategies and analytic methods presented.

As Quality of Life becomes an increasingly important aspect of clinical trials, it becomes essential for clinicians, statisticians, and designers of these studies to understand and meet the challenges this kind of data present. In this book, SAS and S-PLUS programs, checklists, numerous figures, and a clear, concise presentation combine to provide readers with the tools and skills they need to successfully design, conduct, analyze, and report their own studies.

Reviews

"This book not only provides a comprehensive review of the growing literatures and research activities, but also sheds light on new strategic and research directions. Throughout the book chapters, many real examples or simulation studies are used to illustrate the concepts, which make the contents relevant for the reader sand easier to understand. … In general, this book provides an excellent source of references for anyone, including statisticians and non-statisticians, who are interested in global drug development and registration."

—William W.B. Wang,Merck Research Laboratories, Merck & Co., Inc., Beijing, China

Contents

INTRODUCTION

Health-Related Quality of Life

Measuring Health-Related Quality of Life

Example 1: Adjuvant Breast Cancer Trial

Example 2: Advanced Non-Small-Cell Lung Cancer (NSCLC)

Example 3: Renal Cell Carcinoma Trial

Summary

STUDY DESIGN AND PROTOCOL DEVELOPMENT

Introduction

Background and Rationale

Research Objectives

Selection of Subjects

Longitudinal Designs

Selection of a Quality of Life Measure

Conduct

Summary

MODELS FOR LONGITUDINAL STUDIES

Introduction

Building the Analytic Models

Building Repeated Measures Models

Building Growth Curve Models

Summary

MISSING DATA

Introduction

Patterns of Missing Data

Mechanisms of Missing Data

Summary

ANALYTIC METHODS FOR IGNORABLE MISSING DATA

Introduction

Repeated Univariate Analyses

Multivariate Methods

Baseline Assessment as a Covariate

Change from Baseline

Empirical Bayes Estimates

Summary

SIMPLE IMPUTATION

Introduction

Mean Value Substitution

Explicit Regression Models

Last Value Carried Forward

Underestimation of Variance

Sensitivity Analysis

Summary

MULTIPLE IMPUTATION

Introduction

Overview of Multiple Imputation

Explicit Univariate Regression

Closest Neighbor and Predictive Mean Matching

Approximate Bayesian Bootstrap

Multivariate Procedures for Nonmonotone Missing Data

Combining the M Analyses

Sensitivity Analyses

Imputation vs. Analytic Models

Implications for Design

Summary

PATTERN MIXTURE MODELS

Introduction

Bivariate Data (Two Repeated Measures)

Monotone Dropout

Parametric Models

Additional Reading

Algebraic Details

Summary

RANDOM-EFFECTS MIXTURE, SHARED-PARAMETER, AND SELECTION MODELS

Introduction

Conditional Linear Model

Joint Mixed-Effects and Time to Dropout

Selection Model for Monotone Dropout

Advanced Readings

Summary

SUMMARY MEASURES

Introduction

Choosing a Summary Measure

Constructing Summary Measures

Summary Statistics across Time

Summarizing Across HRQoL Domains or Subscales

Advanced Notes

Summary

MULTIPLE ENDPOINTS

Introduction

Background Concepts and Definitions

Multivariate Statistics

Univariate Statistics

Resampling Techniques

Summary

DESIGN: ANALYSIS PLANS

Introduction

General Analysis Plan

Models for Longitudinal Data

Multiplicity of Endpoints

Sample Size and Power

Reported Results

Summary

APPENDICES

BIBLIOGRAPHY

Name: Design and Analysis of Quality of Life Studies in Clinical Trials (eBook)Chapman and Hall/CRC 
Description: By Diane L. Fairclough. More and more frequently, clinical trials include the evaluation of Health-Related Quality of Life (HRQoL), yet many investigators remain unaware of the unique measurement and analysis issues associated with the assessment of HRQoL. At the end of a...
Categories: Statistics for the Biological Sciences, Clinical Implementation