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Introduction to Statistical Methods for Clinical Trials : Texts in Statistical Science - Thomas D. Cook

Introduction to Statistical Methods for Clinical Trials

Texts in Statistical Science

By: Thomas D. Cook (Editor), David L. DeMets (Editor)

Hardcover

Published: 26th November 2007
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Clinical trials have become essential research tools for evaluating the benefits and risks of new interventions for the treatment and prevention of diseases, from cardiovascular disease to cancer to AIDS. Based on the authors' collective experiences in this field, Introduction to Statistical Methods for Clinical Trials presents various statistical topics relevant to the design, monitoring, and analysis of a clinical trial.

After reviewing the history, ethics, protocol, and regulatory issues of clinical trials, the book provides guidelines for formulating primary and secondary questions and translating clinical questions into statistical ones. It examines designs used in clinical trials, presents methods for determining sample size, and introduces constrained randomization procedures. The authors also discuss how various types of data must be collected to answer key questions in a trial. In addition, they explore common analysis methods, describe statistical methods that determine what an emerging trend represents, and present issues that arise in the analysis of data. The book concludes with suggestions for reporting trial results that are consistent with universal guidelines recommended by medical journals.

Developed from a course taught at the University of Wisconsin for the past 25 years, this textbook provides a solid understanding of the statistical approaches used in the design, conduct, and analysis of clinical trials.

! There is much good material in this book. The individual chapters are well written and cover the technical aspects as well. A major strength is the ordering of topics to follow the thought process used in the development and implementation of a protocol from defining the question to reporting results. There are careful discussions on fundamental principles and the pivotal role played by statistics is well brought out. ! there is much that practicing pharmaceutical statisticians will find useful in this book. They will find the coverage of fundamental principles useful and the technical content of the book a good reference source. ! --Pharmaceutical Statistics, 2010 ! fits the need for a contemporary text and handbook that is oriented toward the clinical trial statistician. I highly recommend it and look forward to using it as both a primary and supplemental text in our curriculum, as well as a research resource. --James J. Dignam, University of Chicago, JASA, March 2009 The (technical) statistical content is the main focus of the book and this is what helps it to stand apart from most others on clinical trials (even the more obviously statistically orientated ones). It takes the reader to quite a technical background that would serve him or her well if moving on to research problems in the various areas covered, yet does not lose sight of practical issues. ! For those of us with the interest (and need) to grapple with these more statistical issues, I wholeheartedly recommend it. --Biometrics, December 2008 !The book is very well written and clear. ! the authors generally strike the right balance for the intended audience. The inclusion of many historically important as well as contemporary examples to illustrate various points throughout the text is a major strength, as is the inclusion of several modern topics not seen in other texts. As a basis for a course in clinical trials for graduate students in biostatistics, this book is outstanding. In addition, statisticians in the pharmaceutical industry, government, or academia ! will find this text extremely informative and useful." --Michael P. McDermott, University of Rochester Medical Center, Journal of Biopharmaceutical Statistics, 2008

List of figuresp. xi
List of tablesp. xv
Prefacep. xix
Author Attributionp. xxiii
Introduction to Clinical Trialsp. 1
History and Backgroundp. 3
Ethics of Clinical Researchp. 5
Types of Research Design and Types of Trialsp. 9
The Need for Clinical Trialsp. 15
The Randomization Principlep. 18
Timing of a Clinical Trialp. 18
Trial Organizationp. 20
Protocol and Manual of Operationsp. 22
Regulatory Issuesp. 22
Overview of the Bookp. 26
Defining the Questionp. 29
Statistical Frameworkp. 31
Elements of Study Questionp. 40
Outcome or Response Measuresp. 44
The Surrogate Outcomep. 56
Composite Outcomesp. 64
Summaryp. 73
Problemsp. 73
Study Designp. 75
Early Phase Trialsp. 76
Phase III/IV Trialsp. 85
Non-inferiority Designsp. 101
Screening, Prevention, and Therapeutic Designsp. 106
Adaptive Designsp. 109
Conclusionsp. 112
Problemsp. 112
Sample Sizep. 115
Sample Size versus Informationp. 116
A General Setup for Frequentist Designsp. 118
Loss to Follow-up and Non-adherencep. 122
Survival Datap. 124
Clustered Datap. 134
Tests for Interactionp. 136
Equivalence/Non-inferiority Trialsp. 137
Other Considerationsp. 138
Problemsp. 139
Randomizationp. 141
The Role of Randomizationp. 141
Fixed Randomization Proceduresp. 148
Treatment- and Response-Adaptive Randomization Proceduresp. 155
Covariate-Adaptive Randomization Proceduresp. 161
Summary and Recommendationsp. 165
Problemsp. 168
Data Collection and Quality Controlp. 171
Planning for Collection of Clinical Trial Datap. 172
Categories of Clinical Datap. 185
Data Quality Controlp. 194
Conclusionsp. 199
Survival Analysisp. 201
Backgroundp. 201
Estimation of Survival Distributionsp. 203
Comparison of Survival Distributionsp. 213
Regression Modelsp. 219
Composite Outcomesp. 227
Summaryp. 228
Problemsp. 229
Longitudinal Datap. 231
A Clinical Longitudinal Data Examplep. 232
The Subject-specific Modelp. 234
Two-stage Estimationp. 237
The Random-effects, Subject-specific Modelp. 242
The Population-average (Marginal) Modelp. 246
Restricted Maximum Likelihood Estimation (REML)p. 252
Standard Errorsp. 253
Testingp. 255
Additional Levels of Clusteringp. 258
Generalized Estimating Equations for Non-normal Datap. 260
Missing Datap. 263
Summaryp. 264
Quality of Lifep. 267
Defining QoLp. 268
Types of QoL Assessmentsp. 268
Selecting a QoL Instrumentp. 271
Developing a QoL Instrumentp. 273
Quality of Life Datap. 273
Analysis of QoL Datap. 275
Summaryp. 286
Data Monitoring and Interim Analysisp. 289
Data and Safety Monitoringp. 290
Examplesp. 292
The Repeated Testing Problemp. 293
Group Sequential Testsp. 299
Triangular Testp. 311
Curtailment Proceduresp. 315
Inference Following Sequential Testsp. 322
Discussionp. 329
Problemsp. 336
Selected Issues in the Analysisp. 339
Bias in the Analysis of Clinical Trial Datap. 339
Choice of Analysis Populationp. 340
Missing Datap. 354
Subgroup Analysesp. 364
Multiple Testing Proceduresp. 370
Summaryp. 375
Problemsp. 376
Closeout and Reportingp. 377
Closing Out a Trialp. 377
Reporting Trial Resultsp. 378
Problemsp. 392
Delta Method, Maximum Likelihood Theory, and Informationp. 393
Delta Methodp. 393
Asymptotic Theory for Likelihood Based Inferencep. 393
Hypothesis Testingp. 395
Computing the MLEp. 399
Informationp. 400
Brownian Motionp. 403
Referencesp. 405
Indexp. 427
Table of Contents provided by Ingram. All Rights Reserved.

ISBN: 9781584880271
ISBN-10: 1584880279
Series: Texts in Statistical Science
Audience: Tertiary; University or College
Format: Hardcover
Language: English
Number Of Pages: 464
Published: 26th November 2007
Publisher: Taylor & Francis Inc
Country of Publication: US
Dimensions (cm): 24.13 x 16.51  x 2.54
Weight (kg): 0.77
Edition Number: 1