Matthews Introduction to Randomized Controlled Clinical Trials, Second Edition

2. Auflage 2010
ISBN: 978-1-4200-1130-2
Verlag: Taylor & Francis
Format: PDF
Kopierschutz: Adobe DRM (»Systemvoraussetzungen)

E-Book, Englisch, 302 Seiten

Reihe: Chapman & Hall/CRC Texts in Statistical Science

Evidence from randomized controlled clinical trials is widely accepted as the only sound basis for assessing the efficacy of new medical treatments. Statistical methods play a key role in all stages of these trials, including their justification, design, and analysis. This second edition of Introduction to Randomized Controlled Clinical Trials provides a concise presentation of the principles applied in this area. It details the concepts behind randomization and methods for designing and analyzing trials and also includes information on meta-analysis and specialized designs, such as cross-over trials, cluster-randomized designs, and equivalence studies.

This latest edition features new and revised references, examples, exercises, and a new chapter dedicated to binary outcomes and survival analysis. It also presents numerous examples taken from the medical literature, contains exercises at the end of each chapter, and offers solutions in an appendix. The author uses Minitab and R software throughout the text for implementing the methods that are presented.

Comprehensive and accessible, Introduction to Randomized Controlled Clinical Trials is well-suited for those familiar with elementary statistical ideas and methods who want to further their knowledge of the subject.

Zielgruppe

Advanced undergraduate and graduate students in statistics, biostatistics, and mathematics, medical science researchers, and statisticians working on clinical trials in healthcare and the pharmaceutical industry.

Autoren/Hrsg.

Matthews, John N.S.

Fachgebiete

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

Inhaltsverzeichnis

WHAT IS A RANDOMIZED CONTROLLED TRIAL?

Definition and Key Features

Historical Context and the Nature of RCTS

Structure and Justification of RCTs

Exercises

BIAS

What is meant by bias in RCTs?

Types of Bias

Exercises

HOW MANY PATIENTS DO I NEED?

Criteria for Sample Size Calculations

Hypothesis Tests

Sample Size for a Normally Distributed Variable

Sample Size for a Binary Variable

General Remarks About Sample Size Calculations

Exercises

METHODS OF ALLOCATION

Simple Randomization

Random Permuted Blocks

Biased Coin Designs and Urn Schemes

Unequal Randomization

Stratification

Minimization

Exercises

ASSESSMENT, BLINDING AND PLACEBOS

Double and Single Blindness

Placebos

Practical Considerations

Exercises

ANALYSIS OF RESULTS

Example

Use of Confidence Intervals

Baselines: Uses and Abuses

Analysis of Covariance

Exercises

FURTHER ANALYSIS: BINARY AND SURVIVAL DATA

Binary Data: An Example and a Statistical Model

Point Estimates and Hypothesis Tests

Interval Estimates for the Binary Case

Adjusting Binary Outcomes for Baseline Observations
Survival Analysis

Analyses Using Randomization Models

Exercises

MONITORING ACCUMULATING DATA

Motivation and Problems with Repeated Analysis of Data

Sequential and Group-Sequential Methods

Other Approaches to Accumulating Data

Data Monitoring Committees

Exercises
SUBGROUPS AND MULTIPLE OUTCOMES

The Role of Sub-groups in Randomized Clinical Trials

Methods for Comparing Sub-groups

Methods of Selecting Sub-groups

Qualitative Interactions

Multiple outcomes

Correction of P-values

Some Alternative Methods for Multiple Outcomes

Exercises

PROTOCOLS AND PROTOCOL DEVIATIONS
Protocols: Their Nature and Role
Protocol Deviation
Analysis by Intention-to-Treat
Exercises

SOME SPECIAL DESIGNS: CROSSOVERS, EQUIVALENCE AND CLUSTERS
Crossover Trials and Parallel Group Trials
The AB/BA Design
Analysis of AB/BA Design for Continuous Outcomes
The Issue of Carryover
Equivalence Trials
Cluster Randomized Trials
Exercises

META-ANALYSES OF CLINICAL TRIALS
What Are Meta-Analyses and Why Are They Needed?
Some Methodology for Meta-Analysis
Some Graphical Methods for Meta-Analysis
Some General Issues in Meta-Analysis
Exercises

FURTHER READING
SOLUTIONS TO EXERCISES
REFERENCES