Planning and Analyzing Clinical Trials with Composite Endpoints

✍ Scribed by Geraldine Rauch, Svenja Schüler, Meinhard Kieser

Publisher: Springer International Publishing
Year: 2017
Tongue: English
Leaves: 254
Series: Springer Series in Pharmaceutical Statistics
Edition: 1st ed.
Category: Library

No coin nor oath required. For personal study only.

✦ Synopsis

This book addresses the most important aspects of how to plan and evaluate clinical trials with a composite primary endpoint to guarantee a clinically meaningful and valid interpretation of the results. Composite endpoints are often used as primary efficacy variables for clinical trials, particularly in the fields of oncology and cardiology. These endpoints combine several variables of interest within a single composite measure, and as a result, all variables that are of major clinical relevance can be considered in the primary analysis without the need to adjust for multiplicity. Moreover, composite endpoints are intended to increase the size of the expected effects thus making clinical trials more powerful.

The book offers practical advice for statisticians and medical experts involved in the planning and analysis of clinical trials. For readers who are mainly interested in the application of the methods, all the approaches are illustrated with real-world clinical trial examples, and the software codes required for fast and easy implementation are provided. The book also discusses all the methods in the context of relevant guidelines related to the topic. To benefit most from the book, readers should be familiar with the principles of clinical trials and basic statistical methods.

✦ Table of Contents

Front Matter ....Pages i-xvi
Front Matter ....Pages 1-1
Definition and Rationale (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 3-7
Challenges of Composite Endpoints (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 9-12
Guideline View: Recommendations and Requirements (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 13-19
Clinical Trial Examples (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 21-27
Front Matter ....Pages 29-29
The Single-Stage Design (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 31-51
Group-Sequential and Adaptive Designs (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 53-63
Related Software Code (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 65-83
Front Matter ....Pages 85-86
Correlation Between Test Statistics (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 87-90
The Single-Stage Design (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 91-99
Group-Sequential and Adaptive Designs (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 101-106
Related Software Code (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 107-141
Front Matter ....Pages 143-143
Weighted Composite Binary Endpoint (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 145-149
Weighted Composite Time-to-Event Endpoint (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 151-155
Other Weighted Effect Measures (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 157-162
Related Software Code (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 163-187
Front Matter ....Pages 189-189
Descriptive Analysis of the Components (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 191-204
Supplementary Confirmatory Analyses of the Components (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 205-207
Related Software Code (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 209-216
Front Matter ....Pages 217-217
Clinical Trial Examples with (Composite) Binary Endpoints (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 219-224
Clinical Trial Examples with (Composite) Time-to-Event Endpoints (Geraldine Rauch, Svenja Schüler, Meinhard Kieser)....Pages 225-248
Back Matter ....Pages 249-255

✦ Subjects

Statistics; Statistics for Life Sciences, Medicine, Health Sciences; Biostatistics; Pharmaceutical Sciences/Technology

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