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© 2017

Planning and Analyzing Clinical Trials with Composite Endpoints

  • Discusses methods for planning and evaluating clinical trials with a composite primary endpoint

  • Offers software codes for the implementation of methods

  • Summarizes current medical and methodology guideline documents on composite endpoints

  • Includes recommendations and guidance for planning clinical trials with composite endpoints

  • Provides numerous examples from clinical trials illustrating the different methods

Book

Part of the Springer Series in Pharmaceutical Statistics book series (SSPS)

Table of contents

  1. Front Matter
    Pages i-xvi
  2. Part I

    1. Front Matter
      Pages 1-1
    2. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 3-7
    3. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 9-12
    4. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 13-19
    5. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 21-27
  3. Part II

    1. Front Matter
      Pages 29-29
    2. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 31-51
    3. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 53-63
    4. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 65-83
  4. Part III

    1. Front Matter
      Pages 85-86
    2. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 87-90
    3. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 91-99
    4. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 101-106
    5. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 107-141
  5. Part IV

    1. Front Matter
      Pages 143-143
    2. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 145-149
    3. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 151-155
    4. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 157-162
    5. Geraldine Rauch, Svenja Schüler, Meinhard Kieser
      Pages 163-187

About this book

Introduction

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.

Keywords

62L05, 62P10 composite endpoints clinical trials sample size calculation sequential designs multiple testing survival analysis competing risks

Authors and affiliations

  1. 1.Institute of Biometry and Clinical EpidemiologyCharité - Universitätsmedizin BerlinBerlinGermany
  2. 2.Institute of Medical Biometry and InformaticsUniversity of HeidelbergHeidelbergGermany
  3. 3.Institute of Medical Biometry and InformaticsUniversity of HeidelbergHeidelbergGermany

About the authors

Since 2017, Prof. Dr. Geraldine Rauch has been a professor of Medical Biometry and Director of the Institute of Biometry and Clinical Epidemiology at the Charité Universitätsmedizin Berlin. Prior to that, she had a professorship at the University Medical Center Hamburg-Eppendorf. From 2009 to 2016, she worked at the Institute of Medical Biometry and Informatics, Heidelberg, where she finished her habilitation thesis in 2015. She studied Mathematics at the University of Bremen, and prepared her PhD thesis at Roche Diagnostics GmbH in Penzberg.

Prof. Dr. Meinhard Kieser studied Mathematics at the University of Heidelberg and received his PhD in Medical Biometry in 1992. After that, he worked for more than 15 years as a biostatistician and Head of Biometrics in the pharmaceutical industry. In 2001, he completed his habilitation thesis. Since 2008, he has been a professor of Medical Biometry and Director of the Institute of Medical Biometry and Informatics at the University of Heidelberg.

Svenja Schüler studied Mathematics at the University of Kiel and graduated in 2013. Since then, she has been working as a research fellow at the Institute of Medical Biometry and Informatics at the University of Heidelberg. In her doctorial thesis, funded by the German Research Foundation, she is developing statistical methods for the analysis of composite endpoints.

Bibliographic information

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