Randomised Clinical Trials. David Machin
537
Randomised Clinical Trials
Design, Practice and Reporting
Second Edition
David Machin
Leicester Cancer Research Centre
University of Leicester, Leicester, UK
Medical Statistics Group, School of Health and Related Research
University of Sheffield, Sheffield, UK
Peter M. Fayers
Institute of Applied Health
University of Aberdeen, Scotland, UK
Bee Choo Tai
Saw Swee Hock School of Public Health
National University of Singapore and National University Health System Singapore
Singapore, Singapore
This edition first published 2021, © 2021 John Wiley & Sons Ltd
Edition History John Wiley & Sons, Ltd (1e, 2010)
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Library of Congress Cataloging‐in‐Publication Data
Names: Machin, David, 1939- author. | Fayers, Peter M., author. | Tai, Bee Choo, author.
Title: Randomized clinical trials : design, practice and reporting / David Machin, Peter M. Fayers, Bee Choo Tai.
Description: Second edition. | Hoboken, NJ : Wiley-Blackwell, 2021. | Includes bibliographical references and index.
Identifiers: LCCN 2020044539 (print) | LCCN 2020044540 (ebook) | ISBN 9781119524649 (paperback) | ISBN 9781119524656 (adobe pdf) | ISBN 9781119524670 (epub)
Subjects: MESH: Randomized Controlled Trials as Topic–methods | Biomedical Research–methods | Research Design | Data Interpretation, Statistical
Classification: LCC R853.C55 (print) | LCC R853.C55 (ebook) | NLM W 20.55.C5 | DDC 610.72/4–dc23
LC record available at https://lccn.loc.gov/2020044539 LC ebook record available at https://lccn.loc.gov/2020044540
Cover Design: Wiley
Cover Image: © Kenishirotie/Shutterstock
To
Lorna Christine Machin
Tessa and Emma Fayers
Isaac Xu‐En and Kheng‐Chuan Koh
Preface
It is now more than 10 years since the first edition of this book was published. In the intervening years, while many things have remained unchanged, there have also been many new developments over the period. This second edition refreshes the first, refining some of the sections to better explain their contents and, at the same time, replacing some examples with more current illustrations. To reflect the changes, we have created new chapters by splitting and then expanding old chapters. Thus, we now include full chapters on data and safety monitoring including interim analyses of accumulating data, cluster designs, repeated measures, and noninferiority designs as there has been a rapid increase in the use of such trial designs along with some methodological developments and improvements in statistical software available for analysis. In addition, we have included entirely new chapters on stepped wedge designs, genomic targets and, feasibility and pilot studies. The chapter on stepped wedge designs reflects the growing importance of such complex intervention designs while that on genomic targets highlights the research focus directed towards more individualised medicine. In contrast, the new chapter concerned with feasibility and pilot studies brings us back to the early planning stages of the clinical trial which is planned. The chapter is included as there is increasing recognition that perhaps