Textbook of Clinical Trials in Oncology : A Statistical Perspective book cover
1st Edition

Textbook of Clinical Trials in Oncology
A Statistical Perspective

ISBN 9781138083776
Published May 16, 2019 by Chapman and Hall/CRC
626 Pages - 103 B/W Illustrations

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Book Description

There is an increasing need for educational resources for statisticians and investigators. Reflecting this, the goal of this book is to provide readers with a sound foundation in the statistical design, conduct, and analysis of clinical trials. Furthermore, it is intended as a guide for statisticians and investigators with minimal clinical trial experience who are interested in pursuing a career in this area. The advancement in genetic and molecular technologies have revolutionized drug development. In recent years, clinical trials have become increasingly sophisticated as they incorporate genomic studies, and efficient designs (such as basket and umbrella trials) have permeated the field. This book offers the requisite background and expert guidance for the innovative statistical design and analysis of clinical trials in oncology.

Key Features:

  • Cutting-edge topics with appropriate technical background
  • Built around case studies which give the work a "hands-on" approach
  • Real examples of flaws in previously reported clinical trials and how to avoid them
  • Access to statistical code on the book’s website
  • Chapters written by internationally recognized statisticians from academia and pharmaceutical companies
  • Carefully edited to ensure consistency in style, level, and approach
  • Topics covered include innovating phase I and II designs, trials in immune-oncology and rare diseases, among many others

Table of Contents

Section I Introduction

Introduction to Clinical Trials - Susan Halabi, Stefan Michiels

Section II General Issues

Selection of Endpoints - Katherine S Panageas and Andrea Knezevic

Section III Early Development

Innovative Phase I Trials - Cody Chiuzan and Nathaniel O’Connell

Section IV Middle Development

Current Issues in Phase II Cancer Clinical Trials - Sin-Ho Jung

Design and Analysis of Immunotherapy Clinical Trials - Megan Othus

Adaptive Designs - William T. Barry

Section V Late Phase Clinical Trials

Sample Size Calculations for Phase III Trials in Oncology - Koji Oba, Aye Kuchiba

Non-inferiority Trial - Keyue Ding, Chris O’Callaghan

Design of Multi-arm, Multi-stage Trials in Oncology - James Wason

Multiple Comparisons, Multiple Primary Endpoints and Subpopulation Analysis - Ekkehard Glimm, Dong Xi, Paul Gallo

Cluster Randomized Trials - Catherine M. Crespi

Statistical Monitoring of Safety and Efficacy - Jay Herson, Chen Hu

Section VI Personalized Medicine

Biomarker-Based Phase II and III Clinical Trials in Oncology - Shigeyuki Matsui, Masataka Igeta, Kiichiro Toyoizumi

Genomic Biomarker Clinical Trial Designs - Richard Simon

Trial designs for rare diseases and small samples in oncology - Robert A. Beckman, Cong Chen, Martin Posch and Sarah Zohar

Statistical Methods for Biomarker and Subgroup Evaluation in Oncology Trials - Ilya Lipkovich, Alex Dmitrienko, Bohdana Ratitch

Developing and Validating Prognostic Models of Clinical Outcomes - Susan Halabi, Lira Pi, and Chen-Yen Lin

High-Dimensional Penalized Regression Models in Time-to-Event Clinical Trials - Federico Rotolo, Nils Ternes, Stefan Michiels

Sequential Multiple Assignment Randomized Trials - Kelly Speth, Kelley M. Kidwell

Section VII Advanced Topics

Assessing the value of surrogate endpoints - Xavier Paoletti, Federico Rotolo, Stefan Michiels

Competing Risks - Aurelien Latouche, Gang Li, Qing Yang

Cure models in cancer clinical trials - Catherine Legrand, Aurelie Bertrand

Interval Censoring - Yuan Wu

Methods for analysis of trials with changes from randomised treatment - Nicholas R. Latimer and Ian R. White

The analysis of adverse events in randomized clinical trials - Jan Beyersmann, Claudia Schmoor

Analysis of quality of life outcomes in oncology trials - Stephen Walters

Missing Data - Stephanie Pugh,  James J. Dignam, Juned Siddique

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Susan Halabi, Ph.D. is Professor of Biostatistics and Bioinformatics, Duke University, USA. She has extensive experience in the design and analysis of clinical trials in oncology. Dr. Halabi is a fellow of the American Statistical Association, the Society of Clinical Trials, and the American Society of Clinical Oncology. She serves on the Oncologic Drugs Advisory Committee for the Food and Drug Administration.

Stefan Michiels, Ph.D. is Head of the Oncostat team of the Center for research in epidemiology and population health (INSERM U1018, University Paris-Saclay, University Paris-Sud) at Gustave Roussy, Villejuif, France. His areas of expertise are clinical trials, meta-analyses and prediction models in oncology. Stefan is the currently the chair of the biostatisticians at Unicancer, a French collaborative cancer clinical trials group. Stefan holds a PhD in Biostatistics from the School of Public Health at the University Paris-Sud and Master Degrees in Statistics and in Applied Mathematics from the University of Leuven. His previous positions include the Université Libre de Bruxelles- Institut Jules Bordet (Belgium), the National Cancer Institute (France) and the University of Leuven (Belgium). He is currently member of the editorial board of the Journal of the National Cancer Institute and Annals of Oncology.

Featured Author Profiles

Author - Stefan  Michiels

Stefan Michiels

Head Department Biostatistics & Epidemiology, Gustave Roussy - Oncostat U1018, Gustave Roussy, Gustave Roussy, CESP U1018, Inserm, University Paris-Sud

Learn more about Stefan Michiels »


"This highly anticipated book focuses on clinical trials in oncology, ranging from early, middle, and late phase trials to advanced topics such as precision medicine and immunotherapy. This textbook is expected to be extremely useful for statisticians and investigators who have been doing clinical trials for years, and for future clinical researchers and statisticians who are eager to learn about the design, conduct, analysis, and interpretation of clinical trials in oncology.

We strongly recommend this textbook for four reasons. First, it covers multiple stages of clinical trials in oncology, from early, middle, to late development. Second, it examines various designs of clinical trials, including traditional study designs, flexible designs, and SMART (Sequential Multiple Assignment Randomized Trials) designs. Third, it gives insights into unique aspects of clinical trials in oncology compared with other therapeutic areas, such as time-to-event endpoints and censoring. Fourth, it consists of different types of materials that are suitable to different groups of readers, with some materials for readers who like to have an aerial view of the practical considerations and the other materials for readers who like to have deep understanding to motivate their theoretical research. In the following, we explain these four reasons in detail...To summarize, because of the above four reasons, we strongly recommend this book to clinical researchers and statisticians who are interested in the development, design, conduct and analysis of oncology clinical trials. This book is well-balanced between practical considerations and statistical theories involved in oncology clinical trials. We believe that this book will help advance the design and analysis of oncology clinical trials with the ultimate goal to improve the care of oncology patients and their quality of life."
- Man Jin and Yixin Fang, Journal of Biopharmaceutical Statistics, November 2019

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