Stephen L. George - Cancer clinical trials: current and controversial issues in design and analysis

Cancer Clinical Trials

Current and Controversial
Issues in Design and Analysis

Chapman & Hall/CRC Biostatistics Series

Editor-in-Chief

Shein-Chung Chow, Ph.D., Professor, Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina

Series Editors

Byron Jones, Biometrical Fellow, Statistical Methodology, Integrated Information Sciences, Novartis Pharma AG, Basel, Switzerland

Jen-pei Liu, Professor, Division of Biometry, Department of Agronomy, National Taiwan University, Taipei, Taiwan

Karl E. Peace, Georgia Cancer Coalition, Distinguished Cancer Scholar, Senior Research Scientist and Professor of Biostatistics, Jiann-Ping Hsu College of Public Health, Georgia Southern University, Statesboro, Georgia

Bruce W. Turnbull, Professor, School of Operations Research and Industrial Engineering, Cornell University, Ithaca, New York

Published Titles

Adaptive Design Methods in Clinical Trials, Second Edition

Shein-Chung Chow and Mark Chang

Adaptive Designs for Sequential Treatment Allocation

Alessandro Baldi Antognini and Alessandra Giovagnoli

Adaptive Design Theory and Implementation Using SAS and R, Second Edition

Mark Chang

Advanced Bayesian Methods for Medical Test Accuracy

Lyle D. Broemeling

Advances in Clinical Trial Biostatistics

Nancy L. Geller

Applied Meta-Analysis with R

Ding-Geng (Din) Chen and Karl E. Peace

Basic Statistics and Pharmaceutical Statistical Applications, Second Edition

James E. De Muth

Bayesian Adaptive Methods for Clinical Trials

Scott M. Berry, Bradley P. Carlin, J. Jack Lee, and Peter Muller

Bayesian Analysis Made Simple: An Excel GUI for WinBUGS

Phil Woodward

Bayesian Methods for Measures of Agreement

Lyle D. Broemeling

Bayesian Methods for Repeated Measures

Lyle D. Broemeling

Bayesian Methods in Epidemiology

Lyle D. Broemeling

Bayesian Methods in Health Economics

Gianluca Baio

Bayesian Missing Data Problems: EM, Data Augmentation and Noniterative Computation

Ming T. Tan, Guo-Liang Tian, and Kai Wang Ng

Bayesian Modeling in Bioinformatics

Dipak K. Dey, Samiran Ghosh, and Bani K. Mallick

Benefit-Risk Assessment in Pharmaceutical Research and Development

Andreas Sashegyi, James Felli, and Rebecca Noel

Benefit-Risk Assessment Methods in Medical Product Development: Bridging Qualitative and Quantitative Assessments

Qi Jiang and Weili He

Biosimilars: Design and Analysis of Follow-on Biologics

Shein-Chung Chow

Chapman & Hall/CRC Biostatistics Series

Edited by

Stephen L. George

Duke University School of Medicine
Durham, North Carolina, USA

Xiaofei Wang

Duke University School of Medicine
Durham, North Carolina, USA

Herbert Pang

The University of Hong Kong
Hong Kong, China

CRC Press
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Version Date: 20160414

International Standard Book Number-13: 978-1-4987-0688-9 (Hardback)

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To Ed Gehan and Marvin Zelen, who early in my
career taught me much about the art and science of
cancer biostatistics and served as role models while
doing so.

Stephen L. George

To my beloved family and in memory of my father.

Xiaofei Wang

To my parents and mentors at Duke, the CALGB, and
the Alliance for their support and guidance in my
quest for knowledge in the field of cancer clinical trials.

Herbert Pang

Stephen L. George, Xiaofei Wang, and Herbert Pang

Simon Wandel, Heinz Schmidli, and Beat Neuenschwander

Dong Xi, Ekkehard Glimm, and Frank Bretz

Steven Snapinn and Qi Jiang

Michael C. Sachs and Lisa M. McShane

Mark R. Conaway and Nolan A. Wages

Sin-Ho Jung

Edward Lakatos

Rajeshwari Sridhara and Thomas Gwise

Diane Fairclough

Edward L. Korn and Boris Freidlin

J. Jack Lee and Lorenzo Trippa

Marie Davidian, Anastasios (Butch) Tsiatis, and Eric Laber

There are many important and controversial topics in the design and analysis of cancer clinical trials, including adaptive approaches, biomarker-based trials, and dynamic treatment regime trials. This book provides readers with a current understanding of the critical issues in these and other topics with state-of-the-art approaches. Each chapter is written by well-known statisticians from academic institutions, regulatory agencies (FDA), the National Cancer Institute, or the pharmaceutical industry, all with extensive experience in cancer clinical trials. Examples are taken from actual cancer clinical trials. The topics covered are: