Designs for Clinical Trials: Perspectives on Current Issues (Applied Bioinformatics and Biostatistics in Cancer Research)

This book will examine current issues and controversies in the design of clinical trials, including topics in adaptive and sequential designs, the design of correlative genomic studies, the design of studies in which missing data is anticipated. Each chapter will be written by an expert conducting research in the topic of that chapter. As a collection, the chapters would be intended to serve as a guidance for statisticians designing trials. Statistical methods for clinical trials have been an area of active research in Biostatistics since the first modern clinical trials were mounted in 1946 by the British Medical Research Council in whooping cough and tuberculosis. Often, the participants in clinical trials suffer from potentially fatal chronic diseases, and it is especially important that these experiments in medical research use designs that are efficient, can be understood by physicians, policy makers and patients, respond quickly new ideas in medicine and statistics, and, perhaps above all, show respect for the complex and important ethical issues that arise in these settings. This book explores some recent thinking in designs for clinical trials, including alternative designs for phase I studies, interim monitoring for futility, adaptive designs based on accumulating outcome data, and designs of new, targeted therapies. The book is intended for both the statistical practitioner, who may be too busy to stay abreast of the literature on statistical methods, as well as statisticians conducting research in clinical trials Front Matter....Pages i-xiv Designs for Phase I Trials....Pages 1-27 Randomized and Balancing Allocation Schemes for Clinical Trials: Computational Perspectives on Design and Deployment....Pages 29-55 Sequential Designs for Clinical Trials....Pages 57-79 Sample Size Reestimation for Confirmatory Clinical Trials....Pages 81-108 On Stopping a Randomized Clinical Trial for Futility....Pages 109-137 Molecular Gene-Signatures and Cancer Clinical Trials....Pages 139-155 Targeted Clinical Trials....Pages 157-177 Design Issues for Quality of Life Studies Subject to Dropout....Pages 179-198 Back Matter....Pages 199-204 Designs for Clinical Trials 2 Preface 8 Contents 12 Contributors 14 1 Designs for Phase I Trials 16 2 Randomized and Balancing Allocation Schemes for Clinical Trials: Computational Perspectives on Design and Deployment 43 3 Sequential Designs for Clinical Trials 70 4 Sample Size Reestimation for Confirmatory Clinical Trials 93 5 On Stopping a Randomized Clinical Trial for Futility 121 6 Molecular Gene-Signatures and Cancer Clinical Trials 150 7 Targeted Clinical Trials 167 8 Design Issues for Quality of Life Studies Subject to Dropout 188 Index 208