Table of Contents

Introduction to Clinical Trials.- Ethical Issues.- What is the Question?.- Study Population.- Basic Study Design.- The Randomization Process.- Blinding.- Sample Size.- Baseline Assessment.- Recruitment of Study Participants.- Data Collection and Quality Control.- Assessment and Reporting of Harm.- Assessment of Health Related Quality of Life.- Participant Adherence.- Survival Analysis.- Monitoring Committee Structure & Function.- Statistical Methods Used in Interim Monitoring.- Issues in Data Analysis.- Closeout.- Reporting and Interpreting of Results.- Multicenter Trials.- Regulatory Issues.

About the Author

Lawrence M. Friedman received his M.D. from the University of Pittsburgh. After training in internal medicine, he went to the National Heart, Lung and Blood Institute of the National Institutes of Health. During his many years there, Dr. Friedman was involved in numerous clinical trials and epidemiology studies, having major roles in their design, management and monitoring. While at the NIH and subsequently, he served as a consultant on clinical trials to various NIH institutes and to other governmental and nongovernmental organizations. Dr. Friedman has been a member of many data monitoring and other safety committees.

Curt D. Furberg is Professor Emeritus of the Division of Public Health Sciences of the Wake Forest University School of Medicine. He received his M.D. and Ph.D. at the University of Umea, Sweden, and is a former chief, Clinical Trials Branch and Associate Director, Clinical Applications and Prevention Program, National Heart, Lung, and Blood Institute. Dr. Furberg established the Department of Public Health Sciences and served as its chair from 1986 to 1999. He has played major scientific and administrative roles in numerous multicenter clinical trials and has served in a consultative or advisory capacity on others. Dr. Furberg’s research activities include the areas of clinical trials methodology and cardiovascular epidemiology. 

David L. DeMets, PhD is currently the Max Halperin Professor of Biostatistics and former Chair of the Department of Biostatistics and Medical Informatics at the University of Wisconsin – Madison He has co-authored numerous papers on statistical methods and four texts on clinical trials, two specifically on data monitoring. He has served on many NIH and industry-sponsored data monitoring committees for clinical trials in diverse disciplines. He served on the Board of Directors of the American Statistical Association, as well as having been President of the Society for Clinical Trialsand President of the Eastern North American Region (ENAR) of the Biometric Society. In addition he was Elected Fellow of the International Statistics Institute, the American Statistical Association, the Association for the Advancement of Science, the Society for Clinical Trials and the American Medical Informatics Association. In 2013, he was elected as a member of the Institute of Medicine.

Christopher B. Granger is Professor of Medicine at Duke University, where he is an active clinical cardiologist and a clinical trialist at the Duke Clinical Research Institute. He received his M.D. at University of Connecticut and his residency training at the University of Colorado. He has had Steering Committee, academic leadership, and operational responsibilities for many clinical trials in cardiology. He has been on numerous Data Monitoring Committees. He serves on the National Heart, Lung, and Blood Institute Board of External Experts. He works with the Clinical Trials Transformation Initiative, a partnership between the U.S. Food and Drug Administration and Duke aiming to increase the quality and efficiency of clinical trials. He is a founding member of the Sensible Guidelines for the Conduct of Clinical Trials group, a collaboration between McMaster, Oxford, and Duke Universities.

David M. Reboussin is a Professor in the Department of Biostatistical Science at the Wake Forest University School of Medicine, where he has worked since 1992. He has a master’s degree in Statistics from the University of Chicago and received his doctorate in Statistics from the University of Wisconsin at Madison. He is currently Principle Investigator for the Systolic Blood Pressure Intervention Trial Coordinating Center and has been a co-investigator in the coordinating centers for several NIH and industry funded clinical trials including Action to Control Cardiovascular Risk in Diabetes (ACCORD), Action for Health in Diabetes (Look AHEAD), the Combined Oral and Nutritional Treatment of Late-Onset Diabetes Trial (CONTROL DM) and the Estrogen Replacement and Atherosclerosis (ERA) Trial. Dr. Reboussin has served on the Data and Safety Monitoring Boards for many National Institutes of Health trials within areas including cardiology, diabetes, nephrology, pulmonology, liver disease, psychiatry, pediatrics, weight loss and smoking cessation. His work in statistical methodology has included techniques and software for sequential monitoring of clinical trials.

Reviews

“This book aims to assist investigators in improving the quality of their clinical trials and protocols by discussing fundamental concepts with examples and in-depth review of the literature. … This is a valuable resource for students, clinicians, and researchers who are interested in designing a clinical trial or in critically appraising the published literature on clinical trials.” (Pooja Sethi, Doody’s Book Reviews, December, 2015)