Fundamentals of Clinical Trials

by Lawrence M. Friedman, Curt D. Furberg, David L. DeMets

Overview

The clinical trial is “the most definitive tool for evaluation of the applicability of clinical research.” It represents “a key research activity with the potential to improve the quality of health care and control costs through careful comparison of alternative treatments” [1]. It has been called on many occasions, “the gold st- dard” against which all other clinical research is measured. Although many clinical trials are of high quality, a careful reader of the medical literature will notice that a large number have deficiencies in design, conduct, analysis, presentation, and/or interpretation of results. Improvements have occurred over the past few decades, but too many trials are still conducted without adequate attention to its fundamental principles. Certainly, numerous studies could have been upgraded if the authors had had a better understanding of the fundamentals. Since the publication of the first edition of this book, a large number of other texts on clinical trials have appeared, most of which are indicated here [2–21]. Several of them, however, discuss only specific issues involved in clinical trials. Additionally, many are no longer current. The purpose of this fourth edition is to update areas in which major progress has been made since the publication of the third edition. We have revised most chapters considerably and added one on ethical issues.

Product Details

ISBN-13:	9781441915863
Publisher:	Springer-Verlag New York, LLC
Publication date:	09/09/2010
Sold by:	Barnes & Noble
Format:	eBook
Pages:	445
File size:	2 MB

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.

Table of Contents

to Clinical Trials.- Ethical Issues.- What Is the Question?.- Study Population.- Basic Study Design.- The Randomization Process.- Blindness.- Sample Size.- Baseline Assessment.- Recruitment of Study Participants.- Data Collection and Quality Control.- Assessing and Reporting Adverse Events.- Assessment of Health-Related Quality of Life.- Participant Adherence.- Survival Analysis.- Monitoring Response Variables.- Issues in Data Analysis.- Closeout.- Reporting and Interpreting of Results.- Multicenter Trials.

Editorial Reviews

3 Stars from Doody

This book presents fundamental concepts and activitiesassociated with the design and implementation of clinical researchtrials. In biomedical research, clinical trials have emerged as one ofthe most important and commonly used research designs. This bookseeks to serve both as a guide and resource for investigators involvedin clinical trials both in academic and industrial settings. It hitsthe mark on both accounts. Although the primary audience is clearlymany different categories of health professionals involved in activeresearch, this book's topics and straightforward style will also appealto students and clinicians as well. The basic concepts andapplications are presented in a manner that clearly explains therationale and key steps of the research process. As a result, thisbook facilitates understanding and appreciation of clinical trialsamong noninvestigators even while it guides and assists the efforts ofexperienced researchers. The authors all have extensive experience inthe subject matter; this volume is their third edition and has beenupdated substantially. This is a very solid addition to theliterature and a title that is likely to be read and appreciated bymany different categories of health professionals. This will be veryuseful to most health science libraries.

Bernard J. Turnock

Reviewer: Bernard J. Turnock, MD, MPH (University of Illinois at Chicago)
Description: This book presents fundamental concepts and activities associated with the design and implementation of clinical research trials. In biomedical research, clinical trials have emerged as one of the most important and commonly used research designs.
Purpose: This book seeks to serve both as a guide and resource for investigators involved in clinical trials both in academic and industrial settings. It hits the mark on both accounts.
Audience: Although the primary audience is clearly many different categories of health professionals involved in active research, this book's topics and straightforward style will also appeal to students and clinicians as well.
Features: The basic concepts and applications are presented in a manner that clearly explains the rationale and key steps of the research process. As a result, this book facilitates understanding and appreciation of clinical trials among noninvestigators even while it guides and assists the efforts of experienced researchers. The authors all have extensive experience in the subject matter; this volume is their third edition and has been updated substantially.
Assessment: This is a very solid addition to the literature and a title that is likely to be read and appreciated by many different categories of health professionals. This will be very useful to most health science libraries.

Doody's Review Service

This book aims to assist investigators in improving the quality of their clinical trials and prools 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)

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