Understanding Clinical Research

262 Pages·8.59 MB·english

by  Lopes R.D.

Understanding
Clinical
Research
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Understanding
Clinical
Research
Editors
Renato D. Lopes, MD, MHS, PhD
Executive Director, Brazilian Clinical Research Institute
Federal University of São Paulo, São Paulo, Brazil
Associate Professor of Medicine, Division of Cardiology
Department of Medicine, Duke University Medical Center
Associate Program Director, Fellowship Program
Duke Clinical Research Institute
Durham, North Carolina
Robert A. Harrington, MD
Chair, Department of Medicine
Stanford University
Stanford, California
New York Chicago San Francisco Lisbon London Madrid Mexico City
Milan New Delhi San Juan Seoul Singapore Sydney Toronto
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ISBN: 978-0-07-179265-3
MHID: 0-07-179265-1
The material in this eBook also appears in the print version of this title: ISBN: 978-0-07-174678-6,
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their portion of the sales proceeds to the Duke Clinical Research Institute’s Fellowship Program, to
enhance the education and support of future generations of clinical researchers
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Contents
Contributors . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . ix
Foreword . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . xiii
Preface . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .xv
SECTION I: EVOLUTION OF CLINICAL RESEARCH
Renato D . Lopes, editor
CHAPTER ONE
A Brief History of Clinical Trials, Drug Regulations,
and the Food and Drug Administration . . . . . . . . . . . . . . .3
Nidhi Tripathi, Kate Davis, Catherine Bodine, and Daniel K . Benjamin Jr .
CHAPTER TWO
Information Technology, Access,
ClinicalTrials.gov . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 17
Anjan K . Chakrabarti and C . Michael Gibson
CHAPTER THREE
The Role of Academic Research Organizations
in Clinical Research . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 27
Craig J . Reist, Tyrus L . Rorick, Lisa G . Berdan, and Renato D . Lopes
CHAPTER FOUR
Ethics of Clinical Research: An Overview
and Emerging Issues . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 39
Yee Weng Wong and Kevin A . Schulman
v
vi Contents
SECTION II: PRINCIPLES OF CLINICAL
EXPERIMENTATION
Daniel K . Benjamin Jr . and Karen S . Pieper, editors
CHAPTER FIVE
Introduction to Clinical Experimentation . . . . . . . . . . 65
Robert Bigelow
CHAPTER SIX
Phase I Trials: First in Human . . . . . . . . . . . . . . . . . . . . . . . . 83
Kevin M . Watt, Karen Chiswell, and Michael Cohen-Wolkowiez
CHAPTER SEVEN
Phase II Clinical Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .101
Jeffrey T . Guptill and Karen Chiswell
CHAPTER EIGHT
Phase III and IV Clinical Trials . . . . . . . . . . . . . . . . . . . . . . .113
Gail E . Hafley, Sergio Leonardi, and Karen S . Pieper
CHAPTER NINE
Challenges of Clinical Trials in Pediatrics . . . . . . . . .127
Kevin N . Turner and P . Brian Smith
SECTION III: OBSERVATIONAL RESEARCH
Lesley H . Curtis and Laine E . Thomas, editors
CHAPTER TEN
Observational Research . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .143
Michaela A . Dinan
CHAPTER ELEVEN
Data Resources . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .157
Ying Xian
CHAPTER TWELVE
Observational Study Designs . . . . . . . . . . . . . . . . . . . . . . . . .169
Bradley G . Hammill
Contents vii
CHAPTER THIRTEEN
Challenges of Observational Designs . . . . . . . . . . . . . . .179
David M . Vock
CHAPTER FOURTEEN
Specific Regression Techniques . . . . . . . . . . . . . . . . . . . . . .189
Phillip J . Schulte and Laine E . Thomas
CHAPTER FIFTEEN
Analytical Methods of Addressing
Confounding . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .209
Eric M . Reyes and Laine E . Thomas
CHAPTER SIXTEEN
Lessons from Notable Examples in
Observational Research . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .227
Zubin J . Eapen
Index . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .237
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Contributors
Daniel K. Benjamin Jr., MD, PhD Anjan K. Chakrabarti, MD
Professor of Pediatrics Clinical Fellow in Medicine
Duke University School of Medicine Department of Medicine
Faculty Associate Director Division of Cardiology
Duke Clinical Research Institute Beth Israel Deaconess Medical Center
Durham, North Carolina Boston, Massachusetts
Chapter 1 Chapter 2
Lisa G. Berdan, MHS, PA-C Karen Chiswell, PhD
Associate Director, Cardiovascular Senior Biostatistician
Megatrials Duke Clinical Research Institute
Duke Clinical Research Institute Durham, North Carolina
Durham, North Carolina Chapters 6 and 7
Chapter 3
Michael Cohen-Wolkowiez, MD,
Robert Bigelow, PhD PhD
Associate Director Assistant Professor
Duke Clinical Research Institute Department of Pediatrics
Durham, North Carolina Division of Infectious Disease
Chapter 5 Duke Clinical Research Institute
Durham, North Carolina
Catherine Bodine, RN Chapter 6
Clinical Research Communications
Specialist III Lesley H. Curtis, PhD
Duke Clinical Research Institute Associate Professor in Medicine
Durham, North Carolina Division of General Medicine
Chapter 1 Department of Medicine
Duke University Medical Center
Durham, North Carolina
ix