Pharmacoepidemiology, Fifth Edition

965 Pages·2012·6.94 MB·English

Pharmacoepidemiology
Pharmacoepidemiology
EDITED BY
Brian L. Strom MD, MPH
George S. Pepper Professor of Public Health and Preventive Medicine
Professor of Biostatistics and Epidemiology, of Medicine, and of Pharmacology
Chair, Department of Biostatistics and Epidemiology
Director, Center for Clinical Epidemiology and Biostatistics
Vice Dean for Institutional Affairs
Perelman School of Medicine at the University of Pennsylvania
Philadelphia, PA, USA
Stephen E. Kimmel MD, MSCE
Professor of Medicine and of Epidemiology
Senior Scholar, Center for Clinical Epidemiology and Biostatistics
Perelman School of Medicine at the University of Pennsylvania
Philadelphia, PA, USA
Sean Hennessy PHARMD, PHD
Associate Professor of Epidemiology and of Pharmacology
Senior Scholar, Center for Clinical Epidemiology and Biostatistics
Perelman School of Medicine at the University of Pennsylvania
Philadelphia, PA, USA
FIFTH EDITION
A John Wiley & Sons, Ltd., Publication
This edition fi rst published 2012, © 2012 by John Wiley & Sons, Ltd.
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Library of Congress Cataloging-in-Publication Data
Pharmacoepidemiology / edited by Brian L. Strom, Stephen E. Kimmel, Sean Hennessy. – 5th ed.
p. ; cm.
Includes bibliographical references and index.
ISBN-13: 978-0-470-65475-0 (hard cover : alk. paper)
ISBN-10: 0-470-65475-9 (hard cover : alk. paper)
1. Pharmacoepidemiology. 2. Pharmacology. I. Strom, Brian L. II. Kimmel, Stephen E. III. Hennessy,
Sean.
[DNLM: 1. Pharmacoepidemiology–methods. QZ 42]
RM302.5.P53 2012
615'.7042–dc23
2011019285
A catalogue record for this book is available from the British Library.
Wiley also publishes its books in a variety of electronic formats. Some content that appears in print may not
be available in electronic books.
Set in 9/12pt Meridien by Toppan Best-set Premedia Limited, Hong Kong
1 2012
Contents
List of Contributors, ix
Preface, xvi
Acknowledgements, xx
PART I Introduction, 1
1 What is Pharmacoepidemiology?, 3
Brian L. Strom
2 Basic Principles of Clinical Pharmacology Relevant to
Pharmacoepidemiologic Studies, 23
Jeffrey S. Barrett and Athena F. Zuppa
3 Basic Principles of Clinical Epidemiology Relevant to
Pharmacoepidemiologic Studies, 38
Brian L. Strom
4 Sample Size Considerations for Pharmacoepidemiologic Studies, 52
Brian L. Strom
5 When Should One Perform Pharmacoepidemiologic Studies?, 62
Brian L. Strom
PART II The Role of Pharmacoepidemiology in
Different Sectors, 71
6 The Role of Pharmacoepidemiology in the Health-Care System and Academia, 73
Jerry Avorn
7 The Role of Pharmacoepidemiology in Industry, 84
Jingping Mo, Nicolle M. Gatto, Rachel E. Sobel, and Robert F. Reynolds
8 The Role of Pharmacoepidemiology in Regulatory Agencies, 107
Gerald J. Dal Pan and Peter Arlett
9 Pharmacoepidemiology and the Law, 117
Aaron S. Kesselheim
PART III Sources of Data for
Pharmacoepidemiologic Studies, 135
Section A: Spontaneous Reporting
10 Postmarketing Spontaneous Pharmacovigilance Reporting Systems, 137
Gerald J. Dal Pan, Marie Lindquist, and Kate Gelperin
v
vi Contents
Section B: Automated Data Systems
11 Overview of Automated Databases in Pharmacoepidemiology, 158
Brian L. Strom
12 Health Maintenance Organizations/Health Plans, 163
Susan E. Andrade, Marsha A. Raebel, Denise Boudreau, Robert L. Davis,
Katherine Haffenreffer, Pamala A. Pawloski, Sengwee Toh, and Richard Platt
13 Commercial Insurance Databases, 189
John Seeger and Gregory W. Daniel
14 US Government Claims Databases, 209
Sean Hennessy, Cristin Palumbo Freeman, and Francesca Cunningham
15 Medical Record Databases, 224
Alexis Ogdie, Sinéad M. Langan, John Parkinson, Hassy Dattani, Karel Kostev, and
Joel M. Gelfand
16 In-hospital Databases, 244
Brian T. Fisher, Peter K. Lindenauer, and Chris Feudtner
17 Canadian Provincial Databases, 259
Yola Moride and Colleen J. Metge
18 Pharmacy-based Medical Record Linkage Systems, 270
Ron M.C. Herings and Lars Pedersen
Section C: Ad Hoc Studies—Ongoing Systems for
Pharmacoepidemiologic Studies
19 Case–Control Surveillance, 287
Lynn Rosenberg, Patricia F. Coogan, and Julie R. Palmer
20 Prescription–Event Monitoring, 301
Deborah Layton and Saad A.W. Shakir
21 Registries, 331
Nancy A. Dreyer and Priscilla Velentgas
Section D: Ad Hoc Studies—De Novo Studies
22 Field Studies, 347
David W. Kaufman
Section E: Choosing Among the Available Alternatives
23 How Should One Perform Pharmacoepidemiologic Studies?
Choosing Among the Available Alternatives, 364
Brian L. Strom
PART IV Selected Special Applications
of Pharmacoepidemiology, 377
24 Studies of Drug Utilization, 379
David Lee and Ulf Bergman
25 Evaluating and Improving Physician Prescribing, 402
Sumit R. Majumdar, Helene Levens Lipton, and Stephen B. Soumerai
Contents vii
26 Pharmacoepidemiologic Studies of Vaccine Safety, 423
Robert T. Chen, Jason M. Glanz, and Claudia Vellozzi
27 Epidemiologic Studies of Medical Devices: Methodologic Considerations for
Implantable Devices, 469
Danica Marinac-Dabic, Sharon-Lise Normand, Art Sedrakyan, and Thomas Gross
28 Studies of Drug-Induced Birth Defects, 487
Allen A. Mitchell
29 Risk Management, 505
Gerald J. Dal Pan, Stella Blackburn, and Claudia Karwoski
30 FDA’s Sentinel Initiative: Active Surveillance to Identify Safety Signals, 534
Judith A. Racoosin, Melissa A. Robb, Rachel E. Sherman, and Janet Woodcock
31 Pharmacoepidemiology and Pharmaceutical Reimbursement Policy, 555
Mitchell Levine and Jacques LeLorier
32 Comparative Effectiveness Research, 561
Brian L. Strom, Rita Schinnar, and Sean Hennessy
PART V Selected Special Methodologic Issues in
Pharmacoepidemiology, 581
33 Assessing Causality of Case Reports of Suspected Adverse Events, 583
Judith K. Jones
34 Molecular Pharmacoepidemiology, 601
Stephen E. Kimmel, Hubert G. Leufkens, and Timothy R. Rebbeck
35 Bioethical Issues in Pharmacoepidemiologic Research, 623
Antoine C. El Khoury, Jason Karlawish, Elizabeth Andrews, and Arthur Caplan
36 The Use of Randomized Controlled Trials for
Pharmacoepidemiologic Studies, 640
Samuel M. Lesko and Allen A. Mitchell
37 The Use of Pharmacoepidemiology to Study Benefi cial Drug Effects, 655
Brian L. Strom and Kenneth L. Melmon
38 Pharmacoeconomics: Economic Evaluation of Pharmaceuticals, 678
Kevin A. Schulman, Henry A. Glick, Daniel Polsky, and Shelby D. Reed
39 Using Quality-of-Life Measurements in
Pharmacoepidemiologic Research, 709
Holger Schünemann, Bradley C. Johnston, Roman Jaeschke,
and Gordon H. Guyatt
40 The Use of Meta-analysis in Pharmacoepidemiology, 723
Jesse A. Berlin, M. Soledad Cepeda, and Carin J. Kim
41 Validity of Pharmacoepidemiologic Drug and Diagnosis Data, 757
Suzanne L. West, Mary Elizabeth Ritchey, and Charles Poole
42 Studies of Medication Adherence, 795
Trisha Acri and Robert Gross
viii Contents
43 Risk Evaluation and Communication, 810
Susan J. Blalock and Betsy L. Sleath
44 Studying Effects of Antibiotics, 827
Darren R. Linkin and Ebbing Lautenbach
45 The Pharmacoepidemiology of Medication Errors, 840
Hanna M. Seidling and David W. Bates
46 Sequential Statistical Methods for Prospective
Postmarketing Safety Surveillance, 852
Martin Kulldorff
47 Advanced Approaches to Controlling Confounding
in Pharmacoepidemiologic Studies, 868
Sebastian Schneeweiss and Samy Suissa
PART VI Conclusion, 893
48 The Future of Pharmacoepidemiology, 895
Brian L. Strom, Stephen E. Kimmel, and Sean Hennessy
Appendix A: Sample Size Tables, 904
Appendix B: Glossary, 921
Index, 931
List of Contributors
Trisha Acri Jeffrey S. Barrett Jesse A. Berlin
Assistant Professor Director Vice President
Department of Family and Laboratory for Applied Department of Epidemiology
Community Medicine Pharmacokinetics and Johnson& Johnson Pharmaceutical
Temple University School of Medicine Pharmacodynamics Research and Development
Philadelphia, PA Director Titusville, NJ
USA Pediatric Pharmacology Research Unit USA
The Children’ s Hospital of
Susan E. Andrade Philadelphia Stella Blackburn
Senior Research Associate and Research Professor, Pediatrics EMA Risk Management Development
Research Associate Professor Kinetic Modeling and Simulation and Scientifi c Lead
Meyers Primary Care Institute (KMAS) Core Director European Medicines Agency
and Perelman School of Medicine at the London, UK
University of Massachusetts Medical University of Pennsylvania
School Philadelphia, PA Susan J. Blalock
Worcester, MA USA Associate Professor
USA Division of Pharmaceutical Outcomes
David W. Bates and Policy
Elizabeth Andrews Chief Eshelman School of Pharmacy
Vice President General Medicine Division University of North Carolina at
Pharmacoepidemiology and Risk Brigham and Women’ s Hospital Chapel Hill
Management and Chapel Hill, NC
Research Triangle Institute Health Professor of Medicine USA
Solutions Harvard Medical School
Research Triangle Park, NC Boston, MA Denise Boudreau
USA USA Scientifi c Investigator
Group Health Research Institute
Peter Arlett Ulf Bergman Seattle, WA
Head Professor, Senior Medical Offi cer USA
Pharmacovigilance and Risk Division of Clinical Pharmacology
Management Department of Laboratory Medicine Arthur Caplan
European Medicines Agency Karolinska Institute Director
London, UK WHO Collaborating Centre for Drug Center for Bioethics
Utilization Research and Clinical Professor of Medical Ethics
Jerry Avorn Pharmacological Services Perelman School of Medicine at the
Chief and University of Pennsylvania
Division of Pharmacoepidemiology Centre for Pharmacoepidemiology Philadelphia, PA
and Pharmacoeconomics Karolinska University USA
Brigham and Women’ s Hospital Hospital-Huddinge
and Stockholm, Sweden
Professor of Medicine
Harvard Medical School
Boston, MA
USA
ix
x List of Contributors
M. Soledad Cepeda Hassy Dattani Nicolle M. Gatto
Director Research Director Senior Director
Department of Epidemiology Cegedim Strategic Data Medical Epidemiology
Johnson& Johnson Pharmaceutical Research Ltd Worldwide Safety Strategy
Research and Development London, UK Pfi zer Inc.
Titusville, NJ New York, NY
USA Robert L. Davis USA
Director of Research
Robert T. Chen Center for Health Joel M. Gelfand
HIV Vaccine and Special Studies Team Research—Southeast Medical Director, Clinical Studies Unit
Leader Kaiser Permanente Georgia Assistant Professor of Dermatology
Division of HIV/AIDS Prevention Atlanta, GA and Epidemiology
Centers for Disease Control and USA Senior Scholar, Center for Clinical
Prevention Epidemiology and Biostatistics
Atlanta, GA Nancy A. Dreyer Departments of Dermatology and
USA Chief of Scientifi c Affairs Biostatistics and Epidemiology
Outcome Sciences Inc. Perelman School of Medicine at the
Patricia F. Coogan Cambridge, MA University of Pennsylvania
Associate Professor of Epidemiology USA Philadelphia, PA
Slone Epidemiology Center USA
Boston University Antoine C. El Khoury
Boston, MA Leader, Outcomes Research Kate Gelperin
USA Global Health Outcomes Medical Offi cer
Merck & co., Inc Division of Epidemiology
Francesca Cunningham West Point, PA Offi ce of Surveillance and
Director USA Epidemiology
Center for Medication Safety Center for Drug Evaluation and
and Chris Feudtner Research
Program Manager Associate Professor US Food and Drug Administration
Outcomes Research PBM Services Department of Pediatrics Silver Spring, MD
Department of Veterans Affairs The Children ’s Hospital of Philadelphia USA
Center for Medication Safety Perelman School of Medicine at the
Hines, IL University of Pennsylvania Jason M. Glanz
USA Philadelphia, PA Epidemiologist
USA Institute for Health Research
Gerald J. Dal Pan Kaiser Permanente Colorado
Director Brian T. Fisher Department of Epidemiology Colorado
Offi ce of Surveillance and Assistant Professor of Pediatrics School of Public Health
Epidemiology Center for Pediatric Clinical Denver, CO
Center for Drug Evaluation and Effectiveness USA
Research The Children’ s Hospital of
US Food and Drug Administration Philadelphia Henry A. Glick
Silver Spring, MA Perelman School of Medicine at the Professor of Medicine
USA University of Pennsylvania Perelman School of Medicine at the
Philadelphia, PA University of Pennsylvania
Gregory W. Daniel USA Philadelphia, PA
Vice President USA
Government and Academic Research Cristin Palumbo Freeman
Healthcore Research Project Manager
Alexandria, VA Center for Clinical Epidemiology and
USA Biostatistics
Perelman School of Medicine at the
University of Pennsylvania
Philadelphia, PA
USA
List of Contributors xi
Robert Gross Ron M.C. Herings Claudia Karwoski
Associate Professor of Medicine and Director Director
Epidemiology PHARMO Institute Division of Risk Management
Center for Clinical Epidemiology and Utrecht Offi ce of Surveillance and
Biostatistics The Netherlands Epidemiology
Perelman School of Medicine at the and Center for Drug Evaluation and
University of Pennsylvania Associate Professor of Research
Philadelphia, PA Pharmacoepidemiology US Food and Drug Administration
USA Department of Health Policy and Silver Spring, MA
Management USA
Thomas Gross Erasmus University Rotterdam
Deputy Director Rotterdam, The Netherlands David W. Kaufman
Offi ce of Surveillance and Biometrics Associate Director
Center for Devices and Radiological Roman Jaeschke Slone Epidemiology Center at Boston
Health Professor University
US Food and Drug Administration Department of Clinical Epidemiology and
Silver Spring, MD and Biostatistics Professor of Epidemiology
USA McMaster University Boston University School of Public
Health Sciences Center Health
Gordon H. Guyatt and Boston, MA
Professor Professor USA
Department of Clinical Epidemiology Department of Medicine
and Biostatistics St Joseph ’s Hospital Aaron S. Kesselheim
McMaster University Hamilton Assistant Professor of Medicine
Health Sciences Center Ontario, Canada Division of Pharmacoepidemiology
and and Pharmacoeconomics
Department of Medicine Bradley C. Johnston Department of Medicine
St Joseph ’s Hospital Assistant Professor Brigham and Women’ s Hospital
Hamilton Department of Clinical Epidemiology Harvard Medical School
Ontario, Canada and Biostatistics Boston, MA
McMaster University USA
Katherine Haffenreffer Health Sciences Center
Project Administrator Hamilton Carin J. Kim
Harvard Pilgrim Health Care Institute Ontario, Canada Mathematical Statistician
and Center for Drug Evaluation and
Department of Population Medicine Judith K. Jones Research
Harvard Medical School President and CEO US Food and Drug Administration
Boston, MA The Degge Group Ltd Silver Spring, MD
USA Arlington, VA USA
USA
Sean Hennessy and Stephen E. Kimmel
Associate Professor of Epidemiology Adjunct Professor and Lecturer Professor of Medicine and
and of Pharmacology University of Michigan School of Epidemiology
Senior Scholar, Center for Clinical Public Health Summer Program Senior Scholar, Center for Clinical
Epidemiology and Biostatistics Georgetown University Epidemiology and Biostatistics
Perelman School of Medicine at the Washington, DC Perelman School of Medicine at the
University of Pennsylvania USA University of Pennsylvania
Philadelphia, PA Philadelphia, PA
USA Jason Karlawish USA
Professor of Medicine
Perelman School of Medicine at the Karel Kostev
University of Pennsylvania Senior Research Analyst
Philadelphia, PA Centre of Excellence Patient Data
USA IMS Health GmbH& Co OHG
Frankfurt/Main, Germany