Textbook of Pharmacoepidemiology

About the EditorsBrian L. Strom is Chancellor, Rutgers Biomedical and Health Sciences, University Professor, and Executive Vice President for Health Affairs, Rutgers University, Newark, NJ, USA. Stephen E. Kimmel is Dean’s Professor and Chair of Epidemiology, College of Public Health and Health Professions and College of Medicine, University of Florida, Gainesville, FL, USA. Sean Hennessy is Professor of Epidemiology and Director of the Center for Pharmacoepidemiology Research and Training, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.

• Contributors xviiPreface xxiAcknowledgements xxvPart I Introduction to Pharmacoepidemiology 11 What is Pharmacoepidemiology? 3Brian L. StromIntroduction 3Definition of Pharmacoepidemiology 3Historical Background 5The Current Drug Approval Process 13Potential Contributions of Pharmacoepidemiology 15Key Points 18Further Reading 182 Study Designs Available for Pharmacoepidemiologic Studies 20Brian L. StromIntroduction 20Overview of the Scientific Method 20Types of Errors that one Can Make in Performing a Study 22Criteria for the Causal Nature of an Association 23Epidemiologic Study Designs 26Discussion 31Conclusion 32Key Points 32Further Reading 333 Sample Size Considerations for Pharmacoepidemiologic Studies 35Brian L. StromIntroduction 35Sample Size Calculations for Cohort Studies 35Sample Size Calculations for Case–Control Studies 40Sample Size Calculations for Case Series 41Discussion 43Key Points 45Further Reading 454 Basic Principles of Clinical Pharmacology Relevant to Pharmacoepidemiologic Studies 47Jeffrey S. BarrettIntroduction 47Clinical Pharmacology and Pharmacoepidemiology 48Basics of Clinical Pharmacology 48Pharmacokinetics 49Special Populations 52Pharmacodynamics 56Pharmacogenomics 59Model-InformedDrug Development 59Conclusion 60Key Points 60Further Reading 615 When Should One Perform Pharmacoepidemiologic Studies? 62Brian L. StromIntroduction 62Reasons to Perform Pharmacoepidemiologic Studies 62Safety Versus Risk 67Risk Tolerance 67Conclusion 70Key Points 70Further Reading 716 Views from Academia, Industry, Regulatory Agencies, and the Legal System 73Joshua J. Gagne, Jerry Avorn, Nicolle M. Gatto, Jingping Mo, Gerald J. Dal Pan, June Raine, Shinobu Uzu, Aaron S. Kesselheim, and Kerstin N. VokingerThe View from Academia 73The View from Industry 81The View from Regulatory Agencies 90The View from the Legal System 98Further Reading 107Part II Sources of Pharmacoepidemiology Data 1137 Postmarketing Spontaneous Pharmacovigilance Reporting Systems 115Gerald J. Dal Pan, Marie Lindquist, and Kate GelperinIntroduction 115Description 116Strengths 128Limitations 129Particular Applications 131The Future 132Key Points 132Further Reading 1348 Overview of Electronic Databases in Pharmacoepidemiology 136Brian L. StromIntroduction 136Description 137Strengths 138Weaknesses 139Particular Applications 140The Future 140Key Points 141Further Reading 1419 Encounter Databases 142Tobias Gerhard, Yola Moride, Anton Pottegård, and Nicole PrattIntroduction 142Description 142Strengths 161Limitations 162Particular Applications 163The Future 166Key Points 167Further Reading 171US Databases 172European Databases 172Canadian Databases 173Asian Databases 17310 Electronic Health Record Databases 174Daniel B. Horton, Harshvinder Bhullar, Francesca Cunningham, Janet Sultana, and Gialuca TrifiròIntroduction 174Description 174Strengths 184Limitations 185The Future 186Summary Points for Electronic Health Record Databases 187Acknowledgment 187Further Readings 18911 Primary Data Collection for Pharmacoepidemiology 192Priscilla VelentgasIntroduction 192Methods of Primary Data Collection 195Strengths 197Limitations 197Particular Applications 198Conclusions 199Key Points 199Further Reading 20112 How Should One Perform Pharmacoepidemiologic Studies? Choosing Among the Available Alternatives 203Brian L. StromIntroduction 203Choosing Among the Available Approaches to Pharmacoepidemiologic Studies 203Examples 215Conclusion 216Key Points 216Further Reading 216Part III Special Issues in Pharmacoepidemiology Methodology 21913 Validity of Drug and Diagnosis Data in Pharmacoepidemiology 221Mary Elizabeth Ritchey, Suzanne L. West, and George MaldonadoIntroduction 221Clinical Problems to be Addressed by Pharmacoepidemiologic Research 221Methodological Problems to be Solved by Pharmacoepidemiologic Research 222Currently Available Solutions 233The Future 242Key Points 242Further Reading 24314 Assessing Causality from Case Reports 246Bernard Bégaud and Judith K. JonesIntroduction 246Clinical Problems to be Addressed by Pharmacoepidemiologic Research 246The Two Paradigms of Causality Assessment 246When is Assessing Causation from Cases Reports Useful? 247Methodological Problems to be Addressed by Pharmacoepidemiologic Research 248Approaches for Assessing Causation from Individual Cases 249Calibration 253Choosing the Appropriate Approach 253The Future 254Key Points 255Further Reading 25515 Molecular Pharmacoepidemiology 257Christine Y. Lu and Stephen E. KimmelIntroduction 257Definitions and Concepts 258The Interface of Pharmacogenetics and Pharmacogenomics with MolecularPharmacoepidemiology 259Clinical Problems to be Addressed by Pharmacoepidemiologic Research 260The Progression and Clinical Application of Molecular Pharmacoepidemiology 264Methodological Problems to be Addressed by Pharmacoepidemiologic Research 264Currently Available Solutions 269The Future 271Key Points 273Further Reading 27416 Bioethical Issues in Pharmacoepidemiologic Research 276Laura E. Bothwell, Annika Richterich, and Jeremy GreeneIntroduction 276Clinical Problems to be Addressed by Pharmacoepidemiologic Research 276Methodologic Problems to be Solved by Pharmacoepidemiologic Research 283Currently Available Solutions 286The Future 289Acknowledgement 291Key Points 291Further Reading 29317 The Use of Randomized Controlled Trials for Pharmacoepidemiology 294Samuel M. Lesko, Allen A. Mitchell, and Robert F. ReynoldsIntroduction 294Clinical Problems to be Addressed by Pharmacoepidemiologic Research 294Methodological Problems to be Solved by Pharmacoepidemiologic Research 296Currently Available Solutions 298Large Simple Trials 298Analysis 303Primary Analysis 303The Future 304Key Points 305Further Reading 30518 Pharmacoeconomics: Economic Evaluation of Pharmaceuticals 307Kevin A. SchulmanIntroduction 307Clinical Problems to be Addressed by Pharmacoeconomic Research 307Methodological Problems to be Addressed by Pharmacoeconomic Research 312The Future 320Acknowledgements 320Key Points 320Further Reading 32019 Patient Engagement and Patient Reported Outcomes 322Esi M. Morgan and Adam C. CarleIntroduction 322Patient Reported Outcomes in Clinical Trials 323Patient Reported Outcomes in Routine Care 323Patient Reported Outcomes as Motivation to Develop New Therapeutic Strategies 325Clinical Problems to be Addressed by Pharmacoepidemiologic Research 326Methodologic Problems to be Solved by Pharmacoepidemiologic Research 328Currently Available Solutions 328The Future 330Key Points 331Further Reading 33120 The Use of Meta-analysis in Pharmacoepidemiology 334Brenda J. Crowe, Stephen J.W. Evans, H. Amy Xia, and Jesse A. BerlinIntroduction 334Clinical Problems to be Addressed by Pharmacoepidemiologic Research 335Methodological Problems to be Solved by Pharmacoepidemiologic Research 336Currently Available Solutions 338The Future 350Key Points 351Further Reading 35221 Studies of Medication Adherence 355Julie Lauffenburger, Trisha Acri, and Robert GrossIntroduction 355Clinical Problems to be Addressed by Pharmacoepidemiologic Research 356Methodological Problems to be Addressed by Pharmacoepidemiologic Research 357Currently Available Solutions 357Analysis Issues in Adherence 362Use of Adherence Data in Clinical Trials and Comparative Effectiveness Studies 362The Future 365Key Points 365Further Reading 36622 Advanced Approaches to Controlling Confounding in Pharmacoepidemiologic Studies 368Sebastian Schneeweiss and Samy SuissaIntroduction 368Clinical Problems to be Addressed by Pharmacoepidemiologic Research 368Methodological Problems to be Addressed by Pharmacoepidemiologic Research 368Currently Available Solutions 370Conclusion 382Key Points 382Further Reading 384Part IV Special Applications and the Future of Pharmacoepidemiology 38723 Special Applications of Pharmacoepidemiology 389David Lee, Björn Wettermark, Christine Y. Lu, Stephen B. Soumerai, Robert T. Chen, Sharon-Lise T.Normand, Art Sedrakyan, Danica Marinac-Dabic, Daniel B. Horton, Sonia Hernandez-Diaz, Tamar Lasky, Krista F. Huybrechts, Claudia Manzo, Emil Cochino, Hanna M. Seidling, David W. Bates, Bennett Levitan, Rachael L. DiSantostefano, and Scott EvansStudies of Drug Utilization 389Introduction 389Evaluating and Improving Prescribing 398Special Methodological Issues in Pharmacoepidemiologic Studies of Vaccine Safety 403Epidemiologic Studies of Implantable Medical Devices 408Research on the Effects of Medications in Pregnancy and in Children 418Risk Management 427The Pharmacoepidemiology of Medication Errors 436Benefit–Risk Assessments of Medical Treatments 442Further Reading 45424 The Future of Pharmacoepidemiology 464Brian L. Strom, Stephen E. Kimmel, and Sean HennessyIntroduction 464The View from Academia 465Scientific Developments 465The View from Industry 471The View from Regulatory Agencies 472The View from the Law 473Conclusion 473Key Points 473Further Reading 474Appendix A — Sample Size Tables 475Appendix B — Glossary 493Index 505