Textbook of Pharmacoepidemiology (eBook)

Edited by Brian L. Strom, MD, MPH Executive Vice Dean for Institutional Affairs, George S. Pepper Professor of Public Health and Preventive Medicine, Professor of Biostatistics and Epidemiology, of Medicine, and of Pharmacology, Center for Clinical Epidemiology and Biostatistics, Center for Pharmacoepidemiology Research and Training, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA Stephen E. Kimmel, MD, MSCE Professor of Medicine and of Epidemiology, Center for Clinical Epidemiology and Biostatistics, Center for Pharmacoepidemiology Research and Training, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA Sean Hennessy, PharmD, PhD Associate Professor of Epidemiology and of Pharmacology, Center for Clinical Epidemiology and Biostatistics, Center for Pharmacoepidemiology Research and Training, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA

Textbook of Pharmacoepidemiology 1
Contents 7
Contributors 9
Preface 15
Acknowledgements 19
Part I: Introduction to Pharmacoepidemiology 21
1 What is Pharmacoepidemiology? 23
Introduction 23
Definition of pharmacoepidemiology 23
Historical background 25
The current drug approval process 31
Potential contributions of pharmacoepidemiology 33
Key points 35
Further reading 35
2 Study Designs Available for Pharmacoepidemiologic Studies 37
Overview of the scientific method 37
Types of errors that one can make in performing a study 38
Criteria for the causal nature of an association 40
Epidemiologic study designs 42
Case reports 42
Discussion 47
Conclusion 48
Key points 48
Further reading 49
3 Sample Size Considerations for Pharmacoepidemiologic Studies 50
Introduction 50
Sample size calculations for cohort studies 50
Sample size calculations for case-control studies 53
Sample size calculations for case series 56
Discussion 57
Key points 59
References and further readings 59
4 Basic Principles of Clinical Pharmacology Relevant to Pharmacoepidemiologic Studies 60
Clinical pharmacology and pharmacoepidemiology 60
Basics of clinical pharmacology 61
Pharmacokinetics 61
Special populations 64
Pharmacodynamics 69
Pharmacogenomics 70
Conclusion 71
Key points 72
Further reading 72
5 When Should One Perform Pharmacoepidemiologic Studies? 74
Reasons to perform pharmacoepidemiologic studies 74
Safety versus risk 78
Risk tolerance 79
Conclusion 81
Key points 81
Further reading 82
6 Views from Academia, Industry, Regulatory Agencies, and the Legal System 83
The view from academia 83
Summary points for the view from academia 89
The view from industry 91
The view from regulatory agencies 99
The view from the legal system 106
Further reading 114
Part II: Sources of Pharmacoepidemiology Data 119
7 Postmarketing Spontaneous Pharmacovigilance Reporting Systems 121
Introduction 121
Description 122
Strengths 132
Limitations 133
Particular applications 134
The future 135
Key points 135
Further reading 137
8 Overview of Automated Databases in Pharmacoepidemiology 138
Introduction 138
Description 139
Strengths 140
Weaknesses 140
Particular applications 141
The future 142
Key points 142
Further reading 142
9 Examples of Existing Automated Databases 143
US Health maintenance organizations/health plans 143
US government claims databases 153
Canadian provincial databases 164
Medical record databases 172
Pharmacy-based medical record linkage systems 183
Further reading 193
10 Field Studies 198
Strengths 198
Weaknesses 199
Particular applications 200
Conclusions 208
Key points 208
Further Reading 209
11 How Should One Perform Pharmacoepidemiologic Studies? Choosing Among the Available Alternatives 210
Introduction 210
Choosing among the available approaches to pharmacoepidemiologic studies 210
Examples 220
Conclusion 221
Key points 221
Further reading 221
Part III: Special Issues in PharmacoepidemiologyMethodology 223
12 Validity of Pharmacoepidemiologic Drug and Diagnosis Data 225
Introduction 225
Clinical problems to be addressed by pharmacoepidemiologic research 225
Methodological problems to be solved by pharmacoepidemiologic research 226
Methodological problems in pharmacoepidemiologic research 232
Currently available solutions 238
The future 245
Key points 246
Further reading 246
13 Assessing Causality of Case Reports of Suspected Adverse Events 248
Introduction 248
Clinical problems to be addressed by pharmacoepidemiologic research 248
Historical perspectives: development of concepts 249
Uses of causality assessment 251
Methodological problems to be addressed by pharmacoepidemiologic research 252
Current tools 253
Unstructured clinical judgment/global introspection 253
Algorithm/criterial method with verbal judgments 253
Algorithms requiring scoring of individual judgments 253
Probabilistic methods 254
Comparison among the different methods 255
The future 256
Key points 257
Further reading 257
14 Molecular Pharmacoepidemiology 259
Introduction 259
Definitions and concepts 259
The interface of pharmacogenetics and pharmacogenomics with molecular pharmacoepidemiology 260
Clinical problems to be addressed by pharmacoepidemiologic research 261
Three ways that genes can affect drug response 261
The progression and application of molecular pharmacoepidemiologic research 264
Methodological problems to be addressed by pharmacoepidemiologic research 265
Currently available solutions 270
The future 272
Key points 273
Further reading 274
15 Bioethical Issues in Pharmacoepidemiologic Research 276
Introduction 276
Clinical problems to be addressed by pharmacoepidemiologic research 277
Methodological problems to be addressed by pharmacoepidemiologic research 281
Currently available solutions 283
Conclusion 285
Key points 286
Further reading 288
16 The Use of Randomized Controlled Trials for Pharmacoepidemiologic Studies 289
Introduction 289
Clinical problems to be addressed by pharmacoepidemiologic research 289
Methodological problems to be solved by pharmacoepidemiologic research 291
Currently available solutions 293
The future 297
Key points 298
Further reading 298
17 Pharmacoeconomics: Economic Evaluation of Pharmaceuticals 300
Clinical problems to be addressed by pharmacoeconomic research 300
Economic evaluation and the drug development process 301
Methodological problems to be addressed by pharmacoeconomic research 301
Methodological issues in the pharmacoeconomic assessment of therapies 305
The future 309
Key points 310
Further Reading 310
18 Using Quality-of-Life Measurements in Pharmacoepidemiologic Research 311
Introduction 311
Clinical problems to be addressed by pharmacoepidemiologic research 311
Methodological problems to be addressed by pharmacoepidemiologic research 312
Currently available solutions 312
The future 318
Key points 318
Further reading 318
19 The Use of Meta-analysis in Pharmacoepidemiology 320
Introduction 320
Clinical problems to be addressed by pharmacoepidemiologic research 320
Methodological problems to be addressed by pharmacoepidemiologic research 321
Currently available solutions 322
Currently available solutions 328
The future 331
Key points 332
Further reading 332
20 Studies of Medication Adherence 334
Introduction 334
Clinical problems to be addressed by pharmacoepidemiologic research 334
Methodologic problems to be solved by pharmacoepidemiologic research 335
Currently available solutions 336
Future directions 341
Key points 342
Further Reading 342
21 Advanced Approaches to Controlling Confounding in Pharmacoepidemiologic Studies 344
Clinical problems to be addressed by pharmacoepidemiologic research 344
Methodological problems to be addressed by pharmacoepidemiologic research 344
Currently available solutions 345
Conclusion 354
Key points 354
Further reading 356
Part IV: Special Applications 357
22 Special Applications of Pharmacoepidemiology 359
Studies of drug utilization 359
Evaluating and improving physician prescribing 366
Special methodological issues in pharmacoepidemiologic studies of vaccine safety 370
Key points for special methodological issues in pharmacoepidemiologic studies of vaccine study 374
Epidemiologic studies of implantable medical devices 375
Studies of drug-induced birth defects 383
Risk management 390
The use of pharmacoepidemiology to study medication errors 398
FDA’s Sentinel Initiative: enhancing safety surveillance 404
Comparative effectiveness research 413
Further reading 421
23 The Future of Pharmacoepidemiology 427
The view from academia 428
The view from industry 433
The view from regulatory agencies 433
The view from the law 434
Conclusion 434
Key points 434
Further Reading 435
Appendix A—Sample Size Tables 437
Appendix B—Glossary 455
Index 465