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Concepts and Principles of Pharmacology pp 433–451Cite as

Pharmacoepidemiology

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Part of the book series: Handbook of Experimental Pharmacology ((HEP,volume 260))

Abstract

At the time of their marketing authorization, the effects of drugs and especially their efficacy have been mostly studied in randomized controlled clinical trials (RCT), comparing them to placebo or to existing drugs. However, RCT are by nature limited in their extent, and the often stringent inclusion and exclusion criteria destined to provide for homogeneous study populations reduce the generalizability of RCT results.

The post-authorization evaluation of drugs (pharmacoepidemiology or real-world evidence (RWE)) covers the description of drug utilization and population risks or benefits of these drugs after they have been marketed and provided to their target populations. Though field studies have existed for a long time, modern pharmacoepidemiology has been made possible essentially by the emergence of large population databases compiled from claims data or electronic health records. The methods can be exposure or disease-based cohorts or event-driven case-based studies, tailored to the specific questions to be answered. They rely on scrupulous analysis and execution of impeccable methodology, to ensure the most reliable results possible.

Pharmacoepidemiology requires knowledge of the pharmacology of drugs, of the clinical aspects of diseases and disease management, and of the epidemiological methods that can apply.

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References

  • Bezin J, Girodet PO, Rambelomanana S, Touya M, Ferreira P, Gilleron V et al (2015) Choice of ICD-10 codes for the identification of acute coronary syndrome in the French hospitalization database. Fundam Clin Pharmacol 29(6):586–591

    CAS  PubMed  Google Scholar 

  • Bezin J, Duong M, Lassalle R, Droz C, Pariente A, Blin P et al (2017) The national healthcare system claims databases in France, SNIIRAM and EGB: powerful tools for pharmacoepidemiology. Pharmacoepidemiol Drug Saf 26(8):954–962

    PubMed  Google Scholar 

  • Bezin J, Klungel OH, Lassalle R, Dureau-Pournin C, Moore N, Pariente A (2018) Medications recommended for secondary prevention after first acute coronary syndrome: effectiveness of treatment combinations in a real-life setting. Clin Pharmacol Ther 103(6):1038–1046

    PubMed  Google Scholar 

  • Blin P, Dureau-Pournin C, Lassalle R, Jove J, Thomas-Delecourt F, Droz-Perroteau C et al (2017) Outcomes in patients after myocardial infarction similar to those of the PEGASUS-TIMI 54 trial: a cohort study in the French national claims database. Br J Clin Pharmacol 83(9):2056–2065

    CAS  PubMed  PubMed Central  Google Scholar 

  • Blin P, Dureau-Pournin C, Benichou J, Bonello L, Dallongeville J, Danchin N et al (2019a) Secondary prevention of acute coronary events with antiplatelet agents (SPACE-AA): one-year real-world effectiveness and safety cohort study in the French nationwide claims database. Atherosclerosis 281:98–106

    CAS  PubMed  Google Scholar 

  • Blin P, Dureau-Pournin C, Benichou J, Cottin Y, Mismetti P, Abouelfath A et al (2019b) Comparative real-life effectiveness and safety of dabigatran or rivaroxaban vs. vitamin K antagonists: a high-dimensional propensity score matched new users cohort study in the French National Healthcare Data System SNDS. Am J Cardiovasc Drugs. e-pub ahead of print 2019/06/30; https://doi.org/10.1007/s40256-019-00359-z

  • Blin P, Dureau-Pournin C, Cottin Y, Benichou J, Mismetti P, Abouelfath A et al (2019c) Effectiveness and safety of 110 or 150 mg dabigatran vs. vitamin K antagonists in nonvalvular atrial fibrillation. Br J Clin Pharmacol 85(2):432–441

    CAS  PubMed  Google Scholar 

  • Blin P, Fauchier L, Dureau-Pournin C, Sacher F, Dallongeville J, Bernard MA et al (2019d) Effectiveness and safety of rivaroxaban 15 or 20 mg versus vitamin K antagonists in nonvalvular atrial fibrillation. Stroke 50(9):2469–2476

    CAS  PubMed  PubMed Central  Google Scholar 

  • Blin P, Dureau-Pournin C, Cottin Y, Benichou J, Mismetti P, Abouelfath A et al (2019e) Comparative effectiveness and safety of standard or reduced dose dabigatran vs. rivaroxaban in nonvalvular atrial fibrillation. Clin Pharmacol Ther 105(6):1439–1455

    CAS  PubMed  Google Scholar 

  • Bosco-Levy P, Duret S, Picard F, Dos Santos P, Puymirat E, Gilleron V et al (2019) Diagnostic accuracy of the International Classification of Diseases, Tenth Revision, codes of heart failure in an administrative database. Pharmacoepidemiol Drug Saf 28(2):194–200

    PubMed  Google Scholar 

  • Coloma PM, Schuemie MJ, Trifiro G, Gini R, Herings R, Hippisley-Cox J et al (2011) Combining electronic healthcare databases in Europe to allow for large-scale drug safety monitoring: the EU-ADR project. Pharmacoepidemiol Drug Saf 20(1):1–11

    PubMed  Google Scholar 

  • Czwikla J, Jobski K, Schink T (2017) The impact of the lookback period and definition of confirmatory events on the identification of incident cancer cases in administrative data. BMC Med Res Methodol 17(1):122

    PubMed  PubMed Central  Google Scholar 

  • Depont F, Fourrier A, Merliere Y, Droz C, Amouretti M, Begaud B et al (2007a) Channelling of COX-2 inhibitors to patients at higher gastrointestinal risk but not at lower cardiovascular risk: the Cox2 inhibitors and tNSAIDs description of users (CADEUS) study. Pharmacoepidemiol Drug Saf 16(8):891–900

    CAS  PubMed  Google Scholar 

  • Depont F, Fourrier A, Merliere Y, Droz C, Amouretti M, Begaud B et al (2007b) The CADEUS study: methods and logistics. Pharmacoepidemiol Drug Saf 16(5):571–580

    CAS  PubMed  Google Scholar 

  • Dhainaut JF, Huot L, Pomar VB, Dubray C, Participants of Round Table of Giens X, contributeurs (2018) Using connected objects in clinical research. Therapie 73(1):53–62

    PubMed  Google Scholar 

  • Duong M, Salvo F, Pariente A, Abouelfath A, Lassalle R, Droz C et al (2014) Usage patterns of ‘over-the-counter’ vs. prescription-strength nonsteroidal anti-inflammatory drugs in France. Br J Clin Pharmacol 77(5):887–895

    PubMed  PubMed Central  Google Scholar 

  • Duong M, Gulmez SE, Salvo F, Abouelfath A, Lassalle R, Droz C et al (2016) Usage patterns of paracetamol in France. Br J Clin Pharmacol 82(2):498–503

    CAS  PubMed  PubMed Central  Google Scholar 

  • Duong M, Abouelfath A, Lassalle R, Droz C, Blin P, Moore N (2018) Coronary events after dispensing of ibuprofen: a propensity score-matched cohort study versus paracetamol in the French nationwide claims database sample. Drug Saf 41(11):1049–1058

    CAS  PubMed  Google Scholar 

  • Feinstein AR, Horwitz RI (1981) An algebraic analysis of biases due to exclusion, susceptibility, and protopathic prescription in case-control research. J Chronic Dis 34(8):393–403

    CAS  PubMed  Google Scholar 

  • Fine JP, Gray RJ (1999) A proportional hazards model for the subdistribution of a competing risk. J Am Stat Assoc 94:496–509

    Google Scholar 

  • Flynn RW, MacDonald TM, Hapca A, MacKenzie IS, Schembri S (2014) Quantifying the real life risk profile of inhaled corticosteroids in COPD by record linkage analysis. Respir Res 15:141

    PubMed  PubMed Central  Google Scholar 

  • Fosbol EL, Gislason GH, Jacobsen S, Folke F, Hansen ML, Schramm TK et al (2009) Risk of myocardial infarction and death associated with the use of nonsteroidal anti-inflammatory drugs (NSAIDs) among healthy individuals: a nationwide cohort study. Clin Pharmacol Ther 85(2):190–197

    CAS  PubMed  Google Scholar 

  • Fourrier-Reglat A, Lacoin L, Pariente A, Lassalle R, Robinson P, Droz-Perroteau C et al (2010a) When patients report diseases that prescribers seem unaware of: discordance between patient and physician reporting of risk-related previous history in NSAID users from the CADEUS study. Clin Pharmacol Ther 88(5):668–675

    CAS  PubMed  Google Scholar 

  • Fourrier-Reglat A, Cuong HM, Lassalle R, Depont F, Robinson P, Droz-Perroteau C et al (2010b) Concordance between prescriber- and patient-reported previous medical history and NSAID indication in the CADEUS cohort. Pharmacoepidemiol Drug Saf 19(5):474–481

    PubMed  Google Scholar 

  • Fourrier-Reglat A, Noize P, Facon T, Fermand JP, Fitoussi O, Marit G et al (2014a) Real-life patterns of use and effectiveness of bortezomib: the VESUVE cohort study. Leuk Lymphoma 55(4):848–854

    CAS  PubMed  Google Scholar 

  • Fourrier-Reglat A, Smith D, Rouyer M, Benichou J, Guimbaud R, Becouarn Y et al (2014b) Survival outcomes of bevacizumab in first-line metastatic colorectal cancer in a real-life setting: results of the ETNA cohort. Target Oncol 9(4):311–319

    PubMed  Google Scholar 

  • Garbe E, Kloss S, Suling M, Pigeot I, Schneeweiss S (2013) High-dimensional versus conventional propensity scores in a comparative effectiveness study of coxibs and reduced upper gastrointestinal complications. Eur J Clin Pharmacol 69(3):549–557

    CAS  PubMed  Google Scholar 

  • Graham DJ, Reichman ME, Wernecke M, Zhang R, Southworth MR, Levenson M et al (2015) Cardiovascular, bleeding, and mortality risks in elderly Medicare patients treated with dabigatran or warfarin for nonvalvular atrial fibrillation. Circulation 131(2):157–164

    CAS  PubMed  Google Scholar 

  • Guiard E, Lelievre B, Rouyer M, Zerbib F, Diquet B, Megraud F et al (2019) Bismuth concentrations in patients treated in real-life practice with a bismuth subcitrate-metronidazole-tetracycline preparation: the SAPHARY study. Drug Saf 42:993

    CAS  PubMed  Google Scholar 

  • Gulmez SE, Larrey D, Pageaux GP, Lignot S, Lassalle R, Jove J et al (2013a) Transplantation for acute liver failure in patients exposed to NSAIDs or paracetamol (acetaminophen): the multinational case-population SALT study. Drug Saf 36(2):135–144

    CAS  PubMed  PubMed Central  Google Scholar 

  • Gulmez SE, Larrey D, Pageaux GP, Lignot-Maleyran S, de Vries C, Sturkenboom M et al (2013b) Methodology for a multinational case-population study on liver toxicity risks with NSAIDs: the Study of Acute Liver Transplant (SALT). Eur J Clin Pharmacol 69(3):605–616

    CAS  PubMed  Google Scholar 

  • Gulmez SE, Larrey D, Pageaux GP, Bernuau J, Bissoli F, Horsmans Y et al (2015) Liver transplant associated with paracetamol overdose: results from the seven-country SALT study. Br J Clin Pharmacol 80(3):599–606

    CAS  PubMed  PubMed Central  Google Scholar 

  • Hallas J (1996) Evidence of depression provoked by cardiovascular medication: a prescription sequence symmetry analysis. Epidemiology 7(5):478–484

    CAS  PubMed  Google Scholar 

  • Henry D, Lim LL, Garcia Rodriguez LA, Perez Gutthann S, Carson JL, Griffin M et al (1996) Variability in risk of gastrointestinal complications with individual non-steroidal anti-inflammatory drugs: results of a collaborative meta-analysis. BMJ 312(7046):1563–1566

    CAS  PubMed  PubMed Central  Google Scholar 

  • Horwitz RI, Feinstein AR (1980) The problem of “protopathic bias” in case-control studies. Am J Med 68(2):255–258

    CAS  PubMed  Google Scholar 

  • Idema DL, Wang Y, Biehl M, Horvatovich PL, Hak E (2018) Effect estimate comparison between the prescription sequence symmetry analysis (PSSA) and parallel group study designs: a systematic review. PLoS One 13(12):e0208389

    CAS  PubMed  PubMed Central  Google Scholar 

  • Jick SS, Kaye JA, Vasilakis-Scaramozza C, Garcia Rodriguez LA, Ruigomez A, Meier CR et al (2003) Validity of the general practice research database. Pharmacotherapy 23(5):686–689

    PubMed  Google Scholar 

  • Levesque LE, Hanley JA, Kezouh A, Suissa S (2010) Problem of immortal time bias in cohort studies: example using statins for preventing progression of diabetes. BMJ 340:b5087

    PubMed  Google Scholar 

  • Macdonald TM, Mackenzie IS, Wei L, Hawkey CJ, Ford I, Collaborators SSG (2013) Methodology of a large prospective, randomised, open, blinded endpoint streamlined safety study of celecoxib versus traditional non-steroidal anti-inflammatory drugs in patients with osteoarthritis or rheumatoid arthritis: protocol of the standard care versus celecoxib outcome trial (SCOT). BMJ Open 3(1)

    PubMed  PubMed Central  Google Scholar 

  • MacDonald TM, Ford I, Nuki G, Mackenzie IS, De Caterina R, Findlay E et al (2014) Protocol of the Febuxostat versus Allopurinol Streamlined Trial (FAST): a large prospective, randomised, open, blinded endpoint study comparing the cardiovascular safety of allopurinol and febuxostat in the management of symptomatic hyperuricaemia. BMJ Open 4(7):e005354

    PubMed  PubMed Central  Google Scholar 

  • Mackenzie IS, Ford I, Walker A, Hawkey C, Begg A, Avery A et al (2016) Multicentre, prospective, randomised, open-label, blinded end point trial of the efficacy of allopurinol therapy in improving cardiovascular outcomes in patients with ischaemic heart disease: protocol of the ALL-HEART study. BMJ Open 6(9):e013774

    PubMed  PubMed Central  Google Scholar 

  • Miranda S, Chaignot C, Collin C, Dray-Spira R, Weill A, Zureik M (2017) Human papillomavirus vaccination and risk of autoimmune diseases: a large cohort study of over 2 million young girls in France. Vaccine 35(36):4761–4768

    PubMed  Google Scholar 

  • Moore N, Masson H, Noblet C, Joannidès R (1993) What medicines do patients really take? A comparison of free form vs oriented questionnaires. Post Marketing Surveillance 7:355–362

    Google Scholar 

  • Moore N, Pierfitte C, Pehourcq F, Lagnaoui R, Begaud B (2001) Comparison of patient questionnaires, medical records, and plasma assays in assessing exposure to benzodiazepines in elderly subjects. Clin Pharmacol Ther 69(6):445–450

    CAS  PubMed  Google Scholar 

  • Moore N, Gulmez SE, Larrey D, Pageaux GP, Lignot S, Lassalle R et al (2013) Choice of the denominator in case population studies: event rates for registration for liver transplantation after exposure to NSAIDs in the SALT study in France. Pharmacoepidemiol Drug Saf 22(2):160–167

    PubMed  Google Scholar 

  • Moore N, Duret S, Grolleau A, Lassalle R, Barbet V, Duong M et al (2019) Previous drug exposure in patients hospitalised for acute liver injury: a case-population study in the French National Healthcare Data System. Drug Saf 42(4):559–572

    CAS  PubMed  Google Scholar 

  • Moride Y, Abenhaim L (1994) Evidence of the depletion of susceptibles effect in non-experimental pharmacoepidemiologic research. J Clin Epidemiol 47(7):731–737

    CAS  PubMed  Google Scholar 

  • Neugebauer R, Schmittdiel JA, Zhu Z, Rassen JA, Seeger JD, Schneeweiss S (2015) High-dimensional propensity score algorithm in comparative effectiveness research with time-varying interventions. Stat Med 34(5):753–781

    PubMed  Google Scholar 

  • Neumann A, Weill A, Ricordeau P, Fagot JP, Alla F, Allemand H (2012) Pioglitazone and risk of bladder cancer among diabetic patients in France: a population-based cohort study. Diabetologia 55(7):1953–1962

    CAS  PubMed  PubMed Central  Google Scholar 

  • Noize P, Bazin F, Dufouil C, Lechevallier-Michel N, Ancelin ML, Dartigues JF et al (2009) Comparison of health insurance claims and patient interviews in assessing drug use: data from the Three-City (3C) study. Pharmacoepidemiol Drug Saf 18(4):310–319

    PubMed  Google Scholar 

  • Noize P, Bazin F, Pariente A, Dufouil C, Ancelin ML, Helmer C et al (2012) Validity of chronic drug exposure presumed from repeated patient interviews varied according to drug class. J Clin Epidemiol 65(10):1061–1068

    PubMed  Google Scholar 

  • Noize P, Grelaud A, Bay JO, Chevreau C, Gross-Goupil M, Culine S et al (2017) Real-life patterns of use, safety and effectiveness of sunitinib in first-line therapy of metastatic renal cell carcinoma: the SANTORIN cohort study. Pharmacoepidemiol Drug Saf 26:1561

    CAS  PubMed  Google Scholar 

  • Petri H, de Vet HC, Naus J, Urquhart J (1988) Prescription sequence analysis: a new and fast method for assessing certain adverse reactions of prescription drugs in large populations. Stat Med 7(11):1171–1175

    CAS  PubMed  Google Scholar 

  • Pierfitte C, Macouillard G, Thicoipe M, Chaslerie A, Pehourcq F, Aissou M et al (2001) Benzodiazepines and hip fractures in elderly people: case-control study. BMJ 322(7288):704–708

    CAS  PubMed  PubMed Central  Google Scholar 

  • Pladevall-Vila M, Pottegard A, Schink T, Reutfors J, Morros R, Poblador-Plou B et al (2019) Risk of acute liver injury in agomelatine and other antidepressant users in four European countries: a cohort and nested case-control study using automated health data sources. CNS Drugs 33(4):383–395

    CAS  PubMed  PubMed Central  Google Scholar 

  • Rassen JA, Glynn RJ, Brookhart MA, Schneeweiss S (2011) Covariate selection in high-dimensional propensity score analyses of treatment effects in small samples. Am J Epidemiol 173(12):1404–1413

    PubMed  PubMed Central  Google Scholar 

  • Rouyer M, Francois E, Cunha AS, Monnereau A, Noize P, Robinson P et al (2018) Effectiveness of cetuximab as first-line therapy for patients with wild-type KRAS and unresectable metastatic colorectal cancer in real-life practice: results of the EREBUS cohort. Clin Colorectal Cancer 17(2):129–139

    PubMed  Google Scholar 

  • Schneeweiss S (2018) Automated data-adaptive analytics for electronic healthcare data to study causal treatment effects. Clin Epidemiol 10:771–788

    PubMed  PubMed Central  Google Scholar 

  • Schneeweiss S, Rassen JA, Glynn RJ, Avorn J, Mogun H, Brookhart MA (2009) High-dimensional propensity score adjustment in studies of treatment effects using health care claims data. Epidemiology 20(4):512–522

    PubMed  PubMed Central  Google Scholar 

  • Secrest MH, Platt RW, Dormuth CR, Chateau D, Targownik L, Nie R et al (2019) Extreme restriction design as a method for reducing confounding by indication in pharmacoepidemiologic research. Pharmacoepidemiol Drug Saf. e-pub ahead of print 2019/01/11; https://doi.org/10.1002/pds.4708

  • Steg PG, Lopez-Sendon J, Lopez de Sa E, Goodman SG, Gore JM, Anderson FA Jr et al (2007) External validity of clinical trials in acute myocardial infarction. Arch Intern Med 167(1):68–73

    PubMed  Google Scholar 

  • Suissa S (2008) Immortal time bias in pharmaco-epidemiology. Am J Epidemiol 167(4):492–499

    PubMed  Google Scholar 

  • Trifiro G, Pariente A, Coloma PM, Kors JA, Polimeni G, Miremont-Salame G et al (2009) Data mining on electronic health record databases for signal detection in pharmacovigilance: which events to monitor? Pharmacoepidemiol Drug Saf 18(12):1176–1184

    PubMed  Google Scholar 

  • Wang SV, Schneeweiss S, Berger ML, Brown J, de Vries F, Douglas I et al (2017) Reporting to improve reproducibility and facilitate validity assessment for healthcare database studies V1.0. Pharmacoepidemiol Drug Saf 26(9):1018–1032

    PubMed  PubMed Central  Google Scholar 

  • Wentzell N, Schink T, Haug U, Ulrich S, Niemeyer M, Mikolajczyk R (2018) Optimizing an algorithm for the identification and classification of pregnancy outcomes in German claims data. Pharmacoepidemiol Drug Saf 27(9):1005–1010

    PubMed  Google Scholar 

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Authors and Affiliations

  1. Bordeaux PharmacoEpi, INSERM CIC1401, Université de Bordeaux, Bordeaux, France

    Nicholas Moore, Patrick Blin & Cécile Droz

Authors
  1. Nicholas Moore
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  2. Patrick Blin
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  3. Cécile Droz
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Corresponding author

Correspondence to Nicholas Moore .

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Editors and Affiliations

  1. Center for Substance Abuse Research, Lewis Katz School of Medicine at Temple University, Philadelphia, PA, USA

    James E. Barrett

  2. Sackler Institute of Pulmonary Pharmacology, Institute of Pharmaceutical Science, King’s College London, London, UK

    Clive P. Page

  3. Department of Pharmacology, Johannes Gutenberg University, Mainz, Rheinland-Pfalz, Germany

    Martin C. Michel

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Moore, N., Blin, P., Droz, C. (2019). Pharmacoepidemiology. In: Barrett, J., Page, C., Michel, M. (eds) Concepts and Principles of Pharmacology. Handbook of Experimental Pharmacology, vol 260. Springer, Cham. https://doi.org/10.1007/164_2019_318

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