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European Network of Centres for Pharmacoepidemiology and Pharmacovigilance

Chapter 1: Introduction

Chapter 1 has been revised in August 2026 by the ENCePP co-chairs: Catherine Cohet (European Medicines Agency) and Helga Gardarsdottir (Utrecht University, the Netherlands).

Epidemiology is the study of the distribution and determinants of health-related states or events in specified populations, and the application of this study to the control of health problems (Last JM, editor. A Dictionary of Epidemiology. 4th ed. New York: Oxford University Press; 2001). Guidelines and recommendations for ensuring Good Epidemiological Practice (GEP) (Eur J Epidemiol. 2019;34(3):301-17) provides recommendations for the planning, preparation, execution, analysis, and evaluation of epidemiological research. 

The International Society for Pharmacoepidemiology (ISPE) defines pharmacoepidemiology as a scientific discipline that uses epidemiological methods to evaluate the use, benefits and risks of ‎medical products and interventions in human populations. 

This Guide only briefly addresses general methods of pharmacoepidemiology, as study designs stemming from traditional epidemiological research are fully described in existing textbooks. It rather discusses important aspects of more recent designs and specific analytical approaches to traditional designs, and methods that may be used in studies with drug utilisation, safety, or effectiveness objectives. For some topics, recommendations and references are provided where they differ from general principles.

General guidance on the conduct of pharmacoepidemiology studies is provided in the ISPE Guidelines for Good Pharmacoepidemiology Practices (GPP), which new revision has been launched by the Society in 2026.

The Reflection paper on use of real-word data (RWD) in non-interventional studies (NIS) to generate real-world evidence (RWE) provides methodological guidance on how to design, conduct, analyse, and report NIS for regulatory purposes.

The International Council for Harmonisation (ICH) M14 guideline (General Principles on Planning, Designing, Analysing, and Reporting of Non-interventional Studies That Utilise Real-World Data for Safety Assessment of Medicines. Step 4 Final Guideline. Geneva: ICH; 2025) establishes harmonised principles and best practices for generating RWE on the safety of medicinal products, while key principles can also be applied to effectiveness studies.

The Guideline of good pharmacovigilance practices (GVP) Module VIII - Post-authorisation safety studies (2017) provides general guidance on the development, conduct and reporting of post-authorisation safety studies (PASS) conducted by marketing authorisation holders, voluntarily or pursuant to EU legislation; the new revision is expected to be published in 2026. The Scientific guidance on post-authorisation efficacy studies (2017) provides guidance in the context of EU regulatory decision-making.

Where to begin? Thirty must-read papers for newcomers to pharmacoepidemiology (Pharmacoepidemiol Drug Saf. 2022;31(2):257-9) provides a list of articles touching upon core principles of pharmacoepidemiology and RWE.

The Pharmacoepidemiology and Drug Safety Core Concepts in Pharmacoepidemiology publication series provides concise reviews of the key methods and principles used to study medication use, safety, and effectiveness in real-world settings. It serves as a practical educational resource covering topics such as study design, data sources, bias, and analytical approaches in pharmacoepidemiology.

A selected list of textbooks is provided below. This list is not aimed at being exhaustive and researchers may find other textbooks more appropriate to their needs. Other books are cited in specific chapters of this Guide.

  • Epidemiology: Study Design and Data Analysis, Third Edition (M. Woodward, Chapman & Hall, 2014).
  • Modern Epidemiology, 4th ed. (T. Lash, T.J. VanderWeele, S. Haneuse, K. Rothman. Wolters Kluwer, 2020).
  • Clinical epidemiology: practice and methods, Second Edition (PS Parfrey, BJ Barret, Human Press, 2015).
  • A Dictionary of Epidemiology, 7th edition (M Porta, Editor. Oxford University Press, 2026), sponsored by the International Epidemiological Association (IEA), and related presentation.
  • Dictionary of Pharmacoepidemiology (B. Bégaud, Wiley, 2000).
  • Pharmacoepidemiology, Sixth Edition (B. Strom, S.E. Kimmel, S. Hennessy, Wiley, 2019).
  • Causal Inference: What If (Hernán MA, Robins JM, Chapman & Hall/CRC, 2020.
  • Practical Statistics for Medical Research, Second Edition (D. Altman. Chapman & Hall, 2020).
  • Drug Utilization Research: Methods and Applications, 2nd edition (M. Elseviers, B. Wettermark, T. Mueller, et al., eds., Wiley-Blackwell, 2024).
  • Mann's Pharmacovigilance, Third Edition (EB Andrews, N Moore, Editors, Wiley-Blackwell, 2014).
  • Post-Authorization Safety Studies of Medicinal Products. The PASS Book, 1st Edition (Ayad Ali, Abraham Hartzema, Ed., Academic Press, 2018).
  • Debray, T.P.A., Nguyen, T.-L., & Platt, R.W. (Eds.). (2026). Comparative Effectiveness and Personalized Medicine Research Using Real-World Data (1st ed.). Chapman and Hall/CRC. 
  • Pragmatic Randomized Clinical Trials Using Primary Data Collection and Electronic Health Records (1st Edition - April 8, 2021, Eds: Cynthia Girman, Mary Ritchey).
  • Drug Utilization Research - Methods and Applications (M Elseviers, B Wettermark, AB Almarsdóttir, et al. Editors. Wiley Blackwell, 2016).
  • Applying quantitative bias analysis to epidemiologic data (Lash T, Fox MP, Fink AK. Springer Verlag, New-York, 2009).
  • Hahné, S., Bollaerts, K., & Farrington, P. (2021). Vaccination Programmes: Epidemiology, Monitoring, Evaluation (1st ed.). Routledge. 
  • Halloran ME, Longini IM, Struchiner CJ. Design and Analysis of Vaccine Studies. New York: Springer; 2010. 
  • Higgins JPT, Thomas J, Chandler J, Cumpston M, Li T, Page MJ, Welch VA, editors. Cochrane Handbook for Systematic Reviews of Interventions. Version 6.2. Cochrane; 2021.