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ENCePP Guide on Methodological Standards in Pharmacoepidemiology

 

Chapter 1: Introduction

The choice of epidemiological methods to answer a research question is based on principles and methodological standards to assure validity of the study results. Textbooks describing methodological standards in pharmacoepidemiological research are numerous but cannot incorporate timely all new research developments. ENCePP therefore considered there was a need for a regularly updated resource providing recommendations on the practical implementation of pharmacoepidemiological principles and innovative methods, based on published guidance and illustrative examples.

 

This Guide aims to offer a dynamic and publicly available web resource for methodological English language guidance in pharmacoepidemiology. It provides links to selected published articles and guidelines that illustrate important principles of pharmacoepidemiological research. For each topic covered, recommendations are provided with direct electronic access to textbooks, reference documents and examples chosen by experts from ENCePP. Where relevant, gaps in existing guidance are addressed with what ENCePP considers as being good practice. The Guide is updated annually by a structured review in order to maintain its dynamic nature. It may also be amended as necessary in response to comments received. For this purpose, any comment and additional relevant guidance document may be forwarded to encepp_comments@ema.europa.eu.

 

The Guide does not discuss general methods of pharmacoepidemiology as they are already covered in existing textbooks. For example, it does not describe traditional study designs like the cross-sectional, cohort and case-control designs but it discusses important aspects of more recent designs such as the self-controlled case series (SCCS) design. Chapters 2 to 9 describe methods that may be used in studies with different goals, be they safety, effectiveness, drug utilisation, health technology assessment or any other objective. For some specific topics, specific recommendations and references are provided where they differ from general principles. Chapter 10 provides more extensive guidance on three specific topics, comparative effectiveness research, vaccine safety and effectiveness and pharmacogenetic studies. This chapter may be expanded in future revisions if there is a need for methodological guidance on other topics.

 

Annex 1 has been developed separately by an ENCePP working group and provides methodological guidance addressing the conduct of systematic reviews and meta-analyses of drug safety endpoints.

 

Annex 2 has been developed by an ENCePP Special Interest Group and provides recommendations on methods for measuring the impact of pharmacovigilance activities on patients and public health.

 

General guidance on the conduct of pharmacoepidemiology studies can be found in the ISPE Good Pharmacoepidemiology Practices (GPP) and the IEA Good Epidemiology Practice (GEP). The GPP guidance is especially useful for its recommendations on aspects rarely covered by guidelines, such as data quality issues and archiving. The Guideline of good pharmacovigilance practices (GVP) Module VIII - Post-authorisation safety studies provides a general guidance on the development, conduct and reporting of post-authorisation safety studies (PASS) conducted by marketing authorisation holders voluntarily or pursuant to the EU legislation (Directive 2001/83/EU). It also describes the criteria applicable in the European Union (EU) to define a post-authorisation study as non-interventional, but investigators should be aware that implementation of these criteria may vary at national level in different EU countries.

 

Textbooks on standard methods in pharmacoepidemiology that are considered useful are listed below. The list is not exhaustive, and researchers may find other textbooks more appropriate to their specific needs and others are cited in specific chapters.

  • Modern Epidemiology, Third Edition (K. Rothman, S. Greenland, T. Lash. Lippincott Williams & Wilkins, 2013) is a comprehensive textbook on methods in epidemiology.
  • Epidemiology: Study Design and Data Analysis, Third Edition (M. Woodward, Chapman & Hall, 2014) focuses on the quantitative aspects of epidemiological research.

 

  • A Dictionary of Epidemiology, Fifth Edition (M Porta, Editor. J.M. Last S. Greenland, Associate Editors. Oxford University Press, 2008), sponsored by the International Epidemiological Association (IEA), provides a definition and concise explanation of epidemiologic terms and is a key to understanding epidemiological concepts.

 

  • Clinical epidemiology: practice and methods, Second Edition (PS Parfrey, BJ Barret, Human Press, 2016) focuses on the diagnosis, prognosis and management of human disease using appropriate research design, measurement and evaluation.

 

  • Textbook of Pharmacoepidemiology, Second Edition (B. Strom, S.E. Kimmel, S. Hennessy,Wiley, 2013) provides an introduction to pharmacoepidemiology and the data sources, methods and applications used in clinical research, the pharmaceutical industry and regulatory agencies.

 

  • Pharmacoepidemiology and Therapeutic Risk Management, Third Edition (A.G. Hartzema, H.H. Tilson and K.A. Chan, Editors, Harvey Whitney Books Company, 2008) illustrates practical issues with a large number of real life examples in addition to a general review of drug-specific methodologies.

 

  • Practical Statistics for Medical Research, Second Edition (D. Altman. Chapman & Hall, 2015) presents a problem-based statistical text for medical researchers.

 

  • Drug Utilization Research. Methods and Applications (M Elseviers, B Wettermark, AB Almarsdóttir, et al. Editors. Wiley Blackwell, 2016) provides a comprehensive manual of methodology and applications of drug utilisation research.

 

  • Mann's Pharmacovigilance, Third Edition (EB Andrews, N Moore, Editors, Wiley-Blackwell, 2014) is a reference for the science of detection, assessment, understanding and prevention of the adverse effects of medicines, including vaccines and biologics.

 

  • Post-Authorization Safety Studies of Medicinal Products. The PASS Book, 1st Edition (Ayad Ali, Abraham Hartzema, Ed., Academic Press, 2018) covers the use of observational studies in post-marketing drug safety assessment, presents various types of post-authorisation safety studies and discusses challenges and solutions in the design and conduct of these studies.

 

 

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