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 email@example.com.
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.
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.