The choice of epidemiological methods to answer a research question is based on principles rather than on rules, but certain standards are needed to assure validity and robustness of the study results. Textbooks describing methodological standards in pharmacoepidemiological research are numerous but cannot follow new developments in research. 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 structured review to maintain its dynamic nature. It may also be amended as necessary in response to comments received. For these purposes, any comment and additional relevant guidance document may be forwarded to email@example.com.
The Guide does not discuss general methods of pharmacoepidemiology 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 1 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 to provide methodological guidance addressing the conduct of systematic reviews and meta-analyses of drug safety endpoints generated in completed (published or unpublished) comparative pharmacoepidemiological studies.
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 is especially useful for its recommendations on aspects rarely covered by guidelines, such as data quality issues and archiving. The EU legislation (Directive 2001/83/EU) includes the possibility for regulatory authorities to impose on marketing authorisation holders the conduct of post-authorisation safety study (PASS) as a condition of the marketing authorisation, a PASS being defined as ‘any study relating to an authorised medicinal product conducted with the aim of identifying, characterising or quantifying a safety hazard, confirming the safety profile of the medicinal product, or of measuring the effectiveness of risk management measures.’ The Guideline of good pharmacovigilance practices (GVP) Module VIII - Post-authorisation safety studies provides a general guidance on the development, conduct and reporting of PASS conducted by marketing authorisation holders voluntarily or pursuant to an obligation. 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 such criteria are sometimes interpreted differently at national level in different EU countries.
The following list proposes a list of textbooks on standard methods in pharmacoepidemiology recommended for consultation. The list is not exhaustive, as researchers may find other textbooks more appropriate to their specific needs and others are cited in specific chapters.
|10. Specific topics|
|Annex 1.||Guidance on conducting systematic revies and meta-analyses of completed comparative pharmacoepidemiological studies of safety outcomes|