Pharmacoepidemiologic Studies

Over the last 30 years, observational studies applying epidemiological methods to real-world clinical data from electronic medical records, registries and health insurance claims are highly regarded as generating quality real-world evidence that provides complementary information on safety, particularly post-marketing. This chapter will explore why pharmacoepidemiology is an important part of pharmacovigilance and describe some common epidemiological study designs—namely the cross-sectional study design, the cohort study design and case–control study design—which can be used to study safety (and effectiveness) of medicines. Considerations in objectively assessing such methods will be outlined, including potential confounding and bias, supported by case studies that explore the application of three common epidemiological techniques in support of addressing emergent safety issues. Finally, important initiatives are introduced that collectively aim to improve quality, transparency and reproducibility in generating real-world evidence from observational pharmacoepidemiologic studies that is important for regulatory decision-making.