3. Studies in Pharmacoepidemiology and their classification

1. Studies in Pharmacoepidemiology and their classification


Pharmacoepidemiologic studies offer assessments of potential short and long-term benefits and risks of a medicine and other outcomes within a population with a wide range of health status and demographic characteristics and with a longer follow-up period than in clinical trials, that measure efficacy and safety. Studies clarify clinical and demographic characteristics of diseases and conditions; they identify who is at risk and provide clues to the causes of disease. Studies include the analysis of prescribing medication and its determinant factors, give guidance to preventive measures and interventions, describe and analyse the economics of medicine use and underpin scientific advice to decision-makers.

The abundance of digital data has facilitated pharmacoepidemiology and observational research on the effectiveness of medication use under real-world conditions (providing so-called Real World data, RWD). Advantages of pharmacoepidemiologic research are the availability of data from larger groups of patients, and coverage of under-researched subpopulations, allowing more diverse populations to be looked at compared to clinical trials. Drawbacks of observational pharmacoepidemiologic studies come from the non-randomised nature of group selection, leading to various kinds of possible interference by confounding factors (variables). Potential methods for managing this drawback include for instance active comparison groups, within individual designs. Many of the more rigorous pharmacoepidemiology studies have been strengthened through multiple analytical approaches to improve confidence in inferred causal relationships.

In the following a few principles and types of epidemiological studies as employed in pharmacoepidemiology are covered.