Pharmacoepidemiology research: delivering evidence about drug safety and effectiveness in mental health

Abstract

Research that provides an evidence base for the pharmacotherapy of people with mental disorders is needed. The abundance of digital data has facilitated pharmacoepidemiology and, in particular, observational research on the effectiveness of real-world medication. Advantages of pharmacoepidemiological research are the availability of large patient samples, and coverage of under-researched subpopulations in their naturalistic conditions. Such research is also cheaper and quicker to do than randomised controlled trials, meaning that issues regarding generic medication, stopping medication (deprescribing), and long-term outcomes are more likely to be addressed. Pharmacoepidemiological methods can also be extended to pharmacovigilance and to aid the development of new purposes for existing drugs. Drawbacks of observational pharmacoepidemiological studies come from the non-randomised nature of treatment selection, leading to confounding by indication. Potential methods for managing this drawback include active comparison groups, within-individual designs, and propensity scoring. Many of the more rigorous pharmacoepidemiology studies have been strengthened through multiple analytical approaches triangulated to improve confidence in inferred causal relationships. With developments in data resources and analytical techniques, it is encouraging that guidelines are beginning to include evidence from robust observational pharmacoepidemiological studies alongside randomised controlled trials. Collaboration between guideline writers and researchers involved in pharmacoepidemiology could help researchers to answer the questions that are important to policy makers and ensure that results are integrated into the evidence base. Further development of statistical and data science techniques, alongside public engagement and capacity building (data resources and researcher base), will be necessary to take full advantage of future opportunities.