Nested Case-Control Studies
Nested case-control studies are useful for investigating the prevalence of pre-defined risk factors for an event of interest occurring in patients exposed to a particular drug. In the nested case-control study design, cases of an event occurring in a defined cohort are identified and, for each, a specified number of matched controls are selected from those in the cohort who did not have the event by the time of occurrence in the case.
For many research questions, the nested case-control design potentially offers impressive reductions in costs and efforts of data collection and analysis compared with the full cohort approach, with relatively minor loss in statistical efficiency. This could be particularly advantageous for studies investigating pharmacogenetic susceptibility to adverse drug reactions. The nested case-control study within PEM, subject to appropriate design and statistical projections, offers the opportunity to investigate such susceptibilities and contribute to a greater understanding of the mechanisms behind adverse drug reactions.