Review of studies evaluating effectiveness of risk minimisation measures assessed by the European Medicines Agency between 2016 and 2021.

The European Medicines Agency supervises medicines' safe and effective use throughout the product's life cycle by, for example, monitoring the implementation of risk minimisation measures (RMMs). Limited information is available on factors associated with effectiveness of RMMs. This study reviews post-authorisation safety studies (PASS) evaluating the effectiveness of RMMs assessed by the Pharmacovigilance Risk Assessment Committee (PRAC) between 2016 and 2021. PASS assessment reports finalized by PRAC between 1 st of January 2016 and 31 st of December 2021 were compiled from non-public EMA databases and PASS characteristics were extracted. Of the 94 PASS included, 64.9% aimed to measure healthcare professionals' awareness, knowledge, and behaviour regarding RMMs. 68.1% of the 94 PASS used primary data, 23.4% used secondary data sources, and 8.5% used both. A cross-sectional study design was most frequently applied (76.6%), followed by a cohort study design (27.7%). Nearly 40% of the included PASS did not render a conclusion on RMM effectiveness. Of the 60% that did render a conclusion, 80% were deemed effective. Only minor differences in characteristics were found when stratified by outcome (i.e., effective RMM, ineffective RMM, and no conclusion on RMM effectiveness). To conclude, four out of ten PASS assessing impact of RMMs did not render a conclusion on RMM effectiveness. No clear differences in PASS characteristics were found in relation to their outcomes, indicating that additional research is needed to understand better the underlying reasons for PASS being inconclusive.