Presentation (CTI)

OBJECTIVES: Evaluating the effectiveness of RMMs via Post-Authorization Safety Studies (PASS) is an integral part of pharmacovigilance, and an evolving field for which significant methodological gaps remain. The model proposed by regulators to attest success of RMMs includes process indicators to measure the degree of prescribers’ knowledge of key safety and clinical management information contained in the RMMs, and outcomes to measure the level of clinical actions achieved following the implementation of the RMMs. No previously published review has quantitatively analyzed the agreement between process and outcomes indicators results
METHODS: A literature search was conducted using the HMA-EMA Catalogues of real-world studies, Medline and Embase to identify reports and manuscripts assessing RMM effectiveness completed by December 2025
RESULTS: This review describes the results of 20 survey-only studies assessing process indicators (awareness, knowledge and behaviour), 12 studies using EMR data to assess drug utilization and outcome indicators (pre-post occurrence of adverse reactions, pregnancy, off-label use and medication errors), and 10 studies assessing both indicators (hybrid approach) as recommended by regulators. RMM effectiveness could not be inferred from process indicators in 25% of survey-only studies due to limitations inherent to these studies (selection bias, reliance of respondent recall, self-reported data), whereas it limited generalizability of results in most survey-only studies. Half of the hybrid studies showed discrepancies between process and outcome indicators; only one study correlated survey responses with clinical and safety outcomes
CONCLUSIONS: The inherent limitations of surveys limit the interpretation of results from risk minimization studies, but only a quarter of studies use drug utilization/outcomes evaluations to supplement/help strengthen the validity of survey results. Significant differences between process and outcome indicators results suggest that there may be differences between the actual implementation of the RMM and the attainment of its final objectives, which supports hybrid designs on a case-by-case basis