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Evaluating effectiveness of risk minimization through healthcare professional surveys: A review of post-authorization
Kexin Zhu1, William A Blumentals1, Froilan G Celzo2
1Epidemiology and Benefit-Risk, Sanofi, Morristown, New Jersey, USA.
Aims:
Risk minimization measures (RMMs) are key regulatory tools to ensure safe medicinal product use. Regulatory guidelines recommend pre-specifying the targets, or 'thresholds', for assessing RMM effectiveness. We aimed to review recent industry-sponsored post-authorization safety studies (PASS) that evaluated the effectiveness of RMMs, focusing on the use and justification of predefined thresholds.
Methods:
We searched the Heads of Medicines Agencies-European Medicines Agency Catalogue to identify PASS evaluating the effectiveness of RMM through knowledge-based surveys of healthcare professionals. A total of 17 eligible studies in 2022-2024 were included in the descriptive analysis.
Results:
All studies used cross-sectional designs, and 70.6% were Category 3 PASS. Only 29.4% evaluated RMM effectiveness within the guideline-recommended time frame (12-24 months) following the launch and regulatory implementation of RMMs. Nearly all studies (16 of 17, 94.1%) predefined effectiveness thresholds. Overall, thresholds ranged from 60% to 80%, with the majority (88.2%) being set at 80%. Thresholds for assessing awareness, knowledge, utilization and behaviours were set at 80%, 80%, 60%-80% and 75%-80%, respectively. However, only eight studies (47.1%) provided rationales for threshold determination. Among them, six studies justified thresholds based on success rates applied in comparable studies or regulatory guidance, while two relied on subjective criteria without specifying prior knowledge, experience or established scientific evidence.
Conclusion:
Our findings suggest that although most industry-sponsored PASS evaluating RMM effectiveness included predefined thresholds, these were often applied to the knowledge indicator rather than awareness, utilization and behaviour indicators. Notably, only about half of the studies provided clear justifications for threshold selection. Clear success threshold justifications are essential for interpreting RMM effectiveness.
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