Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, Utrecht, The Netherlands.
European Medicines Agency, Amsterdam, The Netherlands.
Clin Pharmacol Ther. 2023 Dec;114(6):1285-1292. doi: 10.1002/cpt.3034. Epub 2023 Oct 16.
The European Medicines Agency (EMA) supervises medicines' safe and effective use throughout the product's life cycle by, for example, monitoring the implementation of risk minimization measures (RMMs). Limited information is available on factors associated with effectiveness of RMMs. This study reviews post-authorization 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 January 1, 2016, and December 31, 2021, were compiled from non-public EMA databases and PASS characteristics were extracted. Of the 93 PASS included, 62.4% aimed to measure healthcare professionals' awareness, knowledge, and behavior regarding RMMs. There were 67.7% of the 93 PASS that used primary data, 24.7% used secondary data sources, and 7.5% used both. A cross-sectional study design was most frequently applied (77.4%), followed by a cohort study design (29.0%). Nearly 40% of the included PASS did not render a conclusion on RMM effectiveness. Of the 60% that did render a conclusion, 82.1% 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, 4 out of 10 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.
欧洲药品管理局（EMA）通过例如监测风险最小化措施（RMM）的实施情况，监督药品在整个产品生命周期内的安全和有效使用。关于与 RMM 有效性相关的因素，相关信息有限。本研究回顾了 2016 年至 2021 年间，药物警戒风险评估委员会（PRAC）评估 RMM 有效性的事后安全性研究（PASS）。从非公开 EMA 数据库中编译了 PRAC 于 2016 年 1 月 1 日至 2021 年 12 月 31 日之间完成的事后安全性研究评估报告，并提取了 PASS 特征。在 93 项纳入的 PASS 中，有 62.4%旨在衡量医疗保健专业人员对 RMM 的意识、知识和行为。93 项 PASS 中有 67.7%使用了原始数据，24.7%使用了二级数据源，7.5%同时使用了两者。最常应用的是横断面研究设计（77.4%），其次是队列研究设计（29.0%）。近 40%的纳入 PASS 未对 RMM 有效性做出结论。在做出结论的 60%中，82.1%被认为是有效的。当按结局（即有效的 RMM、无效的 RMM 和对 RMM 有效性没有结论）进行分层时，仅发现特征上存在微小差异。总之，在评估 RMM 影响的 10 项 PASS 中，有 4 项未对 RMM 有效性做出结论。在 PASS 特征与其结局之间未发现明显差异，表明需要进一步研究，以更好地理解 PASS 结果不确定的根本原因。
