European Medicines Agency, Domenico Scarlattilaan 6, 1083 HS, Amsterdam, The Netherlands.
Division of Population Health Sciences, University of Dundee, Dundee, UK.
Drug Saf. 2021 Feb;44(2):193-209. doi: 10.1007/s40264-020-01005-3. Epub 2020 Oct 30.
Understanding the impact of regulatory actions for medicines and enablers/barriers for positive health outcomes is fundamental to effective risk minimisation measures (RMM). Therefore, the Impact Strategy of the European Union (EU) Pharmacovigilance Risk Assessment Committee (PRAC) includes engagement with patient communities and healthcare professional (HCP) bodies regarding RMM. However, there is uncertainty on how best to obtain stakeholder input.
The objectives of this study were to (1) analyse stakeholder input at a public hearing and dedicated meeting for the 2017-18 EU procedure on valproate teratogenicity and (2) draw proposals for enhancing PRAC engagement.
For the content analysis, the novel 'Analysing Stakeholder Safety Engagement Tool' (ASSET) was developed with 21 themes in six domains (appropriateness, access, audience, compatibility, integrability, time), based on implementation theories.
Stakeholders provided a wide range of RMM proposals, some beyond the regulatory remit. Patients and most HCPs converged remarkably, but there was some divergence among HCPs on the informed choice objective, the therapeutic place of valproate, the RMM appropriateness, and RMM delivery to HCPs and patients. Ethical aspects emerged as relevant for regulatory decision making, and crucial input gaps were identified from an RMM implementation perspective. Nine pilotable proposals for PRAC were made regarding: (A) Agreeing on appropriate RMM with stakeholders and catalysing healthcare leadership for implementation; (B) Building-up stakeholder input on all elements critical to RMM implementation guided by the ASSET; and (C) Collaborating with all stakeholders for monitoring implementation and evaluating RMM.
New implementation theory-based approaches are promising for enhancing the valuable dialogue between regulators, patients and HCPs and achieving patient safety.
EUPAS35947.
了解药品监管行动对积极健康结果的影响，以及促进/阻碍健康结果的因素，对于实施有效的风险最小化措施（RMM）至关重要。因此，欧盟（EU）药品监管风险评估委员会（PRAC）的影响策略包括就 RMM 与患者群体和医疗保健专业人员（HCP）进行接触。然而，对于如何最好地获取利益相关者的意见，仍存在不确定性。
本研究的目的是：（1）分析 2017-18 年欧盟丙戊酸致畸性程序公共听证会和专门会议上的利益相关者意见；（2）提出增强 PRAC 参与的建议。
为了进行内容分析，基于实施理论，开发了新颖的“分析利益相关者安全参与工具”（ASSET），其中包含 6 个领域的 21 个主题（适当性、可及性、受众、兼容性、可集成性、时间）。
利益相关者提供了广泛的 RMM 建议，其中一些超出了监管范围。患者和大多数 HCP 的意见非常一致，但 HCP 之间在知情选择目标、丙戊酸的治疗地位、RMM 的适当性以及向 HCP 和患者提供 RMM 等方面存在一些分歧。从 RMM 实施的角度来看，伦理方面被认为与监管决策相关，并且确定了关键的意见差距。就 PRAC 提出了 9 个可试行的建议：（A）与利益相关者就适当的 RMM 达成一致，并为实施提供医疗保健领导力；（B）在 ASSET 的指导下，建立利益相关者对所有 RMM 实施关键要素的投入；（C）与所有利益相关者合作，监测实施情况并评估 RMM。
基于新实施理论的方法有望加强监管机构、患者和 HCP 之间的宝贵对话，并实现患者安全。
欧盟 PAS 注册号：EUPAS35947。
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