优化用于监管决策的患者登记系统——来自HMA/EMA多利益相关方研讨会的关键经验教训

Optimizing Patient Registries for Regulatory Decision Making - Key Learnings From an HMA/EMA Multistakeholder Workshop.

作者信息

Plueschke Kelly, Jonker Carla, Kankanen Hanna, Vetter Thorsten, Sepodes Bruno, Naehrlich Lutz, Hillert Jan, Crane Gracy, Straus Sabine, Foggi Paolo, Martin Simona, Kyriakopoulou Christina, Mol Peter, Day Francesca, Breen Kieran, Bennett Neil, de Lemus Belmonte Mencia, Bennett Simon, Verpillat Patrice, Roes Kit C B, Cochino Ana, Schaefer Franz, Hernández-Rivas Jesús María, McGettigan Patricia, Arlett Peter

机构信息

European Medicines Agency, Amsterdam, The Netherlands.

Medicines Evaluation Board (MEB), Utrecht, The Netherlands.

出版信息

Clin Pharmacol Ther. 2025 Sep;118(3):551-560. doi: 10.1002/cpt.3733. Epub 2025 Jun 2.

Abstract

The Joint Heads of Medicines Agencies and European Medicines Agency's (HMA/EMA) big data initiative paves the way for better integration of real-world data, including data from patient registries, into regulatory decisions on medicines. This article focuses on the outcome of a two-day multistakeholder workshop organized by EMA in 2024, which explored ways to optimize the EMA qualification procedure for patient registries, and to establish the value and enable the use of these data across the full spectrum of research questions. Key recommendations include the need to clarify the aim, scope, and added value of the qualification of registries, coupled with a review of the procedural steps to ensure the process is fit-for-purpose to evaluate the use of registries in specific regulatory contexts. Further recommendations focused on strengthening interactions between stakeholders, as well as providing them with enhanced support by increasing awareness of publicly available tools that could leverage the potential of registry data, together with existing guidance. The European Medicines Regulatory Network is now working together with all relevant stakeholders, including the EMA scientific committees and working parties, the Joint HMA/EMA Network Data Steering Group and existing focus groups with external partners, to implement concrete actions that will address these recommendations. Among others, the update of existing guidance, the development of templates and Questions & Answers documents, and the design of appropriate communication and stakeholder engagement plans will aid in achieving the common goal of making optimal use of patient registry data to support public health in the European Union.

摘要

药品管理机构联合负责人及欧洲药品管理局(HMA/EMA)的大数据计划为更好地整合真实世界数据(包括来自患者登记处的数据)以用于药品监管决策铺平了道路。本文重点关注EMA于2024年组织的为期两天的多利益相关方研讨会的成果,该研讨会探讨了优化患者登记处EMA资格认定程序的方法,以及确定这些数据的价值并使其能够用于解决全范围研究问题的方法。关键建议包括需要明确登记处资格认定的目的、范围和附加值,同时审查程序步骤以确保该过程适合评估登记处在特定监管背景下的使用情况。进一步的建议侧重于加强利益相关方之间的互动,并通过提高对可利用登记处数据潜力的公开可用工具以及现有指南的认识,为他们提供更多支持。欧洲药品监管网络目前正与所有相关利益相关方合作,包括EMA科学委员会和工作组、HMA/EMA联合网络数据指导小组以及与外部合作伙伴的现有焦点小组,以实施将解决这些建议的具体行动。其中,更新现有指南、制定模板和问答文件以及设计适当的沟通和利益相关方参与计划将有助于实现最佳利用患者登记处数据以支持欧盟公共卫生这一共同目标。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3c46/12355018/49ed2cee3bdb/CPT-118-551-g001.jpg

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