在七个欧洲国家,通过同情使用计划和命名患者基础获得抗癌和孤儿药物
N Rosenberg1, H C Post2, T Schutte3
1Medicine for Society, Platform at Amsterdam UMC - University of Amsterdam, Amsterdam, The Netherlands; Department of Endocrinology and Metabolism, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, Expertise Centre for Inborn Errors of Metabolism, MetabERN, University of Amsterdam, Amsterdam, The Netherlands.
欧洲药品早期获取计划在同情使用计划 (CUP) 和命名患者基础 (NPB) 获取和报销方面存在显著差异. 尽量减少这些差异对于公平的患者获得重要治疗来说至关重要.
科学领域:
- 卫生政策分析 卫生政策分析
- 药品获取研究 药品获取研究
- 监管科学是一种监管科学.
背景情况:
- 在欧盟,抗癌药物和孤儿药物通常使用代用终点和有限的数据获得授权.
- 由于未满足的医疗需求,诸如同情使用计划 (CUP) 和命名患者基础 (NPB) 等早期获取计划正在获得吸引力.
- 有限的临床证据和高成本可能会阻碍医疗技术评估,报销和患者获取.
研究的目的:
- 评估七个高收入欧洲国家的同情使用计划 (CUP) 和命名患者基础 (NPB).
- 分析这些早期访问计划的监管特征,包括退款.
- 评估抗癌和孤儿药物CUP的可用性,持续时间和地理分布.
主要方法:
- 对比利时,法国,德国,荷兰,挪威,瑞士和英国的CUP和NPB的政策分析.
- 从国家卫生当局资源收集数据和直接咨询.
- 深入检查2021-2022年授权药物的CUP,重点关注关键的运营方面.
主要成果:
- 观察到国家法规的显著变化和CUP和NPB的不一致的报销.
- 在研究的国家中,命名患者基础 (NPB) 的补偿经常不受监管.
- 在欧盟营销许可之前和之后,在同情使用计划 (CUP) 的可用性和持续时间方面发现了差异,在各国发现了36个CUP.
结论:
- 建议包括尽量减少CUP和NPB之间的差异,以确保欧洲各地患者获得公平的医疗服务.
- 减少这些差异至关重要,以防止患者在国外寻求药物或因未经授权的治疗而产生个人成本.
- 协调早期准入政策将改善那些有高未满足医疗需求的人的患者结果.
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