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一个行业对欧洲同情使用的观点:呼吁改变
Philipp Schlatter1, Nina Heiss2, Pedro Franco3
1F. Hoffmann - La Roche Ltd., Grenzacherstrasse 124, CH-4070, Basel, Switzerland.
Therapeutic innovation & regulatory science
|December 15, 2025
概括
欧洲的同情使用 (CU) 途径显示出显著的差异,导致患者延迟. 协调国家法规对于及时获取未经授权的药物至关重要.
科学领域:
- 药物经济学和卫生政策
- 监管科学 监管科学
- 患者获得药品的权利
背景情况:
- 欧洲未经授权的药物的同情使用 (CU) 计划表现出显著的变化.
- 存在两个主要途径:欧盟监管的队列计划和成员国监管的个人患者请求 (IPR).
- 国家法律的差异会给寻求CU药物的患者带来运营挑战和潜在的延误.
研究的目的:
- 为了比较30个欧洲国家的同情使用计划的可用性和运营效率.
- 确定实施CU途径的关键挑战和变化.
- 为改善CU准入和协调提供数据驱动的建议.
主要方法:
- 来自30个欧洲国家的8934个同情使用请求的探索性后期分析.
- 在2020年1月至2023年4月期间,从四家主要制药公司 (默克KGaA,诺华,罗氏,桑诺菲) 收集了数据.
- 包括完整提交,公司批准,监管和出货日期的请求.
主要成果:
- 公司内部批准时间对于队列计划 (中位数为5天) 和IPR (中位数为4天) 类似.
- 从公司批准到药物运输的时间有显著差异:队列的中位数为5天,IPR的中位数为8天.
- 关键的挑战包括不同国家不一致的术语,范围,监管和运营要求.
结论:
- 欧洲不同的国家要求造成了运营障碍和同情使用实施的变化.
- 这些不一致性给参与患者获取药物的利益相关者带来了重大挑战.
- 显然需要在欧洲成员国之间更好地协调同情使用法规和流程.
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