先进疗法药品:意大利的可用性,获取和支出
Pia Rivetti di Val Cervo1, Eva Alessi2, Marilena Lastella2
1HTA and Pharmaceutical Economics Department, Italian Medicines Agency, Via del Tritone 181, 00187, Rome, Italy. p.rivettidivalcervo@aifa.gov.it.
概括
在意大利,先进治疗药物 (ATMP) 越来越多,但患者的获取时间仍然很长. 尽管成本高昂,但ATMP支出目前仍处于控制状态,但预计将增加.
科学领域:
- 生物医学研究生物医学研究
- 卫生经济学 卫生经济学
- 制药政策 制药政策
背景情况:
- 先进治疗药物 (ATMP) 在有效性,安全性,成本和管理方面存在独特的挑战.
- 这些复杂性影响了欧洲授权,国家补偿和患者与传统药物相比获得治疗.
研究的目的:
- 评估2016年至2023年意大利ATMP的可用性,患者获取和支出.
- 了解这些创新疗法的现实世界利用和可访问性障碍.
主要方法:
- 对ATMP监管评估时间表的现实数据的分析.
- 评估患者首次入院的时间和国家卫生系统的支出.
- 包含有关患者流动性和专业医疗机构可用性的数据.
主要成果:
- 在意大利的18个授权的ATMP中,有9个被退还,只有6个被使用,耗资约3亿欧元 (2016-2023年),主要用于CAR-T疗法.
- 在经过漫长的监管评估后,患者获得医疗服务的平均时间是退款决定后的340.6天.
- 107个设施被授权用于ATMP管理,几乎所有意大利地区都有至少一个中心在2023年之前进行治疗.
结论:
- 自2016年以来,意大利的ATMP可用性和全国覆盖率大幅增加.
- 尽管每次治疗成本高,但目前的ATMP支出相对有限,但预计未来会有增长.
- 在ATMP可访问性方面取得的进展是显而易见的,但访问时间表的优化仍然至关重要.
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