关于ATMP首次联合临床评估的ARM的观点:未来道路上的挑战和机遇
Paolo Morgese1, Stephen Majors1, Dilip Patel2
1Alliance for Regenerative Medicine (ARM), B-1040 Brussels, Belgium; smajors@alliancerm.org.
Journal of market access & health policy
|April 25, 2025
概括
先进疗法药物 (ATMP) 为罕见疾病提供治疗方法,但挑战了传统的健康技术评估 (HTA). 欧盟的联合临床评估 (JCA) 旨在统一ATMP的HTA,改善患者的获取.
科学领域:
- 生物医学科学 生物医学科学
- 卫生经济学 卫生经济学
- 监管科学是一种监管科学.
背景情况:
- 先进治疗药物 (ATMPs) 代表了医学的范式转变,针对潜在的治疗疾病的根本原因.
- 这些复杂的,往往是一次性疗法为严重的遗传和罕见疾病,包括罕见癌症提供了显著的好处.
- 传统的医疗技术评估 (HTA) 框架与ATMP的独特特征进行斗争,例如不确定的长期耐用性.
研究的目的:
- 在现有的HTA框架内概述ATMP所面临的挑战.
- 根据欧盟的HTA法规,提供关于联合临床评估 (JCA) 实施的视角.
- 探索"适合目的"的JCA如何促进在欧洲各地获得ATMP的机会.
主要方法:
- 分析当前针对ATMPs的特定HTA挑战.
- 审查欧盟卫生技术评估 (HTA) 条例及其联合临床评估 (JCA) 机制.
- 讨论对ATMPs的JCA实施的潜在好处和担忧.
主要成果:
- ATMPs具有独特的复杂性 (例如,一次性管理,不确定的长期数据),这使传统的HTA受到压力.
- 欧盟的JCA提供了一个统一的框架,以巩固国家HTA流程,解决ATMP的特殊性.
- 通过JCA进行的欧盟范围的合作正在促进更紧密的HTA生态系统,尽管存在持续的方法问题.
结论:
- 一个"适合目的"的JCA对于释放ATMP对欧洲患者的好处至关重要.
- 联合评估机构有潜力简化决策,并将欧洲定位为创新的HTA的领导者.
- 持续的合作和HTA方法的适应对于整合这些变革性疗法至关重要.
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