现实世界证据的可转移性,以支持低收入欧洲国家的HTA建议
Zoltán Kaló1,2,3, Guenka Petrova4, Bertalan Németh3
1Center for Health Technology Assessment Semmelweis University Budapest Hungary.
Health science reports
|January 29, 2026
概括
低收入的欧洲国家可以通过使用目标试验仿真 (TTE) 转移现实世界的证据 (RWE) 来改善罕见病治疗. 这种方法优化了尽管资源有限,骨髓质疏松综合征 (MDS) 的治疗建议.
科学领域:
- 卫生经济学和结果研究研究.
- 治疗罕见疾病 治疗罕见疾病
- 医疗技术评估 (HTA) 是指对健康技术进行评估.
背景情况:
- 低收入欧洲国家 (LIECs) 与高收入国家 (HIECs) 相比,面临着高成本罕见病治疗的财政限制.
- 骨髓质疏松症候群 (MDS) 作为一个案例研究,探索证据的可转移性.
- 该HTx项目研究了将目标试验仿真 (TTE) 整合到常规卫生技术评估 (HTA) 工作中.
研究的目的:
- 探索由TTE产生的现实世界的证据 (RWE) 从HIECs的可转移性,以支持LIECs对罕见疾病的治疗建议.
- 确定挑战,并提出解决方案,以将TTE产生的RWE转移到LIECs.
- 增强LIECs在罕见疾病治疗决策方面的HTA能力.
主要方法:
- 利用目标试验模拟 (TTE) 在高收入欧洲国家 (HIECs) 生成现实世界的证据 (RWE).
- 与HTA联盟合作伙伴和外部专家举行研讨会,以确定将TTE结果转移到低收入欧洲国家 (LIEC) 的挑战.
- 专注于骨髓质疏松症候群 (MDS) 病例研究.
主要成果:
- 缺乏本地真实世界的数据,不同的患者途径,比较技术和有限的专业知识/资源是LIECs中TTE的主要障碍.
- 通过TTE转移RWE比标准HTA方法提供了改进,特别是对于孤儿药物的联合临床评估.
- TTE使LIEC能够评估特定患者子组的高成本技术,但需要先进方法的培训.
结论:
- 这项研究是首次研究在HTA能力有限的国家使用TTE用于罕见疾病的RWE转移.
- 转移TTE产生的RWE对于LIEC来说是可行的和有益的,尽管存在已知的挑战.
- 应对挑战需要培训HTA专业人员和潜在的协作方法,如联合临床评估.
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