优化患者获得孤儿药品的机会:中东欧的经验教训
Tomasz Kluszczynski1,2, Bertalan Nemeth3, Magdalena Władysiuk4
1ACESO Healthcare Consulting, 03-719 Warsaw, Poland.
Journal of market access & health policy
|June 26, 2025
概括
中欧和东欧 (CEE) 地区患者获得孤儿药品 (OMP) 的机会仍然不够理想,延误很大,报销率较低. 主要障碍包括不充分的医疗技术评估 (HTA) 框架和融资挑战.
科学领域:
- 卫生经济学 卫生经济学
- 制药政策 制药政策
- 罕见疾病研究 罕见疾病研究
背景情况:
- 孤儿药品对于治疗罕见疾病至关重要.
- 与西欧相比,中欧和东欧 (CEE) 国家在OMP获取方面面临着独特的挑战.
- 中东欧国家的医疗保健系统经历了快速的转型,影响了OMP的可访问性.
研究的目的:
- 确定障碍物和促进者,以优化患者在中东欧国家获得OMP的机会.
- 为可持续和公平的OMP接入策略提供见解.
- 为了比较七个中东欧国家的OMP访问.
主要方法:
- 混合方法的方法结合了系统的文献评论和深入的采访.
- 利益相关者参与,包括政策制定者,支付者,行业和患者团体.
- 分析中欧和中东欧地区的具体举措和医疗技术评估 (HTA) 框架.
主要成果:
- 在大多数中东欧国家观察到低于最佳的OMP接入,其特点是延迟和较低的退款率.
- 斯洛文尼亚和捷克在OMP访问方面出现了显著的例外.
- 确定了关键障碍:意识有限,HTA不足,资金不足,患者参与度分散.
结论:
- 解决HTA,融资和患者参与方面的障碍对于改善CEE地区的OMP接入至关重要.
- 实施罕见病政策和包括患者的决策可以提高OMP的可访问性.
- 可持续和公平的OMP接入需要为中东欧地区量身定制的战略.
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