使用现实世界的数据来为医疗补助计划中细胞和基因疗法的基于价值的合同提供信息
Antal Zemplenyi1,2, Jim Leonard3, Michael J DiStefano4
1Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Anschutz Medical Campus, Aurora, CO, USA. antal.zemplenyi@cuanschutz.edu.
PharmacoEconomics
|November 22, 2023
概括
血友病A和B的基因治疗费用给医疗补助计划带来了预算挑战. 现实数据显示延长的回报期和成本不确定性,影响了基于价值的合同谈判.
科学领域:
- 卫生经济学 卫生经济学
- 药物经济学 药物经济学
- 血液学 血液学 血液学
背景情况:
- 血友病A和B的基因疗法具有潜力,但由于高额的前期成本和不确定的长期收益,这给医疗补助计划带来了预算挑战.
- 基于价值的合同是一个潜在的解决方案,但它们的设计因成本补偿的不确定性而复杂.
研究的目的:
- 从科罗拉多医疗补助的角度来看,从科罗拉多医疗补助的角度来看,确定valoctocogene roxaparvovec (血友病A) 和etranacogene dezaparvovec (血友病B) 的实际成本补偿.
- 在科罗拉多州医疗补助计划中定义这些基因疗法的回报期和相关的不确定性.
主要方法:
- 使用2018-2022年科罗拉多州医疗补助数据进行成本分析,以评估标准护理成本.
- 成本模拟模型估计了符合条件的患者 (18岁以上的血友病A或B患者) 采用和不采用基因治疗的医疗补助费用.
- 标准治疗包括血友病A的因子VIII/埃米齐祖马布和血友病B的因子IX;模拟患者接受基因疗法.
主要成果:
- 年度标准护理费用为426,000美元 (血友病A) 和546,000美元 (血友病B).
- 现实世界的平衡时间为8年 (血友病A) 和6年 (血友病B),比公布的经济评估的5年更长.
- 成本的变化导致48% (血友病A) 和59% (血友病B) 的几率在10年内实现平衡;资格标准显著影响了平衡时间.
结论:
- 现实世界的数据显示,基因疗法成本存在显著的不确定性和延长的回报期.
- 医疗补助计划可以利用这些数据来谈判基于价值的合同,管理预算,分享风险,并改善患者获得创新治疗的机会.
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