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估计的费用为贝雷马根Geperpavec对于 Dystrophic Epidermolysis Bullosa 的估计支出
Adam J N Raymakers1,2, Aaron S Kesselheim1,2, Arash Mostaghimi2,3
1Program on Regulation, Therapeutics, and Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts.
JAMA dermatology
|January 31, 2024
概括
第一个局部基因疗法,贝雷马基因基因帕维克 (B-VEC),用于表皮溶解,预计将使美国纳税人花费数亿美元. 这项经济评估估计了自体衰退和主导DEB的显著B-VEC支出.
科学领域:
- 药物经济学 药物经济学
- 基因治疗 基因治疗
- 皮肤病学 皮肤病学
背景情况:
- 新的基因疗法为具有有限选择的罕见疾病提供了希望.
- 贝雷玛格尼 (Beremagene geperpavec) (B-VEC) 是第一个获得FDA批准的局部基因疗法,用于缩性表皮溶解牛 (DEB).
- 尽管对后者的数据有限,但FDA的批准包括了自体逆向和主导DEB.
研究的目的:
- 为了估计预计的美国医疗保健支出对B-VEC治疗.
- 从所有付款人的角度评估B-VEC对两种DEB类型的经济影响.
主要方法:
- 经济评价使用来自国家皮肤溶解牛注册表的数据.
- 估计美国符合B-VEC治疗条件的患者人数.
- 基于制造商报告的每年每名患者30万美元的成本成本分析.
主要成果:
- 据估计,在批准后的第一年,有894名美国患者有资格接受B-VEC.
- 预计第一年支出为2.68亿美元,在三年内增加到8.05亿美元.
- 估计的终身治疗费用为1500万美元的自体逆向DEB和1700万美元的自体主导DEB每患者.
结论:
- 对于B-VEC而言,FDA的广泛指示对医疗保健支付人有重大财务影响.
- 这份经济评估强调了与用于罕见遗传疾病的新型基因疗法相关的大量成本.
- 进一步的药物经济分析对于管理B-VEC等先进疗法的采用至关重要.
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