利用真实世界数据，对美国接受聚乙二醇重组凝血因子VIII（FVIII-PEG）或抗血友病因子（重组）FC融合蛋白（rFVIIIFc）治疗的甲型血友病患者的医疗费用进行研究。
Healthcare costs among hemophilia A patients in the United States treated with rurioctocog alfa pegol (FVIII-PEG) or antihemophilic factor (recombinant), FC fusion protein (rFVIIIFc) using real-world data.
作者信息
Farahbakhshian Sepehr, Fan Qi, Schultz Bob G, Princic Nicole, Park Julie, Bullano Michael
机构信息
Takeda Pharmaceuticals USA Inc, Lexington, Massachusetts, USA.
Merative, Ann Arbor, Michigan, USA.
出版信息
J Med Econ. 2023 Jan-Dec;26(1):1278-1286. doi: 10.1080/13696998.2023.2266317. Epub 2023 Oct 25.
AIMS
To compare healthcare costs in patients with non-inhibitor hemophilia A treated with Rurioctocog Alfa Pegol (FVIII-PEG) versus Antihemophilic Factor (Recombinant), FC Fusion Protein (rFVIIIFc).
MATERIALS AND METHODS
Administrative claims data from the Merative MarketScan Commercial (Commerical) and Medicaid (Medicaid) databases were used for these analyses. Males with non-inhibitor hemophilia A treated with FVIII-PEG or rFVIIIFc from 1 January 2016 to 31 March 2021 were identified (earliest treatment = index). Patients were required to have continuous database enrollment for six months before and after the index date. Follow-up was variable in length until disenrollment or study end. All-cause and hemophilia-related healthcare costs were reported per-patient per month [PPPM] and the average weekly dose during follow-up was compared between treatment groups. Generalized linear regressions were used to estimate multivariable-adjusted differences in total costs and weekly dosage in the two treatment groups.
RESULTS
A total of 131 FVIII-PEG (66 Commercial; 65 Medicaid) and 204 rFVIIIFc (111 Commercial; 93 Medicaid) patients were eligible. Mean age was 20.5 and 24.4 for FVIII-PEG and rFVIIIFc in Commercial and 14.9 and 17.5 for FVIII-PEG and rFVIIIFc in Medicaid. PPPM mean (standard deviations [SD]) total healthcare costs in Commercially insured patients were $35,868 [$21,717] for FVIII-PEG vs $40,424 [$25,882] for rFVIIIFc. Costs in Medicaid were $27,495 [$23,243] for FVIII-PEG vs $30,237 [$28,430] for rFVIIIFc. After adjusting for baseline characteristics, the costs for rFVIIIFc (vs FVIII-PEG) were higher by $5,215 in Commercial and $3,895 in Medicaid, but the differences were not statistically significant ( > 0.05). Similar findings were observed for hemophilia-specific healthcare costs. The adjusted mean weekly dose was 6,047 vs 4,892 IU, = 0.21 for FVIII-PEG vs rFVIIIFc in Commercial and 5,549 vs 7,228 IU, = 0.07 for FVIII-PEG vs rFVIIIFc in Medicaid.
CONCLUSIONS
Healthcare costs and treatment dosing were similar ( > 0.05) for non-inhibitor hemophilia A patients treated with FVIII-PEG and rFVIIIFc.
目的
比较接受鲁瑞凝血因子α聚乙二醇（FVIII-PEG）治疗的非抑制物型A型血友病患者与重组抗血友病因子FC融合蛋白（rFVIIIFc）的医疗费用。
材料与方法
这些分析使用了来自默克市场扫描商业数据库（商业数据库）和医疗补助数据库（医疗补助数据库）的管理索赔数据。确定了2016年1月1日至2021年3月31日期间接受FVIII-PEG或rFVIIIFc治疗的非抑制物型A型血友病男性患者（最早治疗时间=索引日期）。患者在索引日期前后需要连续6个月在数据库中登记。随访时间长短不一，直至退出研究或研究结束。报告每位患者每月的全因医疗费用和与血友病相关的医疗费用[PPPM]，并比较治疗组之间随访期间的平均每周剂量。使用广义线性回归估计两个治疗组在总成本和每周剂量方面的多变量调整差异。
结果
共有131例FVIII-PEG患者（66例商业保险；65例医疗补助）和204例rFVIIIFc患者（111例商业保险；93例医疗补助）符合条件。商业保险患者中，FVIII-PEG组和rFVIIIFc组的平均年龄分别为20.5岁和24.4岁；医疗补助患者中，FVIII-PEG组和rFVIIIFc组的平均年龄分别为14.9岁和17.5岁。商业保险患者中，FVIII-PEG的PPPM平均（标准差[SD]）总医疗费用为35,868美元[21,717美元]，rFVIIIFc为40,424美元[25,882美元]。医疗补助患者中，FVIII-PEG为27,495美元[23,243美元]，rFVIIIFc为30,237美元[28,430美元]。在调整基线特征后，rFVIIIFc（相对于FVIII-PEG）的费用在商业保险中高出5,215美元，在医疗补助中高出3,895美元，但差异无统计学意义（>0.05）。在血友病特异性医疗费用方面也观察到类似结果。商业保险中，FVIII-PEG与rFVIIIFc的调整后平均每周剂量分别为6,047 IU和4,892 IU，P=0.21；医疗补助中，FVIII-PEG与rFVIIIFc的调整后平均每周剂量分别为5,549 IU和7,228 IU，P=0.07。
结论
接受FVIII-PEG和rFVIIIFc治疗的非抑制物型A型血友病患者的医疗费用和治疗剂量相似（>0.05）。
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