Cost analysis of hemophilia treatment in a Brazilian public blood center

Abstract Background hemophilia is a rare coagulopathy, treated by replacing the missing blood clotting factor. Objective to assess the direct costs of hemophilia treatment from the perspective of the Unified Health System, highlighting the impact costs of new therapeutic modalities. Method partial economic assessment of the direct costs of hemophilia, in which were collected data from patient records from 2011 to 2015, at the Blood Center in the city of Juiz de Fora. Costs were assigned to consultations, exams, hospitalizations, and medications according to the price list of the National Health Surveillance Agency (in Portuguese ANVISA) and the Table of Procedures, and Medications. Results among 98 patients evaluated, 76 had hemophilia A, and 43.3% presented severe hemophilia. The number of consultations and the consumption of Clotting Factor Concentrates (CFCs) were higher in severe hemophilia. Hospitalizations were rare. Direct costs increased 286.8% from 2011 to 2015. The mean annual cost per patient was R$57,416.43, with no significant difference between hemophilia A and B. The expenditures for factor concentrates amounted to 99.46% of total costs. The actual impact cost was more than R$6,000,000.00. Conclusion the direct costs of hemophilia were high, mainly due to factor concentrates. There was an increase in costs with the incorporation of technologies, although there are some areas with potential inefficiencies.

摘要
## 背景
血友病是一种罕见的凝血功能障碍性疾病，临床治疗以补充缺失的凝血因子为核心手段。
## 目的
从统一卫生体系视角评估血友病治疗的直接成本，重点阐明新型治疗模式的成本影响。
## 方法
本研究采用血友病直接成本部分经济学评估方法，数据采集自巴西茹伊斯迪福拉市血液中心2011至2015年的患者病历。成本核算参照国家卫生监督局（National Health Surveillance Agency，葡萄牙语简称ANVISA）的价目表以及《医疗服务与药品目录》，涵盖门诊就诊、检验检查、住院治疗及药品相关成本。
## 结果
本次共评估98例患者，其中76例为A型血友病，43.3%的患者为重度血友病。重度血友病患者的门诊就诊次数与凝血因子浓缩剂（Clotting Factor Concentrates，CFCs）消耗量均显著更高。住院治疗情况较为罕见。2011至2015年间，直接成本上涨幅度达286.8%。患者年均直接治疗成本为57416.43雷亚尔（R$），A型与B型血友病患者的年均成本无显著差异。凝血因子浓缩剂支出占总直接成本的99.46%。研究期间实际总成本超过6000000.00雷亚尔。
## 结论
血友病治疗的直接成本高昂，其主要支出项为凝血因子浓缩剂。尽管部分领域存在潜在效率低下问题，但随着新技术的引入，治疗成本仍呈现上升趋势。