Formerly Pfizer Limited, Tadworth, Surrey, UK.
HCD Economics, Mere House, Brook St, Knutsford, UK.
BMC Health Serv Res. 2024 Nov 20;24(1):1437. doi: 10.1186/s12913-024-11850-y.
Few studies have evaluated direct medical or societal costs of haemophilia in the United Kingdom (UK), and how patient characteristics impact future costs is uncertain. Cost predictors were identified and examined using cross-sectional data from the CHESS I and II studies.
Patient- and physician-reported outcomes were analysed for UK adult males aged ≤ 65, with haemophilia A or B and no recent clinical trial participation. Demographics, haemophilia type and severity, inhibitors, annual bleed rate (ABR), problem joints (PJs), treatment type, and comorbidities, were utilised in regression analyses. Health-related quality of life was assessed using EQ-5D. Generalised linear models estimated expected non-drug haemophilia-related direct medical costs (DMC) and societal costs (non-drug DMC, direct non-medical and indirect costs). Average marginal effects (AMEs) determined predictors of cost.
Costs for 378 patients were analysed. Mean age was 33 years and 79% (299) had haemophilia A. Mean annual per-patient DMC were £165,001 (including factor treatment costs) and £4,091 when excluding factor replacement treatment costs (non-drug DMC). Mean annual per-patient non-treatment societal costs were £11,550 (standard deviation £20,171) among those with data available (n = 51). Number of PJs, ABR, and treatment regimen were significant determinants of haemophilia-related non-drug DMC (all P < 0.001). Non-drug DMC increased as ABR increased (AMEs were £2,018 for ABR 1-5, £3,101 for ABR 6-10 and £5,785 for ABR ≥ 11, vs. ABR 0) and by £1,869 per additional PJ. No significant predictors of non-drug haemophilia-related societal costs were identified. Mean EQ-5D score was 0.66, with lower scores observed for people with haemophilia B (0.48) compared with haemophilia A (0.71) and with increasing haemophilia severity.
UK direct medical and societal costs of haemophilia are substantial. Non-drug DMC were particularly associated with ABR and number of PJs. These findings may be useful for real-world evaluations of the economic burden of haemophilia in the UK.
在英国（UK），很少有研究评估血友病的直接医疗或社会成本，也不确定患者特征如何影响未来的成本。本研究使用 CHESS I 和 II 研究的横断面数据，确定并检验了成本预测因素。
对年龄≤65 岁的英国成年男性血友病 A 或 B 患者，且无近期临床试验参与的患者进行了患者和医生报告的结局分析。回归分析中使用了人口统计学、血友病类型和严重程度、抑制剂、年出血率（ABR）、问题关节（PJ）、治疗类型和合并症等因素。使用 EQ-5D 评估健康相关生活质量。广义线性模型估计了非药物相关的血友病直接医疗成本（DMC）和社会成本（非药物 DMC、直接非医疗和间接成本）。平均边际效应（AME）确定了成本的预测因素。
对 378 名患者的费用进行了分析。平均年龄为 33 岁，79%（299 人）患有血友病 A。每年每位患者的 DMC 平均为 165,001 英镑（包括因子治疗费用），不包括因子替代治疗费用时为 4,091 英镑（非药物 DMC）。在有数据可用的 51 名患者中，每年每位患者的非治疗性社会成本平均为 11,550 英镑（标准差 20,171 英镑）。PJ 数量、ABR 和治疗方案是非药物相关 DMC 的显著决定因素（均 P<0.001）。随着 ABR 的增加，非药物 DMC 增加（ABR 为 1-5 的 AME 为 2,018 英镑，ABR 为 6-10 的 AME 为 3,101 英镑，ABR≥11 的 AME 为 5,785 英镑，而 ABR 为 0 的 AME 为 2,018 英镑），PJ 每增加一个，非药物相关 DMC 增加 1,869 英镑。未发现非药物相关血友病社会成本的显著预测因素。平均 EQ-5D 评分为 0.66，其中乙型血友病（0.48）的评分低于甲型血友病（0.71），且随着血友病严重程度的增加而降低。
英国血友病的直接医疗和社会成本很高。非药物 DMC 尤其与 ABR 和 PJ 数量相关。这些发现可能有助于在英国对血友病的经济负担进行真实世界的评估。
