Pulmonology Unit, Hospital Geral, Centro Hospitalar e Universitário de Coimbra, Portugal.
Immunoallergology Department, Hospital de Santa Maria, Centro Hospitalar Lisboa Norte (CHLN), Lisbon, Portugal.
Pulmonology. 2021 Mar-Apr;27(2):124-133. doi: 10.1016/j.pulmoe.2020.03.001. Epub 2020 Apr 1.
To estimate the cost-effectiveness of omalizumab compared with standard of care in the treatment and control of severe persistent asthma, using the outcomes from the Portuguese subpopulation of the eXpeRience registry.
This was a pragmatic cost-effectiveness analysis based on real world data from the eXpeRience registry which recruited 62 patients with uncontrolled persistent allergic asthma from 20 participating centers in Portugal. Response to omalizumab treatment was measured prospectively up to 24 months by the physician's Global Evaluation of Treatment Effectiveness (GETE). Retrospective data on patients' clinical symptoms, asthma control, lung function, exacerbations, and healthcare utilization were available for up to 12 months before omalizumab initiation and served as the standard of care comparator. The number of exacerbations (severe and non-severe), the number of clinical episodes, the number of days absent from work and/or school, and GETE response to therapy were considered as effectiveness outcomes. Following a societal perspective, as cost indicators, both direct and indirect costs were considered. Direct costs relate to the cost of omalizumab, standard of care and clinical episodes (emergency room visits, hospitalizations, and unscheduled doctor visits). Indirect costs relate to the societal cost of work absenteeism. Unit costs for clinical episodes and drugs were taken from official sources within the Portuguese Health Authority. A univariate sensitivity analysis was performed.
A rate of 1.5 exacerbations per patient-year was estimated following omalizumab treatment compared with 8.2 exacerbations per patient-year prior to omalizumab initiation, implying an 82.1% reduction in the incidence of exacerbations following omalizumab treatment relative to standard of care alone. A 54.1% reduction in GETE score was also observed in favor of omalizumab treatment. The mean cost per person-year was 3023є in the 12 months of standard of care prior to omalizumab and 16,111є in the period of treatment with omalizumab. The incremental cost-effectiveness ratios were 2244є/exacerbation avoided, and 1750є/unit decrease in GETE classification.
Our results demonstrate that adding omalizumab to the treatment of patients with uncontrolled severe persistent asthma reduces the number of exacerbations, improving overall treatment effectiveness at an acceptable cost from a societal perspective.
利用经验登记处葡萄牙亚人群的结果，评估奥马珠单抗治疗和控制重度持续性哮喘的成本效果。
这是一项基于经验登记处真实数据的实用成本效果分析，该登记处招募了来自葡萄牙 20 个参与中心的 62 名未控制的持续性过敏性哮喘患者。通过医生的总体治疗效果评价（GETE），前瞻性地测量奥马珠单抗治疗的反应，持续 24 个月。在开始奥马珠单抗治疗前的 12 个月内，患者的临床症状、哮喘控制、肺功能、恶化和医疗保健利用情况的回顾性数据可用作标准护理对照。恶化次数（严重和非严重）、临床发作次数、缺勤天数（缺勤工作和/或上学）和治疗的 GETE 反应被视为有效性结果。从社会角度来看，直接和间接成本均被视为成本指标。直接成本与奥马珠单抗、标准护理和临床发作（急诊就诊、住院和非计划就诊）的成本有关。间接成本与工作缺勤的社会成本有关。临床发作和药物的单位成本取自葡萄牙卫生当局的官方来源。进行了单变量敏感性分析。
与奥马珠单抗治疗前相比，奥马珠单抗治疗后估计每位患者每年发生 1.5 次恶化，这意味着与单独的标准护理相比，奥马珠单抗治疗后恶化的发生率降低了 82.1%。还观察到 GTE 评分降低了 54.1%，有利于奥马珠单抗治疗。在奥马珠单抗治疗期间，每个患者的年平均成本为 3023 埃居，而在奥马珠单抗治疗前的 12 个月的标准护理期间，每个患者的年平均成本为 16111 埃居。增量成本效果比为避免恶化的 2244 埃居/次，以及 GTE 分类降低的 1750 埃居/次。
我们的结果表明，在控制不佳的重度持续性哮喘患者的治疗中添加奥马珠单抗可减少恶化次数，从社会角度来看，在可接受的成本下提高总体治疗效果。
