Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Centre, PO Box 22660, Amsterdam, DD 1100, The Netherlands.
Orphanet J Rare Dis. 2013 Feb 19;8:29. doi: 10.1186/1750-1172-8-29.
The cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care was evaluated in the Dutch cohort of patients with Fabry disease.
Cost-effectiveness analysis was performed using a life-time state-transition model. Transition probabilities, effectiveness data and costs were derived from retrospective data and prospective follow-up of the Dutch study cohort consisting of males and females aged 5-78 years. Intervention with ERT (either agalsidase alfa or agalsidase beta) was compared to the standard medical care. The main outcome measures were years without end organ damage (renal, cardiac en cerebrovascular complications), quality adjusted life years (QALYs), and costs.
Over a 70 year lifetime, an untreated Fabry patient will generate 55.0 years free of end-organ damage (53.5 years in males, 56.9 years in females) and 48.6 QALYs (47.8 in males, 49.7 in females). Starting ERT in a symptomatic patient increases the number of years free of end-organ damage by 1.5 year (1.6 in males, 1.3 in females), while the number of QALYs gained increases by a similar amount (1.7 in males, 1.4 in females). The costs of ERT starting in the symptomatic stage are between €9 - €10 million (£ 7.9 - £ 8.8 million, $13.0- $14.5 million) during a patient's lifetime. Consequently, the extra costs per additional year free of end-organ damage and the extra costs per additional QALY range from €5.5 - €7.5 million (£ 4.8 - £ 6.6 million, $ 8.0 - $ 10.8 million), undiscounted.
In symptomatic patients with Fabry disease, ERT has limited effect on quality of life and progression to end organ damage. The pharmaco-economic evaluation shows that this modest effectiveness drives the costs per QALY and the costs per year free of end-organ damage to millions of euros. Differentiation of patients who may benefit from ERT should be improved to enhance cost-effectiveness.
在荷兰法布里病患者队列中，评估了酶替代疗法 (ERT) 与标准医疗相比的成本效益。
使用终生状态转换模型进行成本效益分析。转移概率、疗效数据和成本来自回顾性数据和前瞻性随访的荷兰研究队列，该队列由 5-78 岁的男性和女性组成。ERT（阿加糖酶α或阿加糖酶β）干预与标准医疗进行比较。主要观察指标是无终末器官损伤（肾脏、心脏和脑血管并发症）的年数、质量调整生命年 (QALY) 和成本。
在 70 年的生命周期中，未经治疗的法布里病患者将有 55.0 年无终末器官损伤（男性 53.5 年，女性 56.9 年）和 48.6 QALY（男性 47.8 年，女性 49.7 年）。在有症状的患者中开始 ERT 可使无终末器官损伤的年数增加 1.5 年（男性 1.6 年，女性 1.3 年），同时获得的 QALY 数量也会增加相同数量（男性 1.7 年，女性 1.4 年）。在患者的一生中，从有症状阶段开始 ERT 的成本在 900 万至 1000 万欧元（790 万至 880 万英镑，1300 万至 1450 万美元）之间。因此，每额外增加一年无终末器官损伤和每额外增加一个 QALY 的额外成本范围为 550 万至 750 万欧元（480 万至 660 万英镑，800 万至 1080 万美元），不贴现。
在有症状的法布里病患者中，ERT 对生活质量和终末器官损伤的进展影响有限。药物经济学评价表明，这种适度的疗效导致每 QALY 的成本和每年无终末器官损伤的成本达到数百万欧元。应改善患者受益的区分，以提高成本效益。
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