背景:尼曼-匹克病C型(NP-C)是一种进行性神经退行性疾病,患有早期婴儿(<2岁),晚期婴儿(2-6岁),青少年(7-15岁)和青少年(>15岁)发病。有神经症状的NP-C患者的主要治疗方法是miglustat,一种可以改变疾病进程的药物。
目的:我们的目的是在塞尔维亚共和国的社会经济环境中,与NP-C患者的对症治疗相比,评估miglustat的成本效益。中等收入的欧洲经济体。
方法:本研究选择了塞尔维亚共和国医疗保险基金的视角,时间跨度是80年。该研究的主要结果是使用miglustat和比较器获得的质量调整生命年,和治疗的直接成本。该研究是通过离散事件仿真模型的生成和仿真进行的。模型结果是对1000名虚拟患者的样本进行蒙特卡罗微观模拟后获得的。
结果:与对症治疗相比,米格卢司他治疗的成本效益不高,并且无论神经系统表现如何,净货币收益均为负值(-110,447,627.00±701,614.00RSD,-343,871,695.00±2,577,441.00RSD,-1,397,908,502.00±23,084,235.00RSD和-2,953,680,879.00±33,297,412.00RSD)用于早期婴儿,晚期婴儿,青少年和青少年群体,分别)。
结论:采用传统药物经济学评价时,与治疗NP-C的对症治疗相比,miglustat不是一种具有成本效益的选择。然而,考虑到Miglustat的有效性,有必要找到方法使这种药物可用于所有NP-C患者。
BACKGROUND: Niemann-Pick disease type C (NP-C) is a progressive neurodegenerative disorder with early infantile (< 2 years), late infantile (2-6 years), juvenile (7-15 years) and adolescent (> 15 years) onset. The mainstay of therapy for NP-C patients with neurological symptoms is miglustat, a drug that may modify the course of the disease.
OBJECTIVE: Our aim was to evaluate the cost-effectiveness of miglustat in comparison to symptomatic therapy in patients with NP-C in the socio-economic settings of the Republic of Serbia, an upper-middle-income European economy.
METHODS: The perspective of the Serbian Republic Health Insurance Fund was chosen for this study, and the time horizon was eighty years. The main outcomes of the study were quality-adjusted life years gained with miglustat and comparator, and direct costs of treatment. The study was conducted through the generation and simulation of the Discrete-Event Simulation model. The model results were obtained after Monte Carlo microsimulation of a sample with 1000 virtual patients.
RESULTS: Treatment with miglustat was not cost-effective when compared with symptomatic therapy and was associated with negative values of net monetary benefit regardless of the onset of neurological manifestations (- 110,447,627.00 ± 701,614.00 RSD, - 343,871,695.00 ± 2,577,441.00 RSD, - 1,397,908,502.00 ± 23,084,235.00 RSD and - 2,953,680,879.00 ± 33,297,412.00 RSD) for early infantile, late infantile, juvenile and adolescent cohorts, respectively).
CONCLUSIONS: When traditional pharmacoeconomic evaluation is employed, miglustat is not a cost-effective option in comparison to symptomatic therapy for the treatment of NP-C. However, given the proven efficacy of miglustat, there is a need to find ways to make this drug available to all patients with NP-C.