背景:脊髓性肌萎缩症(SMA)是一种罕见的,进步,神经肌肉紊乱.治疗方面的最新进展需要对负担进行最新评估,以告知报销决定。
目的:量化SMA患者的医疗资源利用率(HCRU)和护理成本。
方法:在瑞典国家患者注册(2007-2018)中确定的SMA患者的队列研究,与分为四种SMA类型(1、2、3,未指明的成人发病[UAO])的参考队列匹配。HCRU包括住院,门诊就诊,程序,和分配药物。直接医疗费用是通过将HRU乘以各自的单位成本来估计的。对SMA和参考队列的平均年度HCRU和医疗费用进行建模,以估计可归因于疾病的差异(即,平均治疗效果估计)。使用合成队列评估了直接成本随时间的轨迹。
结果:我们确定了290例SMA患者。与参考队列相比,SMA患者的年化HCRU更高。对于1型(风险比[RR]:29.2;95%置信区间[CI]:16.0,53.5)和2型(RR:23.3;95%CI:16.4,33.1),观察到住院患者过夜的风险比最高。自首次诊断以来,每位患者每年的平均年度直接医疗费用在1型患者中最高(114,185欧元,SMA归因:113,380欧元),类型2(61,876欧元,可归因于SMA:61,237欧元),类型3(45,518欧元和SMA-归属:44,556欧元),和UAO(4046欧元和SMA-归属:2098欧元)。在所有类型的首次诊断后的2-3年内,费用最高。
结论:可归因于SMA的经济负担是显著的。需要进一步的研究来了解其他欧洲国家的负担以及新疗法的影响。
BACKGROUND: Spinal muscular atrophy (SMA) is a rare, progressive, neuromuscular disorder. Recent advances in treatment require an updated assessment of burden to inform reimbursement decisions.
OBJECTIVE: To quantify healthcare resource utilisation (HCRU) and cost of care for patients with SMA.
METHODS: Cohort study of patients with SMA identified in the Swedish National Patient Registry (2007-2018), matched to a reference cohort grouped into four SMA types (1, 2, 3, unspecified adult onset [UAO]). HCRU included inpatient admissions, outpatient visits, procedures, and dispensed medications. Direct medical costs were estimated by multiplying HCRU by respective unit costs. Average annual HCRU and medical costs were modelled for SMA versus reference cohorts to estimate differences attributable to the disease (i.e., average treatment effect estimand). The trajectory of direct costs over time were assessed using synthetic cohorts.
RESULTS: We identified 290 SMA patients. Annualised HCRU was higher in SMA patients compared with reference cohorts. Highest risk ratios were observed for inpatient overnight stays for type 1 (risk ratio [RR]: 29.2; 95% confidence interval [CI]: 16.0, 53.5) and type 2 (RR: 23.3; 95% CI: 16.4,33.1). Mean annual direct medical costs per patient for each year since first diagnosis were greatest for type 1 (€114,185 and SMA-attributable: €113,380), type 2 (€61,876 and SMA-attributable: €61,237), type 3 (€45,518 and SMA-attributable: €44,556), and UAO (€4046 and SMA-attributable: €2098). Costs were greatest in the 2-3 years after the first diagnosis for all types.
CONCLUSIONS: The economic burden attributable to SMA is significant. Further research is needed to understand the burden in other European countries and the impact of new treatments.