在荷兰，onasemnogene abeparvovec与nusinersen治疗1型脊髓性肌萎缩症患者的成本比较分析。
Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands.
作者信息
van der Schans Simon, Velikanova Rimma, Weidlich Diana, Howells Ruth, Patel Anish, Bischof Matthias, Postma Maarten J, Boersma Cornelis
机构信息
Health-Ecore, Zeist, The Netherlands.
Unit of Global Health, Department of Health Sciences, University Medical Center Groningen, University of Groningen, Groningen, 9173 GZ, The Netherlands.
出版信息
Eur J Health Econ. 2025 Feb 21. doi: 10.1007/s10198-024-01754-3.
BACKGROUND
Spinal muscular atrophy (SMA) is a rare genetic disease resulting in loss of motor function and, in severe cases (e.g., SMA type 1), infantile death. While treatments like nusinersen and onasemnogene abeparvovec improve prognosis for patients with SMA, costs for these medications can contribute to economic burden.
OBJECTIVE
Direct costs were compared for onasemnogene abeparvovec, a one-time gene replacement therapy, versus nusinersen, a lifelong therapy, for patients with SMA type 1 and/or three or more survival motor neuron 2 (SMN2) gene copies in the Netherlands.
METHODS
A cost comparison analysis model of 1-year incident patient population from the Netherlands was used to compare costs of onasemnogene abeparvovec versus nusinersen for patients eligible for onasemnogene abeparvovec immediately after diagnosis. Multiple analyses were conducted for economic outcomes (e.g., base-case, break-even, deterministic sensitivity, probabilistic sensitivity, scenario analyses).
RESULTS
Cost differences of -€2.9 million (undiscounted) and -€1.5 million (discounted) per patient with SMA type 1 treated with onasemnogene abeparvovec versus nusinersen over a 20-year time horizon were identified (base-case). Reduced costs with onasemnogene abeparvovec versus nusinersen were evident after 8.25 years.
CONCLUSION
Onasemnogene abeparvovec was less costly than nusinersen after 8.25 years of treatment of patients with SMA type 1 in the Netherlands.
背景
脊髓性肌萎缩症（SMA）是一种罕见的遗传性疾病，会导致运动功能丧失，在严重情况下（如1型SMA）会导致婴儿死亡。虽然诸如诺西那生钠和onasemnogene abeparvovec等治疗方法可改善SMA患者的预后，但这些药物的成本会加重经济负担。
目的
在荷兰，对1型SMA和/或有三个或更多存活运动神经元2（SMN2）基因拷贝的患者，比较一次性基因替代疗法onasemnogene abeparvovec与终身疗法诺西那生钠的直接成本。
方法
使用来自荷兰的1年新发患者群体的成本比较分析模型，比较确诊后立即符合onasemnogene abeparvovec治疗条件的患者使用onasemnogene abeparvovec与诺西那生钠的成本。对经济结果进行了多项分析（如基础病例分析、盈亏平衡分析、确定性敏感性分析、概率敏感性分析、情景分析）。
结果
在20年的时间范围内，与诺西那生钠相比，接受onasemnogene abeparvovec治疗的1型SMA患者每人的成本差异为-€290万（未贴现）和-€150万（贴现）（基础病例分析）。onasemnogene abeparvovec与诺西那生钠相比，成本在8.25年后开始降低。
结论
在荷兰，对1型SMA患者进行8.25年治疗后，onasemnogene abeparvovec的成本低于诺西那生钠。
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