Longitudinal Dosing Patterns and Treatment Costs Among Patients with Spinal Muscular Atrophy Initiating Nusinersen and Risdiplam.

IF 4.7 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Advances in Therapy Pub Date : 2026-08-06 DOI:10.1007/s12325-026-03687-0

Walter Toro Jimenez, Min Yang, Wei Song, Esteban Lemus Wirtz, Dongni Ye

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引用次数: 0

Abstract

Introduction: Spinal muscular atrophy (SMA) is a neuromuscular disorder historically associated with high morbidity, mortality, and healthcare costs. Disease-modifying therapies have improved outcomes, but their long-term economic burden remains uncertain. Nusinersen and risdiplam, both chronic therapies, differ from onasemnogene abeparvovec, a one-time gene therapy. Understanding real-world treatment costs of chronic SMA therapies is critical to inform therapeutic choice.

Methods: This retrospective cohort study used the Komodo Health Research Database (2016-2024). Patients with SMA aged ≥ 2 years who initiated nusinersen or risdiplam were followed up to 5 years. Dosing patterns and drug acquisition and administration costs (2024 USD) were assessed annually, overall and by age group and scoliosis status.

Results: Among 3864 eligible patients, 2743 initiated nusinersen and 1121 initiated risdiplam (mean age 21.4 vs. 25.3 years, respectively; 51.5% vs. 46.1% male). Median follow-up was 5.5 years for nusinersen and 1.1 years for risdiplam. Persistence declined over time, with 39.5% of patients treated with nusinersen and 63.7% of patients treated with risdiplam remaining on their index therapy at years 5 and 4. Patients treated with nusinersen received 3.6 injections on average in year 1 and 2.8-3.1 annually thereafter. Median annualized nusinersen treatment costs were $504,173 in year 1 and $438,788-548,608 in years 2-5. Patients treated with risdiplam had a median of 96 days of supply in year 1 (51.5% with > 90 days); median annualized drug costs were $143,331 overall in year 1 and $259,419 among those with > 90 days of supply, increasing to $264,701-289,340 in years 2-4. Costs were similar across subgroups.

Conclusion: Nusinersen and risdiplam persistence declined over time. Among patients remaining on their index therapy, both treatments had high long-term costs, highlighting the substantial and ongoing financial burden of chronic SMA therapies and the need for real-world evidence to inform healthcare planning.

Trial registration: ClinicalTrials.gov identifier, NCT07378943.

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脊髓性肌萎缩患者的纵向给药模式和治疗费用：Nusinersen和Risdiplam。

简介：脊髓性肌萎缩症（SMA）是一种历史上与高发病率、高死亡率和高医疗费用相关的神经肌肉疾病。疾病修饰疗法改善了疗效，但其长期经济负担仍不确定。Nusinersen和risdiplam都是慢性疗法，与一次性基因疗法onasemnogene abparvovec不同。了解慢性SMA治疗的实际治疗成本对于告知治疗选择至关重要。方法：采用Komodo健康研究数据库（2016-2024）进行回顾性队列研究。年龄≥2岁的SMA患者开始服用nusinersen或risdiplan，随访至5年。每年评估剂量模式和药物获取和管理成本（2024美元），总体上，按年龄组和脊柱侧凸状况进行评估。结果：在3864例符合条件的患者中，2743例开始使用nusinsen， 1121例开始使用risdiplam（平均年龄分别为21.4岁和25.3岁；男性51.5%和46.1%）。nusinersen的中位随访时间为5.5年，risdiplan的中位随访时间为1.1年。持久性随着时间的推移而下降，39.5%的nusinersen患者和63.7%的risdiplam患者在第5年和第4年仍在进行其指标治疗。接受nusinersen治疗的患者第一年平均注射3.6次，此后每年平均注射2.8-3.1次。nusinersen治疗费用中位数在第一年为504,173美元，在第2-5年为438,788-548,608美元。接受瑞西泮治疗的患者在第1年的平均供应时间为96天（51.5%的患者为90天）；第一年的年化药品成本中位数为143,331美元，在供应90天的患者中为259,419美元，在第2-4年增加到264,701-289,340美元。各亚组的成本相似。结论：Nusinersen和risdiplam的持久性随时间而下降。在继续使用其指数疗法的患者中，两种疗法的长期成本都很高，这突出了慢性SMA疗法的巨大和持续的经济负担，以及为医疗保健计划提供实际证据的必要性。试验注册：ClinicalTrials.gov识别码，NCT07378943。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.