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沙特阿拉伯复发性缓解型多发性硬化症不同疾病修饰疗法的真实世界比较成本效益分析。

Real-World Comparative Cost-Effectiveness Analysis of Different Classes of Disease-Modifying Therapies for Relapsing-Remitting Multiple Sclerosis in Saudi Arabia.

机构信息

Department of Clinical Pharmacy, College of Pharmacy, King Saud University, P.O. Box 2454, Riyadh 11451, Saudi Arabia.

Pharmacoeconomics Research Unit, Department of Clinical Pharmacy, College of Pharmacy, King Saud University, P.O. Box 2454, Riyadh 11451, Saudi Arabia.

出版信息

Int J Environ Res Public Health. 2021 Dec 16;18(24):13261. doi: 10.3390/ijerph182413261.

DOI:10.3390/ijerph182413261
PMID:34948876
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8702157/
Abstract

The very fact that multiple sclerosis (MS) is incurable and necessitates life-long care makes it one of the most burdensome illnesses. The aim of this study was to compare the cost-effectiveness of orally administered medications (e.g., fingolimod, dimethyl fumarate, and teriflunomide), interferon (IFN)-based therapy, and monoclonal antibodies (MABs) (e.g., natalizumab and rituximab) in the management of relapsing-remitting multiple sclerosis (RRMS) in Saudi Arabia using real-world data. This was a retrospective cohort study in which patients with RRMS aged ≥18 years without any other chronic health conditions with non-missing data for at least 12 months were recruited from the electronic health records of a university-affiliated tertiary care center. Multiple logistic regressions controlling for age, sex, and duration of therapy were conducted to examine the odds of disability progression, clinical relapse, MRI lesions, and composite outcome (e.g., relapse, lesion development on MRI, disability progression). The number of patients who met the inclusion criteria and were included in the analysis was 146. Most of the patients were female (70.51%) and young (e.g., ≤35 years of age). There were 40 patients on the orally administered agents (e.g., dimethyl fumarate, teriflunomide, fingolimod), 66 patients were on IFN-based therapy (e.g., Rebif), and 40 patients were on monoclonal antibodies (e.g., rituximab and natalizumab). Patients on MABs had lower odds of the composite outcome (OR = 0.17 (95% CI: 0.068-0.428)). The use of orally administered agents was dominant (e.g., more effective and less costly), with average annual cost savings of USD -4336.65 (95% CI: -5207.89--3903.32) and 8.11% higher rate of effectiveness (95% CI: -14.81-18.07) when compared with Rebif. With regard to the use of MABs in comparison to Rebif, MABs were associated with higher cost but a better rate of effectiveness, with an average additional annual cost of USD 1381.54 (95% CI: 421.31-3621.06) and 43.11% higher rate of effectiveness (95% CI: 30.38-61.15) when compared with Rebif. In addition, the use of MABs was associated with higher cost but a better rate of effectiveness, with an average additional annual cost of USD 5717.88 (95% CI: 4970.75-8272.66) and 35% higher rate of effectiveness (95% CI: 10.0-42.50) when compared with orally administered agents. The use of MABs in the management of RRMS among the young patient population has shown to be the most effective therapy in comparison to both IFN-based therapy (e.g., Rebif) and orally administered agents, but with higher cost. Orally administered agents resulted in better outcomes and lower costs in comparison to IFN-based therapy. Future studies should further examine the cost-effectiveness of different disease-modifying therapies for the management of RRMS using more robust study designs.

摘要

多发性硬化症(MS)无法治愈且需要终身护理,这一事实使其成为最具负担的疾病之一。本研究旨在使用真实世界数据比较口服药物(如芬戈莫德、二甲基富马酸和特立氟胺)、干扰素(IFN)为基础的治疗和单克隆抗体(MABs)(如那他珠单抗和利妥昔单抗)在治疗沙特阿拉伯复发性缓解型多发性硬化症(RRMS)中的成本效益。这是一项回顾性队列研究,纳入了来自大学附属医院三级护理中心电子健康记录中年龄≥18 岁、无其他慢性健康状况且至少有 12 个月非缺失数据的 RRMS 患者。采用多元逻辑回归控制年龄、性别和治疗持续时间,以检查残疾进展、临床复发、MRI 病变和复合结局(如复发、MRI 上的病变发展、残疾进展)的可能性。符合纳入标准并纳入分析的患者人数为 146 人。大多数患者为女性(70.51%)和年轻人(如≤35 岁)。有 40 名患者使用口服药物(如二甲基富马酸、特立氟胺、芬戈莫德),66 名患者使用 IFN 为基础的治疗(如 Rebif),40 名患者使用 MABs(如利妥昔单抗和那他珠单抗)。使用 MABs 的患者发生复合结局的可能性较低(OR=0.17(95%CI:0.068-0.428))。与 Rebif 相比,口服药物的使用更为有效且成本更低,具有平均每年节省 4336.65 美元(95%CI:-5207.89--3903.32)和 8.11%的更高有效率(95%CI:-14.81-18.07)。与 Rebif 相比,MABs 的使用与更高的成本相关,但有效率更高,平均每年额外增加 1381.54 美元(95%CI:421.31-3621.06)和 43.11%的更高有效率(95%CI:30.38-61.15)。此外,与 Rebif 相比,MABs 的使用与更高的成本相关,但有效率更高,平均每年额外增加 5717.88 美元(95%CI:4970.75-8272.66)和 35%的更高有效率(95%CI:10.0-42.50)。在年轻患者人群中,与 IFN 为基础的治疗(如 Rebif)和口服药物相比,MABs 在治疗 RRMS 方面显示出最有效的治疗方法,但成本更高。与 IFN 为基础的治疗相比,口服药物的治疗效果更好,成本更低。未来的研究应进一步使用更稳健的研究设计来研究不同疾病修饰疗法在 RRMS 治疗中的成本效益。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22e2/8702157/f3db5ef965c2/ijerph-18-13261-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22e2/8702157/4fa3d155bd6f/ijerph-18-13261-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22e2/8702157/f3db5ef965c2/ijerph-18-13261-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22e2/8702157/4fa3d155bd6f/ijerph-18-13261-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/22e2/8702157/f3db5ef965c2/ijerph-18-13261-g002.jpg

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COVID-19: Public health issues and ethical dilemmas.新型冠状病毒肺炎:公共卫生问题与伦理困境
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Rituximab for the treatment of multiple sclerosis: a review.利妥昔单抗治疗多发性硬化症:综述
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Clinical presentation and diagnosis of multiple sclerosis.多发性硬化的临床表现和诊断。
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