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奥瑞珠单抗治疗葡萄牙复发型和原发进展型多发性硬化症的成本效益分析。

Cost-effectiveness Analysis of Ocrelizumab for the Treatment of Relapsing and Primary Progressive Multiple Sclerosis in Portugal.

作者信息

Martins Paulo, Vandewalle Björn, Félix Jorge, Capela Carlos M, Cerqueira João J, Salgado António V, Ferreira Diana G, Monteiro Isabel

机构信息

Exigo Consultores, Lda., Lisbon, Portugal.

Neurology Department, Centro Hospitalar Universitário de Lisboa Central, EPE, Lisbon, Portugal.

出版信息

Pharmacoecon Open. 2023 Mar;7(2):229-241. doi: 10.1007/s41669-022-00381-z. Epub 2022 Dec 1.

Abstract

OBJECTIVES

Ocrelizumab demonstrated significant clinical benefit for the treatment of relapsing (RMS) and primary progressive (PPMS) multiple sclerosis (MS), an incurable disease characterized by disability progression. This study evaluated the clinical and economic impact of ocrelizumab relative to current clinical practice, including other disease-modifying therapies (DMT), available in Portugal.

METHODS

Markov models for MS were adapted to estimate the impact of ocrelizumab across three patient populations: treatment-naïve RMS, previously treated RMS, and PPMS. Health states were defined according to the Expanded Disability Status Scale. For RMS, the model further captured the occurrence of relapses and progression to secondary progressive multiple sclerosis (SPMS). A lifetime time-horizon and Portuguese societal perspective were adopted.

RESULTS

For RMS patients, ocrelizumab was estimated to maximize the expected time (years) without progression to SPMS (10.50) relative to natalizumab (10.10), dimethyl fumarate (8.64), teriflunomide (8.39), fingolimod (8.38), interferon β-1a (8.33) and glatiramer acetate (8.18). As the most effective option, with quality-adjusted life year (QALY) gains between 0.3 and 1.2, ocrelizumab was found to be cost-saving relative to natalizumab and fingolimod, and presented incremental cost-effectiveness ratios (ICER) below €16,720/QALY relative to the remaining DMT. For PPMS patients, the ICER of ocrelizumab versus best supportive care was estimated at €78,858/QALY.

CONCLUSIONS

Ocrelizumab provides important health benefits for RMS and PPMS patients, comparing favourably with other widely used therapies. In RMS, ocrelizumab was revealed to be either cost-saving or have costs-per-QALY likely below commonly accepted cost-effectiveness thresholds. In PPMS, ocrelizumab fills a clear clinical gap in the current clinical practice. Overall, ocrelizumab is expected to provide good value for money in addressing the need of MS patients.

摘要

目的

奥瑞珠单抗已证明对复发型(RMS)和原发进展型(PPMS)多发性硬化症(MS)的治疗具有显著临床益处,MS是一种以残疾进展为特征的不治之症。本研究评估了奥瑞珠单抗相对于葡萄牙现有临床实践(包括其他疾病修正疗法(DMT))的临床和经济影响。

方法

对MS的马尔可夫模型进行调整,以估计奥瑞珠单抗对三类患者群体的影响:初治RMS患者、既往接受过治疗的RMS患者和PPMS患者。健康状态根据扩展残疾状态量表进行定义。对于RMS,该模型进一步记录了复发情况以及进展为继发进展型多发性硬化症(SPMS)的情况。采用终身时间范围和葡萄牙社会视角。

结果

对于RMS患者,相对于那他珠单抗(10.10)、富马酸二甲酯(8.64)、特立氟胺(8.39)、芬戈莫德(8.38)β-1a干扰素(8.33)和醋酸格拉替雷(8.18),估计奥瑞珠单抗能使无进展至SPMS的预期时间(年)最大化(10.50)。作为最有效的选择,质量调整生命年(QALY)增益在0.3至1.2之间,发现奥瑞珠单抗相对于那他珠单抗和芬戈莫德具有成本节约效果,相对于其余DMT,其增量成本效果比(ICER)低于16,720欧元/QALY。对于PPMS患者,奥瑞珠单抗与最佳支持治疗相比的ICER估计为78,858欧元/QALY。

结论

奥瑞珠单抗为RMS和PPMS患者带来了重要的健康益处,与其他广泛使用的疗法相比具有优势。在RMS中,奥瑞珠单抗显示出具有成本节约效果或每QALY成本可能低于普遍接受的成本效果阈值。在PPMS中,奥瑞珠单抗填补了当前临床实践中的明显临床空白。总体而言,预计奥瑞珠单抗在满足MS患者需求方面具有良好的性价比。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/257a/10043078/62fdbec77658/41669_2022_381_Fig1_HTML.jpg

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