Hamidi Vida, Couto Elisabeth, Ringerike Tove, Klemp Marianne
Norwegian Institute of Public Health, Norway.
Department of Pharmacology, University of Oslo, Norway.
J Clin Med Res. 2018 Feb;10(2):88-105. doi: 10.14740/jocmr3168w. Epub 2017 Dec 30.
Several disease-modifying drug therapies are available for the treatment of multiple sclerosis (MS). To ensure the most appropriate MS management, we assessed the effectiveness and cost-effectiveness of the disease-modifying medicines used for MS.
We conducted a systematic review including 11 disease-modifying drugs used for treatment of adult patients diagnosed with relapsing-remitting MS. We performed a network meta-analysis using both direct and indirect evidence. We examined the endpoints, annual relapse, disability progression, mortality, serious adverse events and withdrawal from the study due to adverse events. Cost-effectiveness was assessed by developing a decision model. The model calculated costs and quality-adjusted life years (QALYs) with different treatment strategies. Uncertainties in the parameter values were explored with a probabilistic sensitivity analysis and several scenario analyses.
Alemtuzumab 12 mg was the most effective against annual relapse (high quality evidence). For disability progression, dimethyl fumarate 240 mg and fingolimod 0.5 mg and 1.25 mg were more effective treatment alternatives (high quality evidence). For withdrawal due to adverse events, the conclusion is unclear due to the low quality of the available evidence. Peg-interferon beta-1a was associated with more adverse events (than the other treatments). None of the examined treatments had an effect on overall mortality compared to placebo. The economic analysis indicated that alemtuzumab was more effective in terms of QALYs and less costly than the other treatment alternatives. Discarding alemtuzumab, three treatment alternatives (interferon beta-1b (Extavia), peg-interferon beta-1a and natalizumab) could be considered cost-effective depending on the willingness-to-pay (WTP) threshold. Assuming a WTP below EUR 111,690 per QALY, interferon beta-1b (Extavia) was approximately 36% likely to be the most cost-effective treatment, followed by peg-interferon beta-1a (approximately 34% likely).
Our results showed that alemtuzumab can be considered as more effective and less costly than the other treatment alternatives. There is a substantial potential cost saving if more patients start on the more effective and less costly treatment alternatives.
有几种疾病修正药物疗法可用于治疗多发性硬化症（MS）。为确保对MS进行最恰当的管理，我们评估了用于MS的疾病修正药物的有效性和成本效益。
我们进行了一项系统评价，纳入了11种用于治疗确诊为复发缓解型MS的成年患者的疾病修正药物。我们使用直接和间接证据进行了网状Meta分析。我们检查了终点指标、年复发率、残疾进展、死亡率、严重不良事件以及因不良事件退出研究的情况。通过建立决策模型评估成本效益。该模型计算了不同治疗策略的成本和质量调整生命年（QALY）。通过概率敏感性分析和几种情景分析探讨了参数值的不确定性。
阿仑单抗12mg对年复发最有效（高质量证据）。对于残疾进展，富马酸二甲酯240mg、芬戈莫德0.5mg和1.25mg是更有效的治疗选择（高质量证据）。对于因不良事件退出研究，由于现有证据质量低，结论尚不清楚。聚乙二醇干扰素β-1a与更多不良事件相关（与其他治疗相比）。与安慰剂相比，所检查的治疗方法均对总体死亡率无影响。经济分析表明，在QALY方面，阿仑单抗比其他治疗选择更有效且成本更低。不考虑阿仑单抗，根据支付意愿（WTP）阈值，三种治疗选择（干扰素β-1b（Extavia）、聚乙二醇干扰素β-1a和那他珠单抗）可被认为具有成本效益。假设每QALY的WTP低于111,690欧元，干扰素β-1b（Extavia）约有36%的可能性是最具成本效益的治疗方法，其次是聚乙二醇干扰素β-1a（约34%的可能性）。
我们的结果表明，与其他治疗选择相比，阿仑单抗可被认为更有效且成本更低。如果更多患者开始使用更有效且成本更低的治疗选择，存在显著的潜在成本节约。
