Xcenda, Palm Harbor, FL, USA.
J Med Econ. 2011;14(5):617-27. doi: 10.3111/13696998.2011.602444. Epub 2011 Jul 22.
With the addition of new agents for the treatment of multiple sclerosis (MS) (e.g., fingolimod), there is a need to evaluate the relative value of newer therapies in terms of cost and effectiveness, given healthcare resource constraints in the United States.
To assess the cost-effectiveness of natalizumab vs fingolimod in patients with relapsing MS.
A decision analytic model was developed to estimate the incremental cost per relapse avoided of natalizumab and fingolimod from a US managed care payer perspective. Two-year costs of treating patients with MS included drug acquisition costs, administration and monitoring costs, and costs of treating MS relapses. Effectiveness was measured in terms of MS relapses avoided (data from AFFIRM and FREEDOMS trials). One-way and probabilistic sensitivity analyses were conducted to assess uncertainty.
Mean 2-year estimated treatment costs were $86,461 (natalizumab) and $98,748 (fingolimod). Patients receiving natalizumab had a mean of 0.74 relapses avoided per 2 years vs 0.59 for fingolimod. Natalizumab dominated fingolimod in the incremental cost-effectiveness analysis, as it was less costly and more effective in reducing relapses. One-way sensitivity analysis showed the results of the model were robust to changes in drug acquisition costs, administration costs, and costs of treating MS relapses. Probabilistic sensitivity analysis showed natalizumab was cost-effective 95.1% of the time, at a willingness-to-pay (WTP) threshold of $0 per relapse avoided, increasing to 96.3% of the time at a WTP threshold of $50,000 per relapse avoided.
Absence of data from direct head-to-head studies comparing natalizumab and fingolimod, use of relapse rate reduction rather than sustained disability progression as primary model outcome, assumption of 100% adherence to MS treatment, and not capturing adverse event costs in the model.
Natalizumab dominates fingolimod in terms of incremental cost per relapse avoided, as it is less costly and more effective.
随着新型多发性硬化症（MS）治疗药物（如芬戈莫德）的出现，鉴于美国医疗资源有限，需要从成本和效果的角度评估新型疗法的相对价值。
评估那他珠单抗与芬戈莫德治疗复发性多发性硬化症的成本效果。
从美国管理式医疗支付方的角度，开发决策分析模型来评估那他珠单抗和芬戈莫德避免每例复发的增量成本。治疗多发性硬化症患者的两年成本包括药物获得成本、管理和监测成本以及治疗多发性硬化症复发的成本。采用避免的多发性硬化症复发数来衡量有效性（数据来自 AFFIRM 和 FREEDOMS 试验）。进行单因素和概率敏感性分析以评估不确定性。
那他珠单抗治疗的 2 年平均估计治疗费用为 86461 美元，芬戈莫德为 98748 美元。接受那他珠单抗治疗的患者每 2 年平均有 0.74 次复发得到避免，而芬戈莫德为 0.59 次。在增量成本效果分析中，那他珠单抗优于芬戈莫德，因为其成本更低，在减少复发方面更有效。单因素敏感性分析表明，药物获得成本、管理成本和治疗多发性硬化症复发成本的变化不会影响模型结果。概率敏感性分析表明，在意愿支付（WTP）阈值为 0 美元/次避免复发时，那他珠单抗的成本效果为 95.1%，在 WTP 阈值为 50000 美元/次避免复发时增加至 96.3%。
缺乏那他珠单抗与芬戈莫德直接比较的头对头研究数据，模型的主要结果是复发率降低而不是持续性残疾进展，假设对多发性硬化症治疗的依从率为 100%，且未纳入模型中的不良事件成本。
在避免每例复发的增量成本方面，那他珠单抗优于芬戈莫德，因为其成本更低，效果更好。
