Novartis Pharmaceuticals Corporation, Health Economics & Outcomes Research, One HealthPlaza, East Hanover, NJ 07936-1080, USA.
Clin Ther. 2012 Jul;34(7):1583-90. doi: 10.1016/j.clinthera.2012.06.012. Epub 2012 Jun 28.
Fingolimod is a once-daily orally administered disease-modifying therapy (DMT) indicated for treatment of relapsing forms of multiple sclerosis (MS) to reduce the frequency of clinical relapses and delay accumulation of physical disability. In the randomized, double-blind, phase 3 TRANSFORMS trial, 0.5 mg/d oral fingolimod substantially reduced relapse frequency when compared with IM interferon-β1a (IFN-β1a) at 12-months. In a 12-month, double-blind, extension phase of the TRANSFORMS study, patients assigned to receive fingolimod continued to receive the same dosage, whereas patients who originally received IM IFN-β1a were randomized to receive either 0.5 or 1.25 mg/d fingolimod.
To investigate the cost-effectiveness of initiating fingolimod therapy early versus after 1 year of IFN-β1a therapy using TRANSFORMS study extension data.
A Microsoft Excel-based model was used to calculate the cost per relapse avoided for 2 years with continuous treatment with fingolimod compared with first-year treatment with IM IFN-β1a and second-year treatment with fingolimod. One-way sensitivity analyses were conducted on key input variables to assess their effect on cost per relapse avoided.
The 2-year relapse rate in the early fingolimod arm was 0.23, and in the delayed fingolimod arm was 0.53. The cost per relapse avoided was $83,125 in the early fingolimod arm versus $103,624 in the delayed fingolimod arm. Results of the sensitivity analyses showed an effect of drug acquisition cost and number of relapses in patients who received no treatment.
Continuous treatment with fingolimod for 2 years resulted in a lower cost per relapse avoided compared with treatment with IM IFN-β1a for the first year and then switching to fingolimod therapy. Thus, delaying fingolimod therapy does not seem to be cost effective.
芬戈莫德是一种每日口服一次的疾病修正治疗药物（DMT），用于治疗多发性硬化症（MS）的复发形式，以降低临床复发频率并延迟身体残疾的积累。在随机、双盲、III 期 TRANSFORMS 试验中，与 12 个月时的肌内注射干扰素-β1a（IFN-β1a）相比，0.5mg/d 口服芬戈莫德可显著降低复发频率。在 TRANSFORMS 研究的 12 个月双盲扩展阶段，接受芬戈莫德治疗的患者继续接受相同剂量的治疗，而最初接受肌内 IFN-β1a 治疗的患者被随机分配接受 0.5 或 1.25mg/d 芬戈莫德治疗。
利用 TRANSFORMS 研究扩展数据，研究早期开始芬戈莫德治疗与在 IFN-β1a 治疗 1 年后开始治疗的成本效益。
使用基于 Microsoft Excel 的模型计算与第一年肌内 IFN-β1a 治疗和第二年芬戈莫德治疗相比，连续使用芬戈莫德治疗两年的每例复发避免成本。对关键输入变量进行单因素敏感性分析，以评估其对每例复发避免成本的影响。
早期芬戈莫德组的 2 年复发率为 0.23，延迟芬戈莫德组为 0.53。早期芬戈莫德组的每例复发避免成本为 83125 美元，而延迟芬戈莫德组为 103624 美元。敏感性分析结果显示药物获得成本和未接受治疗的患者复发次数的影响。
与第一年肌内 IFN-β1a 治疗然后转换为芬戈莫德治疗相比，连续使用芬戈莫德治疗两年可降低每例复发避免成本。因此，延迟芬戈莫德治疗似乎并不具有成本效益。
