Borg Sixten, Nahi Hareth, Hansson Markus, Lee Dawn, Elvidge Jamie, Persson Ulf
a Health Economics Unit, Department of Clinical Sciences in Malmö , Lund University , Medicon Village , Lund , Sweden .
b The Swedish Institute for Health Economics (IHE) , Lund , Sweden .
Acta Oncol. 2016 May;55(5):554-60. doi: 10.3109/0284186X.2015.1096021. Epub 2015 Nov 17.
Multiple myeloma (MM) patients who have progressed following treatment with both bortezomib and lenalidomide have a poor prognosis. In this late stage, other effective alternatives are limited, and patients in Sweden are often left with best supportive care. Pomalidomide is a new anti-angiogenic and immunomodulatory drug for the treatment of MM. Our objective was to evaluate the cost effectiveness of pomalidomide as an add-on to best supportive care in patients with relapsed and refractory MM in Sweden.
We developed a health-economic discrete event simulation model of a patient's course through stable disease and progressive disease, until death. It estimates life expectancy, quality-adjusted life years (QALYs) and costs from a societal perspective. Effectiveness data and utilities were taken from the MM-003 trial comparing pomalidomide plus low-dose dexamethasone with high-dose dexamethasone (HIDEX). Cost data were taken from official Swedish price lists, government sources and literature.
The model estimates that, if a patient is treated with HIDEX, life expectancy is 1.12 years and the total cost is SEK 179 976 (€19 100), mainly indirect costs. With pomalidomide plus low-dose dexamethasone, life expectancy is 2.33 years, with a total cost of SEK 767 064 (€81 500), mainly in drug and indirect costs. Compared to HIDEX, pomalidomide treatment gives a QALY gain of 0.7351 and an incremental cost of SEK 587 088 (€62 400) consisting of increased drug costs (59%), incremental indirect costs (33%) and other healthcare costs (8%). The incremental cost-effectiveness ratio is SEK 798 613 (€84 900) per QALY gained.
In a model of late-stage MM patients with a poor prognosis in the Swedish setting, pomalidomide is associated with a relatively high incremental cost per QALY gained. This model was accepted by the national Swedish reimbursement authority TLV, and pomalidomide was granted reimbursement in Sweden.
接受硼替佐米和来那度胺治疗后病情进展的多发性骨髓瘤（MM）患者预后较差。在此晚期阶段，其他有效的替代治疗方法有限，瑞典的患者通常只能接受最佳支持治疗。泊马度胺是一种用于治疗MM的新型抗血管生成和免疫调节药物。我们的目标是评估在瑞典复发难治性MM患者中，泊马度胺作为最佳支持治疗补充药物的成本效益。
我们建立了一个健康经济离散事件模拟模型，用于模拟患者从疾病稳定到病情进展直至死亡的病程。该模型从社会角度估计预期寿命、质量调整生命年（QALY）和成本。有效性数据和效用值取自MM - 003试验，该试验比较了泊马度胺加低剂量地塞米松与高剂量地塞米松（HIDEX）。成本数据取自瑞典官方价格表、政府来源和文献。
模型估计，如果患者接受HIDEX治疗，预期寿命为1.12年，总成本为179,976瑞典克朗（19,100欧元），主要为间接成本。使用泊马度胺加低剂量地塞米松治疗时，预期寿命为2.33年，总成本为767,064瑞典克朗（81,500欧元），主要为药物成本和间接成本。与HIDEX相比，泊马度胺治疗使QALY增加0.7351，增量成本为587,088瑞典克朗（62,400欧元），包括药物成本增加（59%）、增量间接成本（33%）和其他医疗成本（8%）。每获得一个QALY的增量成本效益比为798,613瑞典克朗（84,900欧元）。
在瑞典背景下，针对预后较差的晚期MM患者模型中，泊马度胺每获得一个QALY的增量成本相对较高。该模型被瑞典国家报销机构TLV接受，泊马度胺在瑞典获得了报销。
