United BioSource Corporation, London, UK.
J Med Econ. 2011;14(6):690-7. doi: 10.3111/13696998.2011.611841. Epub 2011 Sep 5.
To estimate the cost-effectiveness (cost per additional life-year [LY] and quality-adjusted life-year [QALY] gained) of lenalidomide plus dexamethasone (LEN/DEX) compared with bortezomib for the treatment of relapsed-refractory multiple myeloma (rrMM) in Norway.
A discrete-event simulation model was developed to predict patients? disease course using patient data, best response, and efficacy levels obtained from LEN/DEX MM-009/-010 trials and the bortezomib (APEX) published clinical trial. Predictive equations for time-to-progression (TTP) and post-progression survival (PPS) were developed by identifying the best fitting parametric survival distributions and selecting the most significant predictors. Disease and adverse event management was obtained via survey from Norwegian experts. Costs, derived from official Norwegian pricing data bases, included drug, administration, monitoring, and adverse event management costs.
Complete or partial responders were 65% for LEN/DEX compared to 43% for bortezomib. Derived median TTP was 11.45 months for LEN/DEX compared to 5.15 months for bortezomib. LYs and QALYs were higher for LEN/DEX (4.06 and 2.95, respectively) than for bortezomib (3.11 and 2.19, respectively). The incremental costs per QALY and LY gained from LEN/DEX were NOK 247,978 and NOK 198,714, respectively, compared to bortezomib. Multiple sensitivity analyses indicated the findings were stable. The parameters with the greatest impact were 4-year time horizon (NOK 441,457/QALY) and higher bound confidence intervals for PPS (NOK 118,392).
The model analyzed two therapies not compared in head-to-head trials, and predicted results using an equation incorporating patient-level characteristics. It is a limited estimation of the costs and outcomes in a Norwegian setting.
The simulation model showed that treatment with LEN/DEX leads to greater LYs and QALYs when compared to bortezomib in the treatment of rrMM patients. The incremental cost-effectiveness ratio indicated treatment with LEN/DEX to be cost-effective and was the basis of the reimbursement approval of LEN/DEX in Norway.
评估来那度胺联合地塞米松（LEN/DEX）对比硼替佐米用于挪威复发性/难治性多发性骨髓瘤（rrMM）患者治疗的成本效益（每增加 1 个额外生命年 [LY] 和质量调整生命年 [QALY] 的成本）。
使用来自 LEN/DEX-MM-009/-010 试验和硼替佐米（APEX）发表的临床试验的患者数据、最佳反应和疗效水平，开发了一个离散事件模拟模型来预测患者的疾病进程。通过识别最佳拟合的参数生存分布并选择最显著的预测因素，为进展时间（TTP）和进展后生存（PPS）开发了预测方程。疾病和不良事件管理是通过对挪威专家的调查获得的。成本来自官方挪威定价数据库，包括药物、管理、监测和不良事件管理成本。
完全或部分缓解者 LEN/DEX 为 65%，而硼替佐米为 43%。LEN/DEX 衍生的中位 TTP 为 11.45 个月，而硼替佐米为 5.15 个月。LEN/DEX 的 LYs 和 QALYs 分别为 4.06 和 2.95，而硼替佐米为 3.11 和 2.19。与硼替佐米相比，LEN/DEX 的每增加一个 QALY 和 LY 的增量成本分别为 NOK 247,978 和 NOK 198,714。多项敏感性分析表明，结果是稳定的。影响最大的参数是 4 年时间范围（NOK 441,457/QALY）和 PPS 的较高置信区间（NOK 118,392）。
该模型分析了两种未在头对头试验中进行比较的疗法，并使用包含患者水平特征的方程来预测结果。这只是在挪威环境下对成本和结果的有限估计。
模拟模型表明，与硼替佐米相比，LEN/DEX 治疗复发性 MM 患者可带来更多的 LYs 和 QALYs。增量成本效益比表明 LEN/DEX 的治疗具有成本效益，并为 LEN/DEX 在挪威的报销批准提供了依据。
