The Cost‐Effectiveness of Initial Treatment of Multiple Myeloma in the U.S. With Bortezomib Plus Melphalan and Prednisone Versus Thalidomide Plus Melphalan and Prednisone or Lenalidomide Plus Melphalan and Prednisone With Continuous Lenalidomide Maintenance Treatment

The outlook for transplant‐ineligible multiple myeloma patients has improved enormously over recent years with the incorporation of new agents into standard regimens. Novel regimens combine melphalan and prednisone (MP) with bortezomib (VMP), with thalidomide (MPT), and with lenalidomide with (MPR‐R) and without (MPR) lenalidomide maintenance. The efficacy, safety, and cost‐effectiveness of these regimens have not yet been compared; therefore, we conducted a pharmacoeconomic analysis using data from randomized controlled trials versus MP. Using a Markov model developed from a U.S. payer's perspective, we compared VMP with MPT and MPR‐R over a lifetime horizon. MPT and MPR‐R were chosen because, like VMP, they are superior to MP in response and outcomes. Data from the Velcade as Initial Standard Therapy in Multiple Myeloma (VISTA; VMP), Intergroupe Francophone du Myelome (IFM) 99–06 (MPT), and MM‐015 (MPR‐R) trials were used. The IFM 99–06 study was selected because of the superior activity in this study compared with other MPT studies. Using patient‐level (VMP) and published (MPT, MPR‐R) data, we estimated the health‐state transition and adverse event probabilities for each regimen, related costs, and state‐specific utility estimates. Costs (in 2010 U.S. dollars) and health outcomes were discounted at 3%. Discounted lifetime direct medical costs were lowest with VMP at $119,102. MPT cost $142,452 whereas MPR‐R cost $248,358. Incremental cost‐effectiveness ratio calculations projected that VMP would confer cost savings and better health outcomes relative to MPT and MPR‐R. We conclude that VMP is highly likely to be cost‐effective compared with MP, MPT, and MPR‐R. 摘要 近年来，随着新药被纳入标准治疗方案，不符合移植条件的多发性骨髓瘤患者的前景已大幅改善。新的治疗方案包括美法仑和泼尼松（MP）联合硼替佐米（VMP）、联合沙利度胺（MPT）以及联合来那度胺加来那度胺维持（MPR‐R）或无来那度胺维持（MPR）。尚未比较过这些治疗方案的有效性、安全性以及费用效益；因此，我们采用了对比MP的随机对照试验数据开展了一项药物经济学分析。 采用从美国支付者角度开发的Markov模型，我们在终身范围内将VMP与MPT和MPR‐R进行比较。选择MPT和MPR‐R是由于它们和VMP一样，疗效和转归都优于MP。数据来源于硼替佐米作为初始标准治疗用于多发性骨髓瘤（VISTA；VMP）、法国骨髓瘤组（IFM）99‐06（MPT）和MM‐015（MPR‐R）试验。选用IFM 99‐06研究是因为其活性优于其他MPT研究。我们采用患者水平（VMP）和已发表（MPT，MPR‐R）数据估算了每种方案的转为健康状态概率和不良事件概率、相关费用和不同情况下费用的估计值。按3%贴现计算费用（按2010年美元）和健康转归。 VMP的贴现终身直接医疗费用最低，为119 102美元。MPT费用为142 452美元，而MPR‐R费用为248 358美元。增量费用效益比计算值预计，与MPT及MPR‐R相比，VMP费用更低、健康转归更佳。因此我们得出结论，VMP的费用效益优于MP、MPT和MPR‐R。 A detailed pharmacoeconomic analysis was conducted to estimate the incremental cost‐effectiveness of bortezomib, melphalan, and prednisone versus thalidomide, melphalan, and prednisone versus lenalidomid