Supplementary Material for: Pharmacoeconomic Implications of Lenalidomide Maintenance Therapy in Multiple Myeloma

We compared the three arms of the MM-015 randomized phase III clinical trial [melphalan and prednisone (MP), MP plus lenalidomide (MPR), and MPR plus lenalidomide maintenance (MPR-R)] to determine whether the addition of lenalidomide maintenance therapy for primary treatment of multiple myeloma is cost-effective. We used progression-free survival and adverse event data from the MM-015 study for the analysis. Two novel measures of cost-effectiveness termed the Average Cumulative Cost per Patient (ACCP) and the Average Cumulative Cost per Progression-Free Survivor (ACCPFS) were developed for the purpose of this analysis. The ACCP of MP was USD 18,218, compared to USD 167,862 for MPR and USD 309,173 for MPR-R. The ACCPFS was highest with MPR at USD 1,555,443, while MP was USD 313,592 and MPR-R was USD 690,111. MPR-R is superior to MPR in terms of preventing the first progression after initial therapy. However, the addition of lenalidomide to MP in the induction and also in the maintenance setting leads to significant costs.

本研究针对MM-015随机III期临床试验（randomized phase III clinical trial）的三个试验组展开对比分析，三组分别为美法仑（melphalan）联合泼尼松（prednisone，MP）方案、来那度胺（lenalidomide）联合MP方案（MPR），以及来那度胺维持治疗联合MPR方案（MPR-R），旨在评估在多发性骨髓瘤（multiple myeloma）的初始治疗中加入来那度胺维持疗法是否具备成本效益。本研究采用MM-015试验中的无进展生存期（progression-free survival）与不良事件（adverse event）数据开展分析。本次分析开发了两种全新的成本效益评估指标，分别为每患者平均累计成本（Average Cumulative Cost per Patient, ACCP）与每无进展生存者平均累计成本（Average Cumulative Cost per Progression-Free Survivor, ACCPFS）。MP方案的ACCP为18218美元，MPR方案为167862美元，MPR-R方案则为309173美元。ACCPFS方面，MPR方案最高，达1555443美元；MP方案为313592美元，MPR-R方案为690111美元。在初始治疗后预防首次疾病进展方面，MPR-R方案优于MPR方案。但在诱导治疗及维持治疗阶段均加入来那度胺至MP方案中，会带来显著的成本增加。