Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands.
Institute of Medical Technology Assessment, Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands.
JAMA Netw Open. 2021 Mar 1;4(3):e213497. doi: 10.1001/jamanetworkopen.2021.3497.
Although the number of treatments for elderly patients with non-transplant-eligible (NTE) multiple myeloma (MM) has increased substantially, evidence is lacking on the clinical effectiveness and cost-effectiveness of novel treatment sequences.
To determine the optimal sequence of treatment for patients with NTE MM from the perspective of the patient, physician, and society.
DESIGN, SETTING, AND PARTICIPANTS: Using data from a Dutch observational registry, this economic evaluation combined evidence from network meta-analyses in a patient-level simulation model and modeled time-to-event and types of events from a hospital perspective with a lifetime horizon. Data analysis was performed from June 2019 to September 2020.
Thirty treatment sequences, including up to 3 lines of therapy, were compared with bortezomib-melphalan-prednisone (VMP)-lenalidomide-dexamethasone (LenDex)-pomalidomide-dexamethasone (PomDex).
The primary outcomes of the model were overall survival (OS), quality-adjusted life-years (QALYs), costs, and cost-effectiveness.
Sequences starting with daratumumab-VMP (second line: carfilzomib-lenalidomide-dexamethasone or elotuzumab-lenalidomide-dexamethasone) or bortezomib-melphalan-prednisone-thalidomide-maintenance bortezomib-thalidomide (VMPT-VT) (second line: daratumumab-lenalidomide-dexamethasone) had the largest expected OS (7.5 years), which is 3.5 additional life-years compared with VMP-LenDex-PomDex. Total costs per patient for these sequences ranged between $786 024 and $1 085 794. The sequence VMPT-VT-carfilzomib-lenalidomide-dexamethasone-panobinostat-bortezomib-dexamethasone had the most favorable cost-effectiveness ratio ($98 585 per life-year gained and $132 707 per QALY gained vs VMP-LenDex-PomDex).
These findings suggest that sequences including novel treatments were highly effective, but the cost-effectiveness ratios were above currently accepted willingness-to-pay thresholds. Treating MM with novel agents necessitates either a large increase in budget or a substantial reduction of drug costs by price negotiations, and these findings can support these reimbursement decisions and price negotiations.
尽管有大量治疗不适合移植的（NTE）多发性骨髓瘤（MM）老年患者的治疗方法，但缺乏新型治疗方案的临床效果和成本效益的证据。
从患者、医生和社会的角度确定 NTE MM 患者的最佳治疗顺序。
设计、地点和参与者：使用来自荷兰观察性注册的数据，本经济评估将网络荟萃分析中的证据与从医院角度进行的患者水平模拟模型中的时间到事件和事件类型相结合，并在终生范围内进行建模。数据分析于 2019 年 6 月至 2020 年 9 月进行。
比较了 30 种治疗方案，包括最多 3 线治疗，与硼替佐米-美法仑-泼尼松（VMP）-来那度胺-地塞米松（LenDex）-泊马度胺-地塞米松（PomDex）。
模型的主要结局是总生存期（OS）、质量调整生命年（QALYs）、成本和成本效益。
以达雷妥尤单抗-VMP（二线：卡非佐米-来那度胺-地塞米松或依鲁替尼-来那度胺-地塞米松）或硼替佐米-美法仑-泼尼松-噻唑酰胺维持硼替佐米-噻唑酰胺（VMPT-VT）（二线：达雷妥尤单抗-来那度胺-地塞米松）起始的序列具有最大的预期 OS（7.5 年），与 VMP-LenDex-PomDex 相比，这意味着额外增加了 3.5 年的寿命。这些序列的每位患者总费用在 786024 美元至 1085794 美元之间。VMPT-VT-卡非佐米-来那度胺-地塞米松-帕纳比司他-硼替佐米-地塞米松的成本效益比最具优势（每获得 1 个生命年的成本为 98585 美元，每获得 1 个 QALY 的成本为 132707 美元，与 VMP-LenDex-PomDex 相比）。
这些发现表明，包括新型药物的治疗方案具有高度有效性，但成本效益比高于目前可接受的支付意愿阈值。使用新型药物治疗 MM 需要要么通过谈判大幅增加预算，要么通过降低药物成本来大幅降低药物成本，这些发现可以支持这些报销决策和价格谈判。
