Pelligra Christopher G, Parikh Kejal, Guo Shien, Chandler Conor, Mouro Jorge, Abouzaid Safiya, Ailawadhi Sikander
Evidera, Waltham, Massachusetts.
Celgene Corporation, Summit, New Jersey.
Clin Ther. 2017 Oct;39(10):1986-2005.e5. doi: 10.1016/j.clinthera.2017.08.010. Epub 2017 Sep 28.
Pomalidomide plus low-dose dexamethasone (POM-d), daratumumab monotherapy (DARA), and carfilzomib monotherapy (CAR) have been approved for use in the treatment of patients with heavily pretreated relapsed-refractory multiple myeloma (RRMM) in the US, based on findings from the MM-002, SIRIUS, and PX-171-003-A1 studies, respectively. The objective of this study was to assess the cost-effectiveness of POM-d, DARA, and CAR in this patient population from a US payer's perspective.
A cost-effectiveness model was developed to estimate the cost and health outcomes over a 3-year time horizon in 3 health states: progression-free, post-progression, and death. The main efficacy data source was a matching-adjusted indirect comparison using data from the aforementioned studies. Direct medical costs were considered, including: treatment acquisition and administration (initial line and subsequent line), pre- and post-medication, prophylaxis treatment, adverse event management, and health care resource utilization. Sensitivity analyses were conducted. A scenario analysis that assumed equal efficacy across all 3 treatments was conducted. Costs, life-years, and quality-adjusted life-years were estimated and discounted at 3% per annum.
Over 3 years, the use of POM-d was associated with similar life-years and quality-adjusted life-years gained compared with DARA and CAR (incremental: life-years, +0.02 and +0.07, respectively; quality-adjusted life-years, +0.01 and +0.05), and with a cost less than that of DARA (-$8,919) and similar to that of CAR (-$195). Sensitivity analyses illustrated that the results were sensitive to progression-free survival, treatment duration, and drug costs. An equal efficacy scenario resulted in cost-savings relative to those of both DARA and CAR (-$11,779 and -$12,595).
POM-d may be a cost-effective treatment option relative to DARA or CAR in heavily pretreated patients with RRMM in the US.
基于MM - 002、SIRIUS和PX - 171 - 003 - A1研究的结果，泊马度胺联合低剂量地塞米松（POM - d）、达雷妥尤单抗单药治疗（DARA）和卡非佐米单药治疗（CAR）已在美国被批准用于治疗经过多次治疗的复发难治性多发性骨髓瘤（RRMM）患者。本研究的目的是从美国医保支付方的角度评估POM - d、DARA和CAR在该患者群体中的成本效益。
建立了一个成本效益模型，以估计在3种健康状态（无进展、进展后和死亡）下3年时间范围内的成本和健康结果。主要疗效数据源是使用上述研究数据进行的匹配调整间接比较。考虑了直接医疗成本，包括：治疗获取和给药（初始治疗线和后续治疗线）、用药前和用药后、预防性治疗、不良事件管理以及医疗资源利用。进行了敏感性分析。进行了假设所有3种治疗疗效相同的情景分析。估计了成本、生命年和质量调整生命年，并按每年3%进行贴现。
在3年期间，与DARA和CAR相比，使用POM - d获得的生命年和质量调整生命年相似（增量：生命年分别为+0.02和+0.
