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泊马度胺、卡非佐米和达雷妥尤单抗用于治疗美国多次接受治疗的复发难治性多发性骨髓瘤患者的成本效益

Cost-effectiveness of Pomalidomide, Carfilzomib, and Daratumumab for the Treatment of Patients with Heavily Pretreated Relapsed-refractory Multiple Myeloma in the United States.

作者信息

Pelligra Christopher G, Parikh Kejal, Guo Shien, Chandler Conor, Mouro Jorge, Abouzaid Safiya, Ailawadhi Sikander

机构信息

Evidera, Waltham, Massachusetts.

Celgene Corporation, Summit, New Jersey.

出版信息

Clin Ther. 2017 Oct;39(10):1986-2005.e5. doi: 10.1016/j.clinthera.2017.08.010. Epub 2017 Sep 28.

Abstract

PURPOSE

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.

METHODS

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.

FINDINGS

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).

IMPLICATIONS

POM-d may be a cost-effective treatment option relative to DARA or CAR in heavily pretreated patients with RRMM in the US.

摘要

目的

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。

方法

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。

结果

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.

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