Large Samuel, Hettle Robert, Balakumaran Arun, Wu Elise, Borse Rebekah H
a PAREXEL International , London , UK.
b Merck & Co., Inc. , Kenilworth , NJ , USA.
J Med Econ. 2018 Nov 13;22(1):16-25. doi: 10.1080/13696998.2018.1534738.
Patients with classical Hodgkin's lymphoma (cHL) who have relapsed after or are ineligible for autologous stem cell transplantation (ASCT) have limited treatment options and generally a poor prognosis. Pembrolizumab was recently approved in the US for the treatment of such patients having demonstrated clinical benefit and tolerability in relapsed/refractory cHL; however, the cost-effectiveness of pembrolizumab in this population is currently unknown.
A three-state Markov model (progression-free [PF], progressed disease, and death) was developed to assess the cost-effectiveness of pembrolizumab (200 mg) vs brentuximab vedotin (BV; 1.8 mg/kg) in patients with relapsed/refractory cHL after ASCT who have not received BV post-ASCT over a 20-year time horizon from a US payer perspective. PF survival was modeled using a naïve indirect treatment comparison of data from KEYNOTE-087 and the SG035-003 trial. Post-progression survival was modeled using data from published literature. Costs (drug acquisition and administration, disease management, subsequent treatment, and adverse events) and outcomes were discounted at an annual rate of 3.0%. Uncertainty surrounding cost-effectiveness was assessed via probabilistic, deterministic, and scenario analyses.
In the base case, pembrolizumab was predicted to yield an additional 0.574 life-years (LYs) and 0.500 quality-adjusted life-years (QALYs) vs BV and cost savings of $63,278. Drug acquisition costs were the biggest driver of incremental costs between strategies. Pembrolizumab had a 99.6% probability of being cost-effective compared with BV at a willingness-to-pay threshold of $20,000/QALY and dominated BV in all scenarios tested.
The analysis was subject to potential bias due to the use of a naïve indirect treatment comparison and, given the current immaturity of OS in KEYNOTE-087, PPS was assumed equivalent across both treatments.
Pembrolizumab is a cost-effective alternative to BV for patients with relapsed/refractory cHL after ASCT.
经典型霍奇金淋巴瘤（cHL）患者在自体干细胞移植（ASCT）后复发或不符合ASCT条件，其治疗选择有限，总体预后较差。帕博利珠单抗最近在美国被批准用于治疗此类患者，已证明在复发/难治性cHL中具有临床获益和耐受性；然而，帕博利珠单抗在该人群中的成本效益目前尚不清楚。
建立了一个三状态马尔可夫模型（无进展[PF]、疾病进展和死亡），从美国支付方的角度评估帕博利珠单抗（200mg）与本妥昔单抗（BV；1.8mg/kg）在ASCT后复发/难治性cHL且ASCT后未接受BV治疗的患者中，20年时间范围内的成本效益。PF生存情况采用KEYNOTE - 087和SG035 - 003试验数据的简单间接治疗比较进行建模。进展后生存情况采用已发表文献的数据进行建模。成本（药物采购和给药、疾病管理、后续治疗及不良事件）和结局按每年3.0%的贴现率进行贴现。通过概率分析、确定性分析和情景分析评估成本效益的不确定性。
在基础案例中，与BV相比，预计帕博利珠单抗可多产生0.574个生命年（LYs）和0.500个质量调整生命年（QALYs），并节省成本63,278美元。药物采购成本是各策略间增量成本的最大驱动因素。在支付意愿阈值为20,000美元/QALY时，与BV相比，帕博利珠单抗具有成本效益的概率为99.6%，且在所有测试情景中均优于BV。
由于使用了简单间接治疗比较，该分析可能存在潜在偏倚，并且鉴于KEYNOTE - 087中总生存期目前尚不成熟，假设两种治疗的PPS相等。
对于ASCT后复发/难治性cHL患者，帕博利珠单抗是一种比BV更具成本效益的替代方案。
