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从美国医保支付方角度看,博纳吐单抗与挽救性化疗用于复发或难治性费城染色体阴性B前体急性淋巴细胞白血病的成本效益

Cost-effectiveness of blinatumomab versus salvage chemotherapy in relapsed or refractory Philadelphia-chromosome-negative B-precursor acute lymphoblastic leukemia from a US payer perspective.

作者信息

Delea Thomas E, Amdahl Jordan, Boyko Diana, Hagiwara May, Zimmerman Zachary F, Franklin Janet L, Cong Ze, Hechmati Guy, Stein Anthony

机构信息

a Policy Analysis Inc. (PAI) , Brookline , MA , USA.

b Global Development, Amgen Inc. , Thousand Oaks , CA , USA.

出版信息

J Med Econ. 2017 Sep;20(9):911-922. doi: 10.1080/13696998.2017.1344127. Epub 2017 Jul 11.

Abstract

OBJECTIVE

To evaluate the cost-effectiveness of blinatumomab (Blincyto) vs standard of care (SOC) chemotherapy in adults with relapsed or refractory (R/R) Philadelphia-chromosome-negative (Ph-) B-precursor acute lymphoblastic leukemia (ALL) based on the results of the phase 3 TOWER study from a US healthcare payer perspective.

METHODS

The Blincyto Global Economic Model (B-GEM), a partitioned survival model, was used to estimate the incremental cost-effectiveness ratio (ICER) of blinatumomab vs SOC. Response rates, event-free survival (EFS), overall survival (OS), numbers of cycles of blinatumomab and SOC, and transplant rates were estimated from TOWER. EFS and OS were estimated by fitting parametric survival distributions to failure-time data from TOWER. Utility values were based on EORTC-8D derived from EORTC QLQ-C30 assessments in TOWER. A 50-year lifetime horizon and US payer perspective were employed. Costs and outcomes were discounted at 3% per year.

RESULTS

The B-GEM projected blinatumomab to yield 1.92 additional life years and 1.64 additional quality-adjusted life years (QALYs) compared with SOC at an incremental cost of $180,642. The ICER for blinatumomab vs SOC was estimated to be $110,108/QALY gained in the base case. Cost-effectiveness was sensitive to the number and cost of inpatient days for administration of blinatumomab and SOC, and was more favorable in the sub-group of patients who had received no prior salvage therapy. At an ICER threshold of $150,000/QALY gained, the probability that blinatumomab is cost-effective was estimated to be 74%.

LIMITATIONS

The study does not explicitly consider the impact of adverse events of the treatment; no adjustments for long-term transplant rates were made.

CONCLUSIONS

Compared with SOC, blinatumomab is a cost-effective treatment option for adults with R/R Ph - B-precursor ALL from the US healthcare perspective at an ICER threshold of $150,000 per QALY gained. The value of blinatumomab is derived from its incremental survival and health-related quality-of-life (HRQoL) benefit over SOC.

摘要

目的

基于美国医疗保健支付方视角下的3期TOWER研究结果,评估博纳吐单抗(Blincyto)与标准治疗(SOC)化疗方案相比,用于复发或难治性(R/R)费城染色体阴性(Ph-)B前体急性淋巴细胞白血病(ALL)成人患者的成本效益。

方法

使用博纳吐单抗全球经济模型(B-GEM),一种分区生存模型,来估计博纳吐单抗与SOC的增量成本效益比(ICER)。从TOWER研究中估计了缓解率、无事件生存期(EFS)、总生存期(OS)、博纳吐单抗和SOC的疗程数以及移植率。通过将参数生存分布拟合到TOWER研究的失败时间数据来估计EFS和OS。效用值基于TOWER研究中从EORTC QLQ-C30评估得出的EORTC-8D。采用50年的终身视角和美国支付方视角。成本和结果按每年3%进行贴现。

结果

与SOC相比,B-GEM预计博纳吐单抗可额外产生1.92个生命年和1.64个质量调整生命年(QALY),增量成本为180,642美元。在基础案例中,博纳吐单抗与SOC的ICER估计为每获得一个QALY 110,108美元。成本效益对博纳吐单抗和SOC给药的住院天数和成本敏感,在未接受过前期挽救治疗的患者亚组中更具优势。在每获得一个QALY的ICER阈值为150,000美元时,博纳吐单抗具有成本效益的概率估计为74%。

局限性

该研究未明确考虑治疗不良事件的影响;未对长期移植率进行调整。

结论

从美国医疗保健角度来看,在每获得一个QALY的ICER阈值为150,000美元时,与SOC相比,博纳吐单抗是复发或难治性Ph-B前体ALL成人患者的一种具有成本效益的治疗选择。博纳吐单抗的价值源于其相对于SOC在生存期和健康相关生活质量(HRQoL)方面的增量获益。

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