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在爱尔兰医疗环境中,替沙格赛基因治疗复发/难治性弥漫性大B细胞淋巴瘤的成本效用和信息价值分析。

Cost-utility and value of information analysis of tisagenlecleucel for relapsed/refractory diffuse large B-cell lymphoma in the Irish healthcare setting.

作者信息

Carey Niamh, Leahy Joy, Trela-Larsen Lea, Mc Cullagh Laura, Barry Michael

机构信息

National Centre for Pharmacoeconomics, Old Stone Building, St James's Hospital, Dublin, Ireland.

Department of Pharmacology and Therapeutics, Trinity Centre for Health Sciences, Trinity College Dublin, Dublin, Ireland.

出版信息

J Mark Access Health Policy. 2023 Jan 18;11(1):2166375. doi: 10.1080/20016689.2023.2166375. eCollection 2023.

Abstract

BACKGROUND

The evidence base of tisagenlecleucel is uncertain.

OBJECTIVE

To evaluate the cost-effectiveness of tisagenlecleucel. To conduct expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.

STUDY DESIGN

A three-state partitioned survival model. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 5 years; general population mortality with a standardised mortality ratio was then applied. EVPI and EVPPI were scaled up to population according to the incidence of the decision.

SETTING

Irish healthcare payer.

PARTICIPANTS

Patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL).

INTERVENTIONS

Tisagenlecleucel versus Salvage Chemotherapy (with or without haematopoietic stem cell transplant).

MAIN OUTCOME MEASURE

Incremental cost-effectiveness ratio (ICER). Population EVPI and EVPPI.

RESULTS

At list prices, the ICER was €119,509 per quality-adjusted life year (QALY) (incremental costs €218,092; incremental QALYs 1.82). Probability of cost-effectiveness, at a €45,000 per QALY threshold, was 0%. Population EVPI was €0.00. Population EVPI, at the price of tisagenlecleucel that reduced the ICER to €45,000 per QALY, was €3,989,438. Here, survival analysis had the highest population EVPPI (€1,128,053).

CONCLUSION

Tisagenlecleucel is not cost-effective, versus salvage chemotherapy (with or without haematopoietic stem cell transplant), for R/R DLBCL in Ireland. At list prices, further research to decrease decision uncertainty may not be of value.

摘要

背景

替沙格赛基因治疗白血病的证据基础尚不明确。

目的

评估替沙格赛基因治疗白血病的成本效益。进行完全信息期望值(EVPI)和部分EVPI(EVPPI)分析。

研究设计

三状态分区生存模型。一个短期决策树根据输注状态对替沙格赛基因治疗组的患者进行分区。将生存期外推至5年;然后应用标准化死亡率的一般人群死亡率。根据决策发生率将EVPI和EVPPI扩大到人群水平。

研究背景

爱尔兰医疗保健支付方。

研究对象

复发/难治性弥漫性大B细胞淋巴瘤(R/R DLBCL)患者。

干预措施

替沙格赛基因治疗与挽救性化疗(有或无造血干细胞移植)。

主要观察指标

增量成本效益比(ICER)。人群EVPI和EVPPI。

结果

按标价计算,ICER为每质量调整生命年(QALY)1​​19,509欧元(增量成本218,092欧元;增量QALY 1.82)。在每QALY阈值为45,000欧元时,成本效益概率为0%。人群EVPI为0.00欧元。在替沙格赛基因治疗价格将ICER降至每QALY 45,000欧元时,人群EVPI为3,989,438欧元。在此,生存分析的人群EVPPI最高(1,128,053欧元)。

结论

在爱尔兰,对于R/R DLBCL患者,与挽救性化疗(有或无造血干细胞移植)相比,替沙格赛基因治疗不具有成本效益。按标价计算,进一步研究以降低决策不确定性可能没有价值。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/edc3/9858398/df510ac62e24/ZJMA_A_2166375_F0001_OC.jpg

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