阿西米尼用于慢性髓性白血病的三线治疗:基于无治疗缓解方法的成本效益分析。

IF 1 Q4 PHARMACOLOGY & PHARMACY
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引用次数: 0

摘要

简介肿瘤学领域的首个靶向疗法--伊马替尼彻底改变了慢性髓性白血病(CML)的治疗,并推动了各种癌症靶向疗法的研究。慢性髓性白血病由染色体易位引起,形成 BCR-ABL1 融合基因。阿昔米尼最近被批准用于治疗三线难治或不耐受患者。目的:建立一个成本效益模型,将阿西米尼与已获批的第三代酪氨酸激酶抑制剂(TKIs)(博苏替尼和泊纳替尼)进行比较,重点关注实现无治疗缓解(TFR)。此外,还评估了将 asciminib 作为替代治疗药物的预算影响:该模型基于一个有 7 个状态的马尔可夫链。实现 TFR 的条件是在 DMR 状态下保持 5 年。该模型的疗效以 QALY 衡量,基础病例分析中的成本以西班牙为基础。为评估模型的可变性,进行了概率分析(PSA)和确定性分析(DSA)。阿西米尼与博舒替尼和阿西米尼与泊纳替尼进行了比较:阿西米尼与泊纳替尼相比,是一种节约成本的替代疗法,因为两种替代疗法的疗效相似,而且阿西米尼的成本更低,仅为30,275欧元。与博舒替尼相比,阿西米尼的QALY增加了4.33,而成本(203591欧元)却更高,因此每QALY的ICER为47010.49欧元。PSA 显示,对模型变异性影响较大的参数是转为 BP 的概率以及达到 MMR 和 DMR 的概率。单向分析表明,药物成本对两个模型的影响都较大,而贴现率对阿西米尼与博舒替尼模型的影响较大:阿西米尼扩大了难治性或不耐受前两线治疗的患者的治疗选择,具有成本效益。药物成本对疾病的总体成本有重大影响,这就强调了为每种药物选择贴现率的重要性。鉴于 CML 的发病率相对较低,引入 asciminib 对预算的影响有限,因此需要根据患者的临床特征做出个性化决定。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Asciminib for third-line treatment of chronic myeloid leukemia: Cost-effectiveness analysis based on treatment-free remission approach

Introduction

The first targeted therapy in oncology, imatinib, revolutionized chronic myeloid leukemia (CML) treatment and spurred research in targeted therapies for various cancers. CML results from a chromosomal translocation, forming the BCR-ABL1 fusion gene. Asciminib has been recently approved for third-line refractory or intolerant patients. Treatment-free remission (TFR) is attainable with sustained deep molecular response (DMR) and this approach could be incorporated into pharmacoeconomic models.

Aims

To establish a cost-effectiveness model comparing asciminib to approved third-generation tyrosine kinase inhibitors (TKIs) (bosutinib and ponatinib) with a focus on achieving TFR. Additionally, the budgetary impact of incorporating asciminib as a therapeutic alternative is assessed.

Methods

This model is based on a Markov chain with 7 states. The condition for achieving TFR is to remain for 5 years in DMR state. Efficacy of the model was measured in QALYs, and the costs included in the base case analysis are based in Spain. A probabilistic (PSA) and deterministic analysis (DSA) were carried out to assess the variability of the model. There were achieved 2 independent models comparing asciminib vs bosutinib and asciminib vs ponatinib.

Results

Asciminib, when compared with ponatinib, is a cost-saving alternative, as efficacy is similar between alternatives, and asciminib have a lower cost of 30,275€. Asciminib showed 4.33 more QALYs and a higher cost (203,591€) than bosutinib, resulting in an ICER of €47,010.49 per QALY. PSA shows that the parameters with higher influence in the variability of the model were the probability of transitioning to BP and probabilities of achieving MMR and DMR. A one-way analysis reports that the drug cost has a higher influence on both models, and the discount rate significantly affects the asciminib vs bosutinib model.

Conclusion

Asciminib broadens therapeutic choices for patient’s refractory or intolerant to 2 prior lines of treatment in a cost-effectiveness manner. The costs of drugs significantly impact the overall cost of the disease, emphasizing the importance of the selected discount rates for each drug. Given the relatively low incidence of CML, the introduction of asciminib has a limited budgetary impact, warranting individualized decisions based on patient`s clinical characteristics.

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来源期刊
FARMACIA HOSPITALARIA
FARMACIA HOSPITALARIA PHARMACOLOGY & PHARMACY-
CiteScore
1.90
自引率
21.40%
发文量
46
审稿时长
37 days
期刊介绍: Una gran revista para acceder a los mejores artículos originales y revisiones de la farmacoterapia actual. Además, es Órgano de expresión científica de la Sociedad Española de Farmacia Hospitalaria, y está indexada en Index Medicus/Medline, EMBASE/Excerpta Médica, Alert, Internacional Pharmaceutical Abstracts y SCOPUS.
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