Methodology and results of real-world cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone in relapsed multiple myeloma using registry data.

The European Journal of Health Economics Pub Date : 2020-03-01 Epub Date: 2019-10-31 DOI:10.1007/s10198-019-01122-6
M Campioni, I Agirrezabal, R Hajek, J Minarik, L Pour, I Spicka, S Gonzalez-McQuire, P Jandova, V Maisnar
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引用次数: 4

Abstract

Objective: To predict the real-world (RW) cost-effectiveness of carfilzomib in combination with lenalidomide and dexamethasone (KRd) versus lenalidomide and dexamethasone (Rd) in relapsed multiple myeloma (MM) patients after one to three prior therapies.

Methods: A partitioned survival model that included three health states (progression-free, progressed disease and death) was built. Progression-free survival (PFS), overall survival (OS) and time to discontinuation (TTD) data for the Rd arm were derived using the Registry of Monoclonal Gammopathies in the Czech Republic; the relative treatment effects of KRd versus Rd were estimated from the phase 3, randomised, ASPIRE trial, and were used to predict PFS, OS and TTD for KRd. The model was developed from the payer perspective and included drug costs, administration costs, monitoring costs, palliative care costs and adverse-event related costs collected from Czech sources.

Results: The base case incremental cost effectiveness ratio for KRd compared with Rd was €73,156 per quality-adjusted life year (QALY) gained. Patients on KRd incurred costs of €117,534 over their lifetime compared with €53,165 for patients on Rd. The QALYs gained were 2.63 and 1.75 for patients on KRd and Rd, respectively.

Conclusions: Combining the strengths of randomised controlled trials and observational databases in cost-effectiveness models can generate policy-relevant results to allow well-informed decision-making. The current model showed that KRd is likely to be cost-effective versus Rd in the RW and, therefore, the reimbursement of KRd represents an efficient allocation of resources within the healthcare system.

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使用注册数据对卡非佐米联合来那度胺和地塞米松治疗复发性多发性骨髓瘤的实际成本效益进行方法学和结果分析。
目的:预测卡非佐米联合来那度胺和地塞米松(KRd)与来那度胺和地塞米松(Rd)治疗复发的多发性骨髓瘤(MM)患者经过一到三次治疗后的真实世界(RW)成本效益。方法:建立包括无进展、疾病进展和死亡三种健康状态的分区生存模型。Rd组的无进展生存期(PFS)、总生存期(OS)和停药时间(TTD)数据来自捷克共和国的单克隆Gammopathies登记处;KRd与Rd的相对治疗效果是从3期随机ASPIRE试验中估计的,并用于预测KRd的PFS、OS和TTD。该模型是从付款人的角度开发的,包括药品成本、管理成本、监测成本、姑息治疗成本和从捷克收集的不良事件相关成本。结果:与Rd相比,KRd的基本情况增量成本效益比为每获得质量调整生命年(QALY) 73,156欧元。KRd患者在其一生中产生的费用为117,534欧元,而Rd患者为53,165欧元。KRd和Rd患者获得的质量年分别为2.63和1.75。结论:将随机对照试验和观察性数据库在成本效益模型中的优势结合起来,可以产生与政策相关的结果,从而允许做出明智的决策。目前的模型表明,与RW中的Rd相比,KRd可能具有成本效益,因此,KRd的报销代表了医疗保健系统内资源的有效分配。
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