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估算在美国使用多奈单抗对早期有症状阿尔茨海默病进行限期治疗的经济合理价格。

Estimating the Economically Justifiable Price of Limited-Duration Treatment with Donanemab for Early Symptomatic Alzheimer's Disease in the United States.

作者信息

Boustani Malaz, Doty Erin G, Garrison Louis P, Smolen Lee J, Klein Timothy M, Murphy Daniel R, Spargo Andrew W, Belger Mark, Johnston Joseph A

机构信息

Division of Internal Medicine and Geriatrics, Indiana University School of Medicine, Indianapolis, IN, USA.

Lilly Corporate Center, Indianapolis, IN, 46285, USA.

出版信息

Neurol Ther. 2024 Dec;13(6):1641-1659. doi: 10.1007/s40120-024-00649-y. Epub 2024 Sep 18.

Abstract

INTRODUCTION

The goal of this economic model is to estimate an economically justifiable price (EJP) for using donanemab for the treatment of early symptomatic Alzheimer's disease (AD) in the United States based on clinical data from the phase 3 TRAILBLAZER-ALZ 2 trial (NCT04437511).

METHODS

We adapted an AD Markov state-transition model developed by the Institute for Clinical and Economic Review to estimate the EJP for donanemab at different willingness-to-pay (WTP) thresholds from the health care system perspective and the societal perspective as co-base cases.

RESULTS

Assuming a WTP threshold of $150,000 per quality-adjusted life-year (QALY) gained, the model estimates a 1-year (13-dose) EJP for donanemab of $80,538 from the health care system perspective and $91,126 from the societal perspective; at a WTP threshold of $100,000 per QALY gained, the model estimates a 1-year (13-dose) EJP for donanemab of $44,691 from the health care system perspective and $55,419 from the societal perspective. Mean total treatment costs per patient at the $150,000 per QALY gained EJP derived from the health care system perspective were estimated at $77,812 based on the average number of doses of donanemab patients received in the co-base case analysis. One-way sensitivity analysis (OWSA) indicated that treatment efficacy, disease severity at the time of treatment initiation, and duration of treatment effect were the main drivers of the potential EJP.

CONCLUSIONS

Results from this modeling simulation informed by the TRAILBLAZER-ALZ 2 study support an EJP for limited-duration treatment with donanemab that exceeds per-dose list prices for currently available amyloid-targeting therapies, implying potentially lower lifetime costs and better value for money.

摘要

引言

本经济模型的目标是基于3期TRAILBLAZER - ALZ 2试验(NCT04437511)的临床数据,估算在美国使用多奈单抗治疗早期有症状阿尔茨海默病(AD)的经济合理价格(EJP)。

方法

我们采用了临床与经济评论研究所开发的AD马尔可夫状态转换模型,从医疗保健系统视角和社会视角,以不同的支付意愿(WTP)阈值作为共同基础案例,来估算多奈单抗的EJP。

结果

假设每获得一个质量调整生命年(QALY)的WTP阈值为150,000美元,该模型从医疗保健系统视角估算多奈单抗1年(13剂)的EJP为80,538美元,从社会视角为91,126美元;每获得一个QALY的WTP阈值为100,000美元时,该模型从医疗保健系统视角估算多奈单抗1年(13剂)的EJP为44,691美元,从社会视角为55,419美元。基于共同基础案例分析中患者接受多奈单抗的平均剂量数,从医疗保健系统视角得出的每获得一个QALY的EJP为150,000美元时,每位患者的平均总治疗成本估计为77,812美元。单向敏感性分析(OWSA)表明,治疗效果、治疗开始时的疾病严重程度以及治疗效果持续时间是潜在EJP的主要驱动因素。

结论

这项由TRAILBLAZER - ALZ 2研究提供信息的建模模拟结果支持多奈单抗有限疗程治疗的EJP超过目前可用的淀粉样蛋白靶向疗法的每剂标价,这意味着潜在的终身成本更低且性价比更高。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1004/11541978/ba4a69ae1062/40120_2024_649_Fig1_HTML.jpg

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