估算美国使用多奈单抗对早期症状性阿尔茨海默病进行限期治疗的经济合理价格。

IF 3.9 3区 医学 Q1 CLINICAL NEUROLOGY
Neurology and Therapy Pub Date : 2024-12-01 Epub Date: 2024-09-18 DOI:10.1007/s40120-024-00649-y
Malaz Boustani, Erin G Doty, Louis P Garrison, Lee J Smolen, Timothy M Klein, Daniel R Murphy, Andrew W Spargo, Mark Belger, Joseph A Johnston
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引用次数: 0

摘要

简介:本经济模型的目的是根据 TRAILBLAZER-ALZ 2 3 期试验(NCT04437511)的临床数据,估算在美国使用多那尼单抗治疗早期症状性阿尔茨海默病(AD)的经济合理价格(EJP):我们改编了临床与经济研究所(Institute for Clinical and Economic Review)开发的AD马尔可夫状态转换模型,从医疗保健系统的角度和作为共同基础案例的社会角度估算了多那尼单抗在不同支付意愿(WTP)阈值下的EJP:假设每个质量调整生命年(QALY)获得的WTP阈值为150,000美元,那么从医疗保健系统的角度来看,模型估计多那尼单抗1年(13剂)的EJP为80,538美元,从社会角度来看为91,126美元;如果每个质量调整生命年获得的WTP阈值为100,000美元,那么从医疗保健系统的角度来看,模型估计多那尼单抗1年(13剂)的EJP为44,691美元,从社会角度来看为55,419美元。根据联合基础病例分析中患者接受多那尼单抗的平均剂量,从医疗保健系统角度得出的每 QALY 收益 EJP 为 150,000 美元时,每位患者的平均治疗总成本估计为 77,812 美元。单向敏感性分析(OWSA)表明,治疗效果、开始治疗时的疾病严重程度和治疗效果持续时间是潜在 EJP 的主要驱动因素:以 TRAILBLAZER-ALZ 2 研究为基础的模型模拟结果表明,使用多那尼单抗进行限期治疗的 EJP 超过了目前可用的淀粉样蛋白靶向疗法的每剂量清单价格,这意味着可能会降低终生成本并提高性价比。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Estimating the Economically Justifiable Price of Limited-Duration Treatment with Donanemab for Early Symptomatic Alzheimer's Disease in the United States.

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.

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来源期刊
Neurology and Therapy
Neurology and Therapy CLINICAL NEUROLOGY-
CiteScore
5.40
自引率
8.10%
发文量
103
审稿时长
6 weeks
期刊介绍: Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Neurology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research. Rapid Publication The journal’s rapid publication timelines aim for a peer review decision within 2 weeks of submission. If an article is accepted, it will be published online 3-4 weeks from acceptance. These rapid timelines are achieved through the combination of a dedicated in-house editorial team, who closely manage article workflow, and an extensive Editorial and Advisory Board who assist with rapid peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model, this allows for the rapid and efficient communication of the latest research and reviews to support scientific discovery and clinical practice. Open Access All articles published by Neurology and Therapy are open access. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning that authors will always have a personal point of contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. We also encourage pre-submission enquiries and are always happy to provide a confidential assessment of manuscripts. Digital Features and Plain Language Summaries Neurology and Therapy offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by key summary points, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article. The journal also provides the option to include various types of digital features including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations. All additional features are peer reviewed to the same high standard as the article itself. If you consider that your paper would benefit from the inclusion of a digital feature, please let us know. Our editorial team are able to create high-quality slide decks and infographics in-house, and video abstracts through our partner Research Square, and would be happy to assist in any way we can. For further information about digital features, please contact the journal editor (see ‘Contact the Journal’ for email address), and see the ‘Guidelines for digital features and plain language summaries’ document under ‘Submission guidelines’. For examples of digital features please visit our showcase page https://springerhealthcare.com/expertise/publishing-digital-features/ Publication Fees Upon acceptance of an article, authors will be required to pay the mandatory Rapid Service Fee of €5250/$6000/£4300. The journal will consider fee discounts and waivers for developing countries and this is decided on a case-by-case basis. Peer Review Process Upon submission, manuscripts are assessed by the editorial team to ensure they fit within the aims and scope of the journal and are also checked for plagiarism. All suitable submissions are then subject to a comprehensive single-blind peer review. Reviewers are selected based on their relevant expertise and publication history in the subject area. The journal has an extensive pool of editorial and advisory board members who have been selected to assist with peer review based on the afore-mentioned criteria. At least two extensive reviews are required to make the editorial decision, with the exception of some article types such as Commentaries, Editorials and Letters which are generally reviewed by one member of the Editorial Board. Where reviews conflict, an Editorial Board Member will be contacted for further advice and a presiding decision. Manuscripts are then either accepted, rejected or authors are required to make major or minor revisions (both reviewer comments and editorial comments may need to be addressed. Once a revised manuscript is re-submitted, it is assessed along with the responses to reviewer comments and if it has been adequately revised, it will be accepted for publication. Accepted manuscripts are then copyedited and typeset by the production team before online publication. Appeals against decisions following peer review are considered on a case-by-case basis and should be sent to the journal editor, and authors are welcome to make rebuttals against individual reviewer comments, if appropriate. Preprints We encourage posting of preprints of primary research manuscripts on preprint servers, authors'' or institutional websites, and open communications between researchers whether on community preprint servers or preprint commenting platforms. Posting of preprints is not considered prior publication and will not jeopardize consideration in our journals. Please see here for further information on preprint sharing: https://www.springer.com/gp/authors-editors/journal-author/journal-author-helpdesk/submission/1302#c16721550 Copyright Neurology and Therapy is published under the Creative Commons Attribution-Noncommercial License, which allows users to read, copy, distribute, and make derivative works for non-commercial purposes from the material, as long as the author of the original work is cited. The author assigns the exclusive right to any commercial use of the article to Springer. For more information about the Creative Commons Attribution-Noncommercial License, click here: http://creativecommons.org/licenses/by-nc/4.0. Contact For more information about the journal, including pre-submission enquiries, please contact managing editor Lydia Alborn at lydia.alborn@springer.com.
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