Licensed Subcutaneous Infusion Therapies in Advanced Parkinson's Disease: An Indirect Treatment Comparison and Cost-Minimisation Analysis.

IF 4.8 3区 医学 Q1 CLINICAL NEUROLOGY
Neurology and Therapy Pub Date : 2025-10-01 Epub Date: 2025-07-08 DOI:10.1007/s40120-025-00789-9
Marc Vérin, Ángel Sesar, Bharat Amlani, Niall Smith, Konstantinos Kipentzoglou, Stephen Montgomery, Alice Reading, Andrei Karlsson, Anna Willis, Andrew J Lees
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引用次数: 0

Abstract

Introduction: Parkinson's disease is the second most common neurodegenerative disorder. In advanced Parkinson's disease, subcutaneous (SC) infusion therapies represent minimally invasive and reversible treatment options. In the United Kingdom (UK), licensed SC infusion therapies include apomorphine and foslevodopa-foscarbidopa; both represent effective and generally well-tolerated therapies, although uncertainties regarding their relative efficacy, safety and costs remain.

Methods: The relative efficacy and safety of apomorphine and foslevodopa-foscarbidopa was assessed via Bucher indirect treatment comparison (ITC) of TOLEDO (NCT02006121) and M15-736 (NCT04380142) data, with findings used to support a cost-minimisation analysis (CMM). Thirteen outcomes were evaluated. Efficacy and safety outcomes were measured as mean differences and risk differences, respectively. The CMM, conducted from a UK healthcare payer perspective, considered treatment acquisition and concomitant therapy costs over a 6.34-year horizon (obtained from a published observational study).

Results: Bucher ITC results provided evidence for a comparable efficacy for apomorphine and foslevodopa-foscarbidopa in advanced Parkinson's disease. ITCs also indicated comparable safety, although a trend in favour of apomorphine was identified for hallucinations and most infusion site reactions assessed. The CMM demonstrated a clear per-patient cost benefit for apomorphine versus foslevodopa-foscarbidopa (£120,173.70), primarily driven by lower drug acquisition costs.

Conclusion: The main difference between UK licensed SC infusion therapies for advanced Parkinson's disease relates to cost as opposed to clinical outcome, with some evidence for improved tolerability of apomorphine. This supports the continued use of apomorphine as first-line SC infusion treatment for advanced Parkinson's disease in UK clinical practice.

Abstract Image

Abstract Image

晚期帕金森病的许可皮下输注疗法:间接治疗比较和成本最小化分析。
帕金森病是第二常见的神经退行性疾病。在晚期帕金森病中,皮下(SC)输注治疗是微创和可逆的治疗选择。在英国,获得许可的SC输注疗法包括阿波啡和foslevodopa-foscarbidopa;尽管它们的相对疗效、安全性和成本仍然存在不确定性,但这两种疗法都是有效且通常耐受性良好的疗法。方法:通过TOLEDO (NCT02006121)和M15-736 (NCT04380142)的Bucher间接治疗比较(ITC)数据,评估阿波吗啡和foslevodopa-foscarbidopa的相对疗效和安全性,结果用于支持成本最小化分析(CMM)。评估了13项结果。疗效和安全性结果分别以平均差异和风险差异来衡量。CMM是从英国医疗保健支付者的角度进行的,考虑了6.34年的治疗获得和伴随治疗费用(来自一项已发表的观察性研究)。结果:Bucher ITC结果为阿波啡和fosleovdopa -foscarbidopa治疗晚期帕金森病的疗效相当提供了证据。ITCs也显示出相当的安全性,尽管有一种倾向于阿波啡治疗幻觉的趋势,并评估了大多数输液部位的反应。CMM显示阿波啡相对于foslevodopa-foscarbidopa有明显的每名患者成本优势(120,173.70英镑),主要是由于较低的药物获取成本。结论:英国许可的SC输注治疗晚期帕金森病的主要区别在于成本而不是临床结果,有一些证据表明阿波啡的耐受性得到改善。这支持在英国临床实践中继续使用阿波啡作为晚期帕金森病的一线SC输注治疗。
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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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