澳大利亚、新加坡、韩国、英国和美国国家罕见病资助政策比较分析:范围界定审查。

IF 2.7 3区 经济学 Q1 ECONOMICS
Qin Xiang Ng, Clarence Ong, Kai En Chan, Timothy Sheng Khai Ong, Isabelle Jia Xuan Lim, Ansel Shao Pin Tang, Hwei Wuen Chan, Gerald Choon Huat Koh
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引用次数: 0

摘要

背景:罕见病由于发病率低、伴有残疾以及随之而来的治疗费用,给医疗系统带来了巨大挑战。基因疗法的进步,如脊髓性肌肉萎缩症(SMA)的治疗,为人们带来了新的治疗选择,但高昂的费用(Zolgensma®的治疗费用为 210 万美元)带来了巨大的经济障碍。本范围界定综述旨在比较澳大利亚、新加坡、韩国、英国和美国的高绩效卫生系统中罕见病治疗的资助方法,以确定最佳实践和未来研究领域:根据 PRISMA-ScR 指南、Arksey 和 O'Malley 提出的方法框架及随后的建议,对电子数据库(Medline、EMBASE 和 Cochrane)以及这些国家卫生部门网站和致力于罕见病研究的主要国家组织的灰色文献进行了全面检索。选择进行比较的国家都是经济发达、卫生系统运行良好的高收入国家:澳大利亚、新加坡、韩国、英国和美国。纳入标准侧重于 2010 年至 2024 年间发表的详细介绍药物审批流程、报销决定和筹资机制的研究:根据对 18 篇已发表论文和灰色文献的全面审查,各国采用了各种策略来平衡预算限制和罕见病治疗的可及性。澳大利亚利用 "救生药物计划 "和风险分担协议。新加坡依靠罕见病基金,为公众捐款提供配比。韩国的国民健康保险服务通过风险分担协议覆盖特定的孤儿药。英国在创新药物基金的支持下,依靠国家健康与护理卓越研究所(NICE)对治疗的成本效益进行评估。在美国,则结合使用联邦和州计划、私人保险和非营利性支持:以成果为基础的风险分担协议为管理昂贵治疗的经济压力提供了一个切实可行的解决方案。这些协议将付款与实际疗效挂钩,从而分散了财务风险并促进了持续的数据收集。各国应考虑采用并扩大这些协议,以平衡眼前支出与长期利益,最终确保罕见病患者公平地获得关键治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review.

Background: Rare diseases pose immense challenges for healthcare systems due to their low prevalence, associated disabilities, and attendant treatment costs. Advancements in gene therapy, such as treatments for Spinal Muscular Atrophy (SMA), have introduced novel therapeutic options, but the high costs, exemplified by Zolgensma® at US$2.1 million, present significant financial barriers. This scoping review aimed to compare the funding approaches for rare disease treatments across high-performing health systems in Australia, Singapore, South Korea, the United Kingdom (UK), and the United States (US), aiming to identify best practices and areas for future research.

Methods: In accordance with the PRISMA-ScR guidelines and the methodological framework by Arksey and O'Malley and ensuing recommendations, a comprehensive search of electronic databases (Medline, EMBASE, and Cochrane) and grey literature from health department websites and leading national organizations dedicated to rare diseases in these countries was conducted. Countries selected for comparison were high-income countries with advanced economies and high-performing health systems: Australia, Singapore, South Korea, the UK, and the US. The inclusion criteria focused on studies detailing drug approval processes, reimbursement decisions and funding mechanisms, and published from 2010 to 2024.

Results: Based on a thorough review of 18 published papers and grey literature, various strategies are employed by countries to balance budgetary constraints and access to rare disease treatments. Australia utilizes the Life Saving Drugs Program and risk-sharing agreements. Singapore depends on the Rare Disease Fund, which matches public donations. South Korea's National Health Insurance Service covers specific orphan drugs through risk-sharing agreements. The UK relies on the National Institute for Health and Care Excellence (NICE) to evaluate treatments for cost-effectiveness, supported by the Innovative Medicines Fund. In the US, a combination of federal and state programs, private insurance and non-profit support is used.

Conclusion: Outcome-based risk-sharing agreements present a practical solution for managing the financial strain of costly treatments. These agreements tie payment to actual treatment efficacy, thereby distributing financial risk and promoting ongoing data collection. Countries should consider adopting and expanding these agreements to balance immediate expenses with long-term benefits, ultimately ensuring equitable access to crucial treatments for patients afflicted by rare diseases.

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来源期刊
CiteScore
3.90
自引率
4.20%
发文量
59
审稿时长
13 weeks
期刊介绍: Health Economics Review is an international high-quality journal covering all fields of Health Economics. A broad range of theoretical contributions, empirical studies and analyses of health policy with a health economic focus will be considered for publication. Its scope includes macro- and microeconomics of health care financing, health insurance and reimbursement as well as health economic evaluation, health services research and health policy analysis. Further research topics are the individual and institutional aspects of health care management and the growing importance of health care in developing countries.
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