美国、加拿大、英格兰和苏格兰通过奥比斯项目审查的 FDA 批准的抗癌药物的临床效益、报销结果和价格:回顾性比较分析。

IF 41.6 1区 医学 Q1 ONCOLOGY
Lancet Oncology Pub Date : 2024-08-01 Epub Date: 2024-07-11 DOI:10.1016/S1470-2045(24)00286-9
Kristina Jenei, Arianna Gentilini, Alyson Haslam, Vinay Prasad
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引用次数: 0

摘要

项目背景奥比斯项目是一项全球性倡议,旨在简化美国、加拿大、澳大利亚、英国、以色列、巴西、新加坡和瑞士等国国际监管机构的监管审查流程,以便更早地为患者提供有前景的抗癌药物。我们探讨了通过该倡议审查的抗癌药物的临床疗效、获得监管部门批准和卫生技术评估建议的时间、报销结果和每月治疗价格:在这项回顾性比较分析中,我们确定了 2019 年 5 月 1 日至 2023 年 11 月 1 日期间美国、加拿大和英国通过奥比斯项目审查的抗癌药物批准情况。Orbis项目审查的抗癌药物批文来自美国食品药品管理局(FDA)肿瘤学卓越中心,FDA的所有其他批文来自Drugs@FDA数据库。共同主要结果包括监管审查时间、从监管审批到卫生技术评估建议(英格兰、苏格兰和加拿大)的时间、报销结果、奥比斯项目审查的抗癌药物审批与其他 FDA 审批程序之间的临床获益(定义为无进展生存期和总生存期的中位收益)以及每月治疗价格。使用Wilcoxon秩和检验和Fisher's Exact检验来检验同期通过Project Orbis审查的批准与其他FDA批准之间的统计显著性:在2019年5月1日至2023年11月1日期间,FDA批准的244种抗癌药物中有81种(33%)是通过Project Orbis审查的。总生存期的中位数为4-1个月(IQR为3-3-5-1),而FDA批准的其他药物为2-7个月(2-1-3-9)。同样,Orbis 项目的无进展生存期收益为 2-6 个月(IQR 1-7-4-9),而 FDA 批准的其他项目为 2-6 个月(0-6-5-1)。总生存期(p=0-11)和无进展生存期(p=0-44)的收益在两组批准项目中均无显著差异。在苏格兰药品联合会(SMC)审查的 14 项英国药品和保健品管理局(MHRA)批准中,该机构对所有 14 项(100%)批准均给出了积极建议。在英国国家健康与护理卓越研究所 (NICE) 审查的 15 项英国医疗与保健品管理局 (MHRA) 批准中,该机构对其中 6 项(40%)给出了积极建议。在加拿大药品和卫生技术局 (CADTH) 审查的 49 项批准中,该机构有条件地推荐了 44 项(90%)。从监管部门批准到 NICE 推荐的时间从 2021 年的中位数 137 天(IQR 102-172)增加到 2023 年的 302 天(184-483),SMC 推荐的时间从 185 天(2021 年仅一种药物)增加到 2023 年的 368 天(IQR 313-476),CADTH 决定的时间从 97 天(2020 年仅一种药物)增加到 2023 年的 202 天(IQR 153-304)。通过奥比斯项目审查的审批价格中位数为每月 20 000 美元(IQR 13 000-37 000):Orbis项目的临床结果与同期美国食品与药物管理局的其他审批结果并无不同,而在成功通过卫生技术评估后,药品的获取却被大大延迟或缺失,这不禁让人质疑Orbis项目的参与是否能让患者更快地获得临床疗效高且成本可控的药品。尽管未来的挑战可能会从监管协调中受益,但其优势目前尚不明确:无。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical benefit, reimbursement outcomes, and prices of FDA-approved cancer drugs reviewed through Project Orbis in the USA, Canada, England, and Scotland: a retrospective, comparative analysis.

Background: Project Orbis is a global initiative that aims to streamline regulatory review processes across international regulators in the USA, Canada, Australia, UK, Israel, Brazil, Singapore, and Switzerland to bring promising cancer drugs to patients earlier. We explored the clinical benefit, time to regulatory approval and health technology assessment recommendations, reimbursement outcomes, and monthly treatment prices of cancer drugs reviewed through this initiative.

Methods: For this retrospective, comparative analysis, we identified cancer drug approvals reviewed through Project Orbis in the USA, Canada, and the UK between May 1, 2019, and Nov 1, 2023. Approvals of cancer drugs reviewed Project Orbis were extracted from the Food and Drug Administration (FDA) Oncology Centre of Excellence and all other FDA approvals from the Drugs@FDA database. The co-primary outcomes were time of regulatory review, time from regulatory approval to health technology assessment recommendation (England, Scotland, and Canada), reimbursement outcomes, clinical benefit (defined as median gains in progression-free survival and overall survival) between cancer drug approvals reviewed by Project Orbis and other FDA approval processes, and monthly treatment prices. The Wilcoxon rank-sum and Fisher's Exact tests were used to examine statistical significance between approvals reviewed through Project Orbis and other FDA approvals during the same period.

Findings: Between May 1, 2019 and Nov 1, 2023, 81 (33%) of 244 cancer drugs approved by the FDA were reviewed through Project Orbis. The median overall survival gains were 4·1 months (IQR 3·3-5·1) compared with 2·7 months (2·1-3·9) for other FDA approvals. Similarly, progression-free survival gains were 2·6 months (IQR 1·7-4·9) for Project Orbis compared with 2·6 months (0·6-5·1) for other FDA approvals. Neither overall survival (p=0·11) nor progression-free survival (p=0·44) gains were significantly different between the two cohorts of approvals. Of the 14 UK Medicines and Healthcare products Regulatory Agency (MHRA) approvals reviewed by the Scottish Medicines Consortium (SMC), the agency gave positive recommendations for all 14 (100%). Of the 15 MHRA approvals reviewed by the National Institute for Health and Care Excellence (NICE), the agency gave positive recommendations for six (40%). Of the 49 approvals reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH), the agency conditionally recommended 44 (90%). The time between regulatory approval to NICE recommendation increased from a median of 137 days (IQR 102-172) in 2021 to 302 days (184-483) in 2023, SMC recommendation increased from 185 days (in 2021 for one drug only) to 368 days (IQR 313-476) in 2023, and CADTH decision increased from 97 days (in 2020 for one drug only) to 202 days (IQR 153-304) in 2023. The median monthly price of approvals reviewed through Project Orbis was US$20 000 per month (IQR 13 000-37 000).

Interpretation: Clinical outcomes of Project Orbis were no different than other FDA approvals during the same time, and access, after a successful health technology assessment, was considerably delayed or absent, raising questions about whether Project Orbis participation translates into faster patient access to medicines with high clinical benefit and sustainable costs. Although future challenges might benefit from regulatory harmonisation, the advantages are currently unclear.

Funding: None.

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来源期刊
Lancet Oncology
Lancet Oncology 医学-肿瘤学
CiteScore
62.10
自引率
1.00%
发文量
913
审稿时长
3-8 weeks
期刊介绍: The Lancet Oncology is a trusted international journal that addresses various topics in clinical practice, health policy, and global oncology. It covers a wide range of cancer types, including breast, endocrine system, gastrointestinal, genitourinary, gynaecological, haematological, head and neck, neurooncology, paediatric, thoracic, sarcoma, and skin cancers. Additionally, it includes articles on epidemiology, cancer prevention and control, supportive care, imaging, and health-care systems. The journal has an Impact Factor of 51.1, making it the leading clinical oncology research journal worldwide. It publishes different types of articles, such as Articles, Reviews, Policy Reviews, Personal Views, Clinical Pictures, Comments, Correspondence, News, and Perspectives. The Lancet Oncology also collaborates with societies, governments, NGOs, and academic centers to publish Series and Commissions that aim to drive positive changes in clinical practice and health policy in areas of global oncology that require attention.
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