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法国和英国新药健康技术评估的比较:以用于复发或难治性多发性骨髓瘤患者的伊沙佐米为例。

Comparison of health technology assessment for new medicines in France and England: an example based on ixazomib for patients with relapsed or refractory multiple myeloma.

作者信息

Armoiry Xavier, Spath Hans-Martin, Clarke Aileen, Connock Martin, Sutcliffe Paul, Dussart Claude

机构信息

Pharmacy Department, University of Lyon, School of Pharmacy (ISPB)/UMR CNRS 5510 MATEIS/Lyon University Hospitals, "Edouard Herriot" Hospital, Lyon, France.

Warwick Medical School, University of Warwick, Coventry, UK.

出版信息

J Mark Access Health Policy. 2019 Jul 30;7(1):1648971. doi: 10.1080/20016689.2019.1648971. eCollection 2019.

DOI:10.1080/20016689.2019.1648971
PMID:31489149
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6711145/
Abstract

: The appraisal of medicines is often a complex and iterative process. We compared the health technology assessment (HTA) process in England and France taking as a case study the example of ixazomib for multiple myeloma. : We undertook an analysis of eight relevant published documents identifed from the websites of the French and English HTA bodies (HAS and NICE, respectively). We analyse patients' availability of ixazomib resulting in the different stages of the appraisal process. : We identified differences in the assessment, one of these being the use of an appraisal scope in England allowing the differentiation of populations and comparators according to previously approved treatments. Ixazomib became available earlier in France as part of an early access programme, but the availability was soon discontinued for newly eligible patients following an HAS determination that Ixazomib yielded no additional benefit. This opinion resulted in long pricing discussions. In England, despite the absence of an early access programme and following a process that included cost-effectiveness evaluation combined with pricing discussions, the medicine was fairly rapidly recommended for use. : Differences in the HTA process may result in appreciable differences in time from marketing authorisation to health service adoption of newly licensed drugs.

摘要

药品评估通常是一个复杂且反复的过程。我们以用于治疗多发性骨髓瘤的伊沙佐米为例,比较了英国和法国的卫生技术评估(HTA)过程。我们对分别从法国和英国的HTA机构(分别为HAS和NICE)网站上识别出的八份相关已发表文件进行了分析。我们分析了伊沙佐米在评估过程不同阶段的可及性情况。我们发现了评估过程中的差异,其中之一是英国使用评估范围,允许根据先前批准的治疗方法区分人群和对照药物。伊沙佐米作为早期准入计划的一部分,在法国更早可及,但在HAS判定伊沙佐米未产生额外益处后,新符合条件的患者很快就无法再使用该药。这一观点引发了漫长的定价讨论。在英国,尽管没有早期准入计划,且经过了包括成本效益评估和定价讨论在内的过程,但该药还是很快被推荐使用。HTA过程的差异可能导致从新药上市许可到被卫生服务采用的时间出现明显差异。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/606c/6711145/19ce7466895b/ZJMA_A_1648971_F0001_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/606c/6711145/19ce7466895b/ZJMA_A_1648971_F0001_OC.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/606c/6711145/19ce7466895b/ZJMA_A_1648971_F0001_OC.jpg

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本文引用的文献

1
Ixazomib for Relapsed or Refractory Multiple Myeloma: Review from an Evidence Review Group on a NICE Single Technology Appraisal.伊沙佐米治疗复发/难治性多发性骨髓瘤:来自 NICE 单一技术评估证据审查组的综述。
Pharmacoeconomics. 2018 Sep;36(9):1073-1081. doi: 10.1007/s40273-018-0644-3.
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NICE, the NHS, and Cancer Drugs.英国国家卫生与临床优化研究所、英国国家医疗服务体系与癌症药物
JAMA. 2018 Feb 27;319(8):767-768. doi: 10.1001/jama.2017.20552.
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Analysis of health economics assessment reports for pharmaceuticals in France - understanding the underlying philosophy of CEESP assessment.
法国药品健康经济学评估报告分析——理解CEESP评估的基本理念
J Mark Access Health Policy. 2017 Jul 16;5(1):1344088. doi: 10.1080/20016689.2017.1344088. eCollection 2017.
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[The French medecine pricing committee].[法国药品定价委员会]
Ann Pharm Fr. 2017 Sep;75(5):373-384. doi: 10.1016/j.pharma.2017.04.005. Epub 2017 Jun 2.
5
Oral Ixazomib, Lenalidomide, and Dexamethasone for Multiple Myeloma.来那度胺、伊沙佐米和地塞米松联合治疗多发性骨髓瘤。
N Engl J Med. 2016 Apr 28;374(17):1621-34. doi: 10.1056/NEJMoa1516282.
6
The NICE cost-effectiveness threshold: what it is and what that means.英国国家卫生与临床优化研究所(NICE)的成本效益阈值:是什么以及意味着什么。
Pharmacoeconomics. 2008;26(9):733-44. doi: 10.2165/00019053-200826090-00004.