[1]王珺琦,殷 悦,唐文熙.医保准入视角下罕见病用药经济价值评估的 难点、方法与建议[J].卫生经济研究,2024,41(09):58-61.
　WANG Junqi,YIN Yue,TANG Wenxi.Difficulties, Methods, and Suggestions for Assessing the Economic Value of Rare Disease Medications from the Perspective of Health Insurance Access[J].Journal Press of Health Economics Research,2024,41(09):58-61.
点击复制

医保准入视角下罕见病用药经济价值评估的 难点、方法与建议
分享到：

卫生经济研究[ISSN:1004-7778/CN:33-1056/F]

卷:
41
期数:
2024年09期
页码:
58-61
栏目:
经济学评价
出版日期:
2024-08-29

文章信息/Info

Title:
Difficulties, Methods, and Suggestions for Assessing the Economic Value of Rare Disease Medications from the Perspective of Health Insurance Access
作者:
王珺琦1; 殷 悦1; 唐文熙1
1.中国药科大学国际医药商学院，江苏 南京 211198
Author(s):
WANG Junqi;  YIN Yue;  TANG Wenxi
School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing Jiangsu 211198, China
关键词:
罕见病; 孤儿药; 经济价值; 医保准入
Keywords:
rare disease;  orphan drugs;  economic value;  health insurance access
分类号:
R95
文献标志码:
A
摘要:
罕见病用药的经济价值评估在临床试验、经济学评价方法、标准及价值维度等方面存在着诸多不足，各国对此积极探索，比如基于自然史数据库构建对照臂、基于健康状态偏好测量健康状态效用、根据健康获益水平调整支付阈值、纳入更多价值维度。借鉴国外经验，我国可建立高质量和高效率的罕见病真实世界数据库，推动效用和偏好研究，引导罕见病单独阈值和多维价值研究，加快构建罕见病多层次医保体系，从而促进罕见病用药经济价值评估的高质量发展。
Abstract:
In the economic value evaluation of rare disease drugs, there are many shortcomings in clinical trials, economic evaluation methods, standards, and value dimensions. Various countries are actively exploring this, such as building control arms based on natural history databases, measuring health status utility based on health status preferences, adjusting payment thresholds based on health benefit levels, and incorporating more value dimensions. Drawing on foreign experience, China can establish a high-quality and efficient real-world database for rare diseases, promote utility and preference studies, guide the research on individual thresholds and multidimensional value for rare diseases, and accelerate the construction of a multilevel health insurance system for rare diseases, so as to promote the high-quality development of the economic value evaluation of medication for rare diseases.

参考文献/References:

[1] 胡嘉浩，康琦，朱琳，等.罕见病用药经济学评价的挑战与中国对策思考[J].世界临床药物，2022，43（02）：109-114.
[2] Michaeli T,Jürges H,Michaeli D T.FDA approval, clinical trial evidence, efficacy,epidemiology,and price for non-orphan and ultra-rare, rare,and common orphan cancer drug indications: cross sectional analysis[J].Bmj,2023,381:e073242.
[3] Wu X,Xu W,Yu M,et al.Clinical trials of orphan drugs in China over the decade 2012-2022:Opportunities and challenges[J].Pharmacol Res,2022,182:106349.
[4] Mellerio J E.The challenges of clinical trials in rare diseases[J]. Br J Dermatol,2022,187(04):453-454.
[5] Paller A S,Browning J,Nikolic M,et al.Efficacy and tolerability of the investigational topical cream SD-101(6% allantoin) in patients with epidermolysis bullosa:a phase 3, randomized,double-blind, vehicle-controlled trial(ESSENCE study)[J].Orphanet J Rare Dis, 2020,15(01):158.
[6] Solis D C,Teng C,Gorell E S,et al.Classification of 2 distinct wound types in recessive dystrophic epidermolysis bullosa:a retrospective and cohort natural history study[J].J Am Acad Dermatol, 2021,85(05):1296-1298.
[7] US Food and Drug Administration.Epidermolysis bullosa: developing drugs for treatment of cutaneous manifestations;guidance for industry[EB/OL].[2024-02-21].https://www.fda.gov/regulatory-information/search-fda-guidance-documents/epidermolysis-bullosa-developing-drugs-treatment-cutaneous-manifestations-guidance-industry.
[8] Improved Methods and Actionable Tools for Enhancing HTA.WP10 - HTA Appraisal of Orphan Medicinal Products[EB/OL].[2024-02-21].https://www.impact-hta.eu/country-vignettes.
[9] Blonda A,Denier Y,Huys I, et al.How to value Orphan drugs?A review of European value assessment frameworks[J].Front Pharmacol, 2021,12:631527.
[10] Zorginstituut Nederland.Cost-effectiveness in practice[EB/OL]. [2024-02-21].https://english.zorginstituutnederland.nl/binaries/zinl- eng/documenten/reports/2015/06/16/cost-effectiveness-in-practice/Cost-effectiveness+in+practice.pdf.
[11] 中国罕见病联盟. 《罕见病药物卫生技术评估专家共识：2019版》[EB/OL]. [2024-02-21].https://yd.qq.com/web/bookDetail/91 732350727e1212917af64.
[12] Meregaglia M,Nicod E,Drummond M.The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? A scoping review[J].Eur J Health Econ,2023,24(07):1151-1216.
[13] Wilson E C,Shulgina L,Cahn A P,et al.Treating idiopathic pulmonary fibrosis with the addition of co-trimoxazole: an economic evaluation alongside a randomised controlled trial[J].Pharmacoeconomics,2014,32(01):87-99.
[14] Bagley N,Berger B,Chandra A,et al.The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century[J].Innovation Policy and the Economy,2019,19:97-137.
[15] 北京协和医院罕见病多学科协作组，中国罕见病联盟.多准则决策分析应用于罕见病药品临床综合评价的专家共识（2022）[J].协和医学杂志，2022，13（02）：235-254.
[16] 李戈，孙凤.设置外对照的单臂试验研究现状及其偏倚风险评估案例分析[J].中国药物评价，2022，39（05）：363-369.
[17] Benton M,Skinner M W,Garrison L P,et al.A preliminary analysis of hemophilia patient utility study of treatment administration impact:a discrete choice experiment(dce) using TIME TRADE-OFF (TTO) methodology[J].Haemophilia,2023,29:59-59.
[18] Institute for Clinical and Economic Review.Gene Therapy for Hemophillia B and An Update on Gene Therapy for Hemophillia A：Effectiveness and Value[EB/OL].[2024-02-21].https://icer.org/ wp-content/uploads/2022/05/ICER_Hemophilia_Draft-Evidence- Report_091322.pdf.
[19] National Institute for Health and Care Excellence.Interim process and methods of the highly specialised technologies Programme updated to reflect 2017 changes[EB/OL].[2024-02-21].https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-highly-specialised-technologies-guidance/HST-interim-methods-process-guide-may-17.pdf.
[20] 刘国恩.中国药物经济学评价指南2020[M].北京：中国市场出版社，2020.
[21] Australian Government Department of Health and Aged Care. About the Life Saving Drugs Program[EB/OL].[2024-02-21].https://www.health.gov.au/our-work/life-saving-drugs-program/about-the-lsdp.
[22] National Institute for Health and Care Excellence.NICE Highly Specialized Technologies Guidance[EB/OL].[2024-02-21].https://www.nice.org.uk/About/What-we-do/Our-Programmes/NICE-guidance/NICE-highly-specialised-technologies-guidance.
[23] Malinowski K P,Kawalec P,Trabka W,et al.Reimbursement of Orphan Drugs in Europe in relation to the type of authorization by the european medicines agency and the decision making based on health technology assessment[J].Front Pharmacol,2018,9:1263.

相似文献/References:

[1]陈馨仪,符 晓,王安石,等.罕见病患者治疗及保障研究[J].卫生经济研究,2020,(09):40.
　CHEN Xin-yi,FU Xiao,WANG An-shi,et al.Research on the Treatment and Guarantee of Patients with Rare Diseases[J].Journal Press of Health Economics Research,2020,(09):40.
[2]王子琪,闵连星,田兴军,等.我国罕见病医保政策的现状及优化路径[J].卫生经济研究,2021,38(11):22.
　WANG Zi-qi,MIN Lian-xing,TIAN Xing-jun,et al.The Situation and Optimization Path of Medical Insurance Policy for Rare Diseases in China[J].Journal Press of Health Economics Research,2021,38(09):22.
[3]陈子梦,王 丽,章 浴,等.基于供应链理论的我国孤儿药可及性分析与建议[J].卫生经济研究,2022,39(6):55.
　CHEN Zimeng,WANG Li,ZHANG Yu,et al.Analysis and Suggestions on the Accessibility of Orphan Medicines in China Based on Supply Chain Theory[J].Journal Press of Health Economics Research,2022,39(09):55.
[4]李诗杨,殷彩琴,周 楠,等.“互联网+”背景下孤儿药可及性提升策略研究——基于长尾效应[J].卫生经济研究,2022,39(9):49.
　LI Shiyang,YIN Caiqin,ZHOU Nan,et al.Study on the Strategy of Improving the Accessibility of "Internet +" Orphan Drug——Based on the Long Tail Effect[J].Journal Press of Health Economics Research,2022,39(09):49.
[5]张永理,张开然.我国罕见病患者医疗保障现状及改进建议[J].卫生经济研究,2023,40(1):53.
　ZHANG Yongli,ZHANG Kairan.Medical Security Status and Improvement Suggestions for Patients with Rare Diseases in China[J].Journal Press of Health Economics Research,2023,40(09):53.
[6]朱 坤sup>.中国罕见病保障机制：现状、挑战与建议[J].卫生经济研究,2024,41(01):23.
　ZHU Kun.Security Mechanism for Rare Diseases in China: Progress，Problems and Suggestions[J].Journal Press of Health Economics Research,2024,41(09):23.
[7]谭清立,丘丽莹.城市普惠型商业医疗保险比较研究[J].卫生经济研究,2024,41(02):23.
　TAN Qingli,QIU Liying.Comparative Study on Urban Inclusive Commercial Medical Insurance[J].Journal Press of Health Economics Research,2024,41(09):23.
[8]林 星,董田甜.罕见病药品保障模式的国际经验与启示 ——以酶替代疗法药品为例[J].卫生经济研究,2024,41(11):54.
　LIN Xing,DONG Tiantian.International Experience and Enlightenment of Drug Security Model for Rare Diseases ——Taking Enzyme Replacement Therapy Drugs as an Example[J].Journal Press of Health Economics Research,2024,41(09):54.
[9]沈杨阳.中国罕见病问题及综合保障机制研究[J].卫生经济研究,2024,41(12):1.
　SHEN Yangyang.Study on the Problems of Rare Diseases and Comprehensive Protection Mechanisms in China[J].Journal Press of Health Economics Research,2024,41(09):1.
[10]刘慧玲,赵恒伯,谢志勤,等.2022年江西省罕见病治疗费用核算分析 ——基于SHA2011[J].卫生经济研究,2025,42(02):46.
　LIU Huiling,ZHAO Hengbo,XIE Zhiqin,et al.Analysis of Rare Disease Treatment Cost Accounting in Jiangxi Province in 2022 ——Based on SHA2011[J].Journal Press of Health Economics Research,2025,42(09):46.
[11]胡善联.国内外罕见病的保障政策研究[J].卫生经济研究,2018,(05):3.
[12]李奕昊,唐贵忠.罕见病用药可负担性评价及医保政策研究[J].卫生经济研究,2022,39(4):53.
　LI Yi-hao,TANG Gui-zhong.Affordability Evaluation of Drugs for Rare Diseases and Research on Medical Insurance Policy[J].Journal Press of Health Economics Research,2022,39(09):53.

更新日期/Last Update: 2024-08-29