作者投稿
专家审稿
编辑办公
主编办公
Practice of the Qualification and Recognition for Orphan Drugs in the World and its Inspiration
-
摘要: 分析目前国内外罕见病药品资格认定现状，包括其负责机构、认定流程、认定及上市数量等。通过横向对比，分析典型发达国家和部分发展中国家在罕见病药品资格认定方面的差异，探究中国在罕见病药品监管方面的不足。借鉴典型发达国家和部分发展中国家的先进经验，以期为中国罕见病药品认定管理提供建议，为加快中国罕见病药品研发上市，为最大程度提高患者治疗可及性提供参考。Abstract: We have analyzed the current status of recognization and qualification of orphan drugs in China and abroad, looking at the aspects of the authority institutions, identification and qualification process, and the number of orphan drugs identified and available in the market. By comparing and analyzing horizontally the differences in orphan drugs identification between representative developed countries vs. some developing countries, we discuss the inadequacy of orphan drugs supervision in China. We introduce the advanced experience from the developed countries and some developing countries to provide suggestions for the identification and management of orphan drugs, hoping to speed up the process of development and market availability of orphan drugs and to maximize patient's accessibility to treatment in China.
-
图 1 美国罕见病药品认定流程
OOPD：罕见病药品研发办公室
Figure 1. Identification process of rare diseases drugs in the United States
图 2 美国罕见病药品认定与上市数量
Figure 2. Identification and marketing quantity of rare disease drugs in the United States
图 3 欧盟罕见病药品认定流程
EMA：欧洲药品管理局；COMP：罕见病药品委员会；EC：欧盟委员会
Figure 3. Identification process of rare diseases drugs in EU
图 4 欧盟罕见病药品认定与上市数量
Figure 4. Identification and marketing quantity of rare disease drugs in EU
图 5 日本罕见病药品认定流程
MHLW：日本厚生劳动省；PMDA：医药品医疗器械综合管理机构；PAFSC：药事和食品卫生委员会
Figure 5. Identification process of rare disease drugs in Japan
图 6 日本罕见病药品认定与上市数量
Figure 6. Identification and marketing quantity of rare disease drugs in Japan
表 2 部分发展中国家罕见病领域发展汇总
Table 2. Summary of development for rare diseases in some developing countries
国家 罕见病定义 罕见病药品认定标准 专项立法 认定监管部门 研发激励政策 印度 无* 治疗＜50万患者的药物 无* 无* 国家罕见病治疗政策；资金支持 墨西哥 采用欧盟定义 无* 《普通卫生法》224条修订 无* 加速批准 巴西 患病率低于0.65‰的疾病 无* 无* 无* 罕见病国家政策；优先审查 哥伦比亚 患病率低于0.2‰的疾病 无* 第1392(2010)号法律 无* 无* 秘鲁 无* 采用欧盟定义 第29698(2011)号法律 无* 无* 阿根廷 采用欧盟定义 无* 第26.689(2011)号法律 无* 无* *截至2021年12月31日尚未颁布相关文件 
下载: 导出CSV
表 1 典型发达国家及地区罕见病药品认定情况汇总
Table 1. Summary of drug identification for rare diseases in typical developed countries and regions
分类 美国 欧盟 日本 罕见病定义 患病人数低于20万(总人口的0.75‰)或超过20万无商业回报的疾病 低于0.5‰；严重性、危及生命的、退化性；无可替代治疗的疾病 患病人数低于5万人(总人口0.4‰)；病因不明；无有效治疗方式；严重经济、心理负担的疾病 立法基础 《孤儿药法案》(Orphan Drug Act，ODA) 《罕见病药品条例》(Regulation(EC) No 141/2000 on orphan medicinal products，EC 141/2000) 《药事法》(Pharmaceutical Affairs Law) 监管部门 FDA EMA MHLW 认定标准 用于预防、诊断、治疗美国罕见病的药物 用于预防、诊断、治疗欧盟罕见病的药物 治疗日本罕见病；具备清楚的药品研发计划和支持上市的科学依据 认定机构 OOPD COMP MHLW 认定总数(个)* 5049 1762 438 上市总数(个)* 859 151 344 上市数量占比(%) 17.0 8.6 78.5 *认定总数与上市总数的时间范围：本国法案颁布之日至2021年12月31日；上市数量占比：上市总数/认定总数×100%；EC：欧盟委员会，FDA：美国食品药品监督管理局，EMA：欧洲药品管理局，MHLW：日本厚生劳动省，OOPD：罕见病药品研发办公室，COMP：罕见病药品委员会
下载: 导出CSV
-
[1] Martínez-deMiguel C, Segura-Bedmar I, Chacón-Solano E, et al. The RareDis corpus: a corpus annotated with rare diseases, their signs and symptoms[J]. J Biomed Inform, 2022, 125: 103961. doi: 10.1016/j.jbi.2021.103961 [2] Chan AYL, Chan VKY, Olsson S, et al. Access and unmet needs of orphan drugs in 194 countries and 6 areas: A Global Policy Review With Content Analysis[J]. Value Health, 2020, 23: 1580-1591. doi: 10.1016/j.jval.2020.06.020 [3] Belousova Olga A, Groen Aard J, Ouendag Aniek M. Opportunities and barriers for innovation and entrepreneurship in orphan drug development[J]. Technological Forecasting & Social Change, 2020, 161: 120333. [4] 冀希炜, 梁家彬, 季双敏. 罕见病治疗国内外的研究现状[J]. 中国临床药理学杂志, 2019, 3: 305-308. https://www.cnki.com.cn/Article/CJFDTOTAL-GLYZ201903032.htm [5] Food and Drug Administration. Orphan Drug Act-Relevant Excerpts[EB/OL]. (2018-09-03)[2022-05-08]. https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-act-relevant-excerpts. [6] Seoane-Vazquez E, Rodriguez-Monguio R, Szeinbach SL, et al. Incentives for orphan drug research and development in the United States[J]. Orphanet J Rare Dis, 2008, 12: 3-33. [7] Patel S, Miller Needleman KI. FDA's Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases[J]. J Pharmacokinet Pharmacodyn, 2019, 46: 387-393. doi: 10.1007/s10928-019-09645-4 [8] Food and Drug Administration. Search Orphan Drug Designations and Approvals[EB/OL]. (2017-10-24)[2022-05-18]. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm#Search_param_field#Search_param_field. [9] European Medicines Agency. Orphan designation: Overview[EB/OL]. (2021-01-01)[2022-05-18]. https://www.ema.europa.eu/en/human-regulatory/overview/orph-an-designation-overview. [10] European Medicines Agency. Legal framework: orphan designation[EB/OL]. (2021-01-01)[2022-05-18]. https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation/legal-framework-orphan-designation#regulation-(ec)-no-141/2000-(the-orphan-regulation)-section. [11] European Medicines Agency. Committee for Orphan Medicinal Products (COMP)[EB/OL]. (2021-10-11)[2022-05-18]. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/comp-rules-procedure_en.pdf. [12] European Medicines Agency. Applying for orphan designa-tion[EB/OL]. (2021-01-01)[2022-05-08]. https://www.ema.europa.eu/en/human-regulatory/research-develop-ment/orphan-designation/applying-orphan-designation. [13] European Commission. Community Register of orphan medicinal products[EB/OL]. (2022-05-08)[2022-05-18]. https://ec.europa.eu/health/documents/community-register/html/reg_od_act.htm?sort=n. [14] 厚生劳动省. 稀有疾病药品、罕见疾病医疗器械、罕见疾病再生医学等产品的认定制度概述[EB/OL]. (2022-05-08)[2022-05-18]. https://www.mhlw.go.jp/stf/seisakunitsuite/bunya/0000068484.html. [15] Sakushima K, Takeda H, Aoi Y. Orphan drug designation and development in Japan: 25 years of experience and assessment[J]. Nat Rev Drug Discov, 2021, 20: 893-894. doi: 10.1038/d41573-021-00045-3 [16] Song P, Gao J, Inagaki Y, et al. Rare diseases, orphan drugs, and their regulation in Asia: current status and future perspectives[J]. Intractable Rare Dis Res, 2012, 1: 3-9. [17] 丁志琛, 韦冠, 丁锦希. 日本罕用药制度及其对中国的启示——基于对日本罕用药可及性的评价[J]. 中国药科大学学报, 2014, 45: 118-124. https://www.cnki.com.cn/Article/CJFDTOTAL-ZGYD201401028.htm [18] Pharmaceuticals and Medical Devices Agency. List of Approved Products[EB/OL]. (2022-05-08)[2022-05-18]. https://www.nibiohn.go.jp/nibio/part/promote/files/orphan%20drug%20E.pdf. [19] Biglia LV, Mendes SJ, Lima TM, et al. Incorporation of drugs for rare diseases in Brazil: is it possible to have full access to these patients?[J]. Cien Saude Colet, 2021, 26: 5547-5560. doi: 10.1590/1413-812320212611.26722020 [20] Mayrides M, Ruiz de Castilla EM, Szelepski S. A civil society view of rare disease public policy in six Latin American countries[J]. Orphanet J Rare Dis, 2020, 15: 60. doi: 10.1186/s13023-020-1314-z [21] Choudhury MC, Saberwal G. The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study[J]. Orphanet J Rare Dis, 2019, 14: 241. doi: 10.1186/s13023-019-1224-0 [22] Khosla N, Valdez R. A compilation of national plans, policies and government actions for rare diseases in 23 countries[J]. Intractable Rare Dis Res, 2018, 7: 213-222. doi: 10.5582/irdr.2018.01085 [23] 牛叔文, 李真, 梁曼. 人文发展的多维测度及其政策启示[J]. 中国软科学, 2021, (05): 99-110. https://www.cnki.com.cn/Article/CJFDTOTAL-ZGRK202105010.htm [24] 国家药品监督管理局. 国家药监局综合司公开征求《中华人民共和国药品管理法实施条例(修订草案征求意见稿)》意见[EB/OL]. (2022-05-09)[2022-05-18]. https://www.nmpa.gov.cn/xxgk/zhqyj/zhqyjyp/20220509222233134.html. -
点击扫码阅读文章
计量
- 文章访问数: 211
- HTML全文浏览量: 85
- PDF下载量: 77
- 被引次数: 0
