摘要
罕见病用药的界定和认定标准是实施罕见病用药研发、生产、供应等激励政策的基础。域外很多国家结合国情制定了本国孤儿药的认定标准。美国孤儿药认定标准最为宽松,符合流行病学或者预期收益不足以弥补研发成本的药品,均可以获得孤儿药认定;欧盟在美国的基础上增加了“疾病严重程度”和“显著收益”要求,对于孤儿亚组的认定也较为严格;日本、韩国均强调孤儿药的研发可能性;澳大利亚更侧重于孤儿药的引进,对其认定的孤儿药设置了6个月的孤儿药资格有效期。结合我国罕见病及罕见病用药的管理现状,建议相关部门应当首先明确罕见病定义,在此基础上从成本-收益、疾病严重程度、显著收益、研发可能性及临床优势等方面对罕见病用药的认定标准进行细化和考量,并制定配套的激励政策,进一步提升罕见病用药的可及性。
The definition and designation standards of rare diseases drugs are the basis for implementing incentive policies such as research and development,production and supply concerning rare diseases.The designation standards for rare diseases drugs(orphan drugs)have been formulated in some foreign countries according to their national conditions.The designation of orphan drugs in the U.S.is loosest,drugs could be designated when it meeting the epidemiology standard or lacking of commercial viability.Based on the U.S.’s standards,the EU not only has stricter epidemiological standards,but also requires the severity of the diseases and significant benefits of the drugs.Both Japan and South Korea emphasize that drugs should be potential to research.Australia is more inclined to introduce drugs that have been developed abroad,therefore,a six-month validity period was set for their orphan drug designation.China should clarify the definition of rare diseases firstly,and refine the designation standards from commercial in viability,severity of the diseases,significant benefits of the drugs,development potential and clinical superiority,etc.Drawing up corresponding incentive policies is also necessary to improve the accessibility of drugs for rare diseases.
作者
谢金平
闫丽羽
杨伊凡
翁婷婷
徐欣
邵蓉
XIE Jin-ping;YAN Li-yu;YANG Yi-fan;WENG Ting-ting;XU Xin;SHAO Rong(Institute of Regulatory Science for Medical Products/NMPA Key Laboratory for Drug Regulatory Innovation and Evaluation,China Pharmaceutical University,Nanjing JIANGSU 211198,China;Shanghai Pharma Rare Disease Medicine Co.Ltd.,SHANGHAI 200072,China)
出处
《中国新药与临床杂志》
CAS
CSCD
北大核心
2023年第2期78-84,共7页
Chinese Journal of New Drugs and Clinical Remedies
关键词
罕见病
孤儿药
认定标准
成本和收益
研究与开发
rare diseases
orphan drugs
designation standards
costs and benefits
research and development
作者简介
谢金平,女,讲师,博士,主要从事医药政策法规的研究,E-mail:15151870834@163.com;责任作者:邵蓉,女,教授,博士生导师,博士,主要从事医药政策法规的研究,E-mail:shaorong118@163.com。
