[1]闫丽羽,杨伊凡,翁婷婷,等.欧美国家罕见病用药市场独占制度研究及启示[J].卫生经济研究,2023,40(7):89-93.
　YAN Liyu,YANG Yifan,WENG Tingting,et al.Study on the Market Exclusivity System of the Drugs for Rare Disease in European and American Countries and its Enlightenment[J].Journal Press of Health Economics Research,2023,40(7):89-93.
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欧美国家罕见病用药市场独占制度研究及启示
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卫生经济研究[ISSN:1004-7778/CN:33-1056/F]

卷:
40
期数:
2023年7期
页码:
89-93
栏目:
他山之石
出版日期:
2023-06-30

文章信息/Info

Title:
Study on the Market Exclusivity System of the Drugs for Rare Disease in European and American Countries and its Enlightenment
作者:
闫丽羽1; 2; 杨伊凡1; 2; 翁婷婷1; 2; 谢金平1; 2; 邵 蓉1; 2
1.中国药科大学药品监管科学研究院，江苏 南京 211198
2.国家药品监督管理局药品监管创新与评价重点实验室，江苏 南京 211198
Author(s):
YAN Liyu;  YANG Yifan;  WENG Tingting;  XIE Jinping;  SHAO Rong
Institute of Regulatory Science for Medical Products, China Pharmaceutical University, Nanjing Jiangsu 211198, China
关键词:
罕见病用药; 市场独占; 数据保护
Keywords:
drugs for rare disease;  market exclusivity;  data protection
分类号:
R19；R95
文献标志码:
A
摘要:
目的：介绍欧美国家罕见病用药市场独占的概念、保护期，市场独占制度的关键要素及实施效果，为我国制定罕见病用药市场独占制度提供参考。方法：通过文献研究，梳理欧美国家罕见病用药市场独占制度的关键要素、制度差异、实施效果。结果：欧美国家罕见病用药市场独占制度关于“相同药物”“临床优势”等关键要素的界定标准相同、制度内涵一致,均显著激励了罕见病用药研发上市，但也存在对“相同适应症”理解差异、市场独占保护作用减弱、定期评估条款无法发挥作用等问题。结论：我国在制定罕见病用药市场独占制度时应明确制度适用范围、儿科用药市场独占性质，以及“相同品种”“相同适应症”“临床优势”等关键要素的界定标准，以更好地发挥罕见病用药市场独占制度的作用。
Abstract:
Objective To introduce the concept, protection period, key elements and implementation effects of market exclusivity system of the drugs for rare disease in European and American countries, and to provide references for China to establish market exclusivity system of the drugs for rare disease. Methods By literature research, the key elements, system differences and implementation effects of market exclusivity system of the drugs for rare disease in European and American countries were reviewed and compared. Results The market exclusive system of rare disease drugs in European and American countries has the same criteria for defining key elements such as "same drug" and "clinical superior", and the same connotation of the system, which has significantly stimulated the research, development and marketing of rare disease drugs. However, there were also some problems, such as the differences in understanding the "same indications", weakened market exclusivity protection, and the failure of the terms of regular evaluation. Conclusion When establishing market exclusive system of rare disease drugs in China, the government should clarify the scope of application of the system, the nature of market exclusive of pediatric drugs, and the standards of key elements such as "same variety", "same indication", "clinical superior", so as to better play the role of market exclusive system of rare disease drugs.

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更新日期/Last Update: 2023-06-30