国内外罕见病领域研究进展对比分析

发布时间：2018-02-04 19:24

本文关键词： 罕见病孤儿药研究进展国内外对比分析　出处：《中国药学杂志》2017年06期 　论文类型：期刊论文

【摘要】：目的研究整理国内外罕见病领域研究文献,并进行对比分析,找出国内罕见病领域的相关研究不足与差距,为国内相关研究提供建议。方法检索Webof Science、中国知网、维普数据库、万方数据库等文献数据库,2011年~2016年6月期间发表有关罕见病研究的相关文献。结果共计检索文献782篇,通过排除筛选,纳入200篇符合要求的文献,从罕见病政策研究、罕见病法律监管研究、罕见病医疗社会保障研究、罕用药可获得性研究、罕用药经济性评价研究、罕用药研发现状研究、罕见病界定标准研究7个方面进行分析对比。结论目前国内外罕见病领域研究偏重于罕见病政策研究。与国外相比,国内对于罕用药可获得性和经济性评价研究较少,尤其是经济性评价研究几乎为空白。建议研究人员对罕见病及其药品的多个方面进行研究,以为制定我国罕见病政策提供依据与参考。在罕见病研究领域之外,罕见病药物在药品研究、生产和供应等方面面临着诸多的困难,解决这些难题的先决条件是国家制定罕见病专门的政策与法规,进而明确罕见病的官方定义标准,建立相关政策激励制药企业开发罕见病药物。
[Abstract]:Objective to study and collate the literature on rare diseases at home and abroad, and to find out the deficiency and gap in the field of rare diseases in China. Methods to search the literature databases such as Webof Science, China knowledge Network, Weip Database, Wanfang Database and so on. From 2011 to June 2016, the related literatures on rare diseases were published. Results A total of 782 articles were retrieved and 200 articles were included by exclusion and screening. From rare disease policy research, rare disease legal supervision research, rare disease medical social security research, rare drug availability research, rare drug economic evaluation research, rare drug R & D status. Conclusion at present, the research in the field of rare diseases at home and abroad pays more attention to the policy of rare diseases, compared with foreign countries. There are few studies on the availability and economic evaluation of rare drugs in China, especially on economic evaluation. It is suggested that researchers study rare diseases and many aspects of drugs. In the field of rare diseases research, rare disease drugs are facing many difficulties in drug research, production and supply. The precondition for solving these problems is that the country should make special policies and regulations on rare diseases, then define the official standards of rare diseases, and establish relevant policies to encourage pharmaceutical enterprises to develop rare disease drugs.
【作者单位】：沈阳药科大学工商管理学院;中国药学会;
【分类号】：R95
【正文快照】： 罕见病是指发病率极低,患病人数较少的疾病。世界卫生组织将罕见病定义为患病人数占总人口的0.65‰~1‰之间的疾病或病变[1]。世界各国对罕见病的定义和认证标准不同:美国将罕见病规定为每年患病人数少于20万人或多于20万人但药物研制和生产无商业回报的疾病;日本规定罕见病为

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