国内外罕见病领域研究进展对比分析

李彤彤, 戴罡, 张方

中国药学杂志 ›› 2017, Vol. 52 ›› Issue (6) : 506-512.

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中国药学杂志
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中国药学杂志 ›› 2017, Vol. 52 ›› Issue (6) : 506-512. DOI: 10.11669/cpj.2017.06.016
论著

国内外罕见病领域研究进展对比分析

  • 李彤彤1, 戴罡1,2, 张方1*
作者信息 +

Comparative Analysis on the Research Progress of Rare Diseases in Domestic and Abroad

  • LI Tong-tong1, DAI Gang1,2, ZHANG Fang1*
Author information +
文章历史 +

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 in domestic and abroad, and comparative analysis, provide a scientific basis for the domestic rare diseases research. METHODS Retrieved the Web of Science, China National Knowledge Infrastructure from January 2011 to June 2016 published literature about rare diseases. RESULTS Through the screening of literature, finally determine the 200 articles for analysis. It is divided into seven research directions:rare diseases policy research, rare diseases legal and regulatory research, rare diseases medical social security study, orphan drugs availability research, orphan drugs economic evaluation study, orphan drug development research, rare diseases defined standard research. CONCLUSION Rare diseases policy research is the focus of research both domestic and abroad. Compared with foreign countries, the domestic research on the availability and economic evaluation of orphan drug is less, especially the economic evaluation research is almost blank. It is suggested that the researchers study the multiple aspects of rare diseases and drugs, and to provide the basis and reference for build rare disease policy in China.In addition to the field of rare diseases research, rare diseases drugs face many difficulties in pharmaceutical research, production and supply.The precondition to solve these problems is the nation formulate specific policies and regulations for rare diseases,and then clear the official definition standards of rare diseases,establish relevant policies to encourage pharmaceutical companies to develop rare diseases drugs.

关键词

罕见病 / 孤儿药 / 研究进展 / 国内外 / 对比分析

Key words

rare disease / orphan drug / research progress / domestic and abroad / comparative analysis

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李彤彤, 戴罡, 张方. 国内外罕见病领域研究进展对比分析[J]. 中国药学杂志, 2017, 52(6): 506-512 https://doi.org/10.11669/cpj.2017.06.016
LI Tong-tong, DAI Gang, ZHANG Fang. Comparative Analysis on the Research Progress of Rare Diseases in Domestic and Abroad[J]. Chinese Pharmaceutical Journal, 2017, 52(6): 506-512 https://doi.org/10.11669/cpj.2017.06.016
中图分类号： R951   

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