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从文献计量学角度看全球孤儿药的可视化研究

Visual Research of Global Orphan Drug from a Bibliometric Perspective.

作者信息

Wen Xiaoya, Jin Guangzhi, Wu Chunxing

机构信息

Department of Pharmacy, The Fourth People's Hospital of GuiYang, Guiyang, Guizhou Province, 550007, People's Republic of China.

Department of Hemodialysis Room, The Second People's Hospital of Guizhou Province, Guiyang, Guizhou Province, 550001, People's Republic of China.

出版信息

Drug Des Devel Ther. 2025 May 20;19:4201-4220. doi: 10.2147/DDDT.S506112. eCollection 2025.

DOI:10.2147/DDDT.S506112
PMID:40416799
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12103199/
Abstract

OBJECTIVE

This study analyzes the current research landscape and trends in orphan drug development, providing insights for future advancements in the field.

METHODS

Gathering pertinent material from the China National Knowledge Infrastructure (CNKI) and Web of Science databases. Microsoft Office Excel 2017, VOSview 1.6.20, and CiteSpace 6.3R2 were utilised to summarise the present research state and offer insights into research hotspots in the domain of orphan drugs.

RESULTS

A total of 3598 research papers were included, with Chinese research showing a continuous upward trend, while international research has entered a slow development stage. The field of orphan drug research has formed a sizable research team, and cooperation between international institutions is relatively mature. Meanwhile, the United States and the United Kingdom have strong influence in this research field, while China lacks international cooperation. The research focus in this field mainly involves the development and clinical application of orphan drugs, and domestic and foreign research also has its own emphasis. Maintain consistency in clinical trials and medication support for orphan drugs both domestically and internationally; And foreign research has obvious advantages in the development of orphan drugs. Keyword emergence research indicates that the clinical accessibility and regulatory approval of orphan drugs have become a prominent issue of interest both nationally and globally.

CONCLUSION

This study systematically summarises and examines the current research status and emerging trends in the global orphan drug sector by analysing relevant literature from 2000 to 2024 through bibliometric methods. It further delineates the similarities and differences in orphan drug research domestically and internationally, offering valuable references for future investigations in this domain.

摘要

目的

本研究分析罕见病药物研发的当前研究格局和趋势，为该领域的未来进展提供见解。

方法

从中国知网（CNKI）和科学网数据库收集相关资料。利用Microsoft Office Excel 2017、VOSview 1.6.20和CiteSpace 6.3R2总结当前研究状况，并深入了解罕见病药物领域的研究热点。

结果

共纳入3598篇研究论文，中国的研究呈持续上升趋势，而国际研究已进入缓慢发展阶段。罕见病药物研究领域已形成规模较大的研究团队，国际机构间的合作相对成熟。同时，美国和英国在该研究领域影响力较强，而中国缺乏国际合作。该领域的研究重点主要涉及罕见病药物的研发和临床应用，国内外研究也各有侧重。国内外在罕见病药物的临床试验和用药支持方面保持一致；且国外在罕见病药物研发方面具有明显优势。关键词出现情况研究表明，罕见病药物的临床可及性和监管批准已成为国内外备受关注的突出问题。

结论

本研究通过文献计量学方法分析2000年至2024年的相关文献，系统总结并审视了全球罕见病药物领域的当前研究现状和新出现的趋势。它进一步阐述了国内外罕见病药物研究的异同，为该领域未来的研究提供了有价值的参考。

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