• 中国核心期刊(遴选)数据库收录期刊
  • 中文科技期刊数据库收录期刊
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中国药物评价 ›› 2020, Vol. 37 ›› Issue (2): 81-84.

• 真实世界证据研究 •    下一篇

罕见病真实世界研究的思考

 李壮琪, 杨悦*   

  1. 沈阳药科大学工商管理学院, 沈阳药科大学药品监管科学研究院, 辽宁 沈阳 110016
  • 收稿日期:2020-04-16 出版日期:2020-04-28 发布日期:2020-04-28

Research of Real World Studies on Rare Diseases

  1. School of Business Administration, Shenyang Pharmaceutical University, International Food & Drug Policy
         and Law Research Center, Liaoning Shenyang 110016, China
  • Received:2020-04-16 Online:2020-04-28 Published:2020-04-28
  • Contact: yang yue E-mail:yyue315@vip.126.com

摘要: 目的:研究探索真实世界证据在罕见病领域的应用及现状。方法:罕见病自然史研究是获取真实世界证据的重要来源,思考罕见病自然史研究的设计和应用。结果:自然史研究获得的数据经过分析总结成为真实世界证据,用来支持孤儿药研发及上市申请。结论:罕见病发病率低,基础研究不足,研发活动面临困境。罕见病自然史研究旨在形成真实世界证据,了解疾病发病机制和疾病进展,加强基础研究向转化医学研究转化。

关键词: font-size:medium, ">罕见病;真实世界数据;真实世界证据;自然史研究;真实世界研究

Abstract: Objective: Research and explore the application and status of real-world evidence in the field of rare diseases. Methods:Natural history studies of rare diseases is an important source for obtaining real-world evidence, thinking about the design and application of natural history studies on rare diseases. Results:The data obtained from natural history studies, used to support orphan drug development and marketing applications, is analyzed and summarized to become real-world evidence. Conclusion:The incidence of rare diseases is low, basic research is insufficient, and research and development activities will face many difficulties. Rare disease natural history studies aims to form real-world evidence, understanding disease pathogenesis and disease progression, then strengthening the transformation of basic research into translational medical research.

Key words: font-size:medium, ">Rare Disease; Real-world data; Real-world evidence; Natural history studies; Real-world studies

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