数智化技术在罕见病药物警戒领域的应用现状及展望  

The Current Status and Prospects of the Application of Digital Technology in the Field of Pharmacovigilance of Rare Diseases

作　　者：曹颖 刘欣茹 王胜锋[2,3,4] 卓琳 CAO Ying;LIU Xinru;WANG Shengfeng;ZHUO Lin(School of Public Health,Peking University,Beijing 100191,China;Department of Epidemiology and Biostatistics,School of Public Health,Peking University,Beijing 100191,China;Key Laboratory of Epidemiology of Major Diseases(Peking University),Ministry of Education,Peking University,Beijing 100191,China;Institute for Artificial Intelligence,Peking University,Beijing 100871,China;Research Center of Clinical Epidemiology,Peking University Third Hospital,Beijing 100191,China)

机构地区：[1]北京大学公共卫生学院,北京100191 [2]北京大学公共卫生学院流行病与卫生统计学系,北京100191 [3]北京大学重大疾病流行病学教育部重点实验室(北京大学),北京100191 [4]北京大学人工智能研究院,北京100871 [5]北京大学第三医院临床流行病学研究中心,北京100191

出　　处：《罕见病研究》2025年第1期22-29,共8页Journal of Rare Diseases

基　　金：国家自然科学基金(72342015,82304242)。

摘　　要：目的梳理数智化技术在药物警戒尤其是罕见病领域的应用现状,为罕见病药物警戒的方法选择及发展提供参考。方法检索中国知网、万方数据知识服务平台、维普中文科技期刊数据库、PubMed、Embase 5个数据库,筛选、总结数智化技术在罕见病药物警戒领域的应用并提取相关信息,进行范围综述。结果数智化技术在药物警戒领域的应用暂未能推广到罕见病这一特殊场景中,相关实例研究匮乏,存在数据来源不足及技术局限性两大困境,有待突破。结论结合罕见病药物特点指出适用于罕见病药物警戒领域的数据来源和数智化技术,并提出了未来可能的发展方向和针对性建议。Objective To summarize the current status in the application of digital and intelligent technologies in the field of pharmacovigilance and to provide reference to the selection and development of methods for pharmacovigilance of rare diseases.Methods Searched five major databases-CNKI,WANFANG,VIP,PubMed,and Embase,selected and summarized the data of application of digital technology in the field of drug vigilance for rare diseases,extracted relevant information and conducted a systematic review.Results The application of digital technology in drug surveillance has not yet been used in the special field of rare diseases.Relevant case studies are insufficient.Two major challenges need to be addressed.One is the insufficient data sources and the other is technical limitations.Conclusions Based on the characteristics of drugs for rare diseases,this paper identifies data sources and intelligent technologies suitable for the field of drug vigilance for rare disease,proposes direction for potential development in the future,and makes targeted suggestions.

关 键 词：数智化技术 罕见病 药物警戒 数据资源 

分 类 号：R9[医药卫生—药学] TP39[自动化与计算机技术—计算机应用技术]