摘要
罕见病诊疗是重要的社会公共卫生工作,由于罕见病用药种类少、可及性差,超说明书用药在罕见病治疗领域比较常见。超说明书用药可能是罕见病患者治疗威胁生命疾病的有效手段,但同时也会引发药品安全性和有效性、医疗责任和药品报销等相关问题,因此对其监管显得尤为重要。本文拟对国内外超说明书用药及其在罕见病治疗领域应用现状作一综述,期望能引起社会广泛关注,并通过医疗和管理机构共同努力,保障罕见病患者用药安全。
The diagnosis and treatment of rare diseases are a significant social public health work.Due to the low variety and poor accessibility of orphan drug,off-label use is common in the field of rare diseases at home and abroad.Off-label use may be the effective treatment for life-threatening diseases in patients with rare diseases,but it also raises issues related to drug safety and efficacy,medical liability and drug reimbursement,so the supervision of off-label drug use is of particular importance.This article reviews the current situation of off-label drug use and their application in the field of rare diseases treatment,hoping to arouse widespread concern in society and ensure medication safety for patients with rare diseases through the joint efforts of medical institutions and regulatory agencies.
作者
刘鑫
唐彦
张波
李建涛
梅丹
LIU Xin;TANG Yan;ZHANG Bo;LI Jian-tao;MEI Dan(Department of Pharmacy,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences,Beijing 100730,China)
出处
《国际药学研究杂志》
CAS
北大核心
2019年第9期685-690,共6页
Journal of International Pharmaceutical Research
基金
国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”资助项目（2016YFC0901500）
中国医学科学院医学与健康科技创新工程项目资助项目（2017-I2M-1-011）
