摘要
近年来,在国家和政府的高度重视下,我国罕见病诊疗事业不断迈上新台阶。然而,全球范围内仅5%的罕见病存在有效治疗方法,罕见病药物可及性差仍是中国乃至全球罕见病领域面临的主要困境。由于罕见病药物相关研究数据较少、临床诊疗经验不足、用药经验缺乏,罕见病诊疗全过程亟需药师的广泛参与,并亟待建立系统化管理和监测体系。为充分发挥医疗机构在罕见病药品应用保障中的主体作用,提升罕见病药学服务质量,规范罕见病药学服务内容与形式,充分挖掘药师在罕见病诊疗中的作用,中国药学会罕见病药物专委会组织相关领域专家共同制订了《医疗机构罕见病药学服务专家共识(2025)》,旨在构建符合中国国情的医疗机构罕见病药学服务标准体系,以保障患者用药的可及性、合理性、有效性和安全性。
In recent years,with strong support from the national government,the diagnosis and treatment of rare diseases in China have achieved significant progress.However,only 5%of rare diseases worldwide have effective treatments,and poor drug accessibility remains a major challenge in the field of rare diseases,both in China and globally.Due to limited research data,insufficient clinical experience,and a lack of medication expertise,there is an urgent need for pharmacists to play an expanded role throughout the rare disease care process,alongside the establishment of systematic management and monitoring systems.To enhance the leading role of medical institutions in securing rare disease drug availability,improve pharmaceutical care quality,standardize service content and delivery models,and fully leverage pharmacistsexpertise in rare disease management,the Rare Disease Drug Committee of the Chinese Pharmaceutical Association organized a panel of experts to develop the Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals(2025).This consensus aims to establish a standardized pharmaceutical care framework tailored to Chinas healthcare context,ensuring the accessibility,appropriateness,efficacy,and safety of rare disease treatments.
作者
中国药学会罕见病药物专委会
张波
王晓玲
王少红
左玮
成晓玲
方会慧
魏然
薛娉娉
Rare Disease Drug Committee of the Chinese Pharmaceutical Association;ZHANG Bo;WANG Xiaoling(不详;Department of Pharmacy,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China;State Key Laboratory of Complex Severe and Rare Diseases,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China;National Center for Healthcare Quality Management in Rare Diseases,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China;Department of Pharmacy,Beijing Childrens Hospital,Capital Medical University,Beijing 100045,China)
出处
《协和医学杂志》
北大核心
2025年第3期659-672,共14页
Medical Journal of Peking Union Medical College Hospital
基金
中央高水平医院临床科研专项(2022-PUMCH-B-059)。
关键词
罕见病
罕见病药物
药学服务
医疗机构
rare diseases
rare disease drugs
pharmaceutical care
hospital
作者简介
通信作者:张波,E-mail:zhangbopumch@163.com;通信作者:王晓玲,E-mail:wangxiaoling@bch.com.cn。