协和医学杂志2025，Vol.16Issue(3)：659-672,14.DOI:10.12290/xhyxzz.2025-0252
医疗机构罕见病药学服务专家共识(2025)
Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals(2025)
摘要
Abstract
In recent years,with strong support from the national government,the diagnosis and treat-ment of rare diseases in China have achieved significant progress.However,only 5％of rare diseases world-wide have effective treatments,and poor drug accessibility remains a major challenge in the field of rare disea-ses,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 dis-ease 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 pharmacists' expertise in rare dis-ease 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 China's healthcare context,ensuring the accessibility,appropriateness,efficacy,and safety of rare disease treatments.关键词
罕见病/罕见病药物/药学服务/医疗机构Key words
rare diseases/rare disease drugs/pharmaceutical care/hospital分类
药学引用本文复制引用
中国药学会罕见病药物专委会,张波,王晓玲..医疗机构罕见病药学服务专家共识(2025)[J].协和医学杂志,2025,16(3):659-672,14.基金项目
中央高水平医院临床科研专项(2022-PUMCH-B-059) National High Level Hospital Clinical Research Funding(2022-PUMCH-B-059) （2022-PUMCH-B-059）
