摘要
目的:形成医疗机构药品遴选中药品临床必需性考察项下罕见病用药判别的方法。方法:以“罕见病用药”“罕见病”和“孤儿药”为中文关键词,以“rare diseases”和“orphan drug”为英文关键词,检索(自建立/建库至2021年2月)发布在我国相关政府网站中的法规政策和解读文件,检索在PubMed、万方数据库、中国知网和维普数据库等数据库中发表的相关文献,经筛选分析后纳入可用于判别罕见病用药的文献或文件,形成可用于医疗机构药品遴选的罕见病用药判别的方法。结果与结论:经筛选共纳入政策文件6篇、文献1篇,本研究形成了可根据药品说明书中有罕见病适应证的药品、《临床急需境外新药名单》中用于治疗罕见病的药品、我国《罕见病诊疗指南(2019年版)》中用于治疗罕见病的药品和“老药新用”用于治疗Orphanet网站纳入的罕见病的国内上市销售药品,来判断待遴选药品为罕见病用药的方法,可为《医疗机构药品遴选指南》中药品临床必需性的评价提供单独针对罕见病用药的评价路径及指标。
OBJECTIVE:To construct a method of recognizing rare diseases drugs in drug clinical necessity assessment of drug selection in medical institutions.METHODS:With“rare diseases drugs”,“rare diseases”and“orphan drugs”as Chinese and English keywords,the laws,policies and interpretation documents published on relevant Chinese government websites(from the inception/establishment of the database to Feb.2021)were retrieved.Related literature were collected from PubMed,Wanfang,CNKI,VIP databases.After screening and analyzing the retrieved literature and documents,the method for recognizing rare diseases drugs in drug selection of medical institutions was formed.RESULTS and CONCLUSIONS:Six policy documents and 1 literature were included after screening.This study has formed the method of judging the drugs to be selected as drugs for rare diseases by drugs with indications for rare diseases in the drug instructions,drugs for the treatment of rare diseases in the List of Clinically Urgent New Drugs from Overseas,and drugs for the treatment of rare diseases in the Guidelines of the Diagnosis and Treatment of Rare Diseases in China(2019 Edition),domestic marketing of drugs of“new use of old drugs”for the treatment of rare diseases included in the Orphanet website,which can provide separate evaluation pathway and index for the evaluation of the clinical necessity of drugs for rare diseases in the Drug Selection Guidelines for Medical Institutions.
作者
禹洁
段蓉
吴佳瑶
仲威龙
李正翔
YU Jie;DUAN Rong;WU Jiayao;ZHONG Weilong;LI Zhengxiang(Dept.of Pharmacy,General Hospital of Tianjin Medical University,Tianjin 300052,China;Dept.of Gastroenterology,General Hospital of Tianjin Medical University,Tianjin 300052,China)
出处
《中国医院用药评价与分析》
2022年第9期1025-1028,共4页
Evaluation and Analysis of Drug-use in Hospitals of China
基金
天津市赵以成医学科学基金会项目。
关键词
罕见病
罕见病用药
孤儿药
药品遴选
指南
Rare diseases
Rare diseases drugs
Orphan drugs
Drug selection
Guidelines
作者简介
禹洁,主管药师,硕士。研究方向:医院药学、临床药学。E-mail:yujie1983rain@163.com;通信作者:李正翔,主任药师。研究方向:医院药学、药事管理、循证药学。E-mail:13820893896@163.com。
