Research on Method of Recognizing Rare Disease Drugs in Drug Clinical Necessity Assessment of Drug Selection in Medical Institutions

YU Jie; DUAN Rong; WU Jiayao; ZHONG Weilong; LI Zhengxiang

doi:10.14009/j.issn.1672-2124.2022.09.001

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Research on Method of Recognizing Rare Disease Drugs in Drug Clinical Necessity Assessment of Drug Selection in Medical Institutions

更新时间：2023-04-16

- Research on Method of Recognizing Rare Disease Drugs in Drug Clinical Necessity Assessment of Drug Selection in Medical Institutions
- Vol. 22, Issue 9, Pages: 1025-1028(2022)
- 作者机构：
  
  1. 天津医科大学总医院药剂科
  2. 天津医科大学总医院消化内科
- 作者简介：
- 基金信息：
- DOI：10.14009/j.issn.1672-2124.2022.09.001
  CLC： R95
- Published：2022，
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YU Jie, DUAN Rong, WU Jiayao, et al. Research on Method of Recognizing Rare Disease Drugs in Drug Clinical Necessity Assessment of Drug Selection in Medical Institutions. [J]. 22(9):1025-1028(2022)
DOI：

YU Jie, DUAN Rong, WU Jiayao, et al. Research on Method of Recognizing Rare Disease Drugs in Drug Clinical Necessity Assessment of Drug Selection in Medical Institutions. [J]. 22(9):1025-1028(2022) DOI： 10.14009/j.issn.1672-2124.2022.09.001.

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。

Abstract

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.

关键词

罕见病罕见病用药孤儿药药品遴选指南

Keywords

Rare diseasesRare diseases drugsOrphan drugsDrug selectionGuidelines

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