Expert consensus for off-label drug use of rare disease:a protocol
10.12173/j.issn.1005-0698.202503180
- VernacularTitle:罕见病超说明书用药专家共识计划书
- Author:
Chaoyang CHEN
1
;
Yuehan DUAN
;
Lin ZHUO
;
Guohua HE
;
Yanqin ZHANG
;
Ying ZHOU
;
Shengfeng WANG
;
Yimin CUI
;
Jie DING
Author Information
1. 北京大学第一医院药剂科(北京 100034)
- Publication Type:Journal Article
- Keywords:
Rare disease;
Off-label use;
Expert consensus;
Study protocol
- From:
Chinese Journal of Pharmacoepidemiology
2025;34(9):1066-1073
- CountryChina
- Language:Chinese
-
Abstract:
Rare diseases are a collective term for diseases with extremely low prevalence and incidence rates.Up to now,China has released two lists identifying a total of 207 rare diseases.Given that most rare diseases do not have drugs with corresponding indications,physicians frequently resort to using off-label drugs when treating patients with rare diseases.However,there is currently no systematic guideline or expert consensus for the use of off-label medications in China.To comprehensively collect existing evidence of off-label drug use for rare diseases,fully analyze and evaluate the rationality of off-label drug use for rare diseases,and standardize the management of off-label drug use for rare diseases,the Rare Disease Branch of Beijing Medical Association,Chinese Pharmaceutical Association,Beijing Pharmaceutical Association,and the School of Public Health,Peking University have jointly initiated the drafting of the Expert Consensus on Off-label Use of Drugs for Rare Diseases.This consensus refer to the WHO Handbook for Guideline Development,the Guidelines for Developing/Revising Clinical Diagnostic and Treatment Guidelines in China(2022 Edition),the AGREE Ⅱ and the STAR tools.This protocol outlines the background and purpose of consensus,as well as the comprehensive framework for consensus development,encompassing panel formation,clinical issue identification,evidence retrieval,data extraction,and evidence-based recommendation formulation.
