罕见病超说明书用药专家共识计划书

打开文本图片集

【中图分类号】R951 【文献标识码】A

【Abstract】Rare diseases are a collective term for diseases with extremely low prevalence and incidence rates.Up to now， China has released two lists identifyinga total of 2O7 rare diseases.Given that most rare diseases do not have drugs with coresponding indications， physicians frequently resort to using offlabel drugs when treating patients with rare diseases.However， there is currently no systematic guideline or expert consensus for the use of of-label medications in China.To comprehensively collect existing evidence of off-label drug use for rare diseases，fully analyze and evaluate the rationality of of-label drug use for rare diseases，and standardize the management of of-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 I and the STAR tools.This protocol outlines the background and purpose of consensus，as wellas the comprehensive framework for consensus development， encompassing panel formation，clinical issue identification，evidence retrieval， data extraction，and evidence-based recommendation formulation.

【Keywords】Rare disease; Off-label use; Expert consensus; Study protocol

罕见病又称“孤儿病”，是发病率极低的各种疾病的统称。（剩余12348字）