Expert consensus on the off-label use of drugs for pediatric rare diseases in China (2025 edition).
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Mo Xiaolan, Deng Houliang, Yang Jinlian, Li Jiale, Ouyang Shan, Chen Feng, Cui Yanqin, Gao Xia, Jiang Hua, Li Xiaojing, Liu Li, Liu Jie, Qiu Lin, Rong Xiaoming, Wen Zhe, Wang Jiayi, Zeng Ping, Zhang Wen, Zhao Xiaopeng, Zhou Juan, Chen Jie, Chen Pan, Chen Wenying, Chen Zebin, Fang Rui, Fan Xiaomei, Jia Yuntao, Li Zhiling, Li Xuejuan, Li Xiaoyan, Lin Yunzhu, Liu Maochang, Mai Haiyan, Miao Jing, Wang Xiaoling, Wang Yong, Wang Jinghao, Wei Li, Wu Junyan, Yu Shanshan, Zeng Linan, Zhang Bo, Zhang Hongliang, Zheng Zhihua, Chen Xiao, Xia Sujian, Zhou Wenhao, Sun Jing, Li Yilei, Rare Disease Expert Committee of Guangdong Pharmaceutical Association
Аннотация
METHODS: Under the guidance of the Rare Disease Expert Committee of the Guangdong Pharmaceutical Association, a multidisciplinary panel of experts from clinical medicine, pharmacy, and related specialties developed the "Expert consensus on the off-label use of drugs for pediatric rare diseases in China (2025 edition)". The consensus integrates available evidence, clinical experience, evidence quality, and medication safety profiles, and was finalized after several rounds of rigorous iterative review.
RESULTS: The consensus presents 73 recommendations on off-label drug use across 21 rare diseases, organized in a tabular format for clarity and ease of reference.
CONCLUSIONS: This consensus aims to standardize the management of off-label drug use in pediatric rare diseases. It supports medical institutions in developing off-label drug formularies, promotes rational drug use, and helps address the diagnostic and therapeutic needs of pediatric rare disease patients. Furthermore, it contributes to the establishment of a structured evaluation and management framework for off-label drug use in this clinical context.