AUTHOR=Zhao Boxin , Zhou Xuan , Zheng Ping , Zhang Bo , Feng Xiaoqin , Chen Jie , Cai Lisheng , Chen Yilu , He Liya , Su Jianfen , Cheng Shuqin , Zeng Yingtong , Li Guowei , Ji Bo , Wu Jianlong , Feng Weiyi , Liu Maobai , Jin Yiran , Liu Taotao , Mo Xiaolan , Wu Junyan , Wu Hui , Zhang Hongliang , Zheng Zhichang , Zheng Zhihua , Sun Jing , Li Yilei , Guangdong Pharmaceutical Association, Hematology Group of Rare Disease Expert Committee of Guangdong Pharmaceutical Association TITLE=Expert consensus on the off-label use in China of drugs for rare hematologic diseases (2024 edition) JOURNAL=Frontiers in Pharmacology VOLUME=15 YEAR=2024 URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2024.1477550 DOI=10.3389/fphar.2024.1477550 ISSN=1663-9812 ABSTRACT=

Drug package inserts are a crucial foundation for clinical medication practices and serve as the legal basis for guiding rational drug use and ensuring patient safety and efficacy. As rare disease treatments evolve, current package inserts often need to meet the clinical requirements for treating such conditions, frequently resulting in off-label drug use. This consensus is derived from discussions between Guangdong Pharmaceutical Association Hematologic Rare Diseases Group experts. The consensus aims to provide a framework and reference for the clinical application of off-label drug use in treating rare hematologic diseases.