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REVIEW article

Front. Pharmacol.
Sec. Drugs Outcomes Research and Policies
Volume 15 - 2024 | doi: 10.3389/fphar.2024.1477550
This article is part of the Research Topic Clinical Pharmacist Service Promotes the Improvement of Medical Quality Volume II View all 9 articles

Expert Consensus on the Off-Label Use in China of Drugs for Rare Hematologic Diseases (2024 Edition)

Provisionally accepted
Bo-xin Zhao Bo-xin Zhao 1Xuan Zhou Xuan Zhou 1*Ping Zheng Ping Zheng 1*Bo Zhang Bo Zhang 2*Xiaoqin Feng Xiaoqin Feng 1Jie Chen Jie Chen 3Lisheng Cai Lisheng Cai 4*Yilu Chen Yilu Chen 5Liya He Liya He 5*Jianfen Su Jianfen Su 6*Shuqin Cheng Shuqin Cheng 6*Yingtong Zeng Yingtong Zeng 7*Guowei Li Guowei Li 8*Bo Ji Bo Ji 9Jianlong Wu Jianlong Wu 4Weiyi Feng Weiyi Feng 10*Maobai Liu Maobai Liu 11Yiran Jin Yiran Jin 12TaoTao Liu TaoTao Liu 13Xiaolan Mo Xiaolan Mo 5Junyan Wu Junyan Wu 14*Hui Wu Hui Wu 15*Hongliang Zhang Hongliang Zhang 13Zhichang Zheng Zhichang Zheng 16*Zhihua Zheng Zhihua Zheng 17*Jing Sun Jing Sun 1*Yilei Li Yilei Li 1*
  • 1 Nanfang Hospital, Southern Medical University, Guangzhou, China
  • 2 Peking Union Medical College Hospital (CAMS), Beijing, Beijing Municipality, China
  • 3 The First Affiliated Hospital of Sun Yat-sen University, Guangzhou, Guangdong Province, China
  • 4 Shenzhen Second People's Hospital, Shenzhen, Guangdong Province, China
  • 5 Guangzhou Women and Children’s Medical Center, Guangzhou Medical University, Guangzhou, Guangdong Province, China
  • 6 Guangzhou Panyu Central Hospital, Guangzhou, China
  • 7 Guangdong Provincial People's Hospital, Guangzhou, Guangdong Province, China
  • 8 Huizhou Central People's Hospital, Huizhou, Guangdong Province, China
  • 9 General Hospital of Southern Theater Command of PLA, Southern Medical University, Guangzhou, Guangdong Province, China
  • 10 The First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, Shaanxi, China
  • 11 Fujian Medical University Union Hospital, Fuzhou, Fujian Province, China
  • 12 Second Hospital of Hebei Medical University, Shijiazhuang, Hebei Province, China
  • 13 First Affiliated Hospital, Guangxi Medical University, Nanning, Guangxi Zhuang Region, China
  • 14 Sun Yat-sen Memorial Hospital, Guangzhou, Guangdong Province, China
  • 15 The First Affiliated Hospital of Kunming Medical University, Kunming, Yunnan Province, China
  • 16 Affiliated Hospital of Guizhou Medical University, Guiyang, Guizhou Province, China
  • 17 Guangdong Pharmaceutical Association, Guangzhou, China

The final, formatted version of the article will be published soon.

    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.

    Keywords: Rare Diseases, Hematological disease, off-label drug use, expert consensus, evidencebased pharmacy

    Received: 08 Aug 2024; Accepted: 17 Sep 2024.

    Copyright: © 2024 Zhao, Zhou, Zheng, Zhang, Feng, Chen, Cai, Chen, He, Su, Cheng, Zeng, Li, Ji, Wu, Feng, Liu, Jin, Liu, Mo, Wu, Wu, Zhang, Zheng, Zheng, Sun and Li. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence:
    Xuan Zhou, Nanfang Hospital, Southern Medical University, Guangzhou, China
    Ping Zheng, Nanfang Hospital, Southern Medical University, Guangzhou, China
    Bo Zhang, Peking Union Medical College Hospital (CAMS), Beijing, 100730, Beijing Municipality, China
    Lisheng Cai, Shenzhen Second People's Hospital, Shenzhen, Guangdong Province, China
    Liya He, Guangzhou Women and Children’s Medical Center, Guangzhou Medical University, Guangzhou, 510623, Guangdong Province, China
    Jianfen Su, Guangzhou Panyu Central Hospital, Guangzhou, China
    Shuqin Cheng, Guangzhou Panyu Central Hospital, Guangzhou, China
    Yingtong Zeng, Guangdong Provincial People's Hospital, Guangzhou, 510317, Guangdong Province, China
    Guowei Li, Huizhou Central People's Hospital, Huizhou, 516001, Guangdong Province, China
    Weiyi Feng, The First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, 710061, Shaanxi, China
    Junyan Wu, Sun Yat-sen Memorial Hospital, Guangzhou, 510000, Guangdong Province, China
    Hui Wu, The First Affiliated Hospital of Kunming Medical University, Kunming, 650032, Yunnan Province, China
    Zhichang Zheng, Affiliated Hospital of Guizhou Medical University, Guiyang, 550004, Guizhou Province, China
    Zhihua Zheng, Guangdong Pharmaceutical Association, Guangzhou, China
    Jing Sun, Nanfang Hospital, Southern Medical University, Guangzhou, China
    Yilei Li, Nanfang Hospital, Southern Medical University, Guangzhou, China

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.