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Volume 14 Issue 1
Jan.  2023
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ZUO Wei, LIU Rongji, SUN Yajia, ZHANG Bo, CHEN Yaolong, ZHANG Shuyang. Interpretation on Management Guideline for the Off-label Use of Drugs in China(2021 Edition)[J]. Medical Journal of Peking Union Medical College Hospital, 2023, 14(1): 86-93. DOI: 10.12290/xhyxzz.2022-0588
Citation: ZUO Wei, LIU Rongji, SUN Yajia, ZHANG Bo, CHEN Yaolong, ZHANG Shuyang. Interpretation on Management Guideline for the Off-label Use of Drugs in China(2021 Edition)[J]. Medical Journal of Peking Union Medical College Hospital, 2023, 14(1): 86-93. DOI: 10.12290/xhyxzz.2022-0588
shu

Interpretation on Management Guideline for the Off-label Use of Drugs in China(2021 Edition)

  • 1.

    Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China

  • 2.

    State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China

  • 3.

    School of Public Health, School of Basic Medical Sciences, Lanzhou University, Lanzhou 730000, China

  • 4.

    Institute of Health Data Science, School of Basic Medical Sciences, Lanzhou University, Lanzhou 730000, China

  • 5.

    Evidence-based Medicine Center, School of Basic Medical Sciences, Lanzhou University, Lanzhou 730000, China

  • 6.

    Research Unit of Evidence-based Evaluation and Guidelines, Chinese Academy of Medical Sciences, School of Basic Medical Sciences, Lanzhou University, Lanzhou 730000, China

  • 7.

    Department of Cardiology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China

Funds: 

CAMS Innovation Fund for Medical Sciences 2021-I2M-1-003

National High Level Hospital Clinical Research Funding 2022-PUMCH-B-059

National High Level Hospital Clinical Research Funding 2022-PUMCH-B-060

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    Graphical Abstract
    • Abstract
  • Off-label drug use represents a reasonable need for diagnosis and treatment in some cases, but it faces potential risks with drug safety and legal challenges. However, there was no guideline on how to manage the off-label drug use in China. Therefore, Peking Union Medical College Hospital, in cooperation with Evidence-based Medicine Center of Lanzhou University, established a working group and an expert group consisting of multidisciplinary group of experts to develop the Management Guideline for the Off-label Use of Drugs in China (2021). The Guideline is guided by the Law on Doctors of the People's Republic of China and the World Health Organization Guideline Development Manual. This is the first management guideline on the off-label use of drugs, aiming to improve the stakeholders' understanding of this topic and establish a standardized management procedure in China. This guideline has determined nine questions and proposed a total of twenty-three recommendations. The English version of the guideline was published in September 2022. This paper offers a detailed interpretation of the key points of the guidelines in order to provide suggestions and references for the management of off-label drug use.
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    • References (32)
  • [1]
    中华医学会儿科学分会临床药理学组, 《中华儿科杂志》编辑委员会. 中国儿科超说明书用药专家共识[J]. 中华儿科杂志, 2016, 54: 101-103. DOI: 10.3760/cma.j.issn.0578-1310.2016.02.007
    [2]
    中华人民共和国中央人民政府. 中华人民共和国医师法[EB/OL]. (2021-08-20)[2022-09-13]. http://www.npc.gov.cn/npc/c30834/202108/d954d9fa0af7458aa862182dc50a0d63.shtml.
    [3]
    Zuo W, Sun Y, Liu R, et al. Management guideline for the off-label use of medicine in China (2021)[J]. Expert Rev Clin Pharmacol, 2022, 11: 1-16.
    [4]
    Reay T, Berta WB, Kohn MK. What's the evidence on evidencebased management?[J]. Acad Manag Perspect, 2009, 23: 5-18. DOI: 10.5465/AMP.2009.45590137
    [5]
    Oxford Centre for Evidence-based Medicine. Oxford centre for evidence-based medicine: levels of evidence[EB/OL]. (2009-03)[2022-09-13]. https://www.cebm.ox.ac.uk/resources/levels-of-evidence/oxford-centre-for-evidence-based-medicine-levels-ofevidence-march-2009.
    [6]
    广东省药学会. 超药品说明书用药药物经济学评价专家共识[J]. 今日药学, 2016, 26: 681-683. https://www.cnki.com.cn/Article/CJFDTOTAL-YAXU201610001.htm
    [7]
    Gazarian M, Kelly M, McPhee JR, et al. Off-label use of medicines: consensus recommendations for evaluating appropriateness[J]. Med J Aust, 2006, 185: 544-548. DOI: 10.5694/j.1326-5377.2006.tb00689.x
    [8]
    Gazarian M, Kelly M, McPhee JR, et al. Off-label use of medicines: consensus recommendations for evaluating appropriateness[J]. Med J Aust, 2006, 185: 544-548. DOI: 10.5694/j.1326-5377.2006.tb00689.x
    [9]
    National Institute for Health and Care Excellence. Evidence Summaries: Unlicensed and Off-Label Medicines-Integrated Process Statement[Internet][R]. London: National Institute for Health and Care Excellence (NICE), 2013: Process and Methods Guides No. 14.
    [10]
    European Commission Enterprise And Industry Directorate-General. A guideline on summary of product characteristics (SmPC)[EB/OL]. (2013-01-21)[2022-09-13]. https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/product-information/how-prepare-review-summary-product-characteristics.
    [11]
    Dooms M, Killick J. Off-label use of medicines: The need for good practice guidelines[J]. Int J Risk Saf Med, 2017, 29: 17-23. DOI: 10.3233/JRS-170737
    [12]
    中华人民共和国中央人民政府. 中华人民共和国民法典[EB/OL]. (2020-06-02)[2022-09-13]. https://epaper.gmw.cn/gmrb/html/2020-06/02/nw.D110000gmrb_20200602_7-01.htm.
    [13]
    Atkins D, Best D, Briss PA, et al. Grading quality of evidence and strength of recommendations[J]. BMJ, 2004, 328: 1490. DOI: 10.1136/bmj.328.7454.1490
    [14]
    Oxford Centre for Evidence-Based Medicine. Explanation of the 2011 OCEBM levels of evidence[EB/OL]. (2021-12-23)[2022-09-13]. https://www.cebm.ox.ac.uk/resour-ces/levels-of-evidence/explanation-of-the-2011-ocebm-levels-of-evidence.
    [15]
    Herrero Fernandez M, Molina Villaverde R, Arroyo Yustos M, et al. The Off-Label Use of Antineoplastics in Oncology Is Limited But Has Notable Scientific Support in a University Hospital Setting[J]. Front Pharmacol, 2019, 10: 1210. DOI: 10.3389/fphar.2019.01210
    [16]
    Yang J, Lin W, Chen Y. Off-label use of tamoxifen in a Chinese tertiary care hospital[J]. Int J Clin Pharm, 2019, 41: 555-562. DOI: 10.1007/s11096-019-00788-5
    [17]
    中华人民共和国卫生部. 药品不良反应报告和监测管理办法[EB/OL]. (2011-05-24)[2022-09-13]. http://www.gov.cn/flfg/2011-05/24/content_1870110.htm.
    [18]
    Cook RJ. Off-label drug use as a consent and health regulation issue in New Zealand[J]. J Bioeth Inq, 2015, 12: 251-258. DOI: 10.1007/s11673-014-9568-6
    [19]
    Lenk C, Duttge G. Ethical and legal framework and regulation for off-label use: European perspective[J]. Ther Clin Risk Manag, 2014, 10: 537-546.
    [20]
    Czaja AS, Ross ME, Liu W, et al. Electronic health record (EHR) based postmarketing surveillance of adverse events associated with pediatric off-label medication use: A case study of short-acting beta-2 agonists and arrhythmias[J]. Pharmacoepidemiol Drug Saf, 2018, 27: 815-822. DOI: 10.1002/pds.4562
    [21]
    王丹. 药品不良反应主动监测及其发展趋势[J]. 中国药物警戒, 2015, 12: 600-602, 610. DOI: 10.19803/j.1672-8629.2015.10.007
    [22]
    Coughlin M, Goldie CL, Tregunno D, et al. Enhancing metabolic monitoring for children and adolescents using second-generation antipsychotics[J]. Int J Ment Health Nurs, 2018, 27: 1188-1198. DOI: 10.1111/inm.12417
    [23]
    Emmerich J, Dumarcet N, Lorence A. France's new framework for regulating off-label drug use[J]. N Engl J Med, 2012, 367: 1279-1281. DOI: 10.1056/NEJMp1208347
    [24]
    医疗机构药事管理规定. 中华人民共和国中央人民政府[EB/OL]. (2011-03-30)[2022-09-13]. http://www.gov.cn/zwgk/2011-03/30/content_1834424.htm.
    [25]
    Vargas-Rivas JE, Sabater-Hernández D, Calleja-Hernández MA, et al. Role of the hospital pharmacy and therapeutics committee in detecting and regulating off-label drug use[J]. Int J Clin Pharm, 2011, 33: 719-721; author reply 722-723. DOI: 10.1007/s11096-011-9532-z
    [26]
    Zheng Z, Zeng Y, Huang H. Pharmacists' role in off-label drug use in China[J]. Eur J Hosp Pharm, 2018, 25: 116.
    [27]
    Skledar SJ, Corman SL, Smitherman T. Addressing innova-tive off-label medication use at an academic medical center[J]. Am J Health Syst Pharm, 2015, 72: 469-477.
    [28]
    Loblova O, Csanadi M, Ozieranski P, et al. Patterns of alternative access: Unpacking the Slovak extraordinary drug reimbursement regime 2012-2016[J]. Health Policy, 2019, 123: 713-720. DOI: 10.1016/j.healthpol.2019.05.021
    [29]
    Teagarden JR, Dreitlein WB, Kourlas H, et al. Influence of pharmacy benefit practices on off-label dispensing of drugs in the United States[J]. Clin Pharmacol Ther, 2012, 91: 943-945. DOI: 10.1038/clpt.2011.367
    [30]
    Todd AE. No need for more regulation payors and their role in balancing the cost and safety considerations of off-label prescriptions[J]. Am J Law Med, 2011, 37: 422-443. DOI: 10.1177/009885881103700209
    [31]
    American Society of Clinical Oncology. Reimbursement for cancer treatment: coverage of off-label drug indications[J]. J Clin Oncol, 2006, 24: 3206-3208. DOI: 10.1200/JCO.2006.06.8940
    [32]
    国家市场监督管理总局. 药品注册管理办法[EB/OL]. (2020-07-01)[2022-09-22]. https://baike.baidu.com/item/%E8%8D%AF%E5%93%81%E6%B3%A8%E5%86%8C%E7%AE%A1%E7%90%86%E5%8A%9E%E6%B3%95/2566520?fr=aladdin.
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