OBJECTIVE: To probe into the ethical problems of off-label drug use in children

and to summarize the key points of ethical practice.METHODS: Retrospective analysis was conducted on 83 applications of off-label drug use submitted to the Ethics Committee of Children’s Hospital of Fudan University from Jun.2012 to Jun.2019.RESULTS:Among the 83 applications for off-label drug use

6. 02%（ 5 pieces） was over administration routes

55. 42%（ 46 pieces） was over indications

and 68. 67%（ 57 pieces） was over ages

of which over indications and over ages accounted for 30. 12%（ 25 pieces）.>80% of clinical departments of our hospital applied for off-label drug use.The top five categories of disease were rheumatic diseases

neurological diseases

malignant solid tumors

neonatal diseases and infectious diseases

respectively.The top five categories of drugs ranked by application of off-label drug use were biological preparations（ 15 pieces）

antibiotics（ 14 pieces）

immunosuppressive drugs（ 13 pieces）

antiepileptic drugs（ 7 pieces）

antitumor drugs（ 3 pieces） and antipsychotics（ 3 pieces）

respectively.With regard to the results of the ethical review

59 cases were approved in the initial review（ 71. 08%）

and 21 cases were approved after revision（ 25. 30%） and 3 cases（ 3. 61%） were disagreed.CONCLUSIONS: The key point of the ethical review of offlabel drug use in children lies in the strict review of the indications

scientific validity and informed consent of off-label drug use

which can only be approved for clinical use after being reviewed and approved by the Ethics Committee and the Pharmaceutical Administration Committee.