OBJECTIVE： To provide suggestions for standardizing and protecting the off-label drug use. METHODS： According to the field interviews with pharmacists and lawyers， the present situation of off-label drug use in judicial practice at home and abroad， and relevant countermeasures were put forward. RESULTS & CONCLUSIONS： At present， there was no mechanism for off-label drug use in the laws and regulations in China； there was only “special circumstances” statement in sporadic terms， while lacking explanation. In judicial practice of medical disputes， judges had different identifications in various references， such as the legality of routine and guidelines， teaching medical materials， clinical pathways， and internal regulations of medical institutions prepared by authoritative departments. Meanwhile， the judicial environment of hearing cases by identification made the off-label drug use had impartial adjudication. Legislation in 6 countries， including America， allows reasonable off-label drug use； FDA required off-label drug use must be for the benefit of patients rather than clinical trials， and it also should protect the patient’s right to know. China should establish the authority specification of off-label drug use， providing scientific identification procedures， protect the patient’s informed consent by law. Medical institutions should develop relevant management system and processes to regulate medical practices and ensure off-label drug use in line with the interests of doctors and patients.