Off-label drug use in the treatment of rare diseases:the current situation / 国际药学研究杂志

ABSTRACT

The diagnosis and treatment of rare diseases are a significant social public health work. Due to the low variety and poor accessibility of orphan drug, off-label use is common in the field of rare diseases at home and abroad. Off-label use may be the effective treatment for life-threatening diseases in patients with rare diseases, but it also raises issues related to drug safety and efficacy, medical liability and drug reimbursement, so the supervision of off-label drug use is of particular importance. This article reviews the current situation of off-label drug use and their application in thefield of rare diseases treatment, hoping to arouse widespread concern in society and ensure medication safety for patients with rare diseases through the joint efforts of medical institutions and regulatory agencies.