Off-Label Medication: From a Simple Concept to Complex Practical Aspects

Off-label use of drugs is widely known as unapproved use of approved drugs, and it can be perceived as a relatively simple concept. Even though it has been in existence for many years, prescribing and dispensing of drugs in an off-label regimen is still a current issue, triggered especially by unmet clinical needs. Several therapeutic areas require off-label approaches; therefore, this practice is challenging for prescribing physicians. Meanwhile, the regulatory agencies are making efforts in order to ensure a safe practice. The present paper defines the off-label concept, and it describes its regulation, together with several complex aspects associated with clinical practices regarding rare diseases, oncology, pediatrics, psychiatry therapeutic areas, and the safety issues that arise. A systematic research of the literature was performed, using terms, such as off-label , prevalence , rare diseases , oncology , psychiatry , pediatrics , and drug repurposing . There are several reasons for which off-label practice remains indispensable in the present; therefore, efforts are made worldwide, by the regulatory agencies and governmental bodies, to raise awareness and to ensure safe practice, while also encouraging further research.

Automated summary

Off-label use of drugs, the unapproved use of approved drugs, has been a longstanding issue due to unmet clinical needs. It presents challenges for physicians in various therapeutic areas, prompting efforts by regulatory agencies to ensure safe practice. Awareness and further research are being promoted globally to address the indispensable nature of off-label practice.