Abstract
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.
Highlights
Off-label practice is a concept that has been in existence for many years
The off-label practice was first recognized in the European Directive 2010/84/EU, which addresses the responsibility of marketing authorization holders (MAHs) to continuously monitor the drug product and provide all the available information, including the ones that are not in the scope of the marketing authorization
This directive emphasizes the adverse events associated with this practice, encouraging the MAHs to redefine and to clarify the phrase ‘adverse event’ as to comprise the effects associated with the usage outside the specifications of Summary of
Summary
Off-label practice is a concept that has been in existence for many years. This concept has raised more and more awareness over the past years due to clinical advantages and liability perspectives, with the main focus on the patient’s safety. The off-label practice was first recognized in the European Directive 2010/84/EU, which addresses the responsibility of marketing authorization holders (MAHs) to continuously monitor the drug product and provide all the available information, including the ones that are not in the scope of the marketing authorization. This directive emphasizes the adverse events associated with this practice, encouraging the MAHs to redefine and to clarify the phrase ‘adverse event’ as to comprise the effects associated with the usage outside the specifications of Summary of. Medicines Agency (EMA), and National Drug Agency and Medical Devices from Romania (ANMDM) were consulted
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