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

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.

Safety of Off-Label Pharmacological Treatment in Pediatric Neuropsychiatric Disorders: A Global Perspective From an Observational Study at an Italian Third Level Children’s Hospital

“…Bellis et al found an increased risk related to off label drugs used in oncological practice and the result became statistically insignificant when oncology patients were excluded ( Bellis et al, 2014 ). Thus, it is possible that the greater risk of ADRs observed by the other studies resulted from a different pattern of off-label drug use ( Rusz et al, 2021 ).…”

Fluoxetine use for the treatment of anxiety-induced vomiting in 3-year-old children with complex trauma and developmental concerns: A Case Report

“…(3) Thus, approaching patient concerns in a multifactorial approach can help target underlying mechanisms for illness and behavior, such as the anxiety component of vomiting seen in the two cases. Second, when research is unavailable for rare, complicated cases, evidence-based decision making is needed 7 . Due to the complex medical history of some cases, off-label prescriptions of psychiatric medications may be warranted.…”

Off-Label Drug Use in Oncology

“…В литературе упоминается синоним термина «off -label use» -«an unlabeled use» [8]. Дефиниция «off -label use» отличается от значения термина «незарегистрированный препарат» [11]. Под незарегистрированным ЛП подразумевается продукт, не имеющий государственной регистрации на территории РФ и отсутствующий в Государственном реестре лекарственных средств.…”