Off-Label Treatment in Pediatrics – Associations, Latest Recommendations

Abstract

Off-label treatment is often seen in pediatrics, especially in pediatric intensive care and neonatal departments. It is linked to a broad range of factors, including restriction of pharmacological studies for ethical reasons, different pharmacokinetic and pharmacodynamic properties of medicines due to age and physiological differences in children, and the difficulty of manufacturing and do­sage determination. These gaps increase the likelihood of adverse reactions. Various measures are being taken to avoid unnecessary risks to children without depriving them of potentially effective pharmacotherapy. This study aimed to analyse the studies reported in literature on the relationship between off-label prescribing, the latest recommendations, and the impact of this treatment on the pediatric population. The literature shows that off-label are often prescribed due to children’s younger age, respiratory or rare dise­ases. Relevant groups of commonly misused medicines are as follows anti-infectives, respiratory medicines or treatment for neurological conditions. It has been ob­served that the most common off-label prescriptions are related to inappropriate dosage, patient age or indications for use. To deal with the problem, researchers are en­couraging the research community to find new, scienti­fically supported and effective treatments by increasing the sharing of high-quality research and data. This is to achieve trustworthy, evidence-based medical care and to protect children from ineffective or even harmful treat­ments. For off-label treatment, the European Academy of Paediatrics and the European Society of Perinatal and Paediatric Pharmacology have issued new guidelines to ensure the safest possible care for children.