Two decades of off-label prescribing in children: a literature review

Abstract

This review highlighted that off-label prescribing in children was found to be highly prevalent throughout the past two decades, persistently in the neonatal intensive care units. This suggests that besides legislative and regulatory initiatives, behavioural, knowledge aspects and efforts to integrate evidence into practice related to off-label prescribing also need to be evaluated and consolidated as part of the concerted efforts to narrow the gaps in prescribing for children.

Is puberty delaying treatment ‘experimental treatment’?

“…The use of a drug for an indication that is different from the one for which the drug is licensed, usually referred to as 'off label use' or 'off label prescription' is common in many areas of medicine, but we will focus here on pediatrics. Off label prescription is both common and necessary in pediatrics, because many drugs have only been tested on adults as part of the development process leading to licensing and are therefore only licensed for use in an adult population (Balan et al, 2018;Cuzzolin et al, 2003;Magalhães et al, 2015). Off-label prescribing in pediatrics is endorsed in general by the Royal College of Pediatrics and Child Health (RCPCH), and even for a sensitive area such as pediatric psychiatry by the British Association for Psychopharmacology (Sharma et al, 2016) which states that:…”

Gaining insight into irrational off‐label use of vidarabine through analysis of a spontaneous reporting system in China

“…This may result in therapeutic failure or toxic effects attributed to the differences between children and adults in drug pharmacokinetics and pharmacodynamics, different stages of ontogenesis of drug receptors, and immaturity of biotransformation pathways 2 . This kind of off‐label drug use has developed as an important issue worldwide 3,4 . Reportedly, only one‐third of drugs used to treat children have been investigated in paediatric populations and present appropriate information for use in children on the product label 1 .…”

Inclusion of key populations in clinical trials of new antituberculosis treatments: Current barriers and recommendations for pregnant and lactating women, children, and HIV-infected persons

“…In pediatrics, off-label drug use is a common practice and is largely based on adult studies without rigorously conducted pharmacokinetics (PK), dose-finding, or formulation studies in children [27]. Children, however, are not small adults.…”