Unlicensed and off-label drug use in children

The purpose of licensing is to ensure that medicines are marketed only after having been examined for safety, efficacy, and quality. When a drug is prescribed outside these parameters, this support is lacking. Despite this, unlicensed and off label drug use in children is widespread. This is ironical to say the least, since the licensing process itself was introduced in the US and European countries mainly as a response to drug-related tragedies [chloramphenicol-induced gray baby syndrome, thalidomide-induced phocomelia and deaths following diethylene glycol poisoning] that occurred in newborn babies, infants and children. Estimated to vary from 10-72% [Table - 1], the magnitude of such use varies amongst others, according to the level of healthcare available, subspecialty concerned and certain patient characteristics. The prevalence of off-label and unlicensed drug use is higher in neonates and infants and in premature and low birth-weight babies. Topical preparations such as eye drops, ear drops and dermatological products also account for much of the off-label and unlicensed drug use, as these are hardly ever subjected to formal evaluation in children. Amongst the systemic drugs, bronchodilators, anti-migraine preparations, gastro-intestinal agents, anti-hypertensive agents, oral hypoglycemic agents, anti-spasmodics, oxymetazoline, ondansetron and amphotericin B, are commonly prescribed off-label.