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Unlicensed and off-label drug use in children: implications for safety
1Academic Division of Child Health, University of Nottingham, Derbyshire Children's Hospital, Uttoxeter Road, Derby, DE22 3NE, UK. imti.choonara@nottingham.ac.uk
Insights
Many children in hospitals receive unlicensed or off-label drug prescriptions. This practice is linked to a higher risk of adverse drug reactions in pediatric patients.
Area of Science:
- Pediatric Pharmacology
- Drug Safety
- Hospital Pharmacy
Background:
- A significant proportion of pediatric hospital patients, including neonates, receive medications that are not licensed or are prescribed off-label.
- Commonly used off-label medications include analgesics, antibiotics, and bronchodilators.
Purpose of the Study:
- To evaluate the association between the use of unlicensed or off-label drugs and the risk of adverse drug reactions in children.
- To synthesize existing evidence on the toxicity risks associated with non-standard drug use in pediatric populations.
Main Methods:
- Review of existing studies examining the incidence and outcomes of unlicensed and off-label drug use in hospitalized children.
- Analysis of prospective and retrospective data linking drug prescription status to adverse drug event occurrence.
Main Results:
- One prospective study indicated a significant association between the percentage of unlicensed/off-label drug use and the risk of adverse drug reactions.
- Two studies suggest an increased risk of severe adverse drug reactions with off-label or unlicensed drug use.
- Specific findings include 5/8 severe adverse drug reactions linked to off-label use and 14/19 severe adverse drug reactions associated with unlicensed or off-label prescriptions.
Conclusions:
- Limited data suggest a potential increased risk of adverse drug reactions, particularly severe ones, when children are treated with unlicensed or off-label medications.
- Further prospective research is needed to clearly define the risks associated with prescribing unlicensed and off-label drugs in pediatric care.
Abstract:
A significant number of children receive either an unlicensed or an off-label drug during their stay in hospital. Studies throughout Europe have shown that at least one-third of children in hospital and up to 90% of neonates in a neonatal intensive care unit receive such drug prescriptions. The medicines that are most frequently used off-label include analgesics, antibiotics and bronchodilators. The purpose of licensing a drug is to ensure safety, efficacy and quality. If a drug is used in a different manner, one would expect a greater risk of toxicity. Only three studies have commented on the risk of toxicity in relation to unlicensed or off-label drug use. Only one of these three studies prospectively tried to evaluate the risk associated with off-label and unlicensed drug prescription. This study suggested that the percentage of unlicensed and off-label drug use was significantly associated with the risk of an adverse drug reaction. Two studies looking at adverse drug reactions suggest that there is a greater risk of a severe adverse drug reaction occurring in association with the off-label or unlicensed use of drugs. One study found that five out of eight severe adverse drug reactions were associated with the off-label use of a medicine. The other study found that 14 of 19 drug prescriptions associated with 17 severe adverse drug reactions were either unlicensed or off-label. The risk of prescribing off-label and unlicensed drugs in children is not clear from the limited data available.