Off label and unlicensed drugs use in paediatric cardiology

Objectives. The use of drugs in an off label or unlicensed manner to treat children is a widespread phenomenon in Europe and the United States. The incidence of unlicensed and off label prescribing in paediatric cardiology practice has not been studied to date. This study was designed to assess the extent and nature of off label and unlicensed drug use in paediatric cardiology inpatients. Methods. In a prospective study, drug prescriptions in a paediatric cardiology ward were reviewed during a 2-year period. Data were collected and analyzed by special software created for this purpose. Results. The children (n=544) studied varied in age from 4 h to 18 years. One or more off label and unlicensed prescriptions were given to 414 (76%) patients. Of the 2,130 prescriptions given during the 2-year period, more than one-half were unlicensed (11%) or off label (47%). While children aged 2-11 years received most of the unlicensed drug prescriptions (17%), neonates, who did not receive unlicensed drugs, led (64%) in the use of off label drugs. Conclusions. This study showed that the problem of off label and unlicensed drug use also exists in paediatric cardiology. The findings imply that the phenomenon of off label and unlicensed use of drugs in children can be correlated with the deficiency of paediatric drug formulations on the global market and insufficient data from clinical studies which must be performed to confirm the efficacy and safety of drugs in the paediatric population. Therefore, efforts to improve paediatric labelling are important and need the full support of all involved.