Off-label and unlicensed use of medicines for children

The lack of specific drugs and labelling recommendations for the paediatric population is a longstanding problem. Physicians frequently prescribe marketed medicines as off-label on the basis of clinical practice and medical knowledge. Moreover ‘unapproved’ drugs are employed in different formulations, routes, combinations to adapt them to children. TEDDY studied the definitions of off-label and unlicensed use of medicines in paediatrics as defined by regulatory agencies and by scientific literature. This study confirmed the need for a common definition for unlicensed and off-label uses to be incorporated into the European legislation. On the basis of the results of this investigation, TEDDY conducted a survey in order to reach a common definition with the intention to favour the use of a European official regulatory terminology and facilitate pharmaco-epidemiological research. The definitions will be circulated within the scientific community and recommended to be adopted by relevant regulatory authorities.