Most drugs prescribed in paediatric practice are used out with their marketing authorisation, or off label. At least a third of hospitalised children and 90% of children in neonatal units are prescribed such medicines. While the assumption has been that the off label use of medicine is safe, recent evidence suggests that it may be associated with an increased level of adverse drug reactions, and possible treatment failure. The aims of this study were to assess current attitudes of Scottish hospital based paediatricians to the use of off label medicines and their attitudes to the performance of clinical trials in children.

All paediatric specialist registrars and consultants based in Scottish hospitals were sent a 25 point questionnaire, comprised of 24 multiple choice questions, 3 of which also allowed written comment, and 1 free text question. Topics covered included knowledge of, reasons for and adverse experiences with off label prescribing, plus attitudes towards the need for and the performance of clinical studies in children. Two hundred and seventy nine questionnaires were sent out and 142 (55%) returned (73% were consultant responses). Approximately 90% of responders admitted to being familiar with the concept of off label prescribing and 97% to knowingly prescribing off label medication. The reasons given for off label prescribing in descending order were prescribing for a younger age, higher dose than recommended, a different indication, a different route of administration and a different formulation. Fifty five percent of responders believed that the use of off label medicines disadvantaged children, and 47% that they had concerns about off label use. The reasons given were lack of safety, efficacy, pharmacokinetic, and long-term data, and concerns about the legal status. While 69% of doctors admitted to concerns about the safety of off label medicines, only 17.5% had experienced an adverse event and 43% treatment failure due to off label usage. Neonatologists expressed least concern about the safety (65%) or efficacy (47%) of off label medicines and 100% reported they had not experienced ADRs or experienced treatment failure (76%). The most common medical areas for off label use were neurological (25%), gastrointestinal (14%), neonatology (13%), respiratory (11%), and infection (7%). While 53% said they told parents they were prescribing an off label medicine, 64% admitted that they did not ask for informed consent from the parents and 60% that they did not inform the GP. The most important sources of prescribing information were the BNF (87%), Medicines for Children (77%) and colleague experience (64%). Only 52% believed that all new medicines should undergo clinical trials in children, and 36% that generic medicines should also undergo clinical trials, however 78% agreed that the government should use incentives to stimulate drug research in children. 53% of replies stated that they would be willing to take part in clinical studies, 61% that they would recruit their own patients and 73% that they would allow their children to take part in such studies.

Clearly among Scottish paediatricians there is concern about off label use of medicines, although the majority take a pragmatic approach to such use and the need for clinical trials in children.