The off-label use of psychotropic medicines in paediatric patients in Australian primary care

Psychotropic medicine use in paediatric patients is increasing across many countries, despite limited regulatory approvals and evidence for safety and efficacy. This study investigated the extent of off-label use (OLU) of psychotropic medicines in paediatric patients in Australian primary care, using data from the Bettering the Evaluation and Care of Health (BEACH) program. We retrospectively analysed data collected from 15,276 general practitioners between 2000 and 2016, encompassing 144,397 encounters with children and adolescents aged 3-17 years. Psychotropic medicines were defined by the Anatomical Therapeutic Chemical Classification codes N05 (Psycholeptics) and N06 (Psychoanaleptics), except for prochlorperazine. OLU was defined as use outside of the approved age or indication as specified in the Australian Product Information. A total of 1650 psychotropic medicines were prescribed, with sertraline (14.55 %), fluoxetine (12.48 %), methylphenidate (9.27 %), diazepam (5.52 %), and citalopram (5.27 %) being the most common. Of these, 75.03 % (95 % CI: 71.98, 78.08) were off-label, predominantly due to age (51.74 %, 95 % CI: 48.22, 55.26), followed by indication (23.29 %, 95 % CI: 20.32, 26.27). Off-label rates were highest for citalopram and fluoxetine (both 100 %), followed by sertraline (97.08 %, 95 % CI: 94.94, 99.23), diazepam (41.76 %, 95 % CI: 31.43, 52.09), and methylphenidate (12.42 %, 95 % CI: 7.13, 17.70). Off-label psychotropic medicine use in paediatric patients is common in Australian primary care, with most OLU classified as age-based rather than indication-based. These findings demonstrate high off-label rates and, given limited paediatric evidence, underscore the need for targeted guidelines, enhanced prescriber education, and further research to improve safe and effective use in this population.