OBJECTIVES: Lots of drugs are commonly prescribed in an off-label manner to pediatric patients under 19 years old in the real practices. The main purpose of this study is to investigate off-label prescription patterns for those patients, using some real-world data in Korea.

METHODS: Nine drugs frequently used in the pediatric clinical practices were selected: Carvedilol, Dopamine, Domperidone Maleate, Enalapril, Levetiracetam, Levodopa/Carbidopa complex, Mosapride Citrate Hydrate, Ramosetron, and Sildenafil. Using the Korean Health Insurance Review and Assessment Service database based on claims data from 2010 to 2015, we examined the main clinical areas and the primary diseases for off-label uses of those drugs in children.

RESULTS: Approximately 96% of the total 23,387,281 prescriptions in pediatric patients for the nine study medications were off-label. Those drugs were prescribed most frequently at the departments of pediatrics or internal medicine ranged between 43.4% and 93.2%. Some drugs were also used often in surgical departments: Thoracic surgery (22.5%) for Dopamine; Neurosurgery (35.3%) for Levetiracetam; Orthopedics (27.2%) for Ramosetron. Dopamine was primarily used in conditions originating in the perinatal period (35.2%) or for congenital malformations, deformations, and chromosomal abnormalities (32.4%). Levetiracetam was prescribed to treat disease of the nervous system (22.1%) and convulsions (20.6%), and Ramosetron was confirmed 31.7% use in diseases of digestive system.

CONCLUSIONS: The administration of off-label drugs is out-of-pocket in principle; however, over 22 million off-label prescriptions were still claimed to the health insurance in Korea. More flexible coverage policies need to be considered for some off-label drugs, especially for those without substitutes or in emergency care.