A significant number of pediatric inflammatory bowel disease patients are exposed to a medication not approved by the Food and Drug Administration for pediatric use.

Objectives: Inflammatory bowel disease (IBD) presents before age 20 years in ~25% of patients. Regulatory approvals of IBD medications for pediatric use are often delayed. Nevertheless, many pediatric patients receive medication not approved for pediatric use. The aim of this study was to summarize the exposure of pediatric patients to IBD medication without regulatory approval for pediatrics.

Methods: A retrospective study of exposure to nonapproved biologic/small molecule medications in patients listed in the ImproveCareNow registry with IBD diagnosed before September 2023 was conducted. Chi-square tests were used to see whether exposure differed by demographics, the cohort of IBD diagnosis (Crohn's disease [CD], ulcerative colitis [UC]), or exposure timeframe (i.e., age at exposure and time from diagnosis to exposure).

Results: 16,085 eligible patients with year of diagnosis from 1993 to 2023 were identified. 2836 patients (17.6%) were exposed to a medication not approved by the Food and Drug Administration (FDA) for pediatric use. The mean age of exposure was 12.5 years and the mean time from diagnosis to exposure was 2.9 years. Patients with UC (23.8%) were significantly more likely to have exposure than CD (15.3%). Female patients (19.1%) were more likely to have exposure than male patients (16.5%). Patients diagnosed with IBD between ages 0-5 years had the highest rate of exposure and shortest time from diagnosis to exposure.

Conclusions: A significant number of pediatric IBD patients were exposed to medication not approved by the FDA for pediatrics. There is an urgent need for more rapid approval of medications for IBD in pediatrics.