# 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

## TL;DR

Many children with inflammatory bowel disease are given medications not approved for pediatric use, with higher rates in those with ulcerative colitis and girls.

## Contribution

This study quantifies the prevalence and risk factors for off-label medication use in pediatric IBD patients using a large registry.

## Key findings

- 17.6% of pediatric IBD patients were exposed to non-FDA-approved medications.
- Ulcerative colitis patients and females had higher exposure rates.
- Exposure occurred at a mean age of 12.5 years, 2.9 years after diagnosis.

## Abstract

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.

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).

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.

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.

Significant delays exist in obtaining pediatric approval for inflammatory bowel disease (IBD) therapies.A large number of pediatric IBD patients are exposed the therapies which are not approved for pediatric use.

Significant delays exist in obtaining pediatric approval for inflammatory bowel disease (IBD) therapies.

A large number of pediatric IBD patients are exposed the therapies which are not approved for pediatric use.

Using the ImproveCareNow pediatric registry, a survey of >16,000 pediatric IBD patients was conducted, revealing that >17% were exposed to a medication not approved for pediatric use.Risk factors for use of nonapproved medications included diagnosis of ulcerative colitis and female sex.

Using the ImproveCareNow pediatric registry, a survey of >16,000 pediatric IBD patients was conducted, revealing that >17% were exposed to a medication not approved for pediatric use.

Risk factors for use of nonapproved medications included diagnosis of ulcerative colitis and female sex.