FDA guidance addresses developing treatments for pediatric IBD
The US Food and Drug Administration (FDA) on Wednesday issued a draft guidance to spur the development of new treatments for pediatric ulcerative colitis (UC) and pediatric Crohn’s disease (CD), two types of inflammatory bowel disease (IBD).The guidance addresses study population, study design, efficacy considerations, and safety assessments. The guidance does not address extraintestinal manifestations, stricturing or fistulizing disease, or treatment of long-term complications of pediatric UC or CD.
FDA has approved three classes of medications for pediatric IBD: steroids (budesonide), mesalamines, and anti-TNF agents (infliximab, adalimumab), according to an agency spokesperson.
In the EU, a study found that there are “unacceptable delays,” or a 7-year gap, between authorizing new IBD drugs for adults and children, according to an article that appeared in the February 2023 issue of the Journal of Crohn’s and Colitis.
According to FDA, the recommendations in the guidance “are based upon the assumption that a robust development program is being conducted in adults and that efficacy data from adults will be available to help inform the pediatric program and to support extrapolation of efficacy.” Sponsors should consult with FDA if they do not have an adult IBD clinical development program.
Pediatric studies should adhere to the adult Phase III program with respect to the study design, patient population, endpoints, and timing of assessments.
Sponsors should enroll patients between 2 and 17 years of age; those suffering from monogenic IBD and inherited conditions should be excluded from trials.
FDA recommends a randomized, double-blind study design that evaluates at least two dose levels for each age or weight cohort for drugs that are administered chronically. It further recommends a blinded treatment period of at least 52 weeks to assess both early efficacy and durability of response over time.
For efficacy assessments, FDA recommends evaluating the proportion of pediatric subjects achieving clinical remission as the primary endpoint. Secondary endpoints can include a clinical response, corticosteroid-free remission, endoscopic improvement, endoscopic remission, and maintenance of remission.
FDA cautions that safety information from adult subjects may help to inform risk but cannot replace primary safety data in pediatric subjects. FDA also encourages sponsors to permit weaning from corticosteroids “at the earliest feasible time point after randomization.”
Sponsors seeking to use real-world evidence to support drug safety should discuss their proposed approaches with the appropriate review division early in drug development.
The deadline for submitting comments is 16 September 2024.
Guidance
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