Support for Pediatric Inflammatory Bowel Disease Drug Developers in New FDA Draft Guidance

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A draft guidance document, “Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment,” was released for public comment by FDA on July 19, 2024. The draft was prepared by FDA’s Division of Gastroenterology, within the Center for Drug Evaluation and Research. The comment period for the guidance for industry ends Sept. 17, 2024 (1).

According to FDA, the intent of the draft guidance is to assist sponsors in the clinical development of drugs that treat pediatric patients with inflammatory bowel disease (IBD)—such as pediatric ulcerative colitis or Crohn’s disease, to name two (1). In the document, FDA provides recommendations for the necessary attributes of clinical studies for these drugs; among those attributes are study design and population, efficacy concerns, and assessments of safety.

What the guidance does not do, FDA said, is address “extraintestinal manifestations,” or stricturing or fistulizing disease. Nor does it comment on either treatment or prevention of long-term pediatric ulcerative colitis or Crohn’s disease complications (1). Treatment goals, according to the document, are to first provide relief to the pediatric patient by reducing or resolving signs and symptoms of active disease, and also heal or at least control any underlying inflammation or resulting complications.

In children, ulcerative colitis symptoms include diarrhea, hematochezia (rectal bleeding, or blood in stool), abdominal pain, or fecal urgency (1). By contrast, FDA cited prior research specifying that pediatric Crohn’s disease can affect any part of the gastrointestinal tract from the mouth to the anus. It shares the symptoms of diarrhea and abdominal pain with ulcerative colitis, but can also cause fatigue, weight loss, perianal disease, and especially relevant to pediatrics, growth impairment. Crohn’s disease is also associated with fibrosis, strictures, and perforations.

On a larger scale, FDA said that the aforementioned extraintestinal manifestations of pediatric IBD may include: aphthous stomatitis (canker sores), uveitis (eye inflammation), skin conditions such as pyoderma gangrenosum, erythema nodosum, and psoriasis, primary sclerosing cholangitis affecting the liver and gallbladder, peripheral arthritis, and decreased bone mass (1).

FDA suggested that the evolution of drugs to treat inflammatory bowel disease in adult patients would provide a parallel blueprint for the process for children.

“The recommendations for clinical study design in this 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,” the document says.

While long-term complications of pediatric inflammatory bowel disease are not covered in this draft guidance document, it does say that drugs intended for long-term treatment of either ulcerative colitis or Crohn’s disease should be given to pediatric subjects at the dosing regimen to be marketed for a period of at least 52 weeks (1).

Online, written comments can be submitted here and should specify the docket number FDA-2024-D-2682 (2).

References

1. FDA, Draft Guidance for Industry, Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment (CDER, July 2024).
2. Regulations.gov. Pediatric Inflammatory Bowel Disease: Developing Drugs for Treatment; Draft Guidance for Industry; Availability. regulations.gov/docket/FDA-2024-D-2682, July 19, 2024 (accessed July 23, 2024).