Pediatric IBD workshop calls for innovative trial design

Participants at an FDA-sponsored workshop called on regulators and drug developers to minimize placebo testing and other hurdles to clinical trial participation for pediatric inflammatory bowel disease, and maximize the use of innovative trial designs, efficacy extrapolation and real world evidence to accelerate approval of new IBD drugs for children. The meeting concluded with a call from presenter Jeffrey Hyams for legislation that requires and incentivizes sponsors to collect pediatric pharmacokinetic/pharmacodynamic, exposure-response and short-term safety data before adult approval. Failure to obtain this data before adult approval is "unconscionable," said Hyams, gastroenterology division head and director of The Inflammatory Bowel Disease Center at Connecticut Children's Medical Center.

Off-label availability of drugs approved for adults, clinician and family aversion to placebo use, long washout periods and the need for multiple invasive endoscopies have limited clinical development of IBD drugs for pediatric patients...