Expediting Drug Development for Pediatric Inflammatory Bowel Disease: A Discussion with Stakeholders.

Objectives: Lack of scientific consensus on efficacy endpoints and outcome assessments presents a hurdle for global drug development in pediatric inflammatory bowel disease (IBD). Multiple stakeholders participated in a meeting November 2015, sponsored by The Pediatric IBD Foundation, which is a 501c3 organization formed by parents of children with IBD whose mission is to improve the lives of children with Crohn's disease and ulcerative colitis by supporting innovative research and educational programs ( www.pedsibd.org). Wth representatives of the Federal Drug Administration (FDA), European Medicines Agency (EMA), pediatric gastroenterologists, and representatives of the pharmaceutical industry, this meeting was organized to harmonize present thinking about various aspects of global drug development in pediatric IBD. The meeting was designed to be interactive, allowing participants from the pharmaceutical industry, regulatory agencies, academia and clinical practice an opportunity to collaborate in a public forum and to identify potential strategies to expedite drug evaluation in children. Methods: Prior to the meeting, a questionnaire focused on the hurdles hindering approval of medications used to treat children with IBD was sent to all participants as well as other pediatric gastroenterologists in North America and Europe with expertise in IBD. Responses were reviewed by the steering committee and results presented at the meeting. Following the presentation of the survey, participants were divided into small groups comprised of representatives from academia, industry, regulatory agencies, and members of the Pediatric IBD Foundation and assigned the task of working together to find solutions to the hurdles that had been identified. Results: Hurdles hindering approval included: (i) pediatric trials start later in the development process; (ii) lack of enrollment in pediatric trials; (iii) lack of monitoring safety registries that might expedite approval; (iv) different priorities among stakeholders. Conclusions: This one-day meeting discussed how to expedite pediatric drug development in IBD therapy. Hurdles for achieving approvals of pediatric indications for treatments of IBD were identified. (C) 2017 by European Society for Pediatric Gastroenterology, Hepatology, and Nutrition and North American Society for Pediatric Gastroenterology,

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