Miguel Saps ¹, Samuel Nurko, Marc Benninga, Carlo Di Lorenzo, Jose M Garza, Nikhil Thapar, Kelly Richards, Jessica J Lee, Tara Altepeter

J Pediatr Gastroenterol Nutr. 2021 Jun 1. doi: 10.1097/MPG.0000000000003191.Online ahead of print.

Author information

• ¹Division of Pediatric Gastroenterology, Hepatology and Nutrition, Holtz Children's Hospital, Miami, FL; University of Miami, Miller School of Medicine, Miami, FL Center for Motility and Functional Gastrointestinal Disorders, Boston Children's Hospital, Boston, MA; Professor of Pediatrics, Harvard Medical School, Boston, MA Department of Pediatric Gastroenterology and Nutrition, Emma Children's Hospital, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands Division of Pediatric Gastroenterology, Hepatology and Nutrition at Nationwide Children's Hospital, Nationwide Children's Hospital, Columbus, OH GI Care for Kids, Atlanta, GA; Neurogastroenterology and Motility Program, Children's Healthcare of Atlanta, Atlanta, GA Neurogastroenterology and Motility Unit, Department of Paediatric Gastroenterology, Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom; UCL Great Ormond Street Institute of Child Health, London, United Kingdom Paediatric Gastroenterology, Hepatology and Liver Transplant, Queensland Children's Hospital, Brisbane, Australia Division of Regulatory Operations for Immunology and Inflammation, Center for Drug Evaluation and Research, U.S. Food and Drug Administration Division of Gastroenterology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration.

Abstract

Pediatric functional gastrointestinal disorders including irritable bowel syndrome with constipation and functional constipation are common conditions in childhood, but no drugs are FDA approved for chronic use in pediatric patients with these disorders. Despite efforts to better standardize the diagnosis of these conditions in children (including recent modifications to the Rome criteria), conducting pediatric clinical trials to support drug approval remains a challenge. In March 2018, the U.S. Food and Drug Administration, in collaboration with the NASPGHAN, AGA, and ACG, convened a public workshop to discuss the challenges and opportunities in conducting pediatric clinical trials in functional GI conditions. The workshop assembled gastroenterologists, psychologists, patients, patient advocates, regulators, and industry representatives to discuss trial design and conduct including alternative designs, eligibility criteria, instruments for patient- and observer-reported outcomes, and optimal primary endpoints to support regulatory approval. This report summarizes the workshop, key challenges and knowledge gaps identified, and outlines areas where further research efforts are needed to overcome barriers to developing drugs to treat these conditions.