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A Collaborative Effort to Advance Drug Development in Pediatric Constipation and Irritable Bowel Syndrome
Miguel Saps1, Samuel Nurko2, M A Benninga3
1Division of Pediatric Gastroenterology, Hepatology and Nutrition, Holtz Children's Hospital, Miami, FL; Miller School of Medicine, University of Miami, Miami, FL.
Insights
Developing new medications for pediatric functional gastrointestinal disorders is difficult due to challenges in clinical trials. This workshop identified key issues and research needs to improve drug development for these common childhood conditions.
Area of Science:
- Pediatric Gastroenterology
- Clinical Trial Design
- Regulatory Science
Background:
- Pediatric functional gastrointestinal disorders (FGIDs) like irritable bowel syndrome with constipation and functional constipation are prevalent in children.
- Currently, no U.S. Food and Drug Administration (FDA)-approved medications exist for the chronic treatment of these FGIDs in pediatric populations.
- Standardizing diagnoses and conducting pediatric clinical trials for drug approval present significant challenges.
Purpose of the Study:
- To address the challenges and explore opportunities in conducting pediatric clinical trials for functional gastrointestinal conditions.
- To facilitate the development of FDA-approved drugs for chronic use in pediatric FGIDs.
- To identify knowledge gaps and areas for future research in pediatric FGID clinical trials.
Main Methods:
- A public workshop was convened in March 2018 by the FDA, in collaboration with major pediatric gastroenterology and gastroenterology societies.
- The workshop brought together diverse stakeholders including clinicians, psychologists, patients, advocates, regulators, and industry representatives.
- Discussions focused on trial design, eligibility criteria, outcome measures, and endpoints for regulatory approval.
Main Results:
- Key challenges and knowledge gaps in pediatric clinical trial design for FGIDs were identified.
- Opportunities for improving trial methodologies and outcome assessments were discussed.
- The need for further research to overcome barriers in drug development for pediatric FGIDs was highlighted.
Conclusions:
- Conducting pediatric clinical trials for FGIDs requires innovative approaches and collaborative efforts.
- Addressing identified challenges is crucial for advancing the development of effective treatments for children with FGIDs.
- Further research is essential to overcome regulatory and logistical hurdles in pediatric drug development for these conditions.
Abstract:
Pediatric functional gastrointestinal disorders including irritable bowel syndrome with constipation and functional constipation are common conditions in childhood, but no drugs are U.S. Food and Drug Administration (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, FDA, in collaboration with the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition, American Gastroenterological Association, and American College of Gastroenterology, convened a public workshop to discuss the challenges and opportunities in conducting pediatric clinical trials in functional gastrointestinal 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.
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