Events

FDA’s Division of Gastroenterology and Inborn Errors Products and Division of Pediatric and Maternal Health in CDER, and the Office of Pediatric Therapeutics in the Office of the Commissioner are announcing a 2-day public workshop, “Assessment of Neurocognitive Outcomes in the Inborn Errors of Metabolism” and  “Advancing the Development of Pediatric Therapeutics: Assessment of Pediatric Neurocognitive Outcomes.” Day 1 of the workshop will focus on approaches for assessing the efficacy of therapeutic products based on neurocognitive outcomes in patients diagnosed with inborn errors of metabolism disorders. On day 2 of the workshop, participants will discuss identification of signals in animal studies and clinical trials that warrant further clinical investigation and testing that may be predictive of neurocognitive outcome in children. Participants will also discuss strategies and methods to address the challenges of assessing long-term neurocognitive outcomes for products used to treat pediatric patients.