Thank you for your interest in the FDA Division of Pediatrics and Maternal Health Research Initiative. Research questions that are submitted will appear on this page and be considered by the FDA.
In some indications the established primary clinical endpoint may be a difficult to obtain (for example because it requires a biopsy). Could one use the FDA data to qualify biomarkers or surrogate endpoints for use in pediatric trials instead? If such data don't exist, could one plan prospectively to generate such data over time?
If a compound has bioavailability or clearance that is a function of dose in adults, indicating saturation of either absorption or elimination above a certain dose, respectively, how can these relationships be scaled to a pediatric population who will be receiving a smaller dose?
Can FDA pool data to provide a list of examples of neonatal and pediatric modeling and simulation used for approval
Can FDA pool data to provide applications pediatric age range that is most often used. Based on the data, could this be used to help standardized pediatric ages (birth to <16, <17, <18 years?) as each FDA center varies.
Can FDA pool data to provide most common pediatric lab normal reference ranges for all age groups?
Can FDA pool data to obtain the number of approved products for pediatric indications that have utilized various sampling methods such as microsampling, dried blood spots, etc
Are information sheets becoming so large that they are defeating the purpose for which they were created? Information sheets are continuing to grow, often because sponsors' legal teams insist upon providing progressively more information to reduce the prospect of litigation, in addition to data privacy requirements. However, a clear difference exists between the provision of data and its assimilation.