If age and weight are not identified as covariates for the model, if the flat exposure-response curve is observed in adult population, if in vitro tests and biology theory claim that there are no effects of growth and maturation on the observed levels of PD-markers (or components of the... more »
How could we maximize use of all available data (e.g., existing clinical data in adults, in pediatric population in other diseases, MOA of drug, etiology of the disease, maturation of drug target, safety data in adults and pediatric patients on similar compounds, preclinical data, in vitro data etc.), to minimize data requirement from clinical trial in neonate and older pediatric population.
How similar is the safety profile in children compared to that previously observed in adults?
Are pediatric trials without a preceding trial in adults more likely to be stopped for safety concerns?
Answers to these questions could help accelerate pediatric... more »
What information has been submitted concerning the palatability and/or acceptability of paediatric products that the agency has found acceptable? Where data has NOT been acceptable what additional data was requested and was that provided?
For drugs metabolized through CYP pathways, are there differences and if so what are they across pediatric populations when compared to adult populations
• Biomarkers may serve as alternatives to invasive diagnostic techniques (e.g. Liver biopsy in NAFLD), early indicators of disease progression, and predictors of treatment efficacy. Does the pooled database support this?
• Specifically, what is the utility of... more »
Some characteristics are known to differ between age groups in aGvHD, such as the... more »
• Considering the recently published paper on the long term complications observed in the TODAY study, long term cardiovascular... more »