Advancing Pediatric Drug Development

Pediatric biomarkers

Briefly describe your research question :

There is extreme interest in identifying pediatric specific biomarkers for multiple disease states including NAFLD, heart failure, chronic kidney disease (CKD).

• 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 imaging studies (ultrasound, Fibroscan, MRI-PDFF, MR elastography in NAFLD; ECHO/MRI in heart failure) as biomarkers? Are these imaging studies suitable as components of composite endpoints as are often used in adult trials?

• Considering the difference in etiology for CKD in children and adults, are there biomarkers or imaging that could be useful for children with congenital forms of CKD?

• How can inter operator variability be effectively addressed in clinical trials?

• Is there a correlation between non invasive methods of diagnosis and biopsy in pediatric NASH?

• Can a risk scale for disease be developed using the combination of biomarkers, imaging, clinical parameters?

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Idea No. 206