Module232025

09/05/2025

Pediatric extrapolation

The Organisation for Professionals in Regulatory Affairs

17

Pediatric extrapolation

• Disease similarity: determine the degree of similarities and/or dissimilarities of the disease • Pathophysiology, disease definition (e.g. diagnostic criteria), course of the disease • Drug pharmacology: potential impact of maturation-related differences • ADME, PD (primary and secondary), mechanism (e.g. expression level and sensitivity of the drug target, downstream effectors) • Response to treatment: changes on the dose/exposure and clinical response • Assess factors that impact response (e.g. maturation, prior treatments, concomitant medications, comorbid disease, organ function, genetic makeup)

The Organisation for Professionals in Regulatory Affairs

18