Module232025

07/05/2025

Dose levels should be selected to achieve some overlap in the range of exposure in adult animals to enable comparison of effects between young and adult animals. However, the high dose should not result in marked toxicity that can confound the growth and development endpoints and complicate the assessment. Dose adjustment (increase or decrease) during the course of a JAS should be considered in cases of substantial changes in systemic exposure due to maturation of the ADME systems.

The Organisation for Professionals in Regulatory Affairs

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A common clinical approach for the development of a paediatric-first/only pharmaceutical starts with a First Time in Human (FTiH) study in healthy adult volunteers prior to any paediatric trial. There are cases, however, where paediatric patients are treated without any prior adult patient or healthy volunteer data ( e.g ., for a life-threatening or debilitating disease that only exists in children or when the pharmaceutical cannot be given safely to adult volunteers). In these cases, the FTiH trial would be in paediatric patients and the nonclinical programme would generally include one JAS in a rodent and one JAS in a non-rodent species.

The Organisation for Professionals in Regulatory Affairs

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