Module232025

07/05/2025

An understanding of the overall clinical development plan is needed to design an appropriate, efficient nonclinical plan. A weight of evidence (WoE) based decision should be made to determine whether additional nonclinical investigations are warranted to support the paediatric population. As clinical development progresses, adjustments to the WoE can be made based on all the available data at that time. The outcome of a WoE assessment can be different for different applications of the same pharmaceutical depending on paediatric age, indication and duration of treatment.

The Organisation for Professionals in Regulatory Affairs

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An early consideration of nonclinical support for paediatric pharmaceutical development is recommended. In this respect, changing the design and/or timing of the traditional nonclinical program is one way to address potential safety concerns for the paediatric patient. For example, dosing can be initiated at a younger age in a repeated-dose toxicity study to support the corresponding developmental stages in paediatric patients. Another approach could be to conduct the Pre- and Postnatal Development (PPND) study earlier than the normal drug development paradigm, with modifications such as toxicokinetics (TK) in offspring and additional endpoints. These changes can replace or refine the design of a juvenile animal study (JAS).

The Organisation for Professionals in Regulatory Affairs

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