Module232025

07/05/2025

Additional nonclinical studies are not warranted when existing clinical safety data and risk mitigation strategies are considered sufficient to support paediatric use. In addition, a JAS is not warranted to confirm toxicity in target organs in which sensitivity to toxicity is not expected to significantly differ between adults and paediatric patients. Differences in target or off-target tissue development are, however, a concern that should be considered. If adult clinical data are available and exposure is not occurring at a vulnerable time of organ development, a JAS is not considered important to support initiation of short-term PK studies in paediatric patients.

The Organisation for Professionals in Regulatory Affairs

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Primary or secondary pharmacological properties of a pharmaceutical can be responsible for unwanted side effects. This can raise concerns for paediatric use if effects occur in systems/organs in development or if developing organs have a different sensitivity from mature organs. If the known pharmacology of a pharmaceutical has the potential to impact development in the intended paediatric population, or the role of the pharmacology on development is not understood or not reasonably predictable, further nonclinical investigations should be considered. Further nonclinical studies might not add value when the existing pharmacology information has already identified a particular hazard.

The Organisation for Professionals in Regulatory Affairs

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