Module232025

07/05/2025

If the pharmaceutical is intended to treat a chronic paediatric disease, chronic toxicity studies should be conducted in one rodent and one non rodent species. In at least one of these studies, dosing should start at an age developmentally matched to the lowest age of the intended patient population. In principle, chronic studies that start dosing from ages that developmentally correlate to the youngest paediatric patient age can be sufficient to cover all ages and durations of paediatric use. These studies can replace adult animal chronic studies and a separate JAS.

The Organisation for Professionals in Regulatory Affairs

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A JAS in postweaning NHP is typically conducted in animals starting at 10 to 12 months of age, thus limiting the coverage of the lowest paediatric age ranges. A JAS in preweaning NHP should only be conducted in the situation of pharmaceuticals with first and primarily neonatal clinical use, and where alternative approaches to nonclinical safety assessment are not feasible. Studies with direct dosing of NHPs prior to weaning can require large numbers of mature dams to populate even a relatively small JAS. Therefore, the design and endpoints should be clearly justified based on the clinical concern.

The Organisation for Professionals in Regulatory Affairs

26