Module232025

5/7/2025

Safety data

Does the toxicity profile and/or its impact differ compared with adults or between different paediatric age groups? Examples include:  When the drug is a new molecular entity for a new class of drugs  When there are known on- and/or off- target age-related safety concerns  When there are significant differences in the paeds population of special importance (background therapy)  When the drug has a narrow therapeutic index Further assess tolerability using age appropriate patient-reported outcomes needed Acceptability of severe or life-threatening ADRs vs those affecting QoL depends on disease setting • Effects on organ maturation, growth and CNS development, including fertility • Post-approval follow-up : CTs and/or disease/drug registries and/or RWE

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Where does extrapolation fit ?

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