Module232025

07/05/2025

ETHICS CHALLENGES IN PAEDIATRIC CLINICAL TRIALS My personal top 10 (based on ~600 protocols reviewed 2017-2023)

5 Endpoints (adult, not child, medical review for SAEs) 6 Pregnancy (child privacy, post-trial follow-up, pregnant partner) 7. Post-Trial Access to IMP (protocol ambiguous or silent, toxicology) 8. Communication of Trial Results (onus on participants, child to adult) 9. “Aging Out” (no process, incorrect process) 10. Paediatric Assent and Information Sheets (length, complexity)

The Organisation for Professionals in Regulatory Affairs

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End-point validity

− Questionnaires taken from adult studies − tobacco and alcohol consumption in a neonatal study − “Johnny’s Mexican so he can translate into Spanish” (the Sellotape story) − 21st Century Cures Act (21CCA) requires psychometric instruments to be validated before deployment in pivotal trials − For children, that means linguistic, cultural and age-appropriate validation − Few instruments validated for under 10s − Can you ethically start a trial if you don’t know whether the instrument you plan to use accurately captures information?

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