Module232025

07/05/2025

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

3 Study Burden: Visit Duration, Content and Schedule 4 Intrusive Assessments (Tanner staging) 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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Blood volumes, patient and parent burden

Study Burden

Burden − 12 and 24 hr post-dose PK samples – repeat visits or overnight stay − Visit schedules - unable to avoid weekends − 7 hrs to complete psychometric questionnaires – exceeds permitted examination periods

− School absence may exceed legally permissible − Protocol requirements –vs- medical practice

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