Module232025

12/05/2025

Common issues discussed at PDCO

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Summary of issues the PDCO often discusses in PIPs

• Medicine should be timely available to all children with therapeutic need  Waiver acceptable? If yes, cut-off age? Waiver granted per condition, not indication • Adolescents in adult studies ? • Formulation must be appropriate for the target population (for example age-appropriate IV volumes, excipients to be carefully evaluated for their suitability) • Weight of evidence approach to justifying (lack of) NC studies • What is the purpose of the proposed M&S study • Unclarity related to use of extrapolation, structured guidance not included in the scientific document • Appropriate endpoints in studies to demonstrate clinical benefit  clinical EPs versus biomarkers • Clinical studies in PIP must primarily aim to provide data sufficient for the CHMP to decide on benefit/risk  study design, study duration, control and sample size  but sometimes need to have two (PIP) objectives (early go/ no-go vs pivotal evidence generation)

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https://www.ema.europa.eu/en/documents/scientific-guideline/structured-guidance-use-extrapolation_en.pdf

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