Module232025

09/05/2025

NCE for management of RD in adults + paeds

Separate studies for treatment and prevention

Study M&S-001 • Aimed at confirming suitability of using the adult dose in adolescents, and to define a body weight cutoff if needed, prior to enrollment of adolescents. • Via simple allometric scaling and using PK/PD thresholds as defined in adults.

Phase II dose-ranging ADULTS

Study M&S-002 • Update model from study M&S-001 using PK/PD data in adolescents. • Apply in simulation to aid in dose selection and study optimization for younger children (2-<12 years).

Phase III ADULTS + ADOLESCENTS

Study M&S-003 • Upon finalization of the paediatric clinical trial, observations gathered from children and adolescents will be used to support the extrapolation of efficacy from adults in a M&S based framework. • Through the development of an integrated PK/PD/clinical outcome model.

PK, safety, Confrimatory efficacy

Phase III CHILDREN 2-<12 yrs

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Thank you for your attention!

ACKNOWLEDGEMENTS Model Informed Development team, CTI https://www.ctifacts.com/

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