Senior Advisor Critical Path Institute North Wales, Pennsylvania
This presentation will focus on the key features that discriminate dosing rationales for pediatric rare diseases from other pediatric and adult indications. Initial dosing rationale for first-time-in pediatrics (FTIP) dosing in rare diseases along with guidance for final dose selection will be discussed. Recent examples will be presented along with methodologies and approaches that facilitate these selections and garner regulatory confidence. Finally, a road-map for a more streamlined approach consistent with a model-informed drug development (MIDD) paradigm will be proposed and discussed.
Understand the key differences between pediatric rare disease populations and mainstream pediatric and adult rare disease populations.
Understand the key factors used to propose dosing for pediatric rare disease populations at different stages of development.
Appreciate the factors that contribute to a final dose selection and recommendation for pediatric rare disease populations.
Understand the various approaches used to evaluate and confirm dose selection for pediatric rare disease populations.
Appreciate the value and purpose of a model-informed drug development approach to facilitate decision-making around dose selection for pediatric rare disease populations.