Module232025

5/7/2025

• Positive results do not evenly spread among all therapeutic areas  concentrated in therapeutic areas were adult needs overlap with paediatric ones. • CTs in special subpopulations scarce, e.g. neonates • Development for off patent commonly used drugs is poor  PK or dose establishing CTs are not considered feasible • High number of CTs in certain areas with limited patient numbers • Lack of CTs in areas and products that might have been useful for children due to waivers , e.g. oncology • Delayed regulatory submissions and long CTs deferrals:  Limited opportunity to influence plans,  impact on timely availability of medicines, off label use vs CT participation • Increased medicines does not always translate to increased availability  CT design suitable also for reimbursement/HTA assessment

Paediatric Drug Development – so far …

7

Guess where paediatrics usually fits ?

8

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