Module232025

12/05/2025

Barriers to Timely Completion of Paediatric Studies

Patients and families may be less willing to take on additional burden of trial participation, when the drug is accessible to them off-label Clinicians may be less inclined to participate or refer patients to participate in a trial for a therapy that is widely regarded as effective based on accumulating anecdotal experience. Also has been raised as an ethical issue, pertaining to the inclusion of vulnerable patients Several medications approved for adults have become standard of care in paediatric clinical practice, leading to difficulties with study enrollment

The Organisation for Professionals in Regulatory Affairs

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M Dehlinger-Kremer, 14 May 2025

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Possible Solution - Including Adolescents Early in Adult Trials

The aim is to close the gap between approvals for adults and children , thereby making innovative, safe, and effective new medicines available to children more quickly. Including adolescents in adult trials enables earlier data generation for study design and facilitates quicker access to innovative treatments for younger paediatric groups (infants, toddlers, children, neonates). This approach may also lead to safer and more effective dosing for these younger age groups.

Objective

Benefits

The Organisation for Professionals in Regulatory Affairs

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M Dehlinger-Kremer, 14 May 2025

26