Module232025

12/05/2025

Early Integration of Paediatric Considerations

The Organisation for Professionals in Regulatory Affairs

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M Dehlinger-Kremer, 14 May 2025

27

Key Considerations for Including Adolescents into Adult Clinical Trials

The disease exists on a continuum between adults and adolescents, with no significant differences identified in pharmacodynamic responses, disease prognosis, or manifestation s

The efficacy and safety data collected from adult patients suggest that the potential benefits for adolescents outweigh the risks associated with their inclusion in the phase 3 clinical trials

The pharmacokinetics of the drug has been characterized in adult patients and is expected to be similar in adolescents and adults (which can be further assessed using modelling and simulation approaches)

Guidelines : In some cases, guidance provided is detailed, allowing for the inclusion of adolescents in early phases of adult trials. o FDA guideline for oncology "Considerations for the Inclusion of Ado. Patients in Adult Oncology CTs." (Adopted by TGA in June 2024).  This guideline suggests that adolescent patients should be eligible for enrolment in adult oncology trials at all stages of drug development, provided that the histology and biological behavior of the cancer being studied are similar in both adults and adolescents, or that the molecular target of the drug is relevant to cancers in both groups.

o ICH E11A guideline on paediatric extrapolation

Consideration o

Initiate discussions with regulators as early as possible in drug development , due to the impact on the paediatric study plans required by the EMA and FDA

The Organisation for Professionals in Regulatory Affairs

28

M Dehlinger-Kremer, 14 May 2025

28