ITCC-P4 contributes to EMA consultation on preclinical evidence in paediatric oncology

The consultation addresses a central question in paediatric oncology: what scientific evidence is needed to support investigating a cancer medicine in children?

A medicine’s mechanism of action may suggest potential relevance to a childhood cancer, even when it was originally developed for a different cancer in adults. Assessing that potential requires consideration of the disease biology, the properties of the medicine and findings from appropriate experimental studies.

EMA’s concept paper sets out plans for a reflection paper on the generation and assessment of proof-of-concept data to support these development decisions. It forms part of a broader scientific discussion on how to build a convincing rationale for paediatric investigation.

A contribution from the ITCC-P4 scientific community

As an EMA stakeholder, ITCC-P4 contributed a response developed with input from its R&D Working Group, preclinical experts and Advisory Board. This collaborative effort brought together expertise in paediatric cancer biology, preclinical research and translational development.

Our participation reflects ITCC-P4’s commitment to supporting scientifically grounded paediatric drug development. We help developers review existing evidence, identify unanswered questions and shape preclinical development plans, alongside supporting experimental studies and interpreting their findings.

Through this combination of scientific advice and experimental evaluation, ITCC-P4 helps developers build the evidence needed to inform the next development decision.

Read EMA’s concept paper and consultation information

Explore the regulatory context and how ITCC-P4 supports preclinical development!