EMA opens consultation on concept paper for proof‑of‑concept data in paediatric cancer medicine development
The European Medicines Agency (EMA) has opened a consultation on a concept paper proposing a reflection paper on proof-of-concept data supporting the development of anti-cancer medicinal products in paediatric patients. The paper identifies high-level recommendations for generating and assessing proof-of-concept data using a weight of evidence approach, considering factors such as mechanism of action, biology of paediatric malignancies, non-clinical disease models, pharmacokinetics and safety considerations. It also highlights challenges in paediatric oncology drug development, including biological differences between adult and paediatric cancers and limited clinical trial populations, and proposes stakeholder input, including academic experts, to inform the assessment of Paediatric Investigation Plans (PIPs). The consultation closes on 30 June 2026.