Development of anti-cancer medicinal products in paediatric patients - Scientific guideline

The concept paper identifies high level recommendations that drive the generation and assessment of meaningful proof-of-concept data to support the development of novel products in paediatric oncology. It also outlines the challenges associated with evaluating such data. 

Key evidence domains for a proposed weight of evidence assessment are listed and will be discussed in detail in the planned reflection paper. 

In addition, a high-level recommendation for a process for consultation of stakeholders, including academic experts/academic consortia is proposed. These stakeholders may contribute scientific input and expertise to inform the assessment of Paediatric Investigation Plans supporting anti-cancer drug development in children.

Keywords: mechanism of action, paediatric oncology, non-clinical, weight of evidence, proof of concept

Current version

Concept paper on the development of a reflection paper on proof-of-concept data to support the development of anti-cancer medicinal products in paediatric patients

Draft: consultation open Consultation dates: 13/03/2026 to 30/06/2026 Reference Number: EMA/32831/2026 Summary:

Comments should be provided using this EUSurvey form. For any technical issues, please contact the EUSurvey Support.