EMA proposes new paper on proof-of-concept studies for pediatric oncology drugs
The European Medicines Agency (EMA) has released a concept paper proposing a future reflection paper on the development of non-clinical proof-of-concept (POC) studies to support novel oncology treatments for the pediatric population.EMA said the aim of the reflection paper is to give a more structured regulatory framework for developing these studies. It is also meant to “provide recommendations that drive the generation and assessment of meaningful proof-of-concept data to support the development of medicinal products in paediatric oncology and to outline the challenges associated with evaluating such data.”
The paper states that a weight-of-evidence (WoE) approach should be utilized to determine whether additional non-clinical investigations are necessary to support pediatric clinical development.
EMA said the concept paper was influenced by changing global regulatory requirements, including amendments to the Pediatric Research Equity Act (PREA) in the US through the FDA Reauthorization Act of 2017 (FDARA). These amendments stipulate that oncological products developed for adult indications must also be assessed for pediatric development if their mechanism of action (MoA) is relevant to pediatric cancers.
It was also driven by the growing number of pediatric investigation plans (PIPs) in oncology, which are based on a product’s mechanism of action (MoH) rather than its intended adult indication, which has prompted the EMA’s Paediatric Committee (PDCO) to take notice. As a result, the Non-clinical Working Party is increasingly involved in assessing these PIPs, where non-clinical proof-of-concept data is presented to support the initiation of clinical development for pediatric oncology.
In the guideline, EMA said that most pediatric cancers differ significantly from adult cancers in their biological and clinical characteristics. Consequently, to support the development of pediatric therapies based on drugs initially designed for adult cancer indications, it may be necessary to gather population-specific proof-of-concept data. For instance, if a product has been developed for adult acute myeloid leukemia (AML), it is important to consider the need for additional proof-of-concept data specifically for pediatric AML due to the biological differences between adult and pediatric forms of the disease.
The paper lists key evidence domains for a proposed WoE approach:
- Mechanism of action of the investigational medicinal product and its relevance to the target or pathway to disease biology;
- Biology of the targeted malignancy such as tumorigenic pathways and knowledge on biomarkers;
- Evidence from non-clinical disease models including their relevance in reflecting the intended clinical context and data on combination treatments;
- Use of other appropriate models for demonstrating primary pharmacology, including new approach methodologies (NAMs); and
- Proof-of-concept data from same-in-class products.
The deadline for submitting comments on the concept paper is 30 June 2026. Comment should be submitted here. The agency plans to hold a public workshop in September 2026.
Concept paper
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