Advancing Paediatric Drug Development
Connecting regulatory requirements, scientific expertise and preclinical evidence.
Developing effective medicines for children with cancer requires a clear understanding of disease biology, robust scientific evidence and a well-founded development strategy. This applies both to therapies developed specifically for paediatric malignancies and to medicines whose potential in childhood cancers emerges from development in adults.
ITCC-P4 supports pharmaceutical companies and academic researchers through scientific advice, preclinical development planning and experimental evaluation. We bring together molecular data, paediatric oncology expertise and the capabilities of our exclusive CRO partners to help developers address the questions that matter for each programme.
United States: the RACE for Children Act
The RACE for Children Act expanded the existing Pediatric Research Equity Act (PREA) requirements for certain new oncology medicines.
For qualifying original applications submitted from 18 August 2020, paediatric investigations are required when a new medicine intended to treat adult cancer targets a molecular mechanism that FDA determines is substantially relevant to the growth or progression of a paediatric cancer. This can apply even when the adult cancer does not occur in children. Orphan designation no longer provides an automatic exemption for these investigations.
Product-specific waivers and deferrals remain possible. The relevance of the target and the evidence available for the individual medicine inform the assessment.
European Union: current requirements and proposed changes
Since 2007, the EU Paediatric Regulation has required paediatric development plans for certain medicines. A Paediatric Investigation Plan (PIP) sets out the studies and other measures needed to support development for children. Waivers and deferrals are possible, including a waiver when the disease occurs only in adults.
The agreed pharmaceutical reform would give greater weight to the relevance of a medicine’s mechanism of action for another paediatric disease within the same therapeutic area. This is particularly important in oncology, where adult and childhood cancers may share relevant biology despite being different diseases.
Political agreement on the reform was reached in December 2025. EMA’s published timetable anticipates application of the new framework in 2028, following the legislative and implementation steps. The new provisions should therefore be distinguished from requirements already applicable today.
EU Paediatric Regulation · Published reform text, Article 75 · EMA implementation updates
Engagement in scientific and regulatory dialogue
As an EMA stakeholder, ITCC-P4 contributes to scientific and regulatory dialogue on paediatric drug development. Drawing on the expertise of our scientific community, we bring perspectives from preclinical research and development planning to discussions on the evidence needed to advance medicines for children with cancer.
From regulatory questions to a scientific strategy
Each development programme starts with a different level of evidence. Some require an early assessment of the biological rationale; others need specific studies to resolve an uncertainty or inform the next development decision.
Key questions include:
- Is the target or pathway relevant to particular paediatric cancers?
- What does existing molecular, preclinical and clinical evidence tell us?
- Which evidence gaps need to be addressed?
- Which experimental approaches and models are appropriate?
- How should the findings inform prioritisation and subsequent development?
Target presence alone does not establish that a medicine will work. Molecular information, functional evidence and clinical context need to be considered together.
ITCC-P4 works closely with ITCC (Innovative Therapies for Children with Cancer) to connect preclinical evidence with clinical needs and expertise. This collaboration helps shape clinically relevant research questions, guide preclinical development planning and interpret experimental findings in the context of potential clinical studies.
How ITCC-P4 supports your programme
We provide support from early scientific discussions and preclinical development planning through to study design, execution and interpretation. The scope is tailored to the scientific question, the available evidence and the intended use of the results.
Scientific advice and development planning
Our paediatric preclinical oncology experts help assess the biological rationale, identify evidence gaps and shape an appropriate preclinical development plan. This may include advice on tumour indications, biomarkers, experimental approaches and the sequencing of studies.
Study design and experimental evaluation
We combine access to approximately 300 paediatric cancer models with molecular characterisation and specialist experimental capabilities. Model selection and study design are guided by the medicine’s mechanism of action and the questions the programme needs to answer.
Studies using the ITCC-P4 PDX model portfolio are conducted through our three exclusive CRO partners. Together, we support appropriate in vitro and in vivo evaluation of individual medicines and rational combinations.
Interpretation and next development steps
We interpret experimental findings alongside molecular information and the relevant paediatric disease context. This helps developers assess treatment activity, explore sensitivity and resistance, and make informed decisions about further investigation.
The resulting evidence and scientific interpretation can support development planning and discussions with regulatory authorities.
Connecting complementary expertise
Effective paediatric development benefits from a close connection between three areas:
- Molecular evidence: identifying target presence and potential relevance in childhood cancers.
- Preclinical evaluation: investigating the activity of a specific medicine in relevant experimental settings.
- Clinical expertise: considering therapeutic need, patient populations and the feasibility of subsequent development.
These perspectives inform one another. Clinical questions can shape preclinical studies from the outset, while experimental findings can refine the biological rationale and help identify which approaches warrant further investigation.
ITCC-P4 contributes to this connection through its scientific network, molecular resources and collaboration with experienced CRO partners.
Engagement with the regulatory communi
ITCC-P4 is an EMA stakeholder and has engaged with EMA and FDA representatives through an Innovation Task Force briefing meeting to discuss its preclinical platform and approach.
This dialogue reflects our commitment to connecting scientific expertise with the evolving requirements of paediatric drug development. We aim to contribute practical perspectives on preclinical evidence generation and the challenges of translating promising therapies into clinical development for children and adolescents with cancer.
Evidence tailored to the decision
Not every programme requires new experiments. Existing data may already answer some questions, while others require targeted additional investigation.
Preclinical findings can contribute to the rationale for clinical development or, where appropriate, to the scientific justification for a waiver or deferral. Their interpretation depends on the quality, scope and relevance of the evidence. Regulatory decisions remain with the responsible authorities.
Early discussion of the intended development question helps ensure that the work undertaken is informative and proportionate.
Discuss your programme with us
Whether you are exploring the paediatric relevance of a medicine, developing a preclinical plan or preparing a specific study, we welcome a conversation about your objectives and the evidence you need.