Developing cancer medicines for children

The European Medicines Agency (EMA) plays a central role in supporting research and development of innovative cancer medicines for children. We work together with international partners to advance treatment for childhood cancers.

EMA’s Paediatric Medicines Office and Paediatric Committee (PDCO) support research and development of cancer medicines for children. 

They enable EMA to implement the EU Paediatric Regulation (Regulation (EC) No 1901/2006). 

The goal is to provide the safest and most effective cancer medicines for children. 

Cancer remains the most common cause of death by disease in children, according to the European Cancer Inequalities Registry (ECIR) at the European Commission.

For more information on EMA's role in supporting research and development for paediatric medicines, see:

• Paediatric medicines: Research and development

Cancer medicines and paediatric investigation plans

EMA is supporting the development of cancer medicines for children in line with agreed paediatric investigation plans (PIPs). They set out a product’s development plan. 

This ensures that medicines for children are of high quality, ethically researched, and adequate to be appropriately authorised.

Examples of authorised cancer medicines for children include:

• Qarziba (previously Dinutuximab beta EUSA and Dinutuximab beta Apeiron) - a monoclonal antibody used to treat neuroblastoma, a cancer of nerve cells, in patients over 1 year of age
• Retsevmo - a tyrosine kinase inhibitor used to treat thyroid cancer in patients from 12 years of age
• Koselugo - used to treat neurofibromatosis, benign (non-cancerous) tumours along the nerves, in children from 3 years of age 

International cooperation on paediatric cancer medicines

EMA supports research and development priorities throughout the lifecycle of paediatric cancer medicines.

International paediatric oncology forums

EMA regularly participates in and hosts international paediatric oncology forums focused on topics such as:

• New treatments for brain tumours
• Anti-GD2 therapies - a type of immunotherapy that targets the 'disialoganglioside GD2', a molecule found on the surface of certain cancer cells
• CDK inhibitors - a class of drugs that can halt the rapid and unchecked growth of cancer cells
• DNA damage pathway inhibitors - a class of drugs that can make cancer cells more vulnerable to treatments like chemotherapy and radiation therapy

These meetings bring together patient advocates, clinicians, academics, regulators and pharmaceutical industry representatives. 

They aim to: 

• provide priority research and development recommendations;
• better meet the needs of paediatric cancer patients;
• and increase the feasibility of paediatric medicine development. 

Meeting results feature in dedicated scientific papers.

Select the expandable panel below to access scientific papers on different paediatric strategy forums:

Paediatric strategy forums: scientific papers

• Paediatric Strategy Forum for medicinal product development of multi-targeted kinase inhibitors in bone sarcomas
• Paediatric Strategy Forum for medicinal product development of chimeric antigen receptor T-cells in children and adolescents with cancer
• Paediatric strategy forum for medicinal product development in diffuse midline gliomas in children and adolescents
• Impact of Accelerate Paediatric Strategy Forums: a review of the value of multi-stakeholder meetings in oncology drug development
• Can a Multistakeholder Prioritization Structure Support Regulatory Decision Making? A Review of Pediatric Oncology Strategy Forums Reflecting on Challenges and Opportunities of this Concept
• Accelerate Paediatric Strategy Forums: an advance for oncological drug development?
• European regulatory strategy for supporting childhood cancer therapy developments

For more information, see:

• Accelerate: Innovation for children and adolescents with cancer - past forums

Paediatric clusters

EMA holds regular meetings with other non-EU regulators. These meetings are called 'clusters'. They focus on special topics and therapeutic areas that require an intensified exchange of information and collaboration.

Through its monthly paediatric cluster, EMA works closely with the United States (US) Food & Drug Administration (FDA) and other international regulatory bodies. Together, they aim to foster the global development of cancer medicines for children. 

In addition, the so-called common commentary that EMA and FDA issue together is meant to speed up the development and authorisation of cancer medicines for children.

For more information on the EMA - FDA joint procedural information, see:

• Paediatric investigation plans: Joint EMA - FDA guidance

Select the expandable panel below to access FDA resources on developing cancer medicines for children:

FDA resources on developing cancer medicines for children

• Clinical Cancer Research - Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma Clinical Trials: An FDA Educational Symposium
• Journal of Clinical Oncology - Accelerating the Global Development of Pediatric Cancer Drugs: A Call to Coordinate the Submissions of Pediatric Investigation Plans and Pediatric Study Plans to the European Medicines Agency and US Food and Drug Administration

Clinical trials for paediatric cancer medicines

EMA is supporting paediatric clinical trials through the European Network of Paediatric Research at EMA (Enpr-EMA).

Enpr-EMA groups together research networks, investigators and centres with recognised expertise in performing clinical studies in children.

Its aim is to foster high-quality, ethical research on the safety and effectiveness of medicines for children.

How can academia, industry, regulators, and patient advocates work together to accelerate platform trials for children and adolescents with cancer?

A multistakeholder initiative has developed the Childhood Cancer Academic-Industry Collaborative Platform Trials (C3PT) Position Narrative and Toolkit which includes guidance and resources covering:

• assessing whether a platform trial is the appropriate study design
• timing of the sponsor and regulatory interactions
• academic and industry collaboration models
• meaningful patient advocate engagement
• intellectual property and contracting considerations
• operational planning and funding approaches

Page update history

An update log is available to show the date and summary of changes to this webpage. It does not include updates to linked documents or minor edits like typos or broken link fixes.

The tracking of updates begins in October 2026.

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6 October 2026

Section 'Clinical trials for paediatric cancer medicines' updated