Maria Cavaller Bellaubi
Patient Engagement & Therapeutic Development Director, EURORDIS – Rare Diseases Europe
Across Europe, too many children with rare diseases are still waiting for effective treatments. The conect4children project has laid the foundations for a faster, more inclusive research system.
Across Europe, children, particularly those living with rare diseases, still face major barriers in accessing the medicines they need.
Urgency of rare disease research
A disease is considered rare when it affects fewer than one in 2,000 people, but with over 6,000 rare diseases identified, they collectively affect at least one in 20 people. Around 75% of rare diseases affect children, and 70% have their onset during childhood — yet most still lack approved, effective treatments.1
Clinical trials for children are often delayed due to fragmented infrastructure, limited expertise and regulatory hurdles. Too often, trials are designed without reflecting the realities of children’s lives or involving families early enough. The result is slower research, lower enrolment and missed opportunities for innovation.
Clinical trials for children are often delayed
due to fragmented infrastructure, limited
expertise and regulatory hurdles.
What c4c changed
The conect4children (c4c) project — a public–private partnership funded under the Innovative Health Initiative — set out to change that. Over seven years, this large-scale collaboration brought together over 200 sites in 21 countries, creating a coordinated, patient-centred model for paediatric research. EURORDIS–Rare Diseases Europe was a key partner in ensuring the voice of children and families helped shape trial design from the outset.
C4c established national hubs, expert advisory groups and trial support services across Europe. It launched a centralised feasibility and site identification service, as well as standardised data and training protocols tailored to paediatric needs. It also embedded young people and caregivers as co-designers — not just participants — in the research process.
Among its achievements are the creation of the foundation c4c Stichting, a Supervisory Board and a fully operational IT platform. Strategic partnerships with five major companies, cooperation agreements with 15 countries and the processing of 24 trial service requests show its scale and readiness.
A legacy to build on
As the project concludes, its challenge now is sustainability. With the right support, c4c’s legacy can serve as a lasting blueprint for faster, smarter and more inclusive medicines development for Europe’s children.
[1] European Commission. Rare Diseases and European Reference Networks. Rare diseases.
