María Cavaller Bellaubi
Patient Engagement & Therapeutic Development Senior Manager, Eurordis
Gulcin Gumus
Research and Policy Project Manager, Eurordis
Patient engagement is key. Young patients and their families involved in paediatric drug development contribute to faster and more efficient medicine development.
Patient engagement in the process of drug development can lead to active collaboration between developers, researchers, clinicians and patients who come together and share their knowledge and expertise throughout a medicine’s lifecycle. This stretches from driving research to co-creating regulatory protocols for health technology assessments and cost reimbursements.
Consider children in medicine development
Unfortunately, only a few patients are engaged in medicine development due to a lack of collaboration between patients and developers and geographical barriers, among other reasons. The reality is more dramatic when it comes to paediatric medicines, even though around 70% of rare diseases have a childhood onset.
Today, many medicines prescribed to children are being used ‘off-label,’ which means they may have not been tested specifically on children. This is mostly because the administration of drugs in the paediatric population is commonly and erroneously assumed. Most medicines exhibit different pharmacokinetics in children compared to adults, so we must ensure that the medicines developed are right for them.
Clinical trials for young people
It wasn’t until the European Union’s Paediatric Regulation came into force in 2007 that clinical trials with children started to become more widespread. But there’s still work to be done. The research infrastructure supporting the delivery of large clinical trials hasn’t been developed equally across Europe. Clinical trials are often delayed, and the rollout of medicines approved for children is painstakingly slow.
Today, many medicines prescribed to children are being used ‘off-label,’ which means they may have not been tested specifically on children.
Across Europe, 35 academic and 10 industry partners — including EURORDIS-Rare Diseases Europe — have joined forces and created the pan-European network ‘Conect4Children’ (C4C) to accelerate the development of innovative medicines for the entire paediatric population. EURORDIS aims to amplify the voices of children and young people living with a rare disease and bring their needs to the attention of researchers, clinicians and developers.
As part of this project, we have discussed the establishment of a framework for clinical trials. We have also launched educational videos for patients and parents about paediatric clinical research, and we have organised events empowering patient organisations.
Address young patients’ needs
Through C4C and other initiatives, we aim to create a common framework that enables structured, effective, meaningful, ethical and sustainable patient engagement — specifically for young patients, similar to that which already exists for adults.
Only by engaging the young population in medicines development can we safely provide them with treatments based on actual evidence, which address their specific needs.
