By Begonya Nafria, Fundació Sant Joan de Déu
Children and young people represent 21% of the European population, yet they represent 100% of the future of our society.
Disease in minors can be considered as “antinatural”. A child is expected to be born and grow up healthy; nevertheless, there are a huge number of conditions that have their onset in the early ages and only affect minors. This is especially so in the case of rare diseases. From the 8,000 conditions that can be considered rare, around 80% of them affect minors.
On the other hand, there is a limitation of treatments for paediatric patients and also an emergent need of medicine designed for children. Half of the treatments prescribed for paediatric patients have never been tested in them and when it comes to neonates this figure increases to around 90% of treatments.
The Paediatric Regulation that came into force in the EU on 26 January 2007, aimed to improve the health of children in Europe by facilitating the development and availability of medicines for children aged 0 to 17 years. Despite the regulation, however, more patient-centered research is required with the aim to design better medicines for children.
Generally, minors have been considered vulnerable populations and historically not included in research projects. New approaches emphasise the need to perform research for paediatric patients and include them in the early stages of drug development to ensure clinical trials are suitable for them. With this aim, eYPAGnet (European Young Person’s Advisory Groups) was established in 2017 to provide a single point of contact in Europe to facilitate activities of PPI (patient and public involvement) in drug development to answer the requests of the different stakeholders involved in the performance of clinical trials. Fundació Sant Joan de Déu is one of the founder members of eYPAGnet, and has an internal policy to include patients in all the research initiatives in which we are participating (from the evaluation of the call for projects to the dissemination and communication of outcomes).
PARADIGM is a unique opportunity to standardise the involvement of vulnerable populations in drug development and also to design metrics to measure its impact. I envision a new landscape in the future paediatric clinical trials where patients will be a stakeholder along the process with voice and opinion to facilitate the development of projects according to their needs and features. We are living the momentum to change and improve the scenario of medicines for children thanks to the support of IMI for this project. With the commitment and contribution of the stakeholders, we will be able to design better medicines for children. Let’s do it together!
Begonya Nafria is Patient Engagement in Research Coordinator at Sant Joan de Déu Children’s Hospital (Spain). She has long experience in the field of the involvement of patients and families in research initiatives. She has also a personal story as a caregiver and patient advocate because is the sister of a young adult with cerebral palsy. Her areas of expertise are focused on paediatric patients involvement in research and specifically in the field of clinical trials. Other relevant background of her profile is: Fellow of EUPATI (first cohort), Coordinator of eYPAGnet (European Young Patients Advisory Group Network – www.eypagnet.eu), Coordinator of Kids Barcelona (YPAG of Sant Joan de Déu Children’s Hospital- www.kidsbarcelona.org), member of Children’s Medicines Working Party of EFPGCP, volunteer member of the Editorial Board of Center for Information & Study on Clinical Research Participation (CISCRP), reviewer of Research Involvement and Engagement Journal and Orphanet Journal of Rare
Diseases, Chair of the Educational Committee of ICAN (International Children’s Advocacy Network) and member of the Patients and Families working group of EnprEMA (European Network of Paediatric Research of EMA). Coordinator of the cross-cutting theme of patients involvement in Conect4Children project (pan-European paediatric clinical trials network) and participant of PARADIGM project, both funded by IMI. Coordinator of the Share4Rare project, funded by the European Commission (H2020) that has the aim to develop a digital platform addressed to improve the research in the field of rare diseases through the collective intelligence of patients and families (www.share4rare.org).
Twitter details @BegonyaNafria