Styrkelse af unge krigere: EJP RD's uddannelse af pædiatriske ekspertpatienter til forskning i og fortalervirksomhed for sjældne sygdomme

The European Joint Programme on Rare Diseases (EJP RD) is a programme aiming to create an effective rare diseases research ecosystem for progress, innovation and for the benefit of everyone with a rare disease. The programme supports rare diseases stakeholders by funding research, bringing together data resources & tools, providing dedicated training courses, and translating high quality research into effective treatments.

The Paediatric Expert Patients Training under the EJP RD provides young rare disease patients with skills enabling them to get involved in clinical research. The activities include short workshops under the coordination of the TEDDY European Network of Excellence for Paediatric Research in collaboration with EURORDIS and the Sant Joan de Déu Research Foundation for 15 paediatric patients focusing on the following areas:

• Genetic /rare disease characteristics and evolution;
• Role for paediatric patients and relevance of clinical and translational research (including patients’ engagement in clinical trials, the consent issue, the patient-reported outcomes in the context of paediatric rare diseases, data protection and children rights);
• Orphan Medicinal Products availability and access for paediatric patients on the EU market, pharmacovigilance and off-label use in paediatric setting.