“The empowered patient is necessary for the challenge of medicine and for drug development – this has to be the medicine of today, not the future.”
It´s essential that patients and their families are involved in research on rare diseases. They have an immense expertise, and their needs must be taken into account.
I’m involved as a research technician in different projects of Hospital Sant Joan de Déu (Barcelona) related to rare diseases. Our aim is the clinical research with the objective to reach the description of the natural history of the disease, study the correlation of genotype-phenotype and also write clinical guidelines with consensus of diagnosis and treatment get with the participation of all the researchers community. But the most important challenge is the promotion of basic research to develop clinical trials.
I’m working very closely with the EUPATI project within the Clinical Trial Unit of the Hospital. In some of the disease areas that we work we have ongoing clinical trials or we are going to start them in a near future. I’m the contact for patients and families that are participating in the clinical research and I have to inform them about available innovative treatments.
Furthermore, I’m the team leader of the project “Kids Barcelona”, a Young Patient Advisory Group that acts a scientific council for the different activities of our hospital: research, innovation and clinical trials. We are going to participate in a working group in EmPrEMA (European Network for Paediatric Research of EMA).
The training course opened to me the door of the close collaboration with the patients of Hospital Sant Joan de Déu that are involved in the team KIDS BARCELONA. It’s our Young Patients’ Advisory Group (YPAG) that works with us as a scientific council. They help to our hospital to improve the different project that we develop, not only in the biomedical field, also related to innovation, research and especially in clinical trials. We are designing guidelines about how we can improve the information to the pediatric patient, related to an easy reading and easy understanding of the informed consent document, etc. For me the course is an amazing opportunity to collaborate with the quality of our services, always focused on the pediatric patient, and with the aim to improve the lives of the kids. We don’t work for the patients, we work with the patients.
Begonya Nafría, Hospital Sant Joan de Déu (Barcelona, Spain), EUPATI Trainee