The Spanish Association of Orphan and Ultra Orphan Drugs was able to visit the Hospital Sant Joan de Déu and Casa de Sofia to learn first-hand about the Únicas SJD project, an essential component of the Únicas network.
Dr. Joan Comella, Director of the Institut de Recerca Sant Joan de Déu (IRSJD) and of Innovation, Research and Knowledge Management of the Hospital Sant Joan de Déu, Joana Claverol, Director of Clinical Research of the SJD Research Foundation, together with the Hospital Manager, Manel del Castillo, explained in detail all the ins and outs of the project, jointly promoted by the Hospital Sant Joan de Déu and the Spanish Federation of Rare Diseases (FEDER). Its main objective is to ensure that children with rare pathologies have access to the best treatment, regardless of their place of residence, improving the treatment and quality of life of patients with rare diseases in Spain.
Also at the Hospital Sant Joan de Déu, and led by Alessandra Magnani, head of the Advanced Therapies Platform at the Hospital Sant Joan de Déu and IRSJD researcher, the Association was able to visit the Advanced Therapies area of the Hospital, which includes four clean rooms to develop and produce advanced therapies for the pediatric population.
On the other hand, Jordi Martinez, assistant to the Digital Strategy and Data Management, was in charge of showing how his data area works, a pioneer in the creation of a federated data ecosystem in which specific and very precise information on the hospital is available, with the aim of moving towards personalized prediction and prevention models, while at the same time making research into rare diseases available to patients and families. This area applies a model of comprehensive care for patients with rare diseases that includes multidisciplinary teams of professionals, the generalization of the figure of the case manager, the incorporation of psychosocial support teams, transition units to adult centers and the monitoring of the census of rare diseases.
The final touch to the visit was provided by La casa de Sofia, which received the AELMHU award for the Best Care Project on Rare Diseases in 2022. This center, led by Sant Joan de Déu and promoted by CatSalut, seeks to avoid, when possible, admission to the children's acute hospital, delay long-term hospitalization and care for the patient to give them the clinical stability or functional improvement necessary to return home, in addition to offering the 'end of life' service so that patients can live this process in a center instead of at home.
The Association would like to express its sincere thanks to the entire team of the Hospital Sant Joan de Déu and Casa de Sofia for their warm welcome and excellent organization. Their dedication and commitment are truly inspiring.
