The president of the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU), Beatriz Perales, participated this Thursday in the third edition of Únicas Talks, a conference organized by Sant Joan de Déu Barcelona Hospital and the “la Caixa” Foundation that brings together professionals, researchers, healthcare administrators, patient representatives, and other stakeholders in the field of rare diseases to share knowledge, experiences, and perspectives on the future.
During her presentation, Beatriz Perales analyzed the profound transformation that the approach to rare diseases has undergone in recent years, driven by scientific and technological advances that are changing both research and patient care.
Among the key factors contributing to this change, he highlighted the development of increasingly personalized therapies, advances in genetic diagnosis, the growing use of health data, and the potential of artificial intelligence to accelerate the research and development of new therapeutic solutions.
However, the president of AELMHU emphasized that the main challenge remains effectively translating these advances into clinical practice and ensuring that people with rare diseases can benefit from them on an equitable basis.
In this regard, he noted that “innovation does not end when a treatment is discovered.”“Its true value is realized when it reaches people with rare diseases quickly and equitably”.
He also emphasized the need to continue moving toward models of assessment, funding, and access tailored to the specific characteristics of these diseases, with the goal of reducing inequalities and ensuring that therapeutic innovations reach all patients, regardless of where they live.
The event highlighted the importance of collaboration among all stakeholders in the rare disease ecosystem to address current and future challenges. In this context, AELMHU has reaffirmed its commitment to promoting initiatives that foster dialogue, the exchange of knowledge, and the joint search for solutions to improve the lives of people with rare diseases.
On behalf of AELMHU , we would like to thank Sant Joan de Déu Barcelona Hospital, the “la Caixa” Foundation, Dr. Encarna Guillén, and all the participating professionals and organizations for organizing events such as Únicas Talks, which help foster collaboration and move toward a more accessible, efficient, and patient-centered model of innovation.
