Sergio Bullón, treasurer and member of the Board of Directors of the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories(AELMHU), participated this Thursday in the 'III Edition of Rare Diseases. Metabolic bone pathology', organized by the Hospital Universitario 12 de Octubre.
This meeting, which has already established itself as a benchmark event in the sector, focused on rare bone metabolism diseases, learning about them through clinical cases and the challenges that still exist to achieve a complete approach.
At the inauguration, Sergio Bullón was accompanied by the hospital's medical director, Dr. Julio Pascual; Dr. Montserrat Morales, head of Internal Medicine; and Dr. Guillermo Martínez Díaz-Guerra, head of the Endocrinology and Nutrition Section.
In his speech, the representative of AELMHU detailed several of the challenges faced by the sector, such as the problems of access to orphan drugs, a subject he analyzed through the latest data in our report for the second four-month period of the year.
"We need a collective effort. Administrations, clinicians, researchers, patient associations and the pharmaceutical industry must continue to work together to break down the barriers that delay the arrival of innovation in our country," he said.
To this end, he asked that the particularities of rare diseases, such as their low prevalence or the lack of therapeutic alternatives, be taken into account in order to evaluate treatments with criteria adapted to this reality, i.e., by the social value they generate.
In addition, Sergio Bullón highlighted the importance of research to advance knowledge, diagnosis and treatment of all rare diseases, with the aim of improving the quality of life of patients. In this regard, he congratulated the Hospital 12 de Octubre, the second hospital that has carried out the most clinical trials in rare diseases in Spain in the last five years, according to our latest report on rare diseases.
During the day, the hospital demonstrated its extensive knowledge in dealing with rare diseases, with presentations on Gaucher disease, hypophosphatasia and bone dysplasias, given by experts such as Dr. Leticia Jiménez and Dr. Pilar Quijada. The main characteristics of pathologies such as osteoporosis and rickets were also discussed by doctors Lucía Garzón and Ramón Costa.
From AELMHU we would like to thank the main promoter of the meeting, Dr. Montserrat Morales, and the medical director of the hospital, Dr. Julio Pascual, for having us to participate in this interesting day, as well as the sponsors, Alexion, Biomarin, Kyowa Kirin, Sanofi and Takeda, all of them associated companies of AELMHU, for having made this meeting possible, and also to all the speakers and attendees who have accompanied us.
