The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) participated this Thursday in the meeting of the Federation of Associations of Hereditary Retinal Dystrophies of Spain (FARPE), held at the State Reference Center for the Care of People with Rare Diseases and Their Families (CREER) in Burgos.
Our executive director, Marian Corral, gave a lecture “Orphan Drugs: Research, Access, and Funding”, in which she provided a comprehensive overview of the journey these treatments take, from their earliest stages of development to their eventual availability to people with rare diseases.
During his remarks, he first addressed the complexity of developing orphan drugs, which is characterized by limited knowledge of many diseases or their low prevalence. He also highlighted the significant R&D efforts undertaken by pharmaceutical companies, which involve high levels of investment and risk, as well as a lengthy process that spans from basic research through clinical trials to marketing authorization.
The conference went on to detail the European regulatory framework, which includes mechanisms that encourage the development of these treatments, such as orphan drug designation and market exclusivity. Finally, the speaker explained the process for access and funding in Spain, which involves multiple levels of decision-making (national and regional). On this point, they emphasized the need to move toward models that ensure innovation reaches people with rare diseases more quickly and equitably.
The event was opened by Aitor Aparicio, director of CREER, and David Sánchez, president of FARPE. Throughout the day, there were various presentations, including the workshop “Grants with Impact,” led by Sandra Caballero, a social worker at the Spanish Federation for Rare Diseases (FEDER), and a presentation by Dr. Carmen Ayuso, head of the Department of Medical Genetics at the Jiménez Díaz University Hospital, which focused on hereditary retinal dystrophies and addressed advances in diagnosis, prevention, and treatment.
