The Minister of Health, Mónica García, has emphasized that the “biggest political challenge” in the field of rare diseases involves turning existing commitments and strategies into earlier diagnoses, effective access to the most advanced treatments, and comprehensive care throughout the lives of the affected people.
She made these statements during her address at the European Health Summit's Rare Diseases Edition, an event held this Tuesday at the Office of the European Parliament in Madrid that brought together political leaders, spokespersons for patient associations, and health specialists from different European countries. The goal was to analyze territorial inequalities in care for rare diseases and to discuss the direction of European policies in this area.
The forum, driven by European Business Summits (EBS), saw the participation of Mónica García and Italy's Health Minister, Orazio Schillaci. The latter reiterated his firm commitment to rare diseases and to patients, and defended the need to roll out a coordinated European action plan on rare diseases.
Both ministers agreed that research in rare diseases can only move forward with strong international cooperation, given that each condition affects a very small number of people. For this reason, they stressed that it is essential for countries to share knowledge, resources, and experience.
At the national level, the Ministry of Health will officially present next Wednesday the new Rare Diseases Strategy of the National Health System, which will set out the lines of action for the coming years.
Equity in access to innovation
During the day, it became clear how much distance there still is between the development of new medical solutions and the actual arrival of these innovations to patients. This is what representatives of patient organizations said, who stressed that equity in access must be integrated into the very definition of innovation.
The president of the Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultra Huérfanos (AELMHU), Beatriz Perales, has said that Spain authorized 216 clinical trials on rare diseases in 2025, approximately 22 percent of the national total. However, she has warned about the existing difficulties for these innovations to be effectively incorporated into the public healthcare system.
Patient associations have also insisted on the importance of people with rare diseases actively participating in all stages of the innovation process: from defining research priorities to the design of clinical trials and the assessment of the results obtained.
Next, the debate has focused on the complex balance between promoting innovation and, at the same time, preserving the financial sustainability of health systems in the long term.
European and global action plans
The speakers analyzed how a future European Union Action Plan and the upcoming Global Action Plan could represent a decisive opportunity to articulate a more coherent framework that connects diagnosis, research, innovation, access to therapies, healthcare, and full social inclusion of people with rare diseases.
Likewise, they highlighted the relevance of the European Reference Networks (ERN, for its English-language acronym) and called for their funding to be strengthened and for them to be structurally integrated into national healthcare systems.
The dialogue on healthcare policies will continue on December 8 and 9 in Brussels (Belgium), the venue for the seventh edition of the European Health Summit, which will be held under the slogan "Health is what matters: better options for Europeans".