The National Association of Health Informers (ANIS) and the Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) have organized this Wednesday the II Training Day on rare diseases, aimed at journalists specialized in health.
The objective of the training, given by professionals of great national prestige in the health field, was to create a meeting place where health reporters could acquire knowledge, in order to achieve better precision and specialization on rare diseases and orphan drugs.
The moderator, Susana Fernández Olleros, vice-president of ANIS, highlighted the importance of training for journalists, who are faced with issues such as rare diseases, legislation, orphan drugs and clinical trials. " Continuous training is vital for health journalists to provide quality and rigorous information on such sensitive and complex issues," she said.
Access to Orphan Drugs in Spain
During the inauguration, María José Sánchez, vice-president of AELMHU, emphasized the need to continue investing "in research and development of orphan drugs, as well as to continue improving the processes of evaluation, access and financing so that patients can benefit from innovation".
At the event, which took place at the Madrid Press Association in a hybrid format, Sánchez presented to journalists the data from the Report on Access to Orphan Drugs for the first four months of 2025The report analyzes the access situation of products with trade names that have an orphan designation in force as of April 30, 2025.
The publication shows that further work needs to be done on the waiting times that occur from the time a drug obtains the National Code, i.e. when a product applies to arrive in Spain, until it is approved for funding.
"At this point, the average has worsened compared to the previous report. If we take into account the drugs approved in this first four-month period of 2025, the average is 33 months. That is, 10 months longer than the average for 2024. Therefore, at AELMHU we believe that we must take advantage of this opportunity to develop and update pharmaceutical policy in order to introduce improvements that favor and encourage the arrival of innovation in Spain and allow patients to have equitable and flexible access to the treatments available in the European Union," said the Vice-President of AELMHU.
During the debate, Isabel Motero, director of the Spanish Federation of Rare Diseases (FEDER), has valued "very positively" initiatives such as this conference, which "allow to build bridges between information professionals and the group of people with rare diseases and in search of diagnosis".
"Collaboration with the media is essential to convey a rigorous and committed vision of the challenges we face in access to diagnosis, treatment and comprehensive care. Today we have been able to bring the voice of patients closer and continue working to promote a more informed, sensitive and committed society. A society that moves towards a more inclusive and equitable future for all", explained the director of FEDER.
Research, key for patients
Eva Bermejo, director of the Institute for Rare Diseases Research (IIER) of the Carlos III Institute of Health (ISCIII), stated during her speech that research on rare diseases must respond to the real needs of patients.
"To be efficient, the involvement of all actors in the rare disease ecosystem is essential, as well as collaboration and coordination in order to optimize the use of resources, which are always limited. A broad and innovative vision is needed, in the short, medium and long term, always seeking equity and inclusion and considering that rare diseases are a global health priority. To this end, research is a cross-cutting nexus that must be used in favor of people with rare diseases and their families," he added.
Jorge Mestre, health economist and university professor, recalled that, at the European level, rare diseases and their corresponding treatments represent a priority. "In Spain, although certain measures have been adopted that have favored access to orphan drugs, relevant challenges remain. It is essential to establish new mechanisms to ensure a more timely and efficient availability of orphan drugs and treatments for rare diseases," he said.
The health economist assured that these mechanisms should "be in line with the procedures and conditions foreseen for the accelerated, conditional and provisional financing authorizations contemplated in the Draft Medicines Law"." Likewise, the economic evaluation models must incorporate a social perspective, transcending the traditional analysis based exclusively on cost-effectiveness," he added.
For his part, Lluís Alcover, a lawyer specializing in pharmaceutical law at Faus & Moliner, analyzed the current regulatory framework for orphan drugs and the important regulatory changes that are coming at European and national level. Thus, he stressed the need to closely follow these developments, given the profound impact they will have on access to these treatments.
Among his key messages, he highlighted the importance of strengthening access to EMA 'early advice' and joint scientific consultations under the Health Technology Assessment (HTA) Regulation. For Alcover, this type of early support is "especially critical" for orphan drugs, given their clinical and regulatory particularities. In this sense, he has supported a more solid and predictable European incentive framework, and has insisted that, at the national level, these drugs require specific treatmenton issues such as the reference price system, the sixth additional provision and the financing procedure.
