In the framework of the International Clinical Trials Day, which is celebrated every May 20, the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has presented its Report on Clinical Trials for Rare Diseases in Spain 2024which reflects a positive and sustained evolution in the research of these minority pathologies in the country.
Last year, 929 clinical trials were approved in Spain, 207 of which focused on rare diseases. This number reflects a 10% growth over the previous year and is a positive sign for the many patients who still have no therapeutic options available.
In addition, the involvement of individuals in these studies has grown significantly: a total of 3,659 participants, an increase of 15 % compared to 2023. Among them, 602 were minors, i.e. 16 % of the total.
This figure highlights the importance of promoting research in the pediatric population, especially in pathologies that manifest themselves early in life.
Industry as a driver of research
One of the report's highlights is the key role of the pharmaceutical industry, which has promoted 95% of clinical trials for rare diseases by 2024. This corporate commitment is essential for advancing treatments for rare diseases which, due to their low prevalence, usually receive little public investment.
"These results confirm that research in rare diseases is not only maintained, but is growing steadily. The pharmaceutical industry continues to strengthen its commitment to the search for new treatments and from AELMHU we ratify our commitment to continue working and joining forces around this common goal," said Beatriz Perales, president of AELMHU.
In terms of territorial distribution, Catalonia, Madrid, Andalusia and the Valencian Community account for more than 60% of the clinical trials authorized in this area, which reinforces their leading role in scientific research.
Early stages and new therapeutic areas
The report notes that the majority of clinical trials on rare diseases are in early stages (I, II and III), reflecting an innovation activity in full swing.
Although oncology continues to be the main area of research, its relative weight has fallen to 26%, four points less than in 2023. In contrast, there has been an increase in activity in fields such as neurological and genetic diseases, which could broaden the range of treatments available for other pathologies that have been less studied until now.
The rise of advanced therapies
One of the novelties of the 2024 report is the specific analysis of clinical trials with advanced therapies -such as gene therapies, cell therapies or tissue-engineered products-. In total, 52 trials with advanced therapies were authorized, of which 27 were directed at rare diseases.
This figure indicates a consolidation of these innovations as a key avenue of research to treat diseases that until recently lacked therapeutic alternatives. In the 2020-2024 period, 199 clinical trials with advanced therapies have been authorized, of which more than half (101) focused on rare diseases.
An upward trend
The balance of the last five years confirms a sustained growth of interest in research on rare diseases. Since 2020, Spain has authorized 4,715 clinical trials, of which 23% have been for rare diseases.
These data consolidate Spain as one of the most active European countries in rare disease research, and reinforce the importance of maintaining a fluid collaboration between public administrations, the pharmaceutical industry and patient organizations.
"From AELMHU we firmly believe that working together will result in more therapeutic opportunities and faster access to treatments for patients," concluded Beatriz Perales.
In a context in which rare diseases affect more than three million people in Spain, the promotion of clinical trials and the commitment to innovative therapies are key steps towards a future with more hope and solutions for those who today live with untreated pathologies.
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