The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories has published its annual report, which analyses the most relevant data on clinical trials authorised for these conditions

consalud.es

The Spanish Association of Laboratories for Orphan and Ultra-Orphan Medicines (AELMHU) has published the ‘Report on Clinical Trials for Rare Diseases in Spain 2025’, which analyses the most relevant data on clinical trials authorised for these conditions in 2025, as well as their development over the last six years in Spain.

According to the report, published to mark International Clinical Trials Day, which is celebrated this Wednesday, 20 May, Catalonia, the Community of Madrid and Andalusia were the autonomous communities with the highest level of research activity in rare diseases, accounting for more than 50% of studies in 2025.

Specifically, Catalonia participated in 171 trials for rare diseases; followed by the Community of Madrid, with 151; Andalusia, with 100 trials; and the Valencian Community, with 79. However, when adjusting the data for the population of each region, other autonomous communities such as Navarre, Galicia and the Basque Country also stand out, exceeding the rate of clinical trials per million inhabitants in Catalonia and Andalusia.

“These figures confirm that research into rare diseases continues to advance and gain ground in Spain, which is particularly good news for people with rare diseases who are still waiting for new treatment options”

Nationwide, according to data from the Spanish Clinical Trials Register (REec) of the Spanish Agency for Medicines and Health Products (AEMPS), a total of 962 clinical trials were authorised in Spain in 2025. Of these, 216 focused on rare diseases, representing a 4% increase compared with the previous year. This means that studies on these conditions account for 22% of the total, a proportion similar to that of last year. Furthermore, there was also an 11% increase in the number of participants in studies for these conditions, reaching 4,088 last year.

Another key finding from the report is that clinical trials for rare diseases sponsored by pharmaceutical companies increased by 8% in 2025, reaching 212 studies, compared to the 196 recorded the previous year. Thus, the pharmaceutical industry accounted for 98% of all authorised research into these conditions.

“These figures confirm that research into rare diseases continues to advance and gain a firm foothold in Spain, which is particularly good news for people with rare diseases who are still waiting for new treatment options. Furthermore, they also demonstrate that the pharmaceutical industry remains firmly committed to innovation in this field. However, we believe that more streamlined administrative processes and greater incentives for R&D are still needed to speed up the launch of trials and improve Spain’s competitiveness in this field,” said Beatriz Perales, president of AELMHU.

Clinical trials for rare diseases in Spain in 2025 were mainly concentrated in phases II and III, accounting for 72% of the total. Compared to 2024, the 22% increase in phase III trials compared to the previous year (from 77 to 94 studies) is particularly noteworthy. In terms of therapeutic areas, the report notes a trend towards greater diversification. Oncology remains the predominant area, with 47 clinical trials for rare diseases in 2025, but its relative share has fallen to 22%, four percentage points lower than in 2024.

Next comes the immune system, which has established itself as the second-largest therapeutic area in terms of the number of trials, having grown by 38%: from 32 trials in 2024 to 44 in 2025. This is followed by the nervous system, with 33 authorised trials, ahead of congenital and hereditary disorders and neonatology, with 27. Also noteworthy is the increase in authorised trials for rare cardiovascular diseases, with 16 clinical trials, doubling the figure from the previous year.

‘We believe that there is still a need for more streamlined administrative procedures and greater incentives for R&D in order to speed up the launch of clinical trials and improve Spain’s competitiveness in this field.’

The report also covers the authorisation of clinical trials involving advanced therapy medicinal products. In 2025, the total number of authorised studies fell by 25% to 40, compared with 53 in 2024. This trend was even more pronounced in the field of rare diseases, where the number of trials fell from 28 to 10, representing a 64% decrease.

In line with this, the number of participants in this type of research also fell, with a total of 870 people included in advanced therapy trials, of whom 138 were patients with rare diseases. Despite the decline compared with the previous year, research into advanced therapies remains key to facilitating access to innovative treatments for patients with rare diseases.

Finally, the report analyses the development of clinical trials in Spain since 2020. Over the last six years, 5,678 clinical trials have been authorised in Spain, of which 1,284 (23% of the total) have focused on rare diseases. During this period, clinical trials targeting rare diseases have shown a positive trend, with an average of around 200 studies per year. This is mainly due to the pharmaceutical industry’s drive to support research into rare diseases. In fact, since 2020, 48 trials have been sponsored by non-commercial sponsors and 1,236 trials by pharmaceutical companies, accounting for 96% of the total.