Spain Financed 58% of Orphan Drugs Approved in the European Union

During 2024, Spain has financed 17 new orphan drugs authorized by the European Union , a figure slightly lower than the previous year, when a record of 21 drugs of this type was reached. This is reflected in the Annual Report on Access to Orphan Drugs , prepared by the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories ( Aelmhu).

With the incorporation of these new medicines into the National Health System (NHS), Spain now has a total of 85 funded orphan designations . In other words, 58% of the orphan medicines authorised in the European Union are funded by the national system, which represents an increase of 5% compared to the previous year.

The analysis shows a positive trend in the number of orphan designations, marketing authorisations and national codes granted last year. The incorporation of 25 orphan designations (19 in 2023), 17 commercialisations (12) and 24 national codes (10) stands out.

The Report points out that one of the pending issues is to improve the model and the access times to treatments, which is 23 months.

The situation in Europe has also shown positive developments, with 25 new drugs registered with orphan designation and trade names, six more than in 2023. In addition, 17 drugs have been authorised for marketing by the European Medicines Agency (EMA), representing a five-point increase in the number of authorised products compared to the previous year.

In total, by the end of 2024, the European Union had 210 medicines with orphan designation and a trade name and 147 with marketing authorisation. Meanwhile, in Spain there are 131 orphan drugs with a national code, 89% of those with marketing authorisation in the EU.

Three new advanced therapies funded

The Aelmhu report also reflects the evolution of advanced therapies for rare diseases. As of 31 December 2024, there are 15 advanced therapies with orphan designation and marketing authorisation. Of these, 14 have a national code, although only half are funded by the SNS. Of the seven remaining therapies with a national code, but not funded, five have an unfavourable resolution, and two are under study or without request for funding.

The report also reflects the evolution of advanced therapies for rare pathologies.
The document highlights that in 2024 Spain has incorporated three therapies with orphan designation into the SNS, a fact that represents a milestone considering that the last advanced therapy with orphan designation funded in Spain was in 2021.

46 unfunded orphan drugs

On the other hand, in Spain there are currently 46 unfunded orphan drugs, which is one more than in 2023. Of these, half have received a negative funding resolution while the other half are either under study or their funding has not yet been requested.

Specifically, 35% of these drugs (16) have been waiting for public funding since the period 2015-2020; 22% (10) with a national code in the period 2021-2023 remain without funding and the remaining 43% (20) received a national code in 2024. Almost 40% of orphan drugs with a national code without funding have been waiting for more than three years to be incorporated into the SNS, although it is true that more than 40% (20) have been in this situation for less than a year.

Regarding the average waiting time between obtaining the national code and approval of funding by the SNS, this has remained at 23 months in 2023. At this point, Aelmhu has highlighted the need for a new pricing and financing model that takes into account the specificity of orphan drugs when evaluating and financing them in Spain.

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First published in iSanidad. A third-party contributor translated and adapted the article from the original. In case of discrepancy, the original will prevail.

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