The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) has published its Report on Access to Orphan Drugs in Spain for the first four months of 2025, with data up to April 30.
This publication, which is published both quarterly and annually, provides a detailed analysis of orphan-designated products with trade names in the European Union and Spain, as well as their availability for patients with rare diseases.
In the first four months of 2025, the European Medicines Agency (EMA) has granted orphan designation to 12 new drugs. In addition, 5 treatments have received marketing authorization, 3 have obtained the National Code and 5 have received funding from the National Health System (NHS).
At the European level, a total of 216 drugs with orphan designation and trade name have already been identified, of which 149 have marketing authorization.
Meanwhile, in Spain 132 drugs have a national code and 90 are financed by the SNS, although 54% are financed with restrictions (limitations in the authorized indications or with some indication not financed).
As for the 41 orphan drugs with a National Code but without funding in Spain, 19 have an express resolution of no funding and 22 are under study or have not yet submitted a request for funding.
The report also highlights as a major concern the time lag between National Code allocation and funding. In the first four months of 2025, this time frame has lengthened to 33 months, compared to the 23 months recorded at the close of 2024. For AELMHU, this figure once again highlights the need to speed up access to these drugs for all patients with rare diseases.
The sources consulted in the preparation of this report include the EMAthe Community Register of Orphan Medicinal Products, the Spanish Agency for Medicinal Products and Medical Devices (AEMPS) and the Ministry of Health..
Consult here the Infographic
Infographics_quarterly access report 1_ 2025_AELMHU
