The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has published its Report on Access to Orphan Drugs in Spain for the second four months of 2024. This publication, which analyzes the situation up to August 31, provides a detailed analysis of orphan-designated products with trade names in the European Union and Spain.
As of the close of the report, 208 products with orphan designation and trade name have been registered . Of these, 149 products already have the necessary marketing authorization in the European Union.
Between May and August of this year, the EMA (European Medicines Agency) has granted orphan designation to 7 new drugs, 4 treatments have obtained marketing authorization, 7 have received National Code and 3 have been funded.
In total, orphan products with a national code number 132, of which 85 are financed, equivalent to 64% of the total.
Overall, 89% of the orphan drugs authorized in the European Union have reached Spain. However, the financing of these treatments continues to be a challenge, as only 57% of them are currently financed by the National Health System. This means that, despite having the necessary authorizations, 47 orphan drugs with a National Code are still awaiting funding.
Another worrying aspect reflected in the report is the waiting time between obtaining the national code and the effective financing of the drugs, which has reached 28 months in the second quarter of this year. This long delay can have a significant impact on the health and quality of life of patients who depend on these treatments. AELMHU reiterates the importance of speeding up access to orphan drugs in Spain for patients and is at the disposal of the authorities to collaborate in the creation of a process to speed up access to them.
This report highlights both the progress made in the field of orphan drugs and the challenges that remain, highlighting the need for continued commitment by all stakeholders to improve access to these treatments for patients with rare diseases.
The sources consulted for this report include the European Medicines Agency(EMA), the Community Register of Orphan Medicinal Products, the Spanish Agency for Medicines and Health Products (AEMPS) and the Ministry of Health.
