The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) today presented the results of its Annual Access Report 2024, a comprehensive analysis of the situation of orphan drugs in Europe and their availability to patients in Spain.
Developments in Europe
The AELMHU report reflects a positive evolution at the European level, with 25 new drugs with orphan designation and trade name registered in 2024, an increase of 6 products over the previous year. In addition, 17 drugs have obtained marketing authorization in the European Union from the European Medicines Agency (EMA), improving the number of authorized products by 5 points compared to 2023.
By the end of 2024, 210 drugs have orphan designation and trade name, and 147 of them have marketing authorization in the EU. With this, AELMHU stresses the importance that the new European legislation continues to promote research and development of treatments for rare diseases, eliminating bureaucratic barriers and improving equitable access to these drugs in all countries of the European Union.
Access to Spain
In our country, the evolution has also been positive, with 24 new drugs obtaining the national code (CN) in 2024, compared to 10 the previous year. However, the number of drugs financed has fallen slightly, from 21 to 17. Despite this, Spain has managed to finance 58% of the orphan drugs authorized in the EU, reaching 85 products financed by the National Health System (NHS), 5 percentage points more than in the previous year.
At the close of the report, Spain had 131 orphan drugs with a national code, representing 89% of the products authorized in the EU. However, 46 orphan drugs remain unfunded, 39% of which have been awaiting a favorable resolution for more than three years.
Waiting times at Financing
The report also shows that waiting times from the time a drug obtains the national code until it is approved for funding remain at an average of 23 months. The Association points out that this data demonstrates the need for a new pricing and financing model that takes into account the specificity of orphan drugs in Spain, and that facilitates earlier and more equitable access for all patients with rare diseases, a strategic therapeutic area in the Pharmaceutical Industry Strategy 2024-2028.
Advanced Therapies for Rare Diseases
A highlight in the 2024 report is the funding of 3 new advanced therapies for rare pathologies, a significant advance as there had been no such funding since 2021. Currently, 15 advanced therapies have marketing authorization in the EU, of which 14 have a national code in Spain and 7 are financed by the SNS.
This report highlights significant advances in access and funding of orphan drugs in Spain, but also highlights persistent challenges, such as waiting times for funding and the need for a more agile and equitable approach for patients with rare diseases. With this, AELMHU expresses its commitment to work to improve the financing and access to orphan drugs, contributing to the welfare of patients with rare diseases and collaborating with public administrations and other entities involved in the process.
You can consult theexecutive summary of the report, theinfographic, andthe press release.
