Spain takes almost three years to fund an orphan drug, “far from the desirable six months as the maximum average time for approval and funding,” according to the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU).
Infographic provided by AELMHU
These are the first data from the 2022 Annual Access Report where every year AELMHU offers an analysis of the situation of orphan drugs in Europe and Spain and before the World Day of Rare Diseases which is commemorated on February 28.
Orphan medicines are intended to establish a diagnosis, prevent or treat patients suffering from a rare or minority disease without a therapeutic alternative. They are innovative drugs, most of them of biotechnological origin.
Data in Europe
According to the first data analyzed by AELMHU, 2022 was “an excellent year” in the European Union, with historical highs in new orphan designations of products with commercial names (29) as well as European marketing authorizations (24) .
This has brought the number of orphan medicines authorized in the European Union (EU) to 146.
Data on orphan drugs in Spain
On the contrary, the study reflects that the indicators in Spain “are far from extraordinary”.
Although 17 new orphan drugs acquired a National Code (CN) in our country, only 9 were funded, which represents a decrease of 37% compared to last year (5 less funded products).
In addition, 100% of the new funded products were price conditioned: all have monitoring conditions, 4 are subject to a maximum cost per patient and 2 have a spending ceiling.
In total, on December 31, 2022, Spain had 123 orphan medicines with a National Code and 63 funded, which means that 23 drugs approved at European level have not yet reached the Spanish market, 16% of the total of those authorized at community, the highest figure in the last 4 years.
And this situation is influenced, according to AELMHU, by the extended waiting times which, far from improving, increased by 42% in the last financial year. Thus, the average time between the National Code and the financing of an orphan product in our country exceeds 34 months, almost 3 years, far from the desirable 6 months as the maximum average time for approval and financing.
A total of 60 orphan medicines remain unfunded in Spain, 5 more than in 2021, of which 45% await funding for more than 3 years. And no advanced orphan therapies were approved for funding last year either.
These first results of our report lead us to claim, once again, that Spain should be able to simplify the approval processes of MM.HH. at national level, especially because its effectiveness has already been accredited by the European Medicines Agency (EMA)”, says Maria José Sánchez Losada, president of AELMHU.