Spain takes almost three years to finance an orphan drug, “very far from the desirable six months as the maximum average time for approval and financing,” according to the Spanish Association of Orphan and Ultra-orphan Drug Laboratories (AELMHU).
These are the first data from the 2022 Annual Access Report in which each year AELMHU offers an analysis of the situation of orphan drugs in Europe and Spain and in view of the World Day for Rare Diseases that is commemorated on February 28.
Orphan drugs are intended to establish a diagnosis, prevent or treat patients suffering from a rare or minority disease with no therapeutic alternative. These are innovative drugs, most of them of biotechnological origin.
The data in Europe
According to the first data analyzed by AELMHU, 2022 was “an excellent year” in the European Union, with all-time highs in new orphan designations of products with a trade name (29) as well as European marketing authorizations (24).
This has brought the number of orphan drugs 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 being extraordinary”.
Although 17 new orphan drugs acquired a National Code (CN) in our country, only 9 were financed, which represents a decrease of 37% compared to last year (5 less financed products).
In addition, 100% of these new financed products obtained a conditional price: all have follow-up conditions, 4 are subject to a maximum cost per patient and 2 have an expense ceiling.
In total, as of December 31, 2022, Spain had 123 orphan drugs with a National Code and 63 financed, which means that 23 drugs approved at the European level have not yet reached the Spanish market, 16% of the total of those authorized to community level, the highest figure in the last 4 years.
And in this situation influences, according to AELMHU, the long waiting times that, far from improving, increased by 42% in the last year. Thus, the average time between the National Code and the financing of an orphan product exceeds 34 months in our country, almost 3 years, far from the desirable 6 months as the maximum average time for approval and financing.
A total of 60 orphan drugs remain unfinanced in Spain, 5 more than in 2021, of which 45% have been waiting for financing for more than 3 years. And no advanced orphan therapies have been approved for funding in the past year either.
“These first results of our report lead us to claim, once again, that Spain should be able to simplify the approval processes for MM.HH. at a national level, especially since its effectiveness has already been accredited by the European Medicines Agency (EMA)”, considers María José Sánchez Losada, president of AELMHU.