2024-01-31 09:44:38
In 2023, Spain improved access to orphan drugs by increasing the number of drugs financed by the National Health System – 53% of those authorized by Europe – and reducing the average time to approve this financing, 11 months less than in 2022.
Infographic provided by AELMHU
These are some of the most notable data from the 2023 Annual Report on Access to Orphan Drugs of the Spanish Association of Orphan and Ultra-orphan Drug Laboratories (AELMHU) which analyzes the situation of these drugs in Europe and their availability for patients in Spain.
Orphan drugs are innovative treatments that are exclusively aimed at diagnosing, preventing or treating patients with rare diseases, those that affect less than 5 per 10,000 people and that lack alternative treatment.
New designations
The report reflects that, at the European level, there has been a decrease in both the number of new orphan designations, going from 29 in 2022 to 19 this year, and in the number of new commercial authorizations, from 24 to 12.
However, in Spain these data have improved: in 2023, 21 new orphan drugs were financed, 12 more than in the previous year, adding 78 the total number of financed medicines for public health, 53% of the total of the 147 drugs that have marketing authorization from the European Medicines Agency (EMA).
According to AELMHU, comparing with previous years, “it is the first time in five years that Spain finances more than half of the orphan drugs authorized by the European Union”, although almost half of these new products are financed with restrictions and conditional prices.
Of the 45 drugs approved in Europe that They are not financed in Spain (15 less than in 2022), 40% correspond to the oncology area and 24% to the metabolic area, among others.
Almost half of these unfunded medications have been waiting more than three years for their release.
incorporation into the National Health System.
The criteria for which it is not financed are due to issues of rationalization of public spending on pharmaceutical provision and its therapeutic, social value and incremental clinical benefit, taking into account its cost-effectiveness relationship.
Infographic provided by AELMHU
Furthermore, of the 78 orphan drugs financed by public health, 38 (49%) are financed with restrictions, that is, with limitation of indications or unfunded indications.
An indicator in which Spain does not improve as of December 31, 2023 is that there are 24 orphan drugs with marketing authorization in Europe but without obtaining the national code (CN) for sale in the Spanish market.
Waiting times are reduced
The average time to approve financing in the last year has been reduced to 23 months, 11 less than in 2022.
“Without a doubt, this represents important progress with respect to the delays that have occurred in recent years. However, it is necessary to continue with this momentum to be able to reach the financing times of 2019, whose average waiting time was 14 months, or even reduce them,” the study indicates.
The president of AELMHU, María José Sánchez Losadaexplains that in 2023 the dialogue with the different public administrations was intensified “to raise awareness about the slowdown and decline that has occurred in access to orphan drugs in recent years.”
“The improvements produced – he points out – both in financing and in waiting times during this last year have represented a significant change that, from our association, we value positively and we hope that it will become a trend for the coming years.”
AELMHU is a non-profit association that brings together pharmaceutical and biotechnology companies with the commitment to invest in discovering and developing innovative therapies capable of improving the health and quality of life of patients suffering from rare and ultra-rare diseases.
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