As of December 31, 2022, Spain had 123 orphan drugs with a National Code and 63 financed, which means that 60 orphan products are still pending financing in our country.
The Spanish Association of Laboratories of Orphan Drugs y Ultrahuérfanos (AELMHU) has released this Monday the ‘Annual Report on Access to Orphan Drugs’ in Spain, a study that -as the entity advanced just a few days ago, with the first data- confirms that 2022 was a year ” very encouraging” for European patients with minority ailments while, in Spain, the delays in the approval of these drugs continue to increase. Moreover, AELMHU figures in 60 drugs for minority ailments that are still pending financing in our country.
Before the celebration of the World Day for Rare Diseases, on February 28, AELMHU makes an assessment of its access indicators. Warns the association that we are before “a decisive course” for the pharmaceutical industry in our country, since important projects such as the new Law of Guarantees and Rational Use of Medicines, the new European Orphan Drug Regulation, the new Pharmaceutical Strategy for Europe, the Strategic Plan for the Pharmaceutical Industry 2023-2025 or the Spanish presidency of the Council of the European Union.
All-time highs in the EU
The executive summary of the AELMHU ‘Annual Access Report 2022’ -an analysis of indicators that the association has been carrying out for seven years- which this year delves into the examination of relevant aspects, such as those orphan treatments that have community marketing authorization but do not yet have a National Code in our country, reviewing the deadlines and the therapeutic areas with which they correspond.
Among the main conclusions, it stands out that the European Union reached all-time highs in 2022 both in new orphan designations of products with a trade name (29) and in new marketing authorizations (24), which has raised the number of products with positive orphan designation 195, and 146 authorized.
However, as of December 31, 2022, Spain had 123 orphan drugs with a National Code and 63 financed, which means that 60 orphan products are still pending financing in our country, five more than in 2021, close to half (45 %) of which carries more than three years waiting its incorporation into the National Health System.
On the other hand, average waiting time to achieve a price and reimbursement decision, in the case of the 9 orphan drugs that were financed, it was raised to 34 months, 10 months more on average than last yearwhich could be slowing down the arrival of innovation in our country, as shown by the 23 drugs with community authorization that, according to this report, have not yet reached the Spanish market.
a collective step
For the first time in its analysis documents, the entity proposes an alternative model for this type of drug with recommendations that have already been made known to the Ministry of Health, and which address aspects such as recognition of the economic and social value provided by research on new products, equity in access to treatment, streamlining bureaucratic processes, the improvement of the evaluation process or the new financing models. They are aimed at “providing greater certainty, transparency and participation” to the entire approval process for these treatments.
Based on the data collected over the past year, AELMHU has summoned all the agents involved to “a great collective agreement” in order to seek consensus around solutions “that really improve the quality of life of all individuals and families suffering from a rare disease”. In Spain, it is estimated that there about three million of people who They have a rare ailment.