A few weeks before the celebration of World Day for Rare Diseases, the Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) publishes the results of its Annual Access Report, which analyzes all orphan medicinal products (OMPs) with a trade name, which have a European orphan designation in force as of December 31, 2023. In it, the Association analyzes in detail the situation of orphan products in Europe and their availability for patients with rare diseases (RD) in Spain.

According to the data available for 2023, at the European level there has been a decrease both in the number of new orphan designations, from 29 in 2022 to 19, and in the number of new commercial authorizations, from 24 in 2022 to 12. However, in this same period, Spain has improved its access data, increasing the number of orphan drugs financed and reducing the times for their financing, thus contributing to improve the decrease that has occurred in recent years.

In 2023, 21 new orphan drugs were financed, 12 more than in the previous year, bringing the total number of orphan drugs financed by the National Health System (NHS) to 78, out of 147 with marketing authorization from the European Medicines Agency (EMA), i.e. 53%.

See the full content of the Press Release in the attached document.

The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) has presented its annual awards in recognition of the contribution of institutions and healthcare professionals in the field of rare diseases. The event took place at the Bentelsmann Space and was attended by a notable representation of industry professionals, patient organizations, scientists and institutional representatives of the health field.

In this fifth edition, AELMHU wanted to highlight, posthumously, the exceptional work carried out by Dr. Julio Sánchez Fierro, awarding him this year's Honorary Award for his contribution in favor of patients in matters such as equity and access to medicines and his contributions in the development of numerous legislative texts. These contributions have confirmed him as one of the most outstanding figures in Spain in the defense of Health and Pharmaceutical Law in Spain.

The doctor, professor, researcher and expert geneticist Ángel María Carracedo Álvarez has also been distinguished in the category of best professional career in the field of rare diseases for his contribution to research and innovation.

In the category of best care project on Rare Diseases, the jury awarded the project 'Servicio de Atención Integral Telemática a las personas afectadas de Distrofia Muscular de Duchenne y Becker y sus familiares' of the Asociación Duchenne Parent Project España. And completing the four categories of these awards, in the category corresponding to the best project for dissemination, diffusion and awareness of RRD, the winner was the 'RetroDravet' project of the Spanish delegation of the Dravet Syndrome Foundation.

One out of every four clinical trials authorized in Spain in 2022, was to investigate some rare disease, increasing by 3% compared to 2021 and marking an all-time high with 233 trials dedicated to these minority pathologies. This is reflected in the results of the Annual Report on CCD in Spain conducted by the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) on the occasion of the celebration of World Clinical Trials Day, which takes place on May 20.

According to this study, an increasing trend in the percentage of trials focused on rare pathologies was maintained for the third consecutive year in 2022, despite the fact that the overall computation of authorized HCCs in Spain fell by 8%, from 996 in 2021 to 923 in 2022.

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has made public the full text of its Annual Report on Access to Orphan Drugs (OMH) in Spain, a study that confirms that 2022 was a very hopeful year for patients with rare diseases (RD) at the European level, while in Spain delays in the approval of these treatments continue to increase.

Thus, before the upcoming commemoration of the World Day for Rare Diseases, AELMHU makes an assessment of its access indicators, warning that "we are facing a decisive year for the pharmaceutical industry in our country", since important projects such as the new Law on Guarantees and Rational Use of Medicines, the new European Regulation on Rare Diseases, the new Pharmaceutical Strategy for Europe, the Strategic Plan for the Pharmaceutical Industry 2023-2025 or the Spanish presidency of the Council of the EU are planned.

The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) publishes the preliminary results of its Annual Report on Access to Orphan Drugs in Spain 2022 in which it analyzes the access situation of trade-name products with orphan designation in force as of December 31, 2022.

At the end of the year, 146 orphan drugs had been authorized for marketing in the EU, of which 123 have already reached Spain (84%) but only 63 (43%) are financed.

During 2022, the SNS has incorporated 9 new orphan treatments and the average time from obtaining the National Code to its funding has been 34 months.

The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) presented its annual awards yesterday evening at a ceremony held at Espacio Bertelsmann in Madrid.

In this fourth edition, the winners were the Hospital San Joan de Déu de Barcelona for the project 'La casa de Sofía', in the category of best care project on Rare Diseases (RRD); the Asociación MPS-Lisosomales España for the project 'Primer certamen de fotografía y cortometraje de EE, Estela's Birthday', in the category of best project for dissemination, diffusion and awareness of RRDs; and the doctor and scientist Francesc Palau Martínez, in the category of best professional career in the field of these rare diseases.

AELMHU also wanted to give special recognition to the Mehuer Foundation by awarding them this year's honorary prize for their commitment to social knowledge and the promotion of research in the field of HR.

The Association is strengthened thanks to the incorporation of four new members: Ferrer, GenSight Biologics, Insmed and UCB, consolidating its influence and representativeness in Spain in the field of orphan and ultra-orphan drugs, indicated for patients with rare diseases.

Together with these new companies, the Association claims to be a reference in the pharmaceutical and biotechnology sector and emphasizes its commitment to contribute to improving the situation and quality of life of people and families suffering from rare diseases, promoting the knowledge of their pathologies and the recognition of the therapeutic and social value of orphan drugs.

The evaluation of orphan drugs is delaying access to innovation for Spanish patients with Rare Diseases (RD). This is the main conclusion drawn from the first Analysis of the Therapeutic Positioning Reports of HMPs produced in our country. A pioneering project developed by the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) in its aim to provide the largest number of data and access indicators in Spain.

Although all orphan drugs pass an exhaustive analysis by the European Medicines Agency (EMA), in which the agencies of the different Member States participate, when they arrive in our country they begin a long process of re-evaluation in which the Spanish institutions re-examine their efficacy, as well as the budgetary impact they cause.

As the Spanish Agency for Medicines and Health Products (AEMPS) itself has been demanding, it is necessary to provide this body with sufficient financial and human resources to deal with a huge volume of dossiers that is growing every year.

Executive Summary

Infographic of the Report

The Spanish Association of Orphan and Ultra-Orphan Medicines Laboratories (AELMHU) has re-elected María José Sánchez Losada as President, following elections to its Board of Directors. This re-election means continuing with the project to improve the quality of life of patients with rare diseases, promoting the therapeutic and social value of orphan drugs.

The new Board of Directors is completed with José Luis Moreno Sánchez, as vice-president; David Moreno Alforcea, secretary and member; Sergio Bullón Avendaño, as treasurer and member; and Jordi Casals Ferragut, as member.

Once again, on 20 May, on the occasion of World Clinical Trials Day, the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) presents its Annual Report on Clinical Trials in Rare Diseases in Spain.

This report shows that Spain continues to exponentially increase its commitment to research in clinical trials focused specifically on rare diseases, with 225 studies authorised. This means that 23% of new studies are focused on rare diseases.