The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) held today the II National Conference The Future of Advanced Therapies for Rare Diseases with the aim of promoting dialogue and critical reflection among the different agents of the healthcare system in order to highlight the development of this therapeutic approach for rare diseases and to continue working together to facilitate their availability and access.

Among the main conclusions, the participants agreed on the therapeutic and social value of these advanced therapies, and the joint work of the different agents involved, which are essential to accelerate their availability and change the lives of people suffering from rare diseases.

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The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) has announced the appointment of Beatriz Perales as the new president of the Association. The new Board of Directors will also include María José Sánchez, general manager in Spain and Portugal at CSL Behring, as vice president; José Luis Moreno, general manager in Italy, Spain and Portugal at Ultragenyx, as secretary; Sergio Bullón, general manager in Spain at Alnylam Pharmaceuticals, who continues as treasurer; and Iván Silva, director of Market Access for the Southern cluster and managing director of Spain at Kyowa Kirin, who joins the Board for the first time as a member.

The new Board of Directors was elected at the Ordinary Assembly, held at Takeda's offices.

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On the occasion of International Clinical Trials Day, which is celebrated on May 20, the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has published its annual report in which it highlights that nearly a quarter of the clinical trials carried out in Spain in 2023 focused on rare diseases, despite a decrease of two points compared to the 25% they represented the previous year.

The report reveals that during 2023, 834 clinical trials were authorized in Spain, of which 190 corresponded to rare diseases, representing a 10% decrease over the previous year. A particularly positive development was the significant increase in clinical trials in the early stages of development, which grew by 19% over the previous year.

On the other hand, trials directed at the pediatric population accounted for 22% of all trials authorized in the field of rare diseases.

According to AELMHU data based on the Spanish Registry of Clinical Trials (REec), dependent on the Spanish Agency of Medicines and Health Products (AEMPS), the pharmaceutical industry continues to be the main driver of clinical trials in rare diseases in Spain, accounting for 96%, one point more than in 2022, showing a clear commitment to research in this field.

The Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) has announced a new edition of its awards, which were created to promote knowledge of rare diseases and orphan drugs, highlighting each year the work of professionals and organizations through the selection and recognition given to the best care, dissemination and awareness projects on this type of pathologies.

As the most outstanding novelty of this VI edition, AELMHU has incorporated a new category to recognize the "Best work of communication, dissemination and / or awareness of rare diseases". This category is open to professionals whose communication work has contributed to a greater knowledge, dissemination and awareness of rare diseases.

This category is completed with the other four categories already existing in previous editions and which will select:

• The "Best Care Project on Rare Diseases".
• The "Best Research Career in the field of rare diseases".
• The "Best Rare Disease Outreach and/or Awareness Raising Project".
• The "AELMHU 2024 Honorary Award for work or contribution to improve the lives of people with rare diseases".

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%.

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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.