Nota prensa web

Con motivo del Día Internacional del Ensayo Clínico que se celebra el 20 de mayo, la Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultrahuérfanos (AELMHU) ha hecho público su informe anual en el que destaca que cerca de una cuarta parte de los ensayos clínicos realzados en España en 2023 se centraron en enfermedades raras, a pesar de haberse registrado un descenso de dos puntos respecto al 25% que representaron el año anterior.

El Informe revela que durante 2023 se autorizaron en España 834 ensayos clínicos, de los cuales 190 correspondieron a enfermedades raras, que supusieron un descenso del 10% respecto al año anterior. Como dato especialmente positivo destaca el aumento significativo de los ensayos clínicos en las primeras fases de desarrollo, que han crecido un 19% respecto al año anterior.

Por otro lado, los ensayos dirigidos a población pediátrica representaron el 22% de todos los autorizados en el ámbito de las enfermedades raras.

Según datos de AELMHU basados en el Registro Español de Estudios Clínicos (REec), dependiente de la Agencia Española de Medicamentos y Productos Sanitarios (AEMPS), la industria farmacéutica sigue siendo la principal impulsora de los ensayos clínicos en enfermedades raras en España, al representar el 96%, un punto más que en 2022, mostrando un claro compromiso con la investigación en este ámbito.


La Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultrahuérfanos (AELMHU) ha anunciado una nueva edición de sus premios, que se crearon para fomentar el conocimiento de las enfermedades raras y los medicamentos huérfanos, destacando cada año la labor de profesionales y organizaciones mediante la selección y reconocimiento que se otorga a los mejores proyectos asistenciales, de divulgación y sensibilización sobre este tipo de patologías.

Como novedad más destacada de esta VI edición, AELMHU ha incorporado una nueva categoría para reconocer la “Mejor labor de comunicación, divulgación y/o concienciación sobre enfermedades raras”. En esta categoría podrán participar profesionales que con su trabajo de comunicación han contribuido a un mayor conocimiento, divulgación y concienciación de las enfermedades raras.

Esta categoría se completa con las otras cuatro categorías ya existentes en ediciones anteriores y que seleccionarán:

  • El “Mejor Proyecto Asistencial sobre enfermedades raras”
  • La “Mejor Trayectoria Profesional investigadora en el campo de las enfermedades raras”
  • El “Mejor Proyecto de difusión y/o sensibilización sobre enfermedades raras”
  • El “Premio Honorífico AELMHU 2024 por su labor o contribución para mejorar la vida de las personas con enfermedades raras”

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.

Foto familia_Premios_AELMHU_2023

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 in four clinical trials in Spain investigates rare diseases

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.

AELMHU demands a change of model in Spain

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.

Orphan Drug Annual Access Report 2022
AELMHU publishes preliminary results of its 2022 Annual Access Report

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.

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AELMHU continues to grow with the addition of new members

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.

AELMHU publishes first report on IPT 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