The Spanish Association of Orphan and Ultra-Herphan Drug Laboratories presented the awards of the sixth edition of the AELMHU Awards, in an emotional evening full of recognition in which the most relevant contributions in the field of rare diseases were highlighted. Institutions, patient associations and healthcare professionals were the main protagonists of an event that took place in Madrid, with the participation of institutional representatives, industry and the scientific and healthcare community.
The president of AELMHU, Beatriz Perales, opened the ceremony by highlighting the crucial role of the people and organizations committed to improving the lives of patients with rare diseases, some of whom were present, such as the Spanish Federation of Rare Diseases (FEDER). The journalist María Rey was in charge of conducting an event that closed with an emotional speech by César Hernández, General Director of the Common Portfolio of Services of the National Health System and Pharmacy of the Ministry of Health. The event included words of support and solidarity for the people affected by the recent DANA, in a gesture that further united those present in the fight for the visibility and treatment of rare diseases.
The jury of the awards, made up of renowned professionals from the sector, and represented on stage by Raquel Sanchez of the Spanish Patients' Forum (FEP), agreed on the need to continue to promote research and on the importance of a multidisciplinary and joint approach to minority diseases and orphan drugs.
Award for Best Assistance Project: Ana Carolina Díez Mahou Foundation.
En un acto cargado de emoción, Javier Pérez-Mínguez, director de la Fundación Ana Carolina Díez Mahou, ha recogido el Premio al Mejor Proyecto Asistencial por su innovador programa, “Primera Estrella María de Villota”, que combina atención médica, psicológica y deportiva para mejorar la calidad de vida de estos pequeños pacientes. Javier, quien, como padre, conoce de cerca la experiencia de tener un hijo con una enfermedad rara, ha hecho especial énfasis en la importancia de continuar trabajando para ofrecer un futuro mejor a los afectados por estas patologías. El premio ha sido entregado por Belén Pérez, vicepresidenta de la Asociación Española de Genética Humana, y Raquel Sánchez (FEP).
Award for the best research career: Dr. Lluís Montoliu
The Award for the Best Professional Research Career went to Dr. Lluís Montoliu, an outstanding researcher in the field of albinism and a pioneer in applied biotechnology and gene editing, who received the award from Mª Esperanza Marcos, president of the Health Law Section of the Madrid Bar Association and Mr. Jorge Mestre, health economist. In his speech, Dr. Montoliu underlined the importance of working together with the pharmaceutical industry and patient associations to advance in the treatment of rare diseases. During the event, he had an emotional memory for ALBA, the association for the help of people with albinism, with whom he has worked closely to improve the quality of life of those affected and whose president, Monica Puerto, entered live by videoconference to have a few words of affection and recognition for the excited awardee.
Best Awareness Raising Project Award: AEFAT
El Premio al Mejor Proyecto de Difusión y Sensibilización ha sido entregado a la Asociación Española de Familias Afectadas por Ataxia Telangiectasia (AEFAT), en reconocimiento a su programa de deporte inclusivo y sensibilización social, “Corre, frena la ataxia telangiectasia”. De la mano de Susana Fernández, vicepresidenta de la Asociación Nacional de Informadores de la Salud (ANIS) y Antoni Gilabert, director de Innovación y Partenariado en el Consorcio de Salud y Social de Cataluña, Rosa Casbas, responsable de Comunicación de la asociación, y Álvaro de Arqués, uno de los asociados, han recogido el galardón, agradeciendo a los voluntarios y participantes en la maratón que convirtió esta actividad en una experiencia de visibilidad y concienciación. Rosa ha destacado que “dar visibilidad a las enfermedades raras es esencial para acelerar su investigación y diagnóstico”.
Recognition of the work of communication: Dr. José Luis Poveda
El Premio a la Mejor Labor de Comunicación, Divulgación y Concienciación ha sido otorgado al Dr. José Luis Poveda por su excepcional trabajo en sensibilizar a la sociedad sobre las enfermedades raras y el acceso a los medicamentos huérfanos. Aunque la DANA le ha impedido asistir presencialmente, el Dr. Poveda ha podido participar por videoconferencia, agradeciendo el reconocimiento y extendiendo su gratitud a todos los pacientes, profesionales de la salud y familiares que, como él, luchan por dar visibilidad a estas patologías. En su intervención, ha destacado que “la esperanza de las enfermedades raras tiene un nombre, y ese nombre es investigación”.
As a finale to the presentation of this award, an emotional Dr. Poveda and the rest of those present were able to watch an emotional video that his loved ones and family had prepared for the occasion. The members of the jury Eva Bermejo, director of the Institute for Rare Diseases Research of the Carlos III Institute of Health, and Manuel Perez, vice president of the Orphan Drugs and Rare Diseases Foundation, will be in charge of guarding his award.
Posthumous tribute to Dr. Josep Torrent-Farnell
One of the most emotional moments of the evening was the AELMHU 2024 Honorary Award, given posthumously to Dr. Josep Torrent-Farnell , presented by César Hernández, Director General of the National Health System Common Portfolio of Services and Pharmacy, and the AELMHU Board of Directors.
Iolanda Arbiol, who had the good fortune to share many years working alongside Dr. Torrent, wanted to dedicate some very emotional words of recognition to him for his tireless work to improve the lives of patients with rare diseases. Afterwards, the award was presented to his family, his sister Carme and his nephew, who were deeply grateful for this tribute.
Dr. Torrent-Farnell was a key figure in the history of Spanish public health, and his legacy lives on through his work at the Spanish Agency for Medicines and Health Products (AEMPS), the European Medicines Agency and in the National Health System's rare disease strategy. His pioneering vision helped rare diseases receive the attention they deserve at the European and global level.
Innovation and commitment to society
Beatriz Perales, presidenta de AELMHU, ha cerrado la ceremonia destacando que “los medicamentos huérfanos proporcionan un gran valor sanitario, pero también económico y social para España”. En su intervención, ha destacado que la innovación en el tratamiento de las enfermedades raras es un activo fundamental para el futuro de la sociedad, reafirmando el compromiso de AELMHU de seguir luchando por un mejor acceso a tratamientos para los pacientes.
Finalmente, César Hernández, director general de Cartera Común de Servicios del Sistema Nacional de Salud y Farmacia del Ministerio de Sanidad, ha clausurado el evento recordando que las enfermedades raras son un compromiso social que requiere la colaboración de todos los sectores para encontrar soluciones. “Es fundamental seguir trabajando juntos para avanzar en este objetivo”, ha añadido, cerrando una noche dedicada a la solidaridad, la investigación y la esperanza para millones de personas afectadas por estas patologías.
The gala concluded with a speech by Cisco Garcia, a lawyer by profession who, after a snowboarding accident in December 2015, decided to reinvent himself and dedicate himself entirely to chair tennis, a sport in which he debuted as a professional in 2018.
Cisco, in addition to thrilling the audience with his story of overcoming his obstacles, has put the icing on the cake to a gala, one more year, tremendously emotional.
