María José Sánchez, President of AELMHU, represented the association at the event organised by the Spanish Federation for Rare Diseases (FEDER) together with the European Alliance representing these pathologies (EURORDIS), to discuss the new European Pharmaceutical Strategy and the challenges and opportunities it poses for rare disease patients and orphan drugs.
The president participated in two of the three tables of the day to talk about the unmet needs, the centralised purchase of orphan drugs and incentives in research. She was joined by: Juan Carrión, Antonio Cabrera and Jordi Cruz, members of the Board of Directors of FEDER; Mercedes Amellam from the GNAO1 association; Nacho Muela, from the National Association of Leber's Optic Nerve Atrophy and Icíar Sanz, Director of the International Department of Farmaindustria.
César Hernández, Head of the Department of Medicines for Human Use of the Spanish Agency of Medicines and Health Products; Pedro Irigoyen, Directorate General of Economic-Financial Management and Pharmacy of the Madrid Health Service; and Vicente Alonso, Directorate General of Health Care of the Extremadura Health Service were the representatives of the administration.
There are currently 6,172 rare diseases, 95% of which have no treatment. Therefore, as has been stated at all the roundtables, it is urgent to involve all affected parties so that together they can find a solution to improve diagnosis and access to treatment. In Spain alone, it is estimated that this could improve the lives of nearly 3 million people.
However, according to the latest data from the Report on Access to Orphan Drugs in Spain, as of 30 April 2022, only 58 orphan drugs are funded in Spain, 44% of those with marketing authorisation from the European Medicines Agency (EMA). Moreover, in addition to this low percentage, many of these products do not have funding approved for all the indications authorised by the EMA, which means that in many cases we cannot speak of full funding. And if a product does get full funding, in many cases it will take years to obtain it. Currently, the average time from the time a medicine has CN until it is funded is 32 months.
AELMHU has once again called for a review of the current funding model for medicines. An urgent review that allows for the modification of the model or, in its place, the creation of a specific one for these treatments.
Improving access for all patients and reducing the time they have to wait for diagnosis and/or treatment will remain our priority.
