The Legislative Observatory for Rare Diseases and Orphan Drugs presented a decalogue in the Congress of Deputies on Thursday, outlining ten «essential» proposals to advance an equitable and comprehensive approach to these conditions. Among these proposals is the call to update the Rare Diseases Strategy of the National Health System and strengthen neonatal screening programs and new diagnostic techniques.
Regarding the update of the Rare Diseases Strategy of the National Health System from 2009, currently under review by the Ministry of Health, the Observatory deems it necessary to conduct periodic evaluations post-renewal to assess its implementation and achieved objectives.
«It is an absolute necessity to update the Strategy and carry out periodic evaluations,» reiterated Ofelia de Lorenzo, President of the Spanish Association of Health Law and member of the Observatory.
Additionally, the decalogue suggests promoting comprehensive training in rare diseases for professionals and healthcare workers, ensuring greater socio-sanitary coordination, improving the Network of Centers, Services, and Reference Units (CSUR), creating a unique registry for rare diseases, and enhancing funding for care and treatments.
«We need specific and earmarked budget allocations, but they must always be respected. Research on orphan drugs and rare diseases should not be compromised,» emphasized Francisco Zaragozá, Professor of Pharmacology at the University of Alcalá and member of the Legislative Observatory for Rare Diseases and Orphan Drugs.
On the fifth anniversary of the Observatory, achievements over the years were highlighted, such as the production of eight reports, including those on Covid-19 and rare diseases, as well as works related to European pharmaceutical legislation.
In the last five years, the Observatory has driven around 35 parliamentary initiatives in the Congress, Senate, and regional parliaments, largely based on their reports, according to Remedios Martel, former Director-General of Public Health in Andalusia and Observatory member.
Consultant and former Health Counselor of the Government of the Canary Islands, José Manuel Baltar, commended the recent advancements in neonatal screening. He noted the increase from screening seven diseases until 2024 to screening twelve, with plans to reach twenty-three in the coming months, as announced by the Ministry of Health.
Baltar also highlighted Health’s intention to improve access to orphan drugs through more agile mechanisms in the future drug law draft.
Lastly, the announcement of a law for patient organizations by the Ministry was lauded as a positive step towards providing them with their legal framework, fostering dialogue among patients, professionals, researchers, and lawmakers.
FUENTE