Edition of 08 August 2025

On 16 July 2025, the European Commission unveiled its proposed budget for 2028 to 2034. Amounting to almost 2 trillion euros, the budget establishes a Multiannual Financial Framework (MFF) which aims to provide more flexible and streamlined financial programs at the EU level. As part of this framework, the Commission also announced a new version of Horizon Europe for the upcoming budgetary period, with a proposed budget of 175 billion euros, double that of the current programme. The new Horizon Europe will continue to support research and innovation, while aiming to increase European competitiveness, foster international collaboration, and connect science with society.

Following the publication of the proposed MFF, EURORDIS welcomed its emphasis on innovation and competitiveness. However, they criticised the lack of a standalone health programme, and expressed concerns that the budget does not go far enough to combat the challenges Europe is currently facing.

“To accelerate innovation, it is essential to preserve strong support for basic research and to secure the funding needed to deliver on the EU’s Life Sciences Strategy. Undervaluing fundamental research today risks stalling tomorrow’s medical breakthroughs.” – Roseline Favresse, EURORDIS Head of Research Policy and Initiatives

Despite these concerns, change is still possible before the budget’s adoption. Moving forward, EURORDIS will continue to advocate for ambitious, inclusive and forward-looking EU investment plans for the 2028-2034 period, ensuring that rare diseases remain a strategic priority across all relevant policy areas.

The European Rare Disease Research Alliance (ERDERA) is conducting a survey to explore the needs of rare disease researchers for consultancy services. Responses aim to support the possible development of an ERDERA Consultancy Service, in order to support projects financed by ERDERA and throughout the broader European rare disease community. The survey takes approximately 5 minutes to complete, and will remain open until 30 September 2025.

On 1 July 2025, European rare disease leaders gathered for the launch of the Nordic Rare Diseases Roadmap. The event, which coincided with Denmark’s assumption of the Presidency of the EU Council, was an opportunity for stakeholders to discuss ongoing and future work to improve the lives of the more than 1 million people living with a rare disease in the Nordic countries, and the more than 30 million such individuals in all of Europe. The Roadmap’s launch also helped set the stage for the Nordic Rare Disease Summit 2025, to be held from 16-17 September in Copenhagen.

The Nordic Rare Disease Summit 2025, which follows in the footsteps of previous editions held in 2021 and 2023, is a collaborative initiative which unites voices from patient organisations, academia, and industry to drive advancement in rare disease care and policy. This year’s theme, “Act 2030: Pushing Boundaries for Rare” aims to place patient empowerment at the centre of discussions.

The Nordic Rare Diseases Roadmap is a call to action, drawing on discussions held during the 2021 and 2023 Summits, which sets out recommendations aimed at improving rare disease diagnosis, care and management in the Nordics.

A new article has been published in Management în Sănăte (Health Management), in both English and Romanian, exploring various aspects of rare disease care in the Republic of Moldova. The study collected qualitative and quantitative data on the experiences of health professionals and people living with a rare disease (PLWRD) in order to evaluate barriers and opportunities for rare disease management within Moldova’s national health system.

The authors identified a number of barriers faced by family doctors in providing assistance to PLWRD. These included insufficient knowledge on rare diseases; difficulty in identifying and diagnosing rare diseases; and difficulties in interaction and collaboration with specialists. In particular, gaps in medical education were identified as a contributing factor to these barriers.

Despite these barriers, more than half of patients surveyed reported a high level of satisfaction with the assistance they received within Moldova’s health system. When reported satisfaction was low, it was correlated with a number of challenges such as a lack of specialists in the relevant field, unavailability of disease-specific information, and lack of communication.

In order to overcome these barriers and improve the quality of health care available to PLWRD in Moldova, the authors outline measures to improve awareness of rare diseases and communication between health professionals. In particular, they recommend the creation of an electronic national register of rare diseases, strengthening genetics services, and improving professional education on rare diseases.

In June, the EMA's Committee for Orphan Medicinal Products (COMP) accorded 19 new orphan designations for the following indications:

• Treatment of amyotrophic lateral sclerosis
• Treatment of Rett syndrome
• Treatment of amyotrophic lateral sclerosis
• Treatment of acute myeloid leukaemia
• Treatment of lymphoplasmacytic lymphoma
• Treatment of congenital adrenal hyperplasia
• Treatment of glioma
• Treatment of multiple system atrophy
• Treatment of fragile X syndrome
• Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
• Treatment of Netherton syndrome
• Treatment of primary sclerosing cholangitis
• Treatment of multifocal motor neuropathy
• Treatment of osteogenesis imperfecta
• Treatment of radiation induced maculopathy
• Treatment of hepatocellular carcinoma
• Treatment of systemic sclerosis
• Treatment of mastocytosis
• Treatment of Becker muscular dystrophy