Brussels, 28 February 2025
On 28 February, marking the 2025 Rare Disease Day, Parliament’s public health committee kicks off its survey on rare diseases.
The purpose of this public consultation is to provide a basis for the forthcoming work of the Committee on Public Health (SANT) in bringing a better understanding and detailed knowledge of the challenges of persons affected by rare diseases and views of persons working with or involved in rare diseases.
The online survey can be filled in by all interested parties and is open for one month. Citizens, but also stakeholders (patient organisations, representatives of industry, NGOs etc.) have the opportunity to provide anonymous input with information about their difficulties, needs and expectations regarding further EU actions in the field of rare diseases.
Parliament has consistently highlighted the need for research into rare diseases and the development of medicines. In a 2021 resolution on the EU’s pharmaceutical strategy, it stressed the importance of advancing research for unmet medical needs. Previously, in a 2020 resolution, it called for the creation of an EU action plan for rare and neglected diseases as part of a post-pandemic public health strategy. The newly established SANT Committee will now lead Parliament’s work on the issue, including through the reform of the pharmaceutical legislation and upcoming initiatives.
Background
Rare Disease Day, observed every year on 28 February (or 29 in leap years) – the rarest day of the year – was set up in 2008 by EURORDIS and partner organisations to raise awareness and generate change for the 300 million people worldwide living with a rare disease, and for their families and carers.
Rare diseases (fewer than five cases per 10 000 people), affect approximately 36 million Europeans. Around 6 000 to 8 000 rare diseases have been identified worldwide. Rare diseases can be of genetic origin (such as Duchenne muscular dystrophy, Huntington’s disease, amyotrophic lateral sclerosis, Creutzfeldt-Jakob disease, metachromatic leukodystrophy), but the group also includes metabolic diseases, infectious diseases, autoimmune diseases and rare cancers.