EURORDIS-Rare Diseases Europe

EURORDIS welcomes the opportunity to contribute to the discussion on the EUs next long-term budget and the proposed European Competitiveness Fund (ECF). This feedback aims to ensure that the unmet needs of the 30 million Europeans living with rare diseases are fully reflected in the EUs financial priorities for 20282034, in line with the Unions health, human rights, and solidarity commitments. 1.

EURORDIS-Rare Diseases Europe welcomes the opportunity to comment on the combined evaluation roadmap / inception impact assessment of the general pharmaceutical legislation and commits to contribute to best of its ability to the outline process. Each of the over 6000 identified rare diseases affects a very small population, but altogether they affect nearly 30 million people in the EU.

EURORDIS-Rare Diseases Europe welcomes the EU Biotech Act as a pivotal opportunity to establish the EU as a leader in biotechnology, particularly through a focus on rare diseases (RD). With 30 million people affected by RDs across Europe, and only 6% of these conditions having approved treatments, the field represents both a significant public health challenge and a strategic area for biotech innovation.

There are 6000 various rare diseases affecting over 30 million people across Europe, but only 6% of all known rare diseases have curative treatment. The rare disease community has many unmet needs. The limited number of patients alongside the scarcity of available knowledge and expertise on these diseases, make rare diseases a field that could greatly benefit from the European action.

In the field of rare diseases (RD), typified by limited knowledge and data on each specific disease, needs and benefits of capturing, using, sharing and federating health data to advance scientific research and improve care are very strong.

EURORDIS represents 30 million people living in Europe with one or more of over 6,000 identified rare diseases. We welcome the opportunity to submit our observations to the Data Governance Act (DGA) as we believe that it should contribute to derive value from health data to improve healthcare services, research and policymaking while protecting people’s privacy and respecting their preferences.

EURORDIS welcomes the opportunity to comment on this proposal for the governance of common European data spaces. The rare disease community is acutely aware of the importance of pooling and sharing data to advance research and improve health outcomes.

EURORDIS-Rare Diseases Europe acknowledges the need to revise existing legislation for Blood, Tissue and Cell derived products as they did not suffice to ensure EU self-sufficiency for these products, while their safety may still be a concern. Also, important disparities exist in the EU, between Member States where these products are available and used, and other MS where they are too difficult to obtain.

EURORDIS acknowledges the need to revise legislations for Blood, Tissue and Cell derived products. Current legislations and rules did not suffice to ensure EU self-sufficiency for these products, and their safety might still be a concern.

EURORDIS Rare Diseases Europe welcomes the opportunity to provide feedback to the European Commission Inception Impact Assessment (IIA) on Paediatric Medicines and Orphan Medicinal Products. EURORDIS is of the opinion that moving forward, any future improvements to the Regulation on Orphan Medicinal Products and Paediatric Medicines are to reinforce the EU processes in a structured and seamless way for all rare…

EURORDIS-Rare Diseases Europe welcomes the proposal for a Directive establishing the European Disability Card and the European Parking Card for persons with disabilities, as well as the complementary proposal extending the Directive to third country nationals legally residing in a Member State.

EURORDIS-Rare Diseases Europe welcomes the procedure to establish a EU Disability Card and would like to offer the following input, to contribute to the inclusion of all voices of the disability community in the future Card: We call for a EU Disability Card that allows for mutual recognition of disability status across EU countries and is accepted by all areas of services which offer preferential conditions to…

EURORDISRare Diseases Europe is a non-profit alliance of over 1000 rare disease (RDs) patient organisations working together to improve the lives of 30 million people living with a RDs in Europe. A EU public health priority due to the unmet needs of RD population, added value of the EU action, RDs have been consistently supported by EU funding programmes, thus helping improve lives of people with RDs, advance…