A draft report in the European Parliament’s Committee on Public Health (SANT), led by MEP Nicolás González Casares, calls on the European Commission to propose legislation establishing a comprehensive and binding European rare disease action framework to tackle fragmentation and inequalities across diagnosis, treatment access, research coordination, data infrastructure, patient support and governance.
The text sketches a Regulationstyle framework with six interdependent pillars and proposes EUlevel coordination mechanisms, common indicators and regular reporting. It also pushes for structural reinforcement of European Reference Networks (ERNs), including stronger governance and more sustainable integration into national systems.
Why it matters for patients and families
For people living with rare diseases, the draft’s clearest signal is its focus on shortening the “diagnostic odyssey”. In the proposed legislative text, it sets an explicit ambition: by 1 January 2030, Member States should ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation, where medically feasible.
The draft also frames rare diseases as more than a clinical challenge. It explicitly links rare diseases to socioeconomic burden on families and carers, calling for integrated, multidisciplinary care pathways and stronger attention to inequalities — including gender and caregiving dimensions — so that policy reflects the “full patient journey” rather than isolated clinical episodes.
The ecosystem shift: access, data and cross-border coordination
On treatment access, the draft argues that authorised therapies do not translate into equal availability across Member States, and it calls for reinforced EU-level coordination on evidence evaluation, along with monitoring of inequalities in availability and reimbursement timelines.
It also encourages voluntary cooperation approaches such as joint clinical evidence generation and solidaritybased mechanisms, while keeping the focus on equity across countries. Data and registries are treated as core infrastructure, with the draft calling for full interoperability of rare disease registries and explicitly positions rare disease data within the rollout of the European Health Data Space, including federated approaches and standardised coding, so that realworld evidence can be used more consistently for research and decisionmaking across borders.
What this means for ERDERA — and what happens next
For patients and families, the draft report’s core message is that rare diseases need an integrated, endtoend approach: diagnosis, access to treatment, research and innovation, interoperable data infrastructure, and patient support should move together, backed by governance, monitoring and measurable targets.
That framing directly echoes what the European Rare Disease Research Alliance (ERDERA) was built to do in practice: connect funding, data and digital services, clinical research capacity, and international alignment — so knowledge and effort are not duplicated, and underrepresented regions are better supported. The Parliament’s February 2026 evidence base (EPRS) uses the same sixtheme structure and explicitly notes that EU-level collaboration — including ERDERA — is already helping to build capacity, while gaps persist, strengthening the case for approaches that link research, data and implementation rather than treating them as separate lanes.
EURORDIS’ response to the draft reinforces the patient-centred implication: policy must reflect realities beyond medicine — on families, work, education and social life — and embed structured patient involvement in governance and evaluation, aligning with ERDERA’s focus on patient benefit as the end point of system-building.
What’s next
This remains a draft legislative owninitiative report (INL): it is the mechanism Parliament uses to formally request that the European Commission submit legislative proposals where EU action is considered necessary.
The immediate steps are therefore institutional — the file progresses through the Parliament’s process, after which any move towards a binding EU framework depends on how the Commission and Member States respond to the request.
In terms of next steps, the draft points to a shift from general calls for “more action” to more concrete questions: what indicators and targets should be used to reduce diagnostic delay; how registry interoperability and EHDS alignment should be operationalised; and how progress is reported and compared over time.
It also follows a consultation that gathered views from more than 4 000 participants, signalling broad public support and setting expectations that next steps will be assessed against measurable improvements rather than goodwill alone.
