Access to treatment for rare diseases still depends heavily on where you live. While patients in larger EU Member States receive new medicines within a year or two, others may wait up to four years – if they get them at all. The European Parliament now wants to turn solidarity rhetoric into a concrete Action Plan with measurable benchmarks.
Rare diseases, defined as affecting fewer than 1 in 2,000 people, impact around 36 million Europeans. Between 6,000 and 8,000 such conditions exist. Approximately 95% still lack an approved therapy.
The European Added Value Assessment presented to the Parliament’s SANT Committee on Wednesday concludes that while health systems remain a national competence, EU coordination can reduce fragmentation, pool scarce expertise, and address cross-border inefficiencies that Member States cannot resolve alone.
The study warns that “the costs of non-action could be high, including missed health gains, widening inequalities and duplication of effort”.
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“This own-initiative report is not legislation,” MEP Tomislav Sokol (EPP, Croatia) said at a rare disease event convened by the European Brain Council. “But we will use it (…) to advocate for the development of the actual strategy.”
“First we will do the own-initiative report and then we use this as a pressure on the Commission to come up with a rare disease strategy or a plan.”
He insisted that any EU strategy must include “critical objectives, critical measures, critical benchmarks, measurable results.”
From projects to system design
Europe is attempting to move rare disease innovation from a patchwork approach to a system-based one.
The Biotech Act is framed by the Commission as a tool to shorten the path from “research, clinical development and industrial scale-up,” particularly for advanced therapies central to many rare genetic conditions.
First we will do the own-initiative report and then we use this as a pressure on the Commission to come up with a rare disease strategy or a plan. – Tomislav Sokol
In parallel, the European Rare Diseases Research Alliance (ERDERA) marks a shift from fragmented projects to permanent infrastructure. “Our goal is really to consolidate the rare disease research ecosystem,” said Daria Julkowska, Scientific Coordinator at ERDERA.
By integrating funding, regulatory support, data platforms and business acceleration, ERDERA embeds translational thinking from the outset. Its governance model is public–public–private, aligning Member States, Commission services, academia, industry and patient organisations in a pre-competitive space.
The architecture is tightening. Whether financing and political coordination match that maturity remains uncertain.
The Commission’s approach: integration over escalation
The Commission resists the narrative that nothing is happening.
Officials point to the Life Sciences Strategy as an umbrella framework integrating the Biotech Act, clinical trials reform, support for advanced therapy medicinal products, Horizon Europe funding, EU4Health, and innovation procurement.
“We are not waiting for an action plan to happen to act,” said Hélène Le Borgne, Policy Officer at the Commission’s Directorate-General for Research and Innovation.
Rather than launching a flagship strategy, the Commission favours aligning existing instruments and strengthening coordination across DG SANTE and DG RTD.
The divergence is subtle but real: Parliament seeks a benchmarked strategy; the Commission prefers incremental consolidation.
Geography still determines access
Europe’s rare disease challenge is no longer primarily scientific. It is territorial.
“In the largest Member States… patients access most newly approved medicines within one or maximum two years,” MEP Sokol said. “While in some countries in Central and Eastern Europe, this can be up to three or four years. Which is completely unacceptable.”
The gap reflects disparities in purchasing power, administrative capacity and negotiating leverage.
Cross-border healthcare, theoretically designed to mitigate such inequality, remains difficult to navigate.
“We have a legal framework (…) but they are so complicated (…) that you have to be an expert in health law to know what your rights are,” he said. “So this is unacceptable.”
For smaller Member States, structural limitations are even sharper.
“I come from a small country (…) a lot of rare diseases cannot be treated for lack of medical capacity.”
Without organised EU referral pathways and coordinated purchasing mechanisms, access remains determined by geography rather than need.
Parliament versus Council: the medicines divide
Tensions are equally visible in pharmaceutical reform.
“In terms of quality of access… the end result, for me, is not satisfactory,” Sokol said, referring to negotiations with the Council.
He argues that access provisions were diluted during trilogue talks.
Put pressure on the Member States. – Tomislav Sokol
Parliament is now attempting to rebalance through the Critical Medicines Act, pushing to include all orphan drugs within its scope and to strengthen joint procurement mechanisms.
Pooling demand, he argues, would improve bargaining power for smaller Member States and accelerate access. Parliament also supports procurement criteria that move beyond lowest-price logic and reward European production.
The decisive battle: the EU budget
The sharpest fault line lies in the next Multiannual Financial Framework.
Under current proposals, health does not have a clearly ring-fenced funding envelope. For Parliament’s rare disease push, that omission is not technical, it is structural.
When it comes to securing dedicated EU health funding, Sokol was explicit: “The biggest obstacle will be the Member States.”
In particular, he pointed to larger countries reluctant to expand redistribution at EU level – a reluctance that, in his view, undermines solidarity in precisely the area where EU coordination is most justified.
Without earmarked funding, European Reference Networks, centres of excellence and research alliances risk remaining structurally advanced but financially fragile.
For Sokol, the next phase is no longer about drafting strategies, it is about political mobilisation.
“Put pressure on the Member States,” he urged.
That means engaging national governments, finance ministries and permanent representations. It means building cross-party alliances beyond health committees. It means raising the visibility of rare diseases in capitals, not only in Brussels.
Parliament can pass reports. The Commission can layer instruments. But if Member States refuse to commit funding, the architecture will stall.
Rare diseases, Sokol argued, are the clearest case where the European Union must act because individual countries cannot solve the problem alone.
The test now is whether governments are prepared to translate solidarity into budgetary commitment.
