Axes Health at the Rare Diseases Forum 2026: Innovation alone is not enough

24/4/26, 10:00

On 21 April 2026, the Axes Health team joined policymakers, patient advocates, industry leaders and clinicians at the first-ever Rare Diseases Forum, hosted by The Parliament Magazine at TownHall Europe in Brussels.

The forum brought together a broad coalition of stakeholders — from MEPs and European Commission officials to EURORDIS, pharma executives and rare disease patient representatives — all united around a pressing question: what policy levers must Europe activate now to ensure no patient with a rare disease is left behind?

Innovation without access is a failure

The opening message of the day set the tone clearly: scientific progress means nothing if it cannot reach the patient. Across sessions, speakers returned to the same uncomfortable truth — Europe has made remarkable strides in rare disease research and drug development, yet access to approved treatments remains deeply unequal across Member States. An innovative medicine that sits beyond a patient's reach is not a solution. It is a missed opportunity, and for many, a life not lived fully.

Predictable and viable pathways for all stakeholders

A recurring theme throughout the day was the need for access pathways that work — not just on paper, but in practice. For patients, this means knowing that a treatment pathway exists and that they can navigate it. For payers, it means having robust frameworks for value assessment and budget planning. For industry, it means regulatory and reimbursement processes that are transparent and consistent enough to justify the investment that rare disease R&D requires.

The ongoing implementation of the EU Joint Clinical Assessment (JCA) under the HTA Regulation was discussed at length. While the JCA holds real promise for reducing duplication and supporting more consistent access decisions across Europe, speakers were candid about the challenges ahead — particularly for rare diseases, where evidence is often limited by definition. Getting the JCA right for rare diseases will require flexibility, meaningful patient involvement, and a genuine commitment from Member States to follow through on its outputs.

Early access: where urgency meets equity

Perhaps the most resonant theme of the day was the role of early access schemes. For patients with a high medical need and few or no alternatives, the time between regulatory approval and national reimbursement can stretch into years. That gap is not an administrative inconvenience — it is a clinical and human reality.

The forum was unambiguous: fair and equitable early access mechanisms are not optional add-ons to a well-functioning health system. They are a core component of one.

Belgium's EEFA scheme: a model to be proud of

This is a moment for Belgium to reflect with some pride. The EEFA scheme — Belgium's framework for Early and Exceptional access to Medicines — is precisely the kind of mechanism the forum was calling for. It provides a structured, patient-centred pathway for those who cannot wait, offering access to medicines that address an unmet medical need before full reimbursement procedures are completed.

In a landscape where many European countries still lack such a framework, or have one that is inconsistent in its application, Belgium's approach stands out. At Axes Health, we work at the interface of access, evidence, and patient outcomes — and we see firsthand how meaningful timely access can be. The EEFA scheme deserves recognition, continued investment, and where possible, serves as a reference point for others.

Looking ahead

The Rare Diseases Forum made clear that the EU has both the ambition and the tools to do better for the 30 million Europeans living with a rare disease. An EU Action Plan, a strengthened HTA framework, better use of real-world data through the European Health Data Space, and continued support for initiatives like ERDERA and JARDIN all form part of the puzzle.

But policy frameworks only deliver when they translate into real, timely access for real patients. That bridge — between innovation and access — is where Axes Health is committed to working.

We look forward to continuing these conversations and contributing to a healthcare landscape where no patient has to wait too long for a medicine that could change their life.