If you’re reading this, you probably already know the sobering scale of the challenge posed by rare diseases. Approximately 30 million people1 in Europe live with a rare disease. Despite progress, it still takes four to eight years to receive an accurate diagnosis2 and 95% of rare diseases remain without treatment options3.
The Rare Diseases Forum 2026 comes at a crucial time for EU health policy: register and join the conversation
Europe is at an inflection point, with two clear reasons to act. First, for patients: rare diseases increasingly affect families within pressurised healthcare systems. Timely access to diagnostics, screening, and breakthrough therapies are essential to improving outcomes. Second, for Europe’s economy: rare disease R&D is a major engine of global biomedical innovation, creating opportunities to fuel scientific advancement, compete for investment and generate high-skilled job growth.
We have seen how a strong European framework can support innovation. Since 2000, the Orphan Medicinal Products Regulation has enabled the development and authorisation of more than 200 orphan drugs4, a pace unlikely if countries worked in isolation. This added between 210,000 and 440,000 of Quality Adjusted Life Years for patients in the EU5, meaning more time living well.
Europe is facing growing challenges in translating innovation into timely patient access
The proposed Biotech Act is a welcome reinforcement of Europe’s commitment to biotechnology, which could make the EU more attractive to cutting-edge scientific research by providing faster regulatory and clinical trials pathways and additional incentives.
However, Europe is facing growing challenges in translating innovation into timely patient access. Fewer orphan medicines are reaching Europe compared to the US, and those that are often encounter complex, fragmented access pathways6. As the Office of Health Economics highlighted, EU regulatory incentives for orphan R&D are not always matched by national HTA and reimbursement processes7.
Europe has proved it can stimulate rare disease innovation; the next test is whether it can convert that innovation into equitable access across member states
Unless Europe aligns its ambition for scientific leadership with faster, more predictable access for patients, it risks missing both the health and competitiveness benefits of orphandrug innovation. To stay at the forefront, frameworks must enable both innovation and access for patients. That means aligning incentives with modern HTA approaches, enabling faster and more consistent reimbursement pathways, and backing the diagnostic infrastructure that gets patients to the right care sooner.
As an innovative company dedicated to delivering breakthroughs in areas of high unmet need, Biogen has pioneered first-in-class treatments for Spinal Muscular Atrophy (SMA), Friedreich Ataxia (FA), and a genetic form of Amyotrophic Lateral Sclerosis (ALS). Along with other stakeholders in the ecosystem, we are committed to playing our part in getting it right in Europe.
Europe has proved it can stimulate rare disease innovation; the next test is whether it can convert that innovation into equitable access across member states. To lead in biotech and deliver for families living with rare diseases, the time is now to make room for breakthroughs in rare.
List of references
- eurordis.org/information-support/what-is-a-rare-disease/
- Wang, X., et al. (2024). The landscape for rare diseases in 2024. The Lancet Global Health, 12(3), e341. https://doi.org/10.1016/S2214-109X(24)00056-1
- Global rare‑disease policy shifts: what are the implications for equitable access to orphan medicines? - OHE
- https://www.ema.europa.eu/en/documents/other/orphan-medicines-figures-2000-2025_en.pdf
- https://health.ec.europa.eu/document/download/386fb8fa-7633-482b-bd1b-5d2a492c9be7_en?filename=orphan-regulation_eval_swd_2020-163_part-1.pdf
- ISPOR Europe 2025 orphan approvals comparison [ispor.org] ; Value in Health / ISPOR HTA timelines [valueinhea...ournal.com] ; Office of Health Economics rare disease access analysis [pharmaceut...nology.com]
- Global rare‑disease policy shifts: what are the implications for equitable access to orphan medicines? - OHE
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