As Europe seeks to strengthen its competitiveness in biotechnology, rare disease innovation will be one of the clearest indicators of success. Rare disease research is redefining what is possible. For the 30 million people in Europe living with rare disease, the question is no longer whether scientific breakthroughs will happen, but how quickly those breakthroughs can reach them and their families.
At Ultragenyx, we focus exclusively on developing medicines for rare and ultra-rare diseases. Our mission is to bring transformative therapies to patients who often have no other options. This ambition is increasingly within reach.
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For biotech companies, investment in rare disease discovery is predicated on a on a time and cost-effective environment for clinical development. Rare disease research involves small patient populations, specialised expertise and urgent medical need. Yet clinical trial and regulatory pathways remain largely designed for medicines targeting much larger populations.
As Europe seeks to strengthen its competitiveness in biotechnology, rare disease innovation will be one of the clearest indicators of success
Strategic amendments to clinical trials regulation through the EU Biotech Act could make a meaningfully difference. Strengthening the role of the Reporting Member State and moving towards a single, harmonised ethics assessment across all Member States will make Europe a more attractive location for research and development.
Accelerating development alone is not enough. The real measure of success is whether diagnosed patients can access treatment in time.
Patients in Europe wait on average six years for a diagnosis and many are misdiagnosed along the way. For progressive paediatric rare diseases, delayed diagnosis can mean children are treated after irreversible damage has occurred. In ultra-rare conditions like Sanfilippo Syndrome, newborn screening can mean the difference between being able to walk and talk or not. Newborn screening is therefore critical.
Yet it can take to six years for Member States to introduce new diseases to the screening programme—in some countries screening may never be implemented. Member State cooperation through Joint Action and Council Conclusions can facilitate faster and broader availability to newborn screening to help prevent a European postcode lottery for families.
For advanced therapies such as gene therapies, additional national requirements and infrastructure needs further slows access
The same fragmentation affects access to treatment. Biotech companies must navigate 27 separate access and reimbursement processes and negotiations. For advanced therapies such as gene therapies, additional national requirements and infrastructure needs further slows access. When it’s for a handful of patients, it can lead to significant inequalities in timely access.
Building on the foundations of cross border healthcare, joint health economic assessment and proposals for collaborative procurement, the EU can streamline access. But to equalise it, more is needed. European Guarantee Fund introduced with the next Multiannual Financial Framework could enable Member States to manage the financial uncertainty of introducing transformative therapies for very small patient populations, ultimately unlocking access equity across Europe.
If Europe is serious about biotech competitiveness, it must ensure that scientific leadership translates into timely access. For families living with rare diseases, speed is not a choice, it shapes whether a child has the chance to grow, learn, and live independently.
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