Europe’s competitiveness in life sciences innovation, investment and patient access is at a decisive moment. Nowhere is this more urgent — or more promising — than in rare diseases.
Advances in genetics, advanced therapies and data are unlocking scientific breakthroughs once thought impossible. If we align these advances with the EU’s important focus on regulatory simplification, industrial resilience and economic growth, Europe can secure its place as a global leader in rare disease research and development.
36 million Europeans are affected by rare diseases, less than 10% have an approved treatment. Rare disease research and development is inherently global. Patient populations are small and dispersed, as are scientific and clinical experts.
That’s why Ultragenyx has built a strong and lasting presence in Europe We invest in local R&D, support European trial infrastructure, and work closely with clinical and patient communities across the Union. We are proud to have over a third of our clinical trial patients in Europe.
The EU Biotech Act can make Europe more competitive in clinical trials with a rare disease 30-day fast track for trial approvals
As European policymakers you have a rare opportunity to truly enhance the environment for rare disease innovation. The EU Biotech Act can make Europe more competitive in clinical trials with a rare disease 30-day fast track for trial approvals. The Multiannual Financial Framework can boost research and patient access by further investing in disease registries and cross-border data access. The Life Science Strategy and Horizon programme can support equity of access by harmonising national newborn screening panels with a platform for mutual recognition pilots in ultra-rare diseases. The Critical Medicines Act needs to support European wide access to treatments for ultra rare diseases, including simplifying travel between countries to specialist centres.
The General Pharmaceutical Legislation must maintain existing incentives for orphan medicinal products, especially those with the high unmet medical need. Most importantly, all these initiatives must come together in a cohesive and future-proof EU ecosystem.
Ultragenyx was founded to advance innovative medicines for rare and ultra-rare diseases that have never been treated before. We do this by examining problems from new angles and move therapy development forward at pace to lead to much-needed breakthroughs. We urge EU policymakers to share this boldness.
The regulatory path for new rare disease treatments is often uncharted. Rare disease treatments like gene therapies are expected to follow reimbursement and value assessment pathways designed for small molecules and larger patient populations. We can overcome these hurdles when there is a willingness by regulators and health systems to engage with innovators, patients and caregivers, and share learning from other countries within and beyond Europe.
The European Parliament has already shown leadership by calling for a Rare Disease Action Plan. We call on the Commission, the Council and the Parliament to use the legislative and policy processes of this mandate to consolidate and enhance the framework for orphan medicinal products— ensuring regulatory pathways, investment incentives, and research funding ultimately leads to faster and broader access to new treatments for European patients.
For them, time matters: the right policies can accelerate the delivery of cutting-edge science to patients, or it’s patients who fall behind if development slows and innovation shifts elsewhere.
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