PSC Support Signs Landmark European Declaration to Accelerate Rare Disease Innovation
PSC Support Signs Landmark European Declaration to Accelerate Rare Disease Innovation
PSC Support Signs Landmark European Declaration to Accelerate Rare Disease Innovation
Joining European Reference Networks and global leaders to transform the rare disease care ecosystem in the UK and EU.
What is the European Innovation and Care Ecosystem Declaration?
The Declaration on the European Innovation and Care Ecosystem for Rare and Complex Diseases is a strategic roadmap developed to address the persistent fragmentation in rare disease diagnosis, care, and research across Europe. It was created in response to urgent unmet needs and is supported by a multi-stakeholder collaboration including European Reference Networks (ERNs).
Why is this declaration important for the PSC community?
For people living with primary sclerosing cholangitis (PSC), this declaration is a vital step toward reducing the average five-year delay in rare disease diagnosis. By fostering scientific excellence and cross-border collaboration, the initiative aims to improve the lives of the 30–36 million people in the EU living with rare conditions, including those in our own community.
How will this affect patients in the UK and the EU?
The declaration calls for a ‘whole-of-society’ approach to overcome information gaps and improve access to innovative medicines, which currently take an average of 578 days to reach patients after approval. While the UK has recently simplified its own regulatory environment, this European alignment is essential for cross-border research and ensuring that patients in both the UK and EU are the first beneficiaries of breakthrough treatments.
Read PSC Support’s Research Strategy
What are the key goals for future rare disease care?
The roadmap prioritises several critical areas for improvement:
- Sustainable Funding: Moving away from short-term, project-based funding toward long-term support for biomedical research.
- Data Harmonisation: Addressing gaps in rare disease registries and improving the sharing of high-quality data across borders.
- Access to Innovation: Streamlining regulatory procedures to prevent the decline of clinical trials within Europe.
- Patient Involvement: Formally including patient representatives and advocates in the decision-making process for therapy development.
Turning Ambition into Action in 2026:
Our Fortnight in Focus
At PSC Support, we want a world without PSC. We work tirelessly behind the scenes to drive research and improve lives. Here is a snapshot of what we've been up to:
Improving Care: We attended a major national clinical conference to equip general gastroenterologists and nurses with specialist knowledge, which helps ensure future work is better aligned with what people with PSC need by improving local care and pathways.
Progressing Research: Our 2024 investment into early-stage research to build necessary scientific evidence enabled Dr James Sun to secure major national funding to explore the genetic drivers of the disease. This foundational knowledge is essential for identifying potential new treatments.
Progressing Research: We supported the development of a research manuscript on nutrition in PSC, so that the findings can be effectively shared with the wider medical community. This will help us advocate for the needs of people with PSC to ensure they get the care they need at the right time.
Improving Care: We hosted a clinical consensus meeting to map out an ideal PSC care pathway, which helps ensure future PSC management is better aligned with what people with PSC need. Establishing standard protocols across the NHS is essential for eliminating regional variations in care.
Progressing Research: We contributed to the strategic planning of an international rare liver disease network, which supports progress towards better care for people with PSC. Broadening our influence ensures patient needs are integrated into wider clinical strategies.
Improving Care: We shared firsthand experiences of living with PSC at an international medical school event, which improves clinical understanding of the disease for healthcare professionals. This is important because ensuring future specialists understand the human impact of PSC helps drive patient-centered care and clinical excellence.
Progressing Research: After just over 48 hours on our Big Give Small Charity Week match funding initiative, your donations helped us get to 78% of the way to our £20,000 goal! Thank you! Can we reach 100%?