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:
Building Partnerships: We contributed a comprehensive chapter to a new medical textbook for nurses and allied health professionals, ensuring that the patient perspective is integrated into clinical education.
Building Partnerships: We participated in a planning meeting for the update of national clinical guidelines for PSC.
Building Partnerships: We prepared strategic questions for an upcoming international forum titled “Putting Patients First: From Research to Treatment.”
Organisational Excellence: We updated our web details to make it less likely information from us sent by email ends up in your spam folders.
Progressing Research: We are collaborating with European partners to develop a simplified research registry for pregnancy outcomes, building the evidence base needed to improve medical guidance for women with PSC.
Progressing Research: We completed a formal submission to the Scottish Medicines Consortium to advocate for the value of a potential new treatment for PSC patients.
Progressing Research: We reviewed the participant information for a new academic clinical trial to ensure it is clear and accessible for patients.