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 led an international discussion on removing barriers that delay research, ensuring that drug development is built around the practical realities of living with PSC.
Building Partnerships: We joined an AMMF webinar to share specialist transplant knowledge with healthcare professionals to help ensure that clinicians are aware of evolving care options and can better support patients facing transplant and CCA.
Building Partnerships: We presented the latest the PSC Working Group's achievements at the European Reference Network meeting of liver experts to drive collaboration and improve clinical standards worldwide.
Empowering our Community: We signed an international declaration advocating for rare disease research to be a priority in health policy, ensuring long-term funding for PSC.
Improving Care: We initiated collaborations with clinical experts to streamline specialist referral pathways, ensuring patients get the right care at the right time.
Organisational Excellence: We refined our strategic objectives to ensure every penny donated is focused on the most impactful work for our community.
Organisational Excellence: We met with our Board of Trustees to report on work so far this year to ensure the charity continues to meet the highest standards of governance and ensure we are delivering the maximum benefit for people with PSC.
Progressing Research: There are now 55 volunteers in our Patient Panel! The ensures that a diverse range of lived experiences directly shapes future PSC research and care.
Progressing Research: We reviewed research summaries for a pharmaceutical partner to ensure that study results are communicated clearly and transparently to our community.
Progressing Research: We provided patient leadership on a major new steering committee to ensure research into liver complications remains grounded in patient needs.
Progressing Research: The UK PSC Care Guidelines are going to be updated! We joined the national steering committee to update them, ensuring the "rulebook" for how doctors treat PSC is co-written by the patients who live with it.