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:
Organisational excellence: We simplified our personal data consent process and streamlined how we record patient stories to ensure that the lived experiences of our community are captured accurately and securely to inform future work.
Progressing Research: We coordinated a series of upcoming meetings with international researchers and pharmaceutical companies, which supports better informed decisions for people with PSC, research, or care. This groundwork helps keep PSC research focused on the primary needs of the patient community.
Progressing Research: We reviewed and contributed to scientific abstracts and posters for the upcoming EASL international liver congress, which strengthens the quality of future research for people with PSC, research, or care.
Progressing Research: We engaged with the MHRA to prepare for the upcoming national consultation on rare disease therapies, representing PSC patients in the wider community. Maintaining this direct dialogue ensures our organisation is prepared to respond formally and protect the interests of those living with PSC
Improving Care: We presented about what it is really like to live with PSC to gastroenterologists This first-hand insight equips healthcare professionals to better understand the day-to-day challenges of PSC.
Income Generation: We secured a place in the prestigious Big Give Small Charity Week match-funding campaign, so that we can maximise future donations from our supporters as every donation between 22 and 29 June will be doubled!
Progressing Research: We recruiting for a research and advocacy officer to expand our core team. This additional capacity ensures we can better support scientific studies and represent the patient voice at every level.