Minister Ashley Dalton, Parliamentary Under Secretary of State for Health and Social Care, spoke to attendees at the Rare Disease Day Westminster reception yesterday, providing a preview of the 2025 England Rare Diseases Action Plan, launched today. In her speech the minister praised Genetic Alliance UK and our members for the role we’ve taken to inform this plan. So what’s new?
NHS England has created a model to incentivise clinics for rare conditions that involve multiple healthcare professionals together in the same session. This should help reduce the number of clinics people living with rare conditions have to attend, and improve the coordination of care between different parts of the health service. Our community have been calling for this type of coordination, so the removal of NHS financial barriers to achieving this is welcome.
The action plan sets out how the clinical trial regulator (Medicines and Healthcare Products Regulatory Agency) is planning to facilitate clinical trials in the UK, including for rare conditions and into innovative technologies like gene and cell therapies, while maintaining the safety of participants. Having clinical trials in the UK benefits us by keeping the best doctors here and offering possible innovation to people with unmet need. The flexibility being introduced should make it easier to run trials for small populations in the UK and in collaboration with other countries.
Antisense-oligonucleotides (ASOs) are a way to ‘silence’ genes where their activity is causing a rare condition. This is cutting-edge innovation, where medicines would be bespoke to individuals. In the third new action of this plan, NHS England is committing to develop a framework on how to deliver these treatments – including how to make funding decisions on these treatments. This would mean that innovation generated by initiatives like the UK Platform for Nucleic Acid Therapies (UPNAT) and the Rare Therapies Launchpad at Genomics England, would have a pathway to people that need the treatments.
NHS England has been building on learning from the pilot of a SWAN (syndromes without a name) clinic in Wales to plan for a pathway in England. The plan unfortunately announces a pause to the implementation of this because of funds, which will be disappointing to families with children with undiagnosed genetic conditions. We do not want to lose momentum on this work, so we will contact NHS England to understand more. This may provide an opportunity to embed a service to people with undiagnosed conditions more centrally in the medical genomics service in England.
What’s next?
There are many other ongoing actions in the Action Plan, and we have a final year to deliver the UK Rare Diseases Framework – but in January 2026, the Framework does come to an end. So we now have one year:
We were delighted that Minister Dalton committed to being part of conversations across the four nations and with all of us as key stakeholders to understand what the next steps should be. Genetic Alliance UK plans to work hard to keep us all involved.
