A chance for the UK to lead the way in rare disease care
I Am Number 17 Changemakers at No.10
Şeyda Atadan Memiş, General Manager UK and Ireland, Takeda
Without a renewed commitment to the UK Rare Diseases Framework, progress for patients is at risk. A dedicated strategy is essential to drive improvements for those living with a rare disease, their families and carers
This content has been initiated and funded by Takeda
Although rare diseases are individually rare, they are collectively common – affecting 1 in 17 people at some point in their lifetime. In the UK, that’s over 3.5 million people living with conditions that can be both life limiting and life threatening.1,2
For people with rare diseases, the care they receive can depend on where they live and which specialists they can access. This postcode lottery can be the difference between a timely diagnosis and treatment, or years of a diagnostic odyssey, fragmented care, and missed opportunities for life-changing therapies.3
The I am Number 17 campaign amplifies the voices of those with a rare disease, increasing understanding of the lived experience. Initiated and funded by Takeda, the campaign was created together with individuals living with a rare disease, carers, and 13 rare disease patient organisations.
This spirit of collaboration aligns closely with the UK Rare Diseases Framework, which, since its launch in 2021, has advanced progress by improving collaboration, accountability, and action across the four nations. However, with the framework set to expire in 2026, there is a risk momentum will stall. Without a strategy, challenges including slow diagnosis and unequal access to treatment could deepen.4
The government’s 10 Year Health Plan for England sets out welcome ambitions to tackle long-term NHS challenges, but it cannot replace a dedicated rare disease strategy. Targeted focus is needed to ensure the complex, multi-specialist care that rare disease patients require is not overlooked within broader system priorities, risking wider health inequalities. This is particularly pertinent given upcoming structural reforms to the health service, with an urgent need to design future services with continued patient input.
A renewed UK-wide rare disease strategy would not only help reduce these care gaps; it can also strengthen the UK’s ambitions in life sciences and personalised medicine. Many breakthroughs in these fields begin in rare disease research, where advances for rare conditions often create benefits for many more patients.
Earlier this year, I had the privilege of joining some of the I am Number 17 Changemakers – individuals with lived experience of a rare disease – to deliver a letter to the Prime Minister, urging the UK government and devolved administrations to re-commit to a UK-wide Rare Diseases Framework beyond 2026.
This call to action underscores a vital truth: the inequities faced by the rare disease community are not inevitable and can be fixed through policy action. Now is the time to build on progress, address ongoing challenges, and ensure no one living with a rare disease is left behind through an ambitious successor to the Rare Diseases Framework.
“The voices of the rare disease community must not be allowed to fade into the background, and I’m honoured to advocate for this through my work with the APPG on Genetic, Rare and Undiagnosed Conditions. Renewing the UK Rare Diseases Framework beyond 2026 is vital to build on progress and ensure every patient receives the timely diagnosis and care they deserve. I’m proud to support the I am Number 17 campaign, which has played an important role in bringing the lived experience of patients to the forefront.”
Peter Dowd MP, Chair of the Genetic, Rare and Undiagnosed Conditions APPG
References
1. Genetic Alliance UK, Facts and figures. Last accessed September 2025, https:// geneticalliance.org.uk/campaigns-and-research/facts-and-figures/
2. UK Government, The UK Rare Diseases Framework (2021). Last accessed September 2025, https://www.gov.uk/government/publications/uk-rare-diseases-framework/the-uk-rare-diseases-framework#:~:text=Rare%20diseases%20can%20be%20both,die%20before%20their%20fifth%20birthday be%20both,die%20before%20their%20fifth%20birthday
3. Specialised Healthcare Alliance. Rare diseases, common inequalities: Bringing rare diseases into the health inequalities agenda (2023). Last accessed September 2025 https://shca.info/wp-content/uploads/2023/10/SHCA-Health-Inequalities-Report.pdf
4. UK Government, England Rare Diseases Action Plan 2024. Last accessed September 2025, https:// www.gov.uk/government/publications/england-rare-diseases-action-plan-2024
C-ANPROM/GB/RDG/0317. September 2025
