Rare Diseases

The UK Rare Diseases Framework was published following the National Conversation on Rare Diseases, which received nearly 6,300 responses. This helped identify the four priorities of the framework in tackling rare diseases: helping patients get a final diagnosis faster; increasing awareness of rare diseases among healthcare professionals; better coordination of care; and improving access to specialist care, treatment, and drugs. We have commissioned a portfolio level evaluation of England’s rare diseases action plans with input from the rare disease community on the design of metrics, which is due to complete in 2026.

The Government remains committed to improving the lives of those living with rare conditions and will be publishing the next England Rare Diseases Action Plan for rare disease day in 2026, as in previous years. We recognise that despite the progress that has been made there remains considerable unmet need for people living with rare conditions. We are carefully considering the future of the UK Rare Disease Framework and will be announcing our intentions at a later date.