The Government published England’s fifth rare diseases action plan on gov.uk on 27 February, ahead of international Rare Disease Day on the 28th. This Government remain committed to improving the lives of people living with rare diseases, and today’s action plan provides more detail on the steps we will be taking over the next year to meet our priorities.
The UK rare diseases framework was published in January 2021 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 co-ordination of care, and improving access to specialist care, treatment and drugs.
The 2026 action plan updates on progress we have made across the system—in the NHS, in health education, in regulation—to improve the lives of people living with rare disease:
The Medicines and Healthcare products Regulatory Agency has set out a position paper with commitments for major reform in regulation of rare therapies.
NHS England is developing a framework for individualised genetic therapies—designed for a specific patient—to lay the way for future commissioning decisions, and we have seen the treatment of the first patient with an individualised therapy in the NHS.
And new funding has been allocated to pilot two centres for people with undiagnosed rare conditions.
A central principle of our approach to rare disease policy is the prioritisation of lived experience. The sustained advocacy and specialist insight of patients, families, and patient organisations have significantly contributed to greater awareness and continued progress in the field. Following the extension of the UK rare diseases framework into 2027, across the coming year we will be continuing to deliver the England rare diseases action plan and engaging with the rare disease community to inform future policy options.
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