Rare diseases Rare diseases have a devastating impact on people’s lives: pharmaceutical companies here in the UK and around the world are working to change that – developing new medicines to give families hope for the future.

Rare diseases have a devastating impact on people’s lives: pharmaceutical companies here in the UK and around the world are working to change that – developing new medicines to give families hope for the future.

The UK government and devolved administrations published the UK Rare Diseases Framework in January 2021, setting out a shared vision for addressing health inequalities and improving the lives of people living with rare diseases across the UK.

The framework outlined 4 key national priorities:

  • helping patients get a final diagnosis faster
  • increasing awareness among healthcare professionals
  • better co-ordination of care
  • improving access to specialist care, treatment and drugs

To turn this vision into action, each of the 4 UK nations developed nation-specific action plans detailing how these priorities will be addressed.

Read England's action plan here. 

Scotland's action plan can be found here

The Wales action plan is here

Northern Ireland's action plan is here

We’re facing three challenges:

  1. The awareness challenge
  2. The diagnosis challenge
  3. The evaluation and access to medicines challenge

Last modified: 22 March 2022

Last reviewed: 1 August 2022