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.

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 the action plans: England / Scotland / Wales / Northern Ireland. 

We’re facing three challenges:

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