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 disease Hub

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 Rare Diseases Framework has been published, a national vision of how the UK will improve the lives of those living with rare diseases. Industry wants to help fulfil its important priorities. Read the ABPI statement.

We’re facing three challenges:

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