Stop cost being a barrier to access for life changing treatments so that children and young adults aren't dying from treatable ultra-rare conditions.
Why is this important?
Children and young adults with ultra-rare conditions including Morquio and rare cancers are being discriminated against as NHS England and NICE have deemed the cost of their life changing drugs too expensive to approve.
No one chooses to be born with health conditions and through no fault of their own drugs for ultra-rare condition are more expensive.
The UK has led the world to develop new therapies and yet England is going to desert patients due to changes in decision making processes. Ultra rare shouldn't mean forgotten so please help us to keep our commitment to these patients.
Please sign if you agree that no one's life is less important and access to treatment should be about fairness rather than luck as to whether your condition is common or rare or cheap to fund or expensive to fund.