* This post is written by a guest blogger - it is not sponsored. *
Living with a rare disease? We need your help!
In 2015, the UK health agency NICE was asked to fund a new drug for children with Duchenne Muscular Dystrophy. Parents waited over two years for a decision to be made. But in that time, their children stopped walking, meaning the drug would no longer help.
This is happening all over the world. Forcing patients and their families onto an emotional rollercoaster leading to preventable heartache. Because of this, reforms are urgently needed.
Each person’s experience of disease is unique. Yet national health agencies often don’t have the right data and tools to assess whether drugs for rare diseases are worth funding. Until now…