Engagement and priorities
Engagement on the content of the framework included the National Conversation on Rare Diseases survey in October 2019, which Muscular Dystrophy UK participated in, and further clinical and patient organisation input.
The framework focuses on four key priorities:
- helping patients to get a final diagnosis faster
- increasing awareness of rare diseases among healthcare professionals
- better coordination of care
- improving access to specialist care, treatments and drugs
Action plans
The framework will be followed by action plans by each of the four UK nations which will provide more detail about the steps which each nation will take to implement the priorities.
Each of the action plans will be developed in close collaboration with the rare diseases community and reviewed every one to two years.
Muscular Dystrophy UK will take an active role in all four of the action plans to ensure that there is sufficient focus on the priorities for people living with muscle-wasting conditions.
Rob Burley, Director of Campaigns, Care and Support at Muscular Dystrophy UK, said:
We welcome the publication of the UK Rare Diseases Framework. The priorities which have been identified closely match Muscular Dystrophy UK’s ongoing work to improve access to specialist neuromuscular care and faster access to treatments.
While there has been progress in specialist care provision and approval for ground-breaking new treatments in recent years, we know that there are still many improvements required, and alongside people living with muscle-wasting conditions we look forward to playing our part in securing them.