Thirty clinicians involved in the care of children and adults with spinal muscular atrophy (SMA) have signed an open letter calling for NICE to approve Spinraza.
NICE is due to meet on Wednesday 6 March to consider the treatment, ahead of making a final decision. In August it said it was minded not to approve the drug, despite recognising its benefits in treating infants with the condition.
The letter, which has been published by The Guardian, highlights the clinicians’ frustration at a medicine that can help patients not being available on the NHS in England, Wales and Northern Ireland.
The signatories – including consultants, physiotherapists and nurses – join Muscular Dystrophy UK and Spinal Muscular Atrophy UK in asking NICE to show flexibility. They also call for a change to the appraisal process.
You can read the letter in full here.
What does Muscular Dystrophy UK say?
Muscular Dystrophy UK is calling for NICE to approve the treatment for use on the NHS. Every day counts for families affected by the condition, and continued delays will cost lives.
Rob Burley, Director of Campaigns, Care and Support at Muscular Dystrophy UK, says:
Spinraza offers families hope, but continued delays in making it available are causing heartbreak. It is unthinkable that a child who is now diagnosed with the condition cannot access this treatment in England, yet could if they lived across the border in Scotland. We have seen how effective Spinraza can be in treating people with spinal muscular atrophy, and it is families who suffer when delays happen.
Time is of the essence, and without access to Spinraza babies with the most serious form of the condition are likely to die, while children with other types of spinal muscular atrophy may irreversibly lose the ability to walk, crawl and swallow. Now more than ever, we need to see urgent action from NICE, Biogen and NHS England, who must sit down together to find a solution before any more lives are cut short.
- If you would like information or support, please call our Information and Support Team on our free helpline: 0800 652 6352 (Mon-Fri, 8.30am-6pm) or email us at: email@example.com
- To find out how you can join our campaign, email Clare Lucas at: firstname.lastname@example.org. And if you would like to share your story, email Laura Burge at: email@example.com
- You can also find our factsheet on SMA here and our FAQs on Spinraza here.