2nd August 2023:

The National Institute for Health and Care Excellence (NICE) has been asked to conduct an evaluation of Nusinersen and Risdiplam for treating spinal muscular atrophy. 

Both of these disease-modifying treatments have been funded by the NHS via time limited Managed Access Agreements. NICE intends to assess these drugs together through its Multiple Technology Appraisal (MTA) process.   

As one of the organisations with an interest in this evaluation, SMA UK have been invited take part in a ‘draft scope’. We have reviewed background information that was provided to all parties involved in the assessment to ensure it is correct and detailed enough from the community’s perspective.  

SMA UK and Muscular Dystrophy UK responded jointly.

SMA UK will join other UK SMA patient group to bring the voice of the community to the NICE committee meeting next year. We will be speaking up about the experiences and outcomes reported by people in the UK SMA Community who have been receiving one of these treatments and the importance of any individual being able to work with their clinical team and choose the disease-modifying treatment that best suits their needs.

Once NICE has all the scoping responses it will review what questions it will be asking at the Multiple Technology Appraisal (MTA) for the two treatments. When these are these confirmed there will be an invitation from NICE to make submission. At this point we will be contacting the community to make sure we reflect your views.