Last updated 19th January 2018.
Access to nusinersen in the UK is still only possible for children with SMA Type 1, through what is called an Expanded Access Programme (EAP). For more up to date information on this, please go here.
Nusinersen’s future availability in the UK will depend upon the outcome of reviews by regulatory authorities of the evidence gained from clinical trials in each subtype of SMA. These authorities also review the costs of provision and consider submissions from the patient community. The authorities include the National Institute for Health and Care Excellence (NICE), NHS England, the Scottish Medicines Consortium (SMC) and other authorities in the devolved nations.
On 18th January 2018, the National Institute for Health and Care Excellence (NICE) started its appraisal of nusinersen for the treatment of all types of SMA. It is now looking at the treatment via the Single Technology Appraisal (STA) route. SMA Support UK, MDUK, the SMA Trust and TreatSMA have been invited to make Patient Group submissions by 15th March 2018.
An STA is designed to assess drugs for both clinical and cost-effectiveness and is usually used for more common conditions. As such, we are concerned that this route is not set up to assess rare disease drugs, like nusinersen.
However, there is some promising news. Biogen, has announced that they are in discussions with NICE and NHS England to put an interim access scheme in place. This scheme, known as a Managed Access Agreement (MAA), could give others with SMA access to the treatment while further data and evidence are gathered over several years.
Time is key and it is crucial nusinersen is not subjected to any delays during the NICE assessment process. Please help us push for the MAA to be implemented as soon as possible by writing to your MP and asking for their support. Find out more about how to do this here.
You can read more about the NICE process and what has happened thus far here.
As soon as we know more about the next steps in the appraisal we will let you know. You can keep up to date by signing up for our monthly E-newsletter.
The SMC has now announced that it will be assessing nusinersen for: "pre-symptomatic infantile-onset and later-onset 5q SMA, but not for before birth or Adult Onset SMA". This means they will be considering the case for SMA Types 1, 2, and 3 only. SMA Support UK, Muscular Dystrophy UK and the SMA Trust have each been invited to provide a Patient Group Submission by 5th February 2018. To find out what we are doing to prepare for our submission, please go here.
- Wales and Northern Ireland
These countries will often follow the guidance issued by NICE but could follow Scotland. As soon as we have any news we will post it here and in our monthly E-newsletter.