Scottish Government Updates On Access To Nusinersen For Those With SMA Types 2 And 3

02 July 2019

On 12th February 2019, Biogen advised the community that the Scottish Medicines Consortium (SMC) had granted nusinersen ultra-orphan designation. This allowed the treatment to be reviewed for those with SMA Types 2 and 3 through its new ultra-orphan appraisal process.

Following recent communication from the Scottish Government, the Scottish Medicines Consortium (SMC) webpage for nusinersen has been updated to advise that, as well as being available for those with SMA Type 1, from July 2019 nusinersen can be prescribed for those with SMA Types 2 and 3 under the ultra-orphan pathway for a period of up to three years while further evidence on its effectiveness is generated.  

You can read the update here.

Dr Catherine Calderwood, Scotland's Chief Medical Officer, explains more in this article in the Scotsman here.

We note that in this article it states, “Doctors will adopt a phased approach, starting with children before moving to adults. This phasing means that services can be safely configured to support the care associated with administering this medicine”.

If you haven’t already made contact and are an adult or family with a child who wishes to be considered for access, or if you have previously asked for treatment via the Peer Approved Clinical System (PACS) Tier One system and been turned down, please contact your clinician to see if access may be possible for you at some point. 

You can read their guidance about the ultra orphan pathway on the SMC website here. Nusinersen is now moving into Stage 3 Evidence Generation.