Following recent communication from the Scottish Government, the Scottish Medicines Consortium have now provided guidance 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.
Since July 2019, those affected with SMA Type 1 have had access to nusinersen, following the SMC appraisal of the first and only available treatment for SMA.
In October 2018, the SMC introduced the new “ultra-orphan pathway”, which was designed specifically for drugs used to treat rare conditions like SMA.
Recently Dr Catherine Calderwood, Scotland’s Chief Medical Officer. published an article in The Scotsman, which stated the following
We have now implemented a new approach, intended to bring promising new medicines for extremely rare conditions more quickly to patients in Scotland, with an emphasis on finding out how well they work in the Scottish population. Known as the ‘ultra-orphan pathway’, these medicines will be available for three years while data is collected on their effectiveness. The SMC will undertake an initial assessment of the medicine, highlighting the uncertainties about its benefits and any gaps in the evidence. This will inform the collection of clinical data over the three years. In return, pharmaceutical companies must offer a discounted price. After three years, the SMC will review the evidence and make a decision on whether the medicine should continue to be used.
Next week the first medicine to become available through the pathway is for the neurological condition spinal muscular atrophy (SMA). SMA destroys nerve cells, leading to severe disability.
Nusinersen (Spinraza®) was the first treatment to be licensed in Europe for this devastating condition. Last year, Scotland was the first country in the UK to make it available for newborn babies with type 1 SMA. Before then as many as 90 per cent of type 1 SMA babies didn’t survive a year. However, whilst the evidence was compelling for improvement for babies with type 1 SMA, the SMC was unable to recommend its use in types 2 and 3 because the evidence wasn’t strong enough to justify its high cost. Doctors will now be able to prescribe nusinersen for patients with types 2 and 3. They 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.
The SMC have also issued the following guidance for those affected with SMA
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 Scotland can now access nusinersen (Spinraza®️) for types 2 and 3if access may be possible for you at some point.
This is fantastic news for SMA sufferers in Scotland. We hope that NICE and NHSE may follow suit, with the MAA criteria due to be published soon.
We will of course update the community if and when we have any further information.
The TreatSMA Team.
