UK Access Now
Last updated 18th February 2020.
On 24th July, NICE published the Final Appraisal Document (FAD) and Managed Access Agreement (MAA) which SMA UK, MDUK and TreatSMA have all signed up to. You can see the final documents NICE has released here or you may wish to go straight to their 'Information For The Public' tab. You may also find our summary of some key aspects of the Managed Access Agreement helpful. You can read this here.
Children who are eligible for treatment are beginning to get access through paediatric neuromuscular centres.
At the end of November, eight hospitals were given contracts to deliver services to adults. You can read more about the adult service, here. To help with planning the roll-out of the nusinersen treatment programme, we are currently running a survey and want to hear from all adults who live in the UK and who have 5q SMA (Types 1, 2 or 3) to find out who does and who doesn’t want to access nusinersen (Spinraza) treatment.
More information about how the MAA will work will be coming out over the following weeks / months. This will be led by NHS England who will continue to consult and work with clinicians, NHS Trusts and Patient Advocacy Groups. We will keep you posted as soon as any information is released publicly.
Though this MAA doesn't at the moment achieve access for all, for which the SMA Community, SMA UK, MDUK and TreatSMA have all strongly advocated, the door is left open for possible future changes to eligibility criteria for those who have SMA Type 3 who don't currently have access.
You can read Biogen’s Community Update (7th August) with their answers to the questions the Patient Groups asked about the MAA, here.
Wales and Northern Ireland
Biogen has advised that since 15th May, infants with SMA Type 1 have access.
For the treatment Translarna, Northern Ireland adopted NICE’s guidance on the day it was issued. In Wales, this was a month later after the All Wales Medicines Strategy Group had met.
You can read Biogen’s Community Update (7th August) with their answers to the questions the Patient Groups asked about the access in Wales and Northern Ireland if you scroll down to the last page, here.
Nusinersen has been routinely available to children with SMA Type 1 in Scotland since May 2018.
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.
You can read Biogen’s Community Update (7th August) with their answers to the questions the Patient Groups asked about the access in Scotland if you scroll down to the last page, 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.
You can read more about all the work that went into getting this result for Scotland here.