UK Access Now
Page last updated 19th May 2020
On 24th July 2019, the Final Appraisal Document (FAD) and five year Managed Access Agreement (MAA) were published by NICE.
On 6th April 2020 we received this important update: How access to nusinersen treatment will be affected by COVID-19
On 19th May 2020 we started to get responses from NHS England/NICE to the many questions the patient groups had raised last August:
Further FAQs coming soon:
- Access for adults FAQs
(see below for information to date)
- Nusinersen Treatment FAQs
- Data Collection FAQs
Access for adults
At the end of November 2019, eight hospitals were given contracts to deliver services to adults:
- Nottingham University Hospitals NHS Trust
- Oxford University Hospitals NHS Foundation Trust
- Salford Royal NHS Foundation Trust
- Sheffield Teaching Hospitals NHS Foundation Trust
- St George’s University Hospitals NHS Foundation Trust
- The Newcastle upon Tyne Hospitals NHS Foundation Trust
- University College London Hospitals NHS Foundation Trust
- University Hospitals Birmingham NHS Foundation Trust
To help with planning, in Feb / March 2020, we worked with the other Patient Groups to gather information from the Community as to who wants access to treatment. 145 people replied and we passed on an overall summary to the adult services network, NICE and NHSE. Due to the COVID-19 crisis, plans for passing on more information to the treatment centres were delayed but we are now trying to do this now. We will let anyone who responded and gave their contact details know which Centre has agreed to review their information when we succeed in passing on their individual response.
Access for all who have SMA Type 3
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. We continue to advocate for strongly for this.
Wales and Northern Ireland
Biogen has advised that since 15th May 2019, infants who have SMA Type 1 have access. We understand others in Northern Ireland are also now either being assessed for, or are receiving, treatment.
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 2019) with their answers to the questions the Patient Groups asked about access in Wales and Northern Ireland if you scroll down to the last page, here.
Nusinersen has been routinely available to children who have SMA Type 1 in Scotland since May 2018.
The Scottish Medicines Consortium (SMC) announced that, as well as being available for those who have SMA Type 1, from July 2019 nusinersen could be prescribed for those who have 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 their update here.
You can read Biogen’s Community Update (7th August 2019) with their answers to the questions the Patient Groups asked about 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. We are very aware that though adults have been assessed for treatment, as of this no one had started treatment and, with MDUK, were following this up.
You can read the SMC 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.