The UK Expanded Access Programme for nusinersen for children with SMA Type 1
(last updated 7th December 2017)
The Expanded Access Programme (EAP) was set up by Biogen and is designed to provide access to nusinersen for eligible children with SMA Type 1. It is only available to children with SMA Type 1 where both the child’s medical team and the child’s parents/guardians have agreed that it could be of potential benefit and that the child is eligible for the treatment.
Deciding whether this is a treatment you want to request is a very personal decision. Our information sheet 'Nusinersen for children with SMA Type 1 – Information for Families' talks more about the treatment, which children are eligible and what happens next if you do request access to the programme.
Under the terms of the EAP, Biogen funds the drug free to eligible children accepted on the EAP for the child’s lifetime.
Until 4th August 2017, funding the hospital costs of administering the EAP has been an issue in many areas of the country and not all areas have been offering the EAP because of this and lack of capacity. On August 4th, NHS England announced an interim policy that provides new funding that will provide all hospital costs for administering nusinersen to children with SMA Type 1. Going forward, until there is a decision from NICE (National Institute for Health and Care Excellence), children must have been diagnosed with SMA Type 1 by 7 months of age and have two copies of the SMN2 gene to be eligible for this funding. Children with SMA Type 1 who were diagnosed before this time, are older than this and are not already on the EAP, will be eligible for treatment if their medical team agrees it could potentially be beneficial. You can read this interim policy here.
We joined with clinicians and the other charities and advocacy groups to appeal these limits. You can read our latest joint letter sent on 30th October to NHS England here. As yet we have had no reply to this latest appeal.
Biogen has said it will keep the EAP open at least until the end of this year. This should mean that, whatever their home address, eligible children who are waiting to join the EAP, or who are newly diagnosed during this time, should be able to access treatment which will be paid for throughout their lifetime. Children already on the programme are also guaranteed access and funding.
We are concerned that Biogen has said it's decision about the future of the EAP at the end of the year will depend on what progress there has been with NICE's appraisal of the treatment. We continue to do all we can to find out why this is so delayed and to strongly urge Biogen to keep the programme open.
As access to this programme is evolving all the time, it is not possible to keep an accurate record on this website of which centres are currently offering the EAP. Your medical team will talk with you about whether the EAP is available locally and, if not, where they would arrange for your child to be assessed for eligibility. They will work with NHS England and other Trusts to organise access. There should be no need for you to make contact separately. Doing so can make it more difficult for NHS England and Trusts to keep track of who in the short term needs to go where and to organise the funding for this.
You can also contact us to see if we have any further updates or if we can help you with personal advocacy for access or other support with caring for your child. Phone 01789 267520 or email email@example.com
You can read previous updates about the work that went on to secure this access to the EAP for eligible children with SMA Type 1:
- 29th September 2017 - NHS England reply to letter appealing the EAP criteria.
- 24th August 2017 - Joint clinicians and charities letter to NHS England appealing EAP criteria
- 18th July 2017 - At the All Party Parliamentary Group (APPG) meeting organised by Muscular Dystrophy UK and attended by families, SMA Support UK, TreatSMA and the SMA Trust, NHS England announced that, following the release of the EMA Assessment Report of the drug received by them week of 19th June, they were now reviewing their interim EAP policy position.
- 13th July 2017 - Biogen announces EAP will remain open to end of the year
- 6th June 2017 - NHS England meeting confirms their position but sets out an action plan
- 16th May 2017 - NICE’s reply to the 5th April letter
- 10th May 2017 - we publish notes from the NHS England meeting held on 24th April which sets out their ‘not commissioning the treatment’ policy at this stage and we publish Biogen’s responses
- 5th April 2017 - North Star clinicians, SMA REACH UK and our open letter to NICE and NHS England asking NHS England to fund the administration costs of the programme
- 7th March 2017 - Biogen to submit proposal to NHS England to consider a commissioning framework to support the implementation of the EAP
- 20th January 2017 - potential for other Centres to offer the EAP
- 21st December 2016 - includes many initial Q and As about the treatment
- 16 September 2016 - from the Newcastle and GOSH UK Clinical Trial Principal Investigators
Nusinersen was tested with infants with SMA Type 1 in a clinical trial called ENDEAR. Newcastle and Great Ormond Street (GOSH) were UK clinical trial sites.
By autumn 2016, the ENDEAR study results hadn’t been fully analysed and there was only limited knowledge about the efficacy of nusinersen across the full spectrum of infants and children with SMA Type 1. However, results had been sufficiently positive for Biogen to work on opening an Expanded Access Programme (EAP).