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The UK Expanded Access Programme for nusinersen for children with SMA Type 1

(last updated 11th May 2018)

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. Now that Scotland has advised the Scottish NHS to fund the treatment there for those with SMA Type 1, it will no longer operate in Scotland. It remains open in England, Wales and Northern Ireland for 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 was an issue in many areas of the country and not all areas were offering the EAP because of this and lack of capacity. On August 4th, NHS England announced an interim policy  that provides new funding to cover all hospital costs for administering nusinersen to children with SMA Type 1.  This was updated on 9th March 2018, following advocacy by clinicians and patient groups. It makes the EAP available to: all children diagnosed by age 7 months, no matter what their SMN2 copy number. You can read this interim policy here.

Children with SMA Type 1 who were diagnosed before 4th August 2017, are older than this and are not already on the EAP, remain eligible for treatment if their medical team agrees it could potentially be beneficial. 

This access to nusinersen will stay in place for as long as Biogen continues to offer the programme and until there is a decision from NICE (National Institute for Health and Care Excellence) about whether NHS England will fund all of the treatment costs, to access this programme. Wales and Northern Ireland often follow England, but may choose to follow Scotland.

Biogen had said it would keep the EAP open, at least until the end of 2017 and has continued to do so. This should mean that, whatever their home address, eligible children should be able to access treatment which will be paid for throughout their lifetime, even when the programme finally closes to further new children. Children already on the programme are also guaranteed access and funding.

As 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 supportservices@smasupportuk.org.uk

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:

  • 30th October 2017 – first letter to NHS England appealing eligibility criteria 
  • 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).

To keep up to date with progress on developing this programme, you can follow us on Facebook and Twitter and sign up to receive our monthly E-news updates.