Tel: 01789 267520

The UK Expanded Access Programme for Nusinersen for Children with SMA Type 1

(last updated 6th September 2018)

The Expanded Access Programme (EAP) was set up by Biogen and was designed as a short-term solution for access to nusinersen for eligible children with SMA Type 1. It will stay open in England, Wales and Northern Ireland until 1st November 2018 for any child 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. Children with SMA Type 1 in Scotland are now able to access NHS funded treatment there. They are not affected by this decision. You can read Biogen’s community update about this decision, here. We strongly urged them not to take this step at this time.

On 28th August, we, jointly with MDUK and The SMA Trust, wrote to Biogen urging them and NHS England to be as flexible as possible in their discussions on price so that, as a matter of urgency, a Managed Access Agreement that merges seamlessly with the current EAP is set up and ensures that patients are able to access this life-changing treatment at the earliest possible opportunity. You can read the letter here.

In our response to NICE’s consultation paper on access in general ,we strongly urged NICE and NHS England to ensure they reach this agreement in time.

Eligible Children

All children diagnosed by age 7 months with SMA Type 1 are eligible for access if the family and clinical team, agree they may potentially benefit. 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. 

Anyone enrolled in the programme by November will be assured of continued treatment unless the family and their clinician consider it appropriate to stop.

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.

Which Centres Are Providing Treatment

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. You can also contact us to see 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

Continued Advocacy

We will continue to focus our advocacy for access to treatment on the urgent need for NICE, NHS England and Biogen to agree the terms of a managed access agreement to replace this EAP ahead of 1 November, as well as the need for a long-term sustainable plan for access. You can find out how you can help, here.

You can read below about the huge amount of work that went on to secure access to the EAP for eligible children with SMA Type 1:

  • 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.
     
  • 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.