Advocacy that Helped to get the EAP Up and Running in the UK

Which children were treated via the EAP, and how did it work?

Until 1st November 2018, all children diagnosed by age 7 months with SMA Type 1 were 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, who were older than this and were not already on the EAP, were also eligible for treatment if their medical team agreed it could potentially be beneficial. 

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

Under the programme, the pharmaceutical company Biogen funded the cost of the drug itself while the NHS provided all the hospital costs of its administration. 

Advocacy that helped to get the EAP up and running in the UK

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 provided 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 made the EAP available to: all children diagnosed by age 7 months, no matter what their SMN2 copy number. 
  • 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).