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

The Expanded Access Programme (EAP) is designed to provide access to nusinersen for eligible children with SMA Type 1 who didn’t participate in the ENDEAR clinical trial. The EAP is aimed at providing access to nusinersen prior to any European Medicines Agency (EMA) approval of the drug.

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 accepting eligible children from which geographical areas for the EAP. Talk first to your medical team about what is possible for your child in your area. You can also contact us to see if we have any further updates to help you. Phone 01789 267520 or email supportservices@smasupportuk.org.uk

You can read what we are doing to secure urgent access to this programme for all eligible children with SMA Type 1 no matter where they live in the UK here and how you can help here.
 

More about what the current position is with the EAP and what else has been happening

Q. What will happen to children currently on the EAP if nusinersen is licensed in Europe?

A. On 21st April, the EMA recommended marketing authorisation of nusinersen, or Spinraza™ as it will be called, for the treatment of "5q spinal muscular atrophy", which includes SMA Types 1, 2 and 3. We asked Biogen what this means for children enrolled in an EAP.

They confirmed that they will continue to support infants enrolled in the UK EAP to access nusinersen, whatever the final UK decision about NHS access and funding. Read their full reply here.  
 

Q. Will new children be enrolled and for how long after marketing authorisation?

A. Biogen replied: “The EAP in Europe will remain open for new patients for a period of time following marketing authorisation. The actual date of closure will be discussed and agreed country by country.  As such, Biogen continues to receive and assess EAP applications from individual hospitals within the process already established. As of May 12th 2017, the EAP is active in 5 sites in England, 1 in Scotland and 1 in Northern Ireland. Additional sites have been approved but are not yet active. The intention is these sites will eventually form a small number of regional hubs.”
 

Q. What about children travelling overseas for treatment?

A. Biogen is not able to say what the position is for infants receiving treatment via a non-UK EAP once that country makes a funding decision about access to nusinersen for its nationals. It is up to the treating physician and that centre to make this decision. Read Biogen's reply in full here. We will continue to try to find out more from any overseas centres we know are treating UK infants.

If you are thinking about going overseas to access the EAP, please do read our checklist of points to discuss and consider.
 

Q. What can I do to help progress access to the EAP?

A. To support our efforts to ensure the EAP continues and access to nusinersen becomes a long-term option, you can write to Simon Stevens, Chief Executive at NHS England, using the template letter here.
 

Q. What have clinicians been doing to speed up access?

A. Clinicians have been taking all steps they can to progress the EAP within the resources they have, but so far progress in England has been significantly constrained by lack of capacity. On 5th April, we joined with them in asking the National Institute for Health and Care Excellence (NICE) and NHS England's (NHSE) Clinical Reference Groups (CRGs) to address this issue. You can read our open letter here and the reply finally received from NICE on the 16th May here.
 

Q. What are NHS England (NHSE) and Biogen doing to speed up access?

A. On 10th May we were able to publish a report of our 24th April meeting with NHSE, GOSH clinical respresentative, Biogen and MDUK which you can read in full here

  • Peer reviewed publication of results

At this meeting NHSE stated that, because of a rule which requires companies to have their trial results published in a peer review journal before they qualify for NHSE support, it is not possible for NHSE to consider the possibility of supporting treatment costs at this point. As soon as Biogen has moved on such a publication, NHSE said, it will review the possibility of support again.

We asked Biogen to respond to this, which they did on 15th May. They advised that they had, at the request of NHSE, provided documentation to support the business case for NHSE supporting the additional aspects of care required to administer treatment. “This involved the provision of publicly available data outlining the clinical evidence base for nusinersen as well as estimates of additional funding that might be required by the NHS to support children receiving the drug.”

Biogen expressed disappointment with the outcome of the 10th May meeting. They advised that, “the request by NHSE to view the clinical data in a peer-reviewed journal is something we are actively pursuing and plans to publish the full results of the Phase III studies are in development. In the interim, Phase III data has been presented at international scientific congresses as is normal prior to full publication. However, it is important to note that the Phase II data has already been published in a peer-reviewed journal, The Lancet.”

“The preparation of data for submission to a peer-reviewed journal requires a number of months for consolidation, analysis and preparation. Biogen’s timelines for this are in line with the standard process for publication of pivotal clinical trial data. Under usual embargo conditions, a manuscript submitted to a medical journal for publication must not be disclosed until the publication date. Biogen is optimistic that publication can be confirmed in the summer.”

You can read Biogen’s full reply here.

  • NHSE asked Biogen to fund ancillary costs

At the 24th April meeting, NHSE suggested Biogen consider funding the costs of treatment along with the actual drug. On 15th May, Biogen advised us of their response to this as follows:

“NHSE has requested that Biogen considers supporting the ancillary costs in addition to provision of drug under the EAP programme. It is fundamental that in partnership with the NHS, Biogen understands specifically how any additional funding would be applied at each existing and potential EAP site to resolve its infrastructure challenges. Biogen has offered to work with individual hospitals to assess this. Biogen remains committed to continue these discussions and to review what is possible.”
 

Q. What else is there to know?

A. You can read previous updates released about the EAP below:

Nusinersen was tested with infants with SMA Type 1 in a clinical trial called ENDEAR. The latest report can be read here. 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).  

  • 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 Q and As about the treatment
     
  • 16 September 2016 - from the Newcastle and GOSH UK Clinical Trial Principal Investigators 

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