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What Is Happening About Access To Nusinersen?

18 October 2017

We share the frustration of the SMA community that although there are now some 60 children with SMA Type 1 receiving Biogen / NHS England funded treatment via the EAP, there has still been no announcement from NICE about whether it will assess the treatment for continued and wider provision by the NHS. The UK continues to lag behind the US and Europe. The UK continues to fail the SMA community.

SMA Support UK:

  • is continuing to try to find out why there is no announcement from NICE - we had expected to hear in July
  • is talking to clinicians to see whether submitting a Policy Proposition Route (see NHSE letter) about expanding the SMA Type 1 EAP criteria would be worthwhile
  • has asked MDUK as part of their Fasttrack campaign, to highlight nusinersen in national media as a strong example of what is wrong with the system. We have asked them to work with Rare Diseases UK which has also been working hard on these wider access issues
  • has contacted RDUK and asked them to work with MDUK so that there is a strong coordinated joint lead
  • has committed to support this work
  • is concerned that Biogen has said it will review the future of the SMA Type 1 EAP at the end of this calendar year and that its decision 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 will urge Biogen to keep the programme open.
  • is in close contact with TreatSMA  - working in our different ways to maintain pressure and give voice to the SMA community