NICE Announcement on Nusinersen

13 August 2018

NICE’s consultation paper was released this morning with the devastating news that they are not recommending it for funding by the NHS. This is a huge blow for families who are so desperate to be able to potentially have access. We always expressed that NICE's single technology appraisal (STA) process wasn't suitable for assessing this treatment and strongly advocated for it to be assessed as a highly specialised technology (HST) which has a higher cost per annum threshold for approval.

The glimmer of hope, however, is that the paper encourages the possibility of a ‘Managed Access Agreement (MAA)’. This would be a 3 – 5-year scheme agreed between Biogen, NICE and NHS England that would enable access to treatment and the opportunity to collect further evidence of its clinical benefits and impact on quality of life. At the moment we don’t know who might be covered by such an agreement, but patient groups and clinicians would take part in the conversations about what might be possible.

The consultation summary document states, "This recommendation is not intended to affect treatment with nusinersen that was started in the NHS before this guidance was published". We therefore understand this to mean that children currently accessing nusinersen via the NHS / Biogen Expanded Access Programme (EAP) will continue to receive treatment until they and their NHS clinician consider it appropriate to stop and that currently the EAP remains open to eligible infants; for more information, please click here.

What can we do?

The consultation paper is now open for anyone to comment by 5th September. You can read it here. It’s vital that as many people as possible give NICE their views on what the paper says. They want your views as to whether:

  • All the relevant evidence has been taken into account
  • The summaries of clinical and cost effectiveness are reasonable interpretations of the evidence
  • The provisional recommendations are a sound and a suitable basis for guidance to the NHS

SMA Support UK, Muscular Dystrophy UK, The SMA Trust and TreatSMA are all working on responses.

If you wish to make a response, you can do so (you will need to either sign in to / create a NICE account first) here

We will share our preliminary thoughts on these questions with the community as soon as possible. We will be urging NICE, NHS England and Biogen to have agreed an MAA with the SMA community by NICE’s original decision date of 21st November 2018.