Spinraza not to be funded – NICE Announce

Spinraza (Nusinersen) will not be recommended for funding by the NHS, is the devastating decision announced today by NICE (National Institute for Health and Care Excellence). One glimmer of hope, however, is that it does leave the door open for a Managed Access Agreement.

NICE’s consultation paper was released this morning with the devastating news that they are not recommending Spinraza for funding by the NHS. This is a huge blow for families and individuals affected. The glimmer of hope, however, is that the paper encourages the possibility of a ‘Managed Access Agreement (MAA)’ – a 3 to 5 year scheme agreed between Biogen the manufacturer, NICE and NHS England, that would enable access to the treatment and the opportunity to collect further evidence of Spinraza’s clinical benefits and impact on quality of life.

At this time, we don’t know who might be covered for treatment by such an agreement, but patient groups and clinicians would take part in the conversations about how access may be structured.

The NICE 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 the EAP remains open to eligible infants. For more information on the EAP, please click here.

The NICE consultation paper is now open for anyone to review and provide any comments or feedback, by 5th September. It’s vital that as many people as possible provide feedback to NICE on their views on the paper and its content. 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

The SMA Trust, SMA Support UK, MDUK and Treat SMA are all working on providing responses. If you wish to make a response, you can sign up 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.



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