17 April 2026 News
The National Institute for Health and Care Excellence (NICE) has started its appraisal of life-changing drug tofersen – a key step towards making it available on the NHS - with the MND Association working to ensure a positive outcome.
Tofersen, developed by Biogen, has been shown to slow, and in some cases even halt, symptom progression in the 2% of people whose MND is caused by a change in the SOD1 gene. The treatment is currently only available through an Early Access Programme (EAP).
Our involvement in the appraisal
The NICE appraisal will review data, research evidence and the testimony of people who have been taking tofersen to assess whether the drug meets the cost-effectiveness requirements to be made available on the NHS.
We’re gathering evidence from people with SOD1 MND and their carers about the positive impact of tofersen, and nominating clinical experts and people affected by MND to contribute evidence directly to NICE through both written submissions and at committee meetings.
They will talk about the incredible benefits of tofersen and their experiences of campaigning for equal access.
This ensures the voices of people with MND are considered, and gives tofersen, the first effective MND treatment for decades, the best chance of being available on the NHS.
This evidence submission phase will last until 4 June, before an additional evidence-gathering period leading into the NICE committee meeting on 22 October.
Possible outcomes
It would not be unusual for NICE to issue a draft recommendation not to make the treatment available on the NHS at that stage.
If this were to happen, the decision would still be under consultation with the opportunity to gather more evidence and community insight to inform a final decision.
Richard Evans, Director of Engagement, added: “This is a pivotal process for the MND community. Tofersen is the first new treatment for MND to emerge in decades.
“We are in a constant dialogue with NICE to provide all the evidence and personal testimony we can to give tofersen the best possible chance of being available on the NHS as soon as possible.
“People with SOD1 MND can’t wait any longer, caught in the middle of drawn-out negotiations when we are dealing with a rapidly progressing disease.”
We’ve recently introduced a new support fund to help people travelling out of their area to receive tofersen.
However, currently not everyone who is eligible has been able to access it – an issue we’ve been campaigning on for the last year.