Prescribe Life: our campaign to find a way forward for tofersen

I’m Eleanor and I have motor neurone disease. It’s fatal.

But for some of us, there’s a chance that might be about to change.

I’m among the 2% of people with a form of MND caused by a fault in the SOD1 gene. A ground-breaking new treatment, tofersen, has been developed for this type of MND. It slows – and could even stop – my devastating neurological disease. But a decision by a government body about how to assess it has caused a standoff between it and the company that owns tofersen, meaning there is now no prospect of it being approved for use on the NHS.

Please sign this petition to unblock the logjam and help make tofersen a step closer to being available on the NHS.

Sign the petition

Dear NICE,

Please make an exception and adopt the HST route to appraising tofersen for SOD1 MND, so this life-saving treatment has the best chance of being made available to patients on the NHS.

What's the issue with tofersen?

I currently take tofersen through an Early Access Programme funded by the manufacturer, but they could close this programme to new people at any time. That would be devastating for future SOD1 patients, which may include other family members – we don't yet know if anyone else in my family may have the change in the SOD1 gene. That's why we need the NHS to fund tofersen.

But the National Institute for Health and Care Excellence (NICE), which decides which medicines the NHS will fund, has decided not to evaluate tofersen using the approach that gives it the best chance of being approved, saying MND isn’t rare enough to qualify. But its own guidance says that it can make exceptions for game-changing treatments for conditions with particularly poor outcomes.

If tofersen isn’t worth making an exception for, what possibly could? If not people with MND, which kills half of people within two years of diagnosis, then who?

That’s why, together with the MND Association, I’m launching the Prescribe Life campaign – calling on NICE to make an exception and use the approach that gives tofersen the best chance of becoming available on the NHS to people, like me, who have the SOD1 gene variation (about 60-100 people in the UK).

What is motor neurone disease (MND)?

Motor neurone disease (MND) is a fatal, often rapidly progressing disease that affects the brain and spinal cord. It kills a third of people within a year and more than half within two years of diagnosis.

MND leaves people locked in a failing body, unable to move, talk and eventually breathe. 

Six people a day die from MND in the UK. There is no cure.

Tofersen is the first treatment to make a significant difference to life expectancy, but can only benefit 2% of people with MND, and while it significantly slows symptom progression, it is not a cure.

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