A new campaign is aiming to help more people living with motor neurone disease (MND) to access a ground-breaking drug.
UK charity the MND Association has launched the Prescribe Life campaign and is urging the National Institute for Health and Care Excellence (NICE) to use its discretionary powers to evaluate the drug tofersen in the way that gives it the best chance possible of being prescribed in the future.
Tofersen has been shown to slow progression of symptoms in people with some types of MND but currently NICE is refusing to appraise it in the way the MND Association believes gives it the best chance of being made available through the NHS.
The charity is urging people to sign its petition asking people to support the call to NICE to make an exception for tofersen.
Tofersen has been shown in clinical trials to slow the progression of symptoms in people living with MND who have alterations in the SOD1 gene.
“This is about two per cent of the MND population, estimated to be between 60-100 people in the UK. MND is a terminal progressive illness. But some people with SOD1 MND who are taking tofersen have reported their symptoms have actually improved.
Leading MND clinical and research experts have called for the drug to be appraised through NICE’s Highly Specialised Technology (HST) route, designed for treatments for serious rare diseases where only a small number of patients would benefit.
NICE has refused and, in March 2024, confirmed it would assess tofersen through the Single Technology Appraisal (STA) route instead, which Biogen, the pharmaceutical company which developed tofersen, has responded to by not progressing its application.
NICE has decided tofersen should not be assessed using the HST because MND is not rare enough to qualify. But NICE’s own guidance states that it can make an exception for treatments that can make a big difference to people with conditions with a poor prognosis.
The MND Association believes tofersen meets those criteria and therefore should be appraised using the HST route.
Currently Biogen is keeping its early access programme open, so people with SOD1 MND can get the treatment if they wish to. However, at any point, that programme could be closed to new patients.
Eleanor Dalley, 49, from Barnet is taking tofersen through the early access programme and has been an integral member of the planning phase of the campaign.
Eleanor (pictured above), who lives in London with her 14-year-old daughter, said: “There have been small but, for me, significant improvements. I can move my legs in bed unaided and can get on the massage table on my own.
“These small things are mini miracles to me. My daughter said to me ‘With this treatment it gives you hope, and it gives us a chance of a cure’.
“For me, it feels like tofersen is giving the research community time to find more answers and find a cure. I feel in a privileged and lucky position to have the drug.”
Tofersen has even given Eleanor, who has seen two of her aunts and her father diagnosed with MND, the chance to consider a long-term future.
She added: “When I was given my diagnosis I was told the stats – 18 months to two years. It’s now more than five years since I was diagnosed. I am outliving the odds.
“I’m 50 next year and I’m planning a girls’ trip with my friends and a holiday with my daughter to celebrate. I never thought I would be able to do that and plan that far ahead.
“If this lifeline drug does not get the necessary approvals, then hope dies for helping future generations affected.”
Tofersen was approved by the European Medicines Agency in May 2024, meaning people with MND in Europe could potentially have access to the drug in the future. But, in the UK, the approval process has stalled.
Tanya Curry, chief executive of the MND Association, said: “The MND community so often talks about hope – tofersen is more than hope, it is life.
“For people with SOD1 MND, tofersen is a game-changer; it gives them the chance to plan for the future in a way that would not be possible without it.
“But we need NICE to show flexibility. Tofersen is an exceptionally exciting new treatment, MND is an exceptionally awful condition, and so NICE needs to make an exception to unblock the impasse and allow us to take a step towards tofersen being available on the NHS.
“We’re launching the Prescribe Life petition because the more people who sign it, the louder the voice will be demanding that tofersen is too important not to be accessible for the people who need it.
“Please join us – be a voice for our community and sign the petition.”
