17 October 2024 News
The MND Association has today (October 17) launched a national campaign focused on creating a route for people with SOD1 motor neurone disease (MND) to access a ground-breaking drug.
The charity’s Prescribe Life campaign is urging the National Institute for Health and Care Excellence (NICE) to use its discretionary powers to evaluate tofersen in the way that gives it the best chance possible of being prescribed in the future.
People with MND have been at the heart of the Association's project from the very start. Eleanor Dalley, 49, from Barnet is taking tofersen through the early access programme and has been an integral member of the planning phase.
The charity is now urging people to sign its petition asking people to support the call to NICE to make an exception for tofersen.
It is the first step in the campaign and is aimed directly at NICE. 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, but some people 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, to which Biogen, the pharmaceutical company which developed tofersen, has responded by not progressing its application.
NICE has decided tofersen should not be assessed using the HST route 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.
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, 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 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.
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.”
If you want to join our fight, please sign and share the petition here.