The Motor Neurone Disease (MND) Association is urging the government to take action as people with a rare form of MND are unable to access a groundbreaking new drug, despite it being currently provided for free to the NHS.
Tofersen is the first effective new MND treatment for decades, however, access to the drug has become a lottery – with people unfairly missing out despite urgently needing the treatment.
Over 30 patients are currently taking tofersen in the UK through an Early Access Programme (EAP) supported by Biogen, the developer of the drug. However, the MND Association estimates 12 people are being refused access, despite having the same level of need.
Tofersen has been shown to slow, and in some cases, halt progression of symptoms in people living with a rare form of MND caused by a variation in the SOD1 gene.
There are estimated to be between 60 and 100 people living with SOD1 MND in the UK. A third of people die within a year of diagnosis, half within two years.
Biogen provides tofersen at no cost to patients or to the NHS but because it is not yet a commissioned NHS treatment – capacity to deliver the EAP is provided on a ‘goodwill’ basis.
The MND Association has learned that many of the care centres and networks where it provides funding have been advised they cannot accept any more people onto the EAP.
One of those affected is Seckin McGuirk, 56, a retired maths teacher from Rugby, who has been told she cannot access tofersen.
Seckin said: “I just struggle to comprehend if such a drug is available, and just because there is no staff to deliver that drug, they can’t take patients in. It is not many people.
“It’s such a terrible illness, it feels like every day there is something going away from you. Every day you’re waiting, you’re losing something.”
Richard McGuirk, 56, Seckin’s husband, added: “The drug is free, but I can’t believe the NHS can’t find a team once a month to do the lumbar puncture. It’s not just the people who have that condition, it’s all their families who could be affected. You are talking about a small number of people.
“The NHS is one of the biggest employers in the world. You’re telling me that within that entire framework, you can’t accommodate 12 people.”
It is hoped tofersen, developed by pharmaceutical company Biogen, will enter the regulatory process in the UK in the coming months.
The drug has already received marketing authorisation from the Federal Drug Administration in the USA and the European Medicines Agency. However, the MND Association argues that people with MND, a rapidly progressive and terminal condition, do not have time to wait.
As the number of eligible patients is low, only a modest amount of additional resource would be needed to address the current inequitable situation where some patients receive it while others miss out.
MND Association CEO, Tanya Curry, and officers from the All-Party Parliamentary Group on Motor Neurone Disease have written to Karin Smyth Minister of State for Health, urging her to work with NHS England and find a solution.
Without this Seckin, and others like her, face a greatly reduced life expectancy. She said: “I’m constantly checking my emails and phone all the time. I’m just hoping that I’m going to get something, give me an appointment and start the treatment. I’m waiting for that.
“When you are diagnosed, all you hear from medical staff is ‘this is the end’ and ‘we will just try to keep you alive as long as possible.
“My quality of life is deteriorating. It’s an awful disease and there is no hope, no hope at all. Since I heard about tofersen, it gives me hope to carry on with my life as long as possible.”
Seckin first noticed the inability to lift her right leg in Easter 2023 after jogging while on holiday in Turkey. She thought it was ‘just one of those pains’ but then she noticed issues walking.
Seckin said: “I just couldn’t work out what was going wrong. I couldn’t walk fast. I organised a meeting with a neurologist in July 2023, and I asked him ‘what do you think it is?’
“[MND] is something they can’t diagnose straight away because it’s hard to pinpoint. Everything started being crossed out and the final diagnosis was made in June 2024.
“At first you just don’t accept this diagnosis, but I mentioned to my consultant that my cousin passed away, and we found out she also had MND.”
Seckin, who has one son Aiden, 25, had tests carried out in Turkey, revealing she has the SOD1 gene which can be inherited from family members.
Tanya Curry, MND Association Chief Executive, said: “It cannot be right that there is a drug being provided for free that could save some people with MND from rapid physical deterioration and death, and yet they cannot get access it because the NHS is refusing to cover the costs of administering it.
“The fact that some people are being refused this drug while others receive it is deeply unfair and amounts to a lottery where some people will live and others condemned to a fate of rapid physical decline and certain death.
“We’re calling on the Government to step in and ensure no one falls through the cracks. Every day that passes without these people being able to access this free drug is another day of avoidable physical decline. We need the Government to act now, because those who need tofersen do not have time to wait.”
Ian Byrne, Chair of the All-Party Parliamentary Group on Motor Neurone Disease (MND), added: “We urge the Government to step in immediately to find a solution to help people facing this horrendous, terminal illness.
“It cannot be right that people lose their lives for lack of a small amount of resource to give them a freely available drug.
“This treatment has the potential to transform lives and must be provided to everyone who can benefit from it as soon as possible.”
For more information about MND and the MND Association please visit www.mndassociation.org and sign the petition here.
Image: Seckin and Richard McGuirk, photo provided by MND Association.
