30 May 2025 News
You may remember that last year, we campaigned to urge the National Institute for Health and Care Excellence (NICE) to change its mind about the way it was going to decide if a drug called tofersen should be available on the NHS.
Tofersen is a new treatment that is effective for the roughly 2% of people with MND who have a particular genetic change. We were delighted that, following our campaign, NICE changed its mind and is now assessing tofersen in a way that gives it a higher chance of being approved.
That approval process could still take many months. But we weren’t concerned about this because Biogen, the company that makes tofersen, has said, for the time being, it will provide the drug for free to people who need it. This is a hugely important interim measure because tofersen significantly slows, in some cases even pausing, the disease’s progression. It can literally mean the difference between life and death.
But a few months ago, we started hearing worrying reports of people with MND who would benefit from tofersen but were being denied access to it.
Is the NHS denying people tofersen?
This seemed inconceivable. How could it be possible that the NHS could be effectively denying a lifesaving drug to people with MND, even though that drug is free? Surely there must be some mistake?
But when we dug deeper, we found it was true. While most people who would benefit from tofersen are getting it, some people – about 12 people but we fear this number will rise as more people are diagnosed - are being told the NHS will not give it to them.
The reason? Because while the drug itself is free, the NHS won’t pay for the hospital bed and the health professional time needed to administer the monthly lumbar punctures to these people.
But while there are costs involved, these are relatively modest ones that pale into insignificance compared to the benefit tofersen brings: stopping or significantly slowing the progress of a disease that, without it, kills half of people within two years of diagnosis.
And even if you were to somehow argue that these modest costs aren’t worth paying, it’s grossly unfair that tofersen is being given to some people with MND who would benefit from it, but not to others.
What the Government has said about tofersen
We have put this case to NHS England and the Government. NHS England has told us it is not their role to direct or influence any NHS Trust decisions on this case, or on any other private activity. The Government has said it has no plans to intervene - they think this should be a decision for each local NHS trust. You can read the Government’s rationale in its response to a question from an MP.
The result is a lottery - each local trust decides how much capacity it can offer to administer tofersen, on a first come, first served basis.
We believe that leaving the decision to individual trusts is not good enough. It is effectively creating a situation where some people with this type of MND are given the hope of many more years of life, while others are arbitrarily left facing the continuing progression of their disease, and, ultimately, death.
This is completely unacceptable.
I recently spoke to Sky News about this, alongside Seckin McGuirk, who is one of the people with MND who would benefit from tofersen but can’t get it.
I can’t imagine what it must be like for Seckin, and other people like her, unable to get a potentially lifesaving drug that the NHS can get for free but won’t pay to administer.
This is why we at the MND Association have launched a campaign calling on the Government to intervene. This lifesaving drug is available, for free, right now from Biogen and it is simply wrong that some people are getting it and some people aren’t.
We need the Government to act now. People like Seckin don’t have time to wait.
If you agree, please take time to sign our petition today
