Imagine being denied a life-saving treatment that’s already available for free. That’s the harsh reality for some patients with motor neurone disease (MND) in the UK, despite a groundbreaking drug being offered at no cost. But here’s where it gets controversial: while the drug itself is free, some NHS trusts are refusing to cover the costs of administering it, leaving patients in limbo. Let’s dive into this complex and emotionally charged issue.
Tofersen, a drug that has shown remarkable potential in reversing symptoms of MND for patients with a specific genetic mutation, has been approved for use in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA) since 2025. It works by preventing the clumping of faulty proteins in the brain and spinal cord, which are the root cause of MND in these patients. For some, it’s not just a treatment—it’s a lifeline. Yet, despite its availability in Europe and the US, its rollout in the UK has been fraught with challenges.
Here’s the catch: while Biogen, the biotech company behind Tofersen, has generously agreed to provide the drug free of charge to the NHS on a compassionate basis, the treatment requires additional resources—such as skilled staff to administer it via lumbar puncture once a month. These non-drug costs are where the problem lies. Some NHS trusts argue they simply can’t afford these expenses, leaving dozens of patients without access to a potentially life-changing therapy.
And this is the part most people miss: the charity MND Association estimates that at least 20 patients are being denied treatment, even though it could mean the difference between life and death. Alex Massey, the charity’s head of campaigning, policy, and public affairs, emphasizes the urgency: ‘People with MND do not have time to wait. We urge the Government to step in and unblock this impasse.’ With an average life expectancy of just two to five years after diagnosis, every day counts.
MND is a devastating condition affecting around 5,000 people in the UK at any given time. It causes the body’s motor neurones to stop working, leading to progressive weakness, mobility issues, and eventually difficulties with breathing, swallowing, and speaking. High-profile figures like physicist Stephen Hawking, comedian Ronnie Corbett, and rugby stars Doddie Weir and Rob Burrow have all succumbed to this relentless disease. Yet, treatment options remain limited, making Tofersen a beacon of hope for those with the SOD1 gene mutation, which accounts for about 2% of MND cases.
In trials, Tofersen has shown promising results, reducing levels of faulty proteins in cerebrospinal fluid and signs of neuron damage in the blood. For some patients, it has stabilized the disease—and in rare cases, even led to functional recovery. But here’s the rub: only eight out of 24 specialist neurology centres in the UK have chosen to offer the treatment, citing hospital capacity and staffing constraints. This disparity means some patients, like one individual traveling from Northern Ireland to Sheffield monthly, are forced to go to extraordinary lengths for care.
Dr. Brian Dickie, chief scientist at the MND Association, highlights the drug’s potential but also the systemic hurdles: ‘It’s great for those who can access it, but the NHS is stretched to its limits. Even with the drug being free, trusts are struggling to cover the costs of administration.’ This raises a thought-provoking question: Should the NHS prioritize funding for treatments like Tofersen, even if it means diverting resources from other areas?
The National Institute for Health and Care Excellence (Nice) is currently assessing Tofersen’s cost-effectiveness, but the process has been slow and contentious. Meanwhile, patients are left in a heartbreaking waiting game. An NHS spokesman encourages Biogen to complete the Nice appraisal process, which could pave the way for widespread adoption. But with time running out for many, is this enough?
What do you think? Is it fair for NHS trusts to refuse funding for administering a free, life-saving drug? Or should the Government intervene to ensure equitable access? Let us know in the comments—this is a conversation that deserves your voice.
