MND Drug Postcode Lottery: Lifeline for Some, 'Mental Torture' for Others
A groundbreaking treatment for motor neurone disease has created a stark postcode lottery within the NHS, offering a lifeline to some patients while leaving others in what they describe as mental torture. The drug Tofersen, which can halt the progression of this devastating condition, is available through an early access programme, but inconsistent implementation across NHS trusts means access depends heavily on where patients live.
Eleanor's Story: A Miracle Treatment
Eleanor Dalley, diagnosed with MND in 2019, feared she would not live to see her 50th birthday. MND, which affects the brain and nerves, typically leads to progressive muscle weakness, loss of movement, and death within a few years. However, Eleanor gained access to Tofersen through a manufacturer-led programme, effectively freezing her symptoms.
It is a miracle to be honest, it is like I have frozen in time, said Eleanor, a mother-of-one. While she has lost some leg movement, her condition has not worsened, allowing her to reach milestones she once thought impossible. My worst fear was that I would be dead within a year, she recalled, noting her relief at seeing her daughter grow up and celebrating her 50th birthday. Having this new drug has meant I have achieved all those things. But I am really lucky – and not everybody is getting that same opportunity.
Lillia's Struggle: The Cruel Lottery
In contrast, 19-year-old Lillia Jakeman, diagnosed with MND in August, has been unable to access Tofersen despite being referred. Her stepmother Rachel, a GP with 30 years of experience, described the situation as very cruel and unjust. It is like a carrot that is dangled, and then it is taken away, she said.
Lillia now uses a wheelchair, with her upper arm weakness affecting her ability to engage in beloved activities like Lego and artwork. Rachel emphasised that Tofersen must be administered early to preserve function, stating, Every day matters. Lillia herself calls the uncertainty mental torture, waking each day unsure if she will lose another function.
Systemic Barriers and Political Action
The issue stems from NHS resource constraints. While Biogen, the manufacturer, offers Tofersen for free, some trusts lack the staff or facilities to administer the regular spinal injections required. This has resulted in at least 20 people being denied access, with four reportedly dying while awaiting treatment, according to the All Party Parliamentary Group on MND.
Caroline Nokes, Lillia's local MP and a senior Conservative backbencher, argues that extending access to this small group – Tofersen targets the inherited SOD1 form of MND, representing just 2% of cases – should not be difficult. She joined Labour MP Ian Byrne and Lillia's family in delivering a petition to Downing Street, urging government intervention.
Broader NHS Challenges
The case highlights wider concerns about NHS red tape in adopting new treatments, despite significant investment in UK life sciences. Tofersen received UK marketing authorisation in July, but the National Institute for Health and Care Excellence (NICE) will not begin its evaluation for routine NHS funding until March, with no clear timeline for completion.
Professor Ammar Al-Chalabi, a consultant neurologist at King's College Hospital, criticised the system, noting, The government would like us to be a science superpower, and for motor neurone disease, we really are. And this drug is available but it is not available to patients despite the scientific breakthrough, what does that say? He stressed the need for a simpler pathway to ensure treatments reach patients promptly.
A Department of Health and Social Care spokesperson stated commitment to improving MND care, confirming that NICE is assessing Tofersen for routine NHS funding. If recommended, NHS England will work to make it available rapidly. However, for patients like Lillia, time is of the essence in this postcode lottery of hope and despair.
