NHS Refuses to Fund Life-Saving Motor Neurone Disease Medication Despite Free Supply From Drugmaker

Individuals living with motor neurone disease (MND) in the United Kingdom are being denied access to a breakthrough treatment, tofersen. This comes despite the biotech firm Biogen is supplying the drug free of charge while it undergoes formal evaluation for standard use within the National Health Service (NHS).

The innovative drug, approved by the UK's Medicines and Healthcare products Regulatory Agency (MHRA), targets a rare genetic form of MND. It has been hailed as the first effective therapy in decades for this condition.

However, NHS trusts are refusing to cover the non-drug expenses linked to its administration, including staffing, specialised equipment, and hospital resources, resulting in numerous eligible patients being unable to access the treatment.

While some specialist centres have offered tofersen through Biogen's Early Access Program (EAP), others have said they can't because they don't have enough space or lack of commissioned services. Campaigners and patient groups report that at least 20 eligible patients have been denied treatment, even though there is an urgent need for it.

This deadlock has drawn sharp criticism from charities, healthcare professionals, and advocates who warn that each delay could lead to irreversible effects for individuals living with MND.

What Is Tofersen and What Is It For?

Tofersen is a new medicine designed to treat a rare genetic form of MND caused by a mutation in the SOD1 gene. This mutation results in a detrimental protein that harms nerve cells, causing muscle weakness and various other symptoms.

Tofersen works by decreasing the production of this harmful protein, helping in the prevention of nerve damage. In certain patients, there have been instances where symptoms have stabilised or function has shown improvement.

The medication is administered monthly via a lumbar puncture, an injection into the spinal fluid, requiring the expertise of trained specialists in a hospital setting.

The drug's approval by the MHRA marked a major milestone in MND care and followed similar authorisations in the United States and European Union.

However, wider NHS funding remains contingent on a final decision from the National Institute for Health and Care Excellence (NICE), which assesses cost‑effectiveness before a treatment can be recommended for routine use.

Understanding Motor Neurone Disease (MND)

Motor neurone disease or MND is a rare and progressive neurodegenerative condition that damages motor neurones, which are the specialised nerve cells that control voluntary muscle movements.

As these cells decline, individuals face escalating muscle weakness, twitching, cramps, and stiffness, rendering daily activities like walking, speaking, swallowing, and even breathing more challenging.

In the UK, there are about 4,000 individuals currently living with MND, with around 1,500 new diagnoses occurring annually, according to NICE.

The progression of MND differs from person to person, yet it typically develops swiftly and relentlessly. Some individuals, including the renowned theoretical physicist Stephen Hawking, have managed to live for decades following their diagnosis.

Meanwhile, numerous patients usually encounter a life expectancy ranging from two to five years after the initial appearance of symptoms. A notable percentage pass away within a year of being diagnosed.

Today, MND remains without a cure, and treatment choices have been scarce. Prior to tofersen, the sole licensed medication in the UK is riluzole that provides only slight improvements in life expectancy.

Calls for Government Action and NHS Reform

Charities and advocacy organisations, such as the MND Association, are calling on the UK Government and NHS leaders to guarantee fair access to tofersen for all qualifying patients. They say that the small group of people who could benefit (about 60 to 100 people in the UK) is not worth the extra resources needed to provide the treatment, especially since it could have a life-changing effect.

Alex Massey, who leads the campaigning efforts at the MND Association, told the Telegraph: 'We are talking about a small amount of extra resource for something that could be the difference between life and death. People with MND do not have time to wait, so we would urge the Government to step in and unblock the impasse.'

Campaigners also highlight the need for clearer NHS pathways for rare disease treatments, ensuring that new medicines can reach patients quickly while waiting for full funding approval. As the NHS awaits NICE's final decision on tofersen, families and healthcare professionals continue to press for urgent action to prevent further delays in care.