Key Summary
- A promising MND drug is not being administered to NHS patients
- Delays stem from staffing and delivery costs
- Families and charities urge urgent government action
Though Medicines and Healthcare products Regulatory Agency (MHRA) has approved Tofersen, used to treat motor neurone disease (MND), NHS patients are yet to access the medicine due to procedural delays and other issues, The Telegraph reports.
Tofersen stops the clumping of proteins in the brain and spinal cord that causes MND in patients with a specific genetic mutation. In some patients it has even reversed the symptoms.
But this breakthrough drug, currently available in Europe and the US, is still being assessed for NHS use by the National Institute for Health and Care Excellence (Nice).
Meanwhile, Biogen, the biotech company behind Tofersen, and the NHS have made an early access deal, making it a free drug.
But some NHS trusts are reluctant to pay the non-drug costs, while some cite lack of skilled staff to administer the medicine.
Tofersen needs to be delivered by a lumbar puncture, an injection into the spinal fluid, once a month.
Hence, many patients are being denied the medicine.
According to MND Association, 20 people are being denied treatment though their condition is very serious.
The charity has urged the government to step in and break the impasse.
MND causes the body’s motor neurones to stop working, creating mobility difficulties and weakness.
The symptoms may vary, but it typically involves weakness in the legs, feet and hands that may make it hard to climb stairs and hold or grip items.
A mutation to the SOD1 gene, which means the disease can be passed down through families, is behind two per cent of MND cases.
