Rob Burrow recently died of Motor Neurone Disease. His father wants to get the new treatment approved for the English NHS. He claims correctly that NICE is rationing care. The article below from Prof Shaw, quite rightly argues for the treatment. Her duty is to her patient. Equally the government duty is to the population, and some treatments (perhaps for many more patients) might not be affordable assuming that we have to ration health care provision. NHSreality believes we have to ration, and overtly. The more we fund new treatments when within patent, the poorer off we are for generic well proven treatments for larger numbers of people. I am not an MND sufferer but i have known friends and patients with the disease. If it was myself or my family with MND i would want treatment. If it was not available for 12 years (until patent expires) I would be angry. For the same reasons we dont let families of murder victims do the sentencing. The treatment will come, its just a question of when. Countries with no interest in fairness and universal care (USA) will approve such drugs first. Insurance based universal care countries (Australia and the EU) will approve next, and the only “free” service (NHS 4 systems) will be slow, unsynchronised and last.
On 20th June 2024 Prof Shaw of Sheffield University Comments: A devastating setback for MND research: Why NICE needs to reconsider its approach to tofersen – Professor Dame Pamela Shaw, Director of the Sheffield Institute for Translational Neuroscience, discusses NICE’s refusal to classify the MND drug tofersen, as a treatment for a highly specialised condition, rather than just a standard drug.
When I started training in neurology as a junior doctor, I thought motor neurone disease (MND) was the cruellest disease in medicine. It always seemed to happen to the nicest people, and it made me feel like a useless doctor because in those days we couldn’t do anything for patients or their families, and we knew nothing about what caused it.
It’s been a long, slow process, but several decades on from my first MND clinics, I really feel that we are making progress. One of the most important treatment milestones in global MND research is the new genetically-targeted therapy, tofersen. Tofersen offers a beacon of hope to those with a SOD1 gene mutation – which affects approximately two per cent of MND patients…….
The Promise of the new treatment. Sheffield University Prof / Dr Shaw
September 2022: ‘Truly remarkable’ drug helps motor neurone disease – BBC News
May 2024: Tofersen given green light by the European Commission (MND Association)
The Biogen – Tofersen drug company website and their recent update (19th April 2024)
The approach in Australia: SOD1 antisense oligonucleotide – Tofersen (ATLAS Preventative Trial)
Daniel Boffey for the Guardian 21st April 2024: People with MND in England and Wales fear losing access to life-extending drug – Exclusive: NHS cost threshold has not been raised by National Institute for Health and Care Excellence since
In The United States the FDA approval has occurred, but then again, the US does not make any attempt to have “universal” cover.
On 13th March 2024 NICE in the UK is put into the “bad news” doghouse by the MND association.
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