Tris Dyson is the Managing Director of Challenge Works, which is part of Nesta, the nnovation foundation. He designs and runs global challenge prizes to incentivise innovation in healthcare, society, and the environment
Last year, the Government announced £100 million for AI-powered drug discovery. That’s in addition to the £75 million invested in precision medicine and £14 million for rare disease research by the Medical Research Council. There’s another £20 million from UKRI in precision medicine, and £22 million for genomic research. This list goes on.
Millions upon millions are being targeted by the Government at advancing new drug discoveries and rare disease treatments, often using AI. This can lower the cost of new drug development and, in turn, the cost of treatment for the NHS. However, a decision last month by the National Institute for Health and Clinical Excellence (NICE) threatens to undermine these massive investments.
Motor Neurone Disease (MND) is one condition that could benefit massively from precision medicine discoveries. Though many assume it’s a rare disease, it’s more common than you think. 1 in 300 people will develop it in their lifetime.
Last year I was diagnosed with Motor Neurone Disease at age 44. I probably only have a few years left with my partner and our baby daughter. Progressively, the signals from my brain to my muscles are being blocked by proteins generated by my own body. This affects my movement, my speech, my ability to swallow water and food, and eventually, my ability to breathe. There is no cure and to date the only medicines available extend lives by a few precious weeks.
Tofersen is a new precision drug that has shown enormous promise in slowing the progression of the disease for a small subset of MND patients. They have a rare inherited form of the disease and represent around 2 per cent of cases. However, Tofersen’s adoption in the UK has been effectively torpedoed.
These patients have a gene known as SOD1, and while Tofersen doesn’t offer a cure, it is one of the first drugs to show significant promise in improving and extending lives. In trials, patients have reported better mobility and lung function after 12 months. Given the existing treatments, this is nothing short of miraculous.
The mutated SOD1 gene produces toxic proteins which block the signals from the brain to the muscles. Tofersen is a small string of DNA letters which bind to the specific molecules in the SOD1 gene to prevent the production of those harmful proteins.
There are maybe only 60-100 people living with SOD1 MND in the UK at any given time. This is the promise of precision medicine, developing new targeted drugs designed specifically to meet the needs of small patient cohorts.
In a baffling turn of events, NICE has recommended to the MHRA (the medicines regulator) that Tofersen should only be authorised in the UK if it is tested in the context of all MND patients, rather than the 1-2 per cent for whom the drug has been designed for at a genetic level.
This decision has doomed the approval process for Tofersen to failure. When a precision medicine is developed for a small group of patients, testing it against the 98 per cent for whom we know it is not designed will only produce a result that says the drug is not value for money and does not work.
Tofersen has been approved by the FDA in the United States and recommended for approval by the European Medicines Agency. This makes the UK an outlier because NICE and the MHRA will not assess it as the targeted drug that it is. It is a bitter irony that Tofersen has largely been developed and clinically trialled in the UK, yet British patients are unlikely to benefit.
There is absolutely no doubting the current government’s commitment to precision medicine – it is investing heavily in research and development to make the UK a world leader. There is a clear direction of travel for medicine (particularly for neurological conditions) supported by AI drug discovery. However regulatory red tape is undermining that investment and patients will be the collateral damage.
As happens all too often in the British health landscape, ambition comes to a clunking halt when it comes up against the bureaucratic institutions that should be accelerating innovation for patients.
The UK doesn’t just talk a big game about precision medicine, the Government and pharmaceutical industry are real players, ploughing millions into the space. However, the regulator seems ill-equipped or just incapable to support it. Unless something changes, we risk killing a new industry and leaving patients behind in the process – there must be a middle way to support drugs for disease sub-groups that are rare.
Drugs like Tofersen are a beacon. Precision drugs for many other conditions are starting to come through thick and fast and producing astounding results.
I don’t have the SOD1 mutation, Tofersen will not work for me, but I can tell you, we need many more drugs like it. Precision medicine, genomic medicine, and AI-discovered drugs are the future – not just for MND, but for cancer treatment, dementia, diabetes, and rare genetic conditions. It is going to transform how we treat and cure disease. The UK’s investments will only pay off if those treatments reach patients.
