The UK’s health regulator has rejected a drug that could slow the progression of Alzheimer’s disease, saying its benefits are too small to justify the cost of the therapy and close monitoring of patients for signs of “serious side effects”.
Lecanemab, given twice a month, removes sticky clumps of the protein amyloid beta from the brain, which is believed to be a hallmark of the disease. The drug is not a cure. But in clinical trialsthe therapy slowed cognitive decline by 27% in early Alzheimer’s patients, compared to a placebo.
The Medicines and Healthcare Products Regulatory Agency (MHRA), the UK’s drug regulator, gave the green light to the drug on Thursday. The National Institute for Health and Care Excellence (Nice), the health regulator, at the same time ruled out the drug being offered on the NHS.
It comes weeks after the EU’s drug regulator also rejected the drug, saying the risk of severe brain swelling did not outweigh its small impact on slowing cognitive decline. It also said the drug’s effect on delaying cognitive decline was small.
The Nice decision is a further blow to the companies behind the drug, Eisai and Biogen, as the treatment faces slow uptake in the US, where it costs around £20,000 per patient per year. It also exposes the cdifficulties linked to a new class of drugs which benefited early stage patients but can cause serious side effects.
The therapy, also known as Leqembi, is approved in the US, China, Hong Kong, Israel, Japan and South Korea. The green light from the MHRA means the UK has become the first country in Europe to license the drug which can treat the neurodegenerative condition rather than its symptoms.
The rejection of its use on the NHS by Nice means only a small number of patients in the UK are likely to benefit, and will need to access the drug privately.
Hilary Evans-Newton, chief executive of Alzheimer’s Research UK, said: “Today’s news is bittersweet for people affected by Alzheimer’s disease.
“It is a remarkable achievement that science is now producing licensed treatments that can slow the devastating effects of Alzheimer’s disease, rather than just relieve its symptoms. However, it is clear that our health system is not ready to embrace this new wave of Alzheimer’s drugs.
“This means that, as things stand, people in the early stages of the disease will be denied access to lecanemab by the NHS, and it will only be available to those who can pay privately.”
Dr Samantha Roberts, CEO of Nice, said: “This is a new and emerging field of medicine that will undoubtedly develop rapidly.
“However, the reality is that the benefits of this first treatment are just too small to justify the significant costs to the NHS.
“It is an intensive treatment to give to patients that involves a hospital visit every two weeks with skilled staff needed to monitor them for signs of serious side effects, plus the cost of purchasing the drug.
“Our independent committee has rigorously assessed the available evidence, including the benefit for carers, but Nice should only recommend treatments that offer good value for the taxpayer.”
According to Nice, clinical trials showed that lecanemab could delay cognitive decline by four to six months, but there was little evidence about its long-term effects. It estimates around 70,000 adults in England would have been eligible for treatment.
A public consultation on Nice’s draft guidelines will close on 20 September.
Julian Beach, Interim Executive Director for Healthcare Quality and Access at MHRA, said: “Licensing medicines that meet acceptable standards of safety, quality and effectiveness is a key priority for us.
“We have been assured that, together with the conditions of the license approval, the relevant regulatory standards for this medicine have been met.
“As with all medical products, we will closely monitor its safety, and with a controlled post-approval safety study to be undertaken, we will ensure that the benefit-risk of lecanemab in clinical use is closely monitored.”
Tara Spires-Jones, professor at the UK Dementia Research Institute at the University of Edinburgh, said the arrival of the drug was “a turning point”, but warned it could come with “dangerous side effects”.
She said on Radio 4’s Today programme: “This is the first time we’ve actually been able to slow down the progression of the disease at all. So from that point of view it’s amazing.
“However, the treatment is not perfect. It slows the progression of the disease only moderately, and it comes with dangerous side effects, and people have to be monitored really, really carefully, and only certain people will be able to use the drug. So together it’s good news, but we have to temper our enthusiasm.”
Asked what the dangerous side effects were, Spiers-Jones said: “Some people who use this drug have brain swelling and bleeding and a few people have died from those side effects.”
