The UK’s health regulator has rejected a drug that can slow the progression of Alzheimer’s disease, saying its benefits are too small to justify the costs of the therapy and close monitoring of patients for signs of “serious side-effects”.
Lecanemab, which is given twice a month, removes sticky clumps of protein amyloid beta from the brain, believed to be a hallmark of the disease. The drug is not a cure. But in clinical trials, the therapy slowed cognitive decline by 27% in early Alzheimer’s patients, compared with a placebo.
The Medicines and Healthcare products Regulatory Agency (MHRA), the UK’s drugs regulator, gave the green light to the drug on Thursday. However, the National Institute for Health and Care Excellence (Nice), the health regulator, simultaneously ruled out offering the drug on the NHS.
It comes weeks after the EU’s drugs regulator also rejected the drug, saying the risk of serious brain swelling did not outweigh its small impact on slowing cognitive decline. It also said the effects of the drug on delaying cognitive decline were small.
The Nice decision is a further blow to the companies behind the drug, Eisai and Biogen, as the treatment faces slow take-up in the US, where it costs about £20,000 per patient per year. It also lays bare the complexities tied to a new class of drugs that have 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 that can treat the neurodegenerative condition rather than its symptoms.
However, the rejection of its use on the NHS by Nice means probably only a small number of patients will benefit in the UK, and will have to access the drug privately.
Hilary Evans-Newton, the chief executive of Alzheimer’s Research UK, said: “Today’s news is bittersweet for people affected by Alzheimer’s disease.
“It’s a remarkable achievement that science is now delivering licensed treatments that can slow down the devastating effects of Alzheimer’s, rather than just alleviating its symptoms. However, it’s clear our health system isn’t ready to embrace this new wave of Alzheimer’s drugs.
“It means that, as things stand, people in the early stages of the disease will be denied access to lecanemab through the NHS, and it will only be available to those who can pay privately.”
Dr Samantha Roberts, the chief executive of Nice, said: “This is a new and emerging field of medicine which will no doubt develop rapidly.
“However, the reality is that the benefits this first treatment provides are just too small to justify the significant cost to the NHS.
“It is an intensive treatment to give to patients involving 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 evaluated the available evidence, including the benefit for carers, but Nice must only recommend treatments that offer good value to the taxpayer.”
According to Nice, clinical trials showed lecanemab can slow cognitive decline by four to six months, but there was little evidence on its long-term effects. It estimates about 70,000 adults in England would have been eligible for treatment.
A public consultation on Nice’s draft guidance will close on 20 September.
Julian Beach, interim executive director for healthcare quality and access at MHRA, said: “Licensing medicines which meet acceptable standards of safety, quality and efficacy is a key priority for us.
“We’re assured that, together with the conditions of the licence approval, the appropriate regulatory standards for this medicine have been met.
“As with all medical products, we will keep its safety under close review, and with a controlled post-authorisation safety study to be undertaken, we will ensure that the benefit risk of lecanemab in clinical use is closely followed up.”
Tara Spires-Jones, professor of the UK Dementia Research Institute at the University of Edinburgh, said the arrival of the drug marked “a turning point” but cautioned that it could be accompanied by “dangerous side-effects”.
Speaking on Radio 4’s Today programme, she said: “It’s the first time we’ve actually been able to slow disease progression at all. So from that standpoint, it’s amazing.
“However, the treatment’s not perfect. It only slows disease progression moderately, and it comes with dangerous side-effects, and people really do need to be monitored very carefully, and only certain people will be able to use the drug. So together, it’s great news, but we have to temper our enthusiasm.”
Asked what the dangerous side-effects were, Spires-Jones said: “Some people who take this drug have brain swelling and bleeding and a few people have died from those side-effects.”
