British health officials have rejected a drug that could slow the progression of Alzheimer's disease, saying the benefits are not big enough to justify the costs of the treatment and close monitoring of patients for signs of “serious side effects”.
Administered twice a month, lecanemab removes sticky clumps of amyloid-beta protein in the brain that are thought to be a hallmark of the disease. The drug is not a cure, but in clinical trials the treatment slowed cognitive decline in early-stage Alzheimer's patients by 27% compared with a placebo.
Britain's medicines regulator, the Medicines and Healthcare products Regulatory Agency (MHRA), gave the drug the green light on Thursday, but the health regulator, the National Institute for Health and Care Excellence (Nice), simultaneously rejected it for availability on the NHS.
A few weeks ago, the EU drug regulator also rejected the drug, saying the risks of severe brain swelling did not outweigh its small benefit in slowing cognitive decline, which it also said was small.
The Nice decision is a further blow to the drug's developers, Eisai and Biogen, who have been slow to introduce the treatment in the US, where it costs around £20,000 per patient per year, and highlights the complexities of a new class of medicine that, while effective for some patients in the early stages, can cause serious side effects.
The treatment, also known as Lukenbi, is approved in the US, China, Hong Kong, Israel, Japan and South Korea. The MHRA's approval means the UK is the first European country to have it approved as a treatment, rather than a symptom, for neurodegenerative diseases.
But Nice's refusal to make it available on the NHS means that only a minority of patients in the UK will likely benefit, and will have to obtain the drug privately.
Hilary Evans-Newton, chief executive of Alzheimer's Research UK, said: “Today's news is bittersweet for those living with Alzheimer's.
“It is a remarkable achievement that science has delivered approved treatments that can not only alleviate the symptoms of Alzheimer's disease but also slow its devastating effects. Yet it is clear that our health care system is not ready for 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 through the NHS, with only those who can pay privately available.”
Dr Samantha Roberts, chief executive of Nice, said: “This is a new area of medicine that will undoubtedly develop rapidly.”
“But the reality is that the benefits of this first treatment are too small to justify the significant costs to the NHS.
“This is an intensive treatment given to patients, requiring skilled staff to attend hospital every two weeks and monitor the patient for any signs of serious side effects, plus the cost of purchasing the medicine.”
“Our independent committee has rigorously assessed the available evidence, including the benefits to carers, and Nice must only recommend treatments that offer good value for taxpayers.”
Clinical trials have shown that lecanemab may slow cognitive decline by four to six months, but there is little evidence about its long-term effects, according to Nice, who estimates that around 70,000 adults in England could be eligible for the treatment.
Public consultation on the Nice draft guidelines closes on September 20th.
Julian Beech, interim executive director for healthcare quality and access at the MHRA, said: “The authorisation of medicines that meet acceptable standards of safety, quality and effectiveness is a key priority for us.”
“We are confident that the appropriate regulatory standards for this medicine have been met, together with the conditions of licence approval.
“As with all medicines, we closely monitor its safety and will closely track the benefit-risk profile of lecanemab in clinical use by conducting controlled post-approval safety studies.”
Prof Tara Spires-Jones, from the University of Edinburgh's Dementia UK Institute, said the drug was a “turning point” but warned it could have “dangerous side effects”.
She told Radio 4's Today programme: “This is the first time we've been able to slow the progression of the disease, so in that sense it's incredible.”
“But this treatment is not perfect. It only slows the progression of the disease modestly, has dangerous side effects, patients need to be monitored very closely, and only a select few can use the drug. So, while this is great news, we must temper our enthusiasm.”
When asked what dangerous side effects there are, Spiers-Jones replied: “Some people who have taken this drug have experienced swelling and bleeding in the brain. Some people have died from these side effects.”
