Alzheimer’s sufferers in Britain will now be able to buy the first drug shown to slow the disease but the NHS cannot afford to provide it.
The Medicines and Healthcare Products Regulatory Agency (MHRA) has approved Lecanemab, which trials indicated is the first-ever disease-modifying drug which can slow mental decline.
However, the National Institute of Health and Care Excellence (NICE) also announced that its benefits were too small to justify the costs to the NHS.
Alzheimer’s Research UK said it was “deeply disappointed” the drug had been rejected on the NHS and has written to Health Secretary Wes Streeting to provide leadership on the issue.
David Thomas, head of policy at Alzheimer’s Research UK, said: “This is a bittersweet moment for people with dementia. That the news that the MHRA has given a licence is a milestone and it moves us one step closer to tackling the culprit that’s caused by dementia.
“But the decision by NICE to not recommend this drug as cost effective is deeply disappointing and means that people who could benefit from this treatment, in England, will not be able to do so. There is now a licensed drug [deemed] safe and effective by the regulator. That needs to be available to NHS patients and not just those with the ability to pay.”
The US list price of the infusions is around £23,000 a year privately, but there would be additional costs for scans and tests which could take annual costs to around £30,000. It would be cheaper for the NHS if it were to fund it but this cost is confidential.
The drug – developed by Eisai and sold under the brand name Leqembi – is administered every fortnight via an IV drip and is already licensed in the US . Trials showed that it slowed cognitive decline by 27% in sufferers if they were diagnosed early enough before significant memory loss.
But it was rejected by the European Medicines Agency in July after the regulator considered its benefits were relatively small and did not outweigh the risk of serious side-effects such as bleeding and swelling in the brain.
Around 70,000 sufferers in England would have been eligible. Helen Knight, director of medicines evaluation at NICE, said: “We recognise that this draft recommendation is going to be deeply disappointing to those affected.”
Dr Samantha Roberts, 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.”
Lecanemab was hailed by experts as “the beginning of the end” for Azheimer’s as it appeared to approve the so-called amyloid hypothesis. Scientists had hoped that clearing the amyloid protein build-up from the brains of people with early-stage Alzheimer’s could halt the progress of the disease.
Julian Beach, MHRA executive director, said: “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.”
A similar drug called donanemab, which is developed by Eli Lilly, is also being assessed for approval in the UK. NHS England had estimated that up to 280,000 patients might theoretically benefit from the new treatments.
Helen Knight, director of medicines evaluation at NICE, said: “Lecanemab and other similar treatments for Alzheimer’s disease now coming on stream have prompted a great deal of debate about the prospects of being able for the first time to slow the effects of a condition that is progressive, life-limiting, complex and distressing.
“For NICE to be able to approve a medicine for use in the NHS it must not only provide benefits to patients, but it must also represent a good use of NHS resources and taxpayers’ money.
“Lecanemab provides on average 4 to 6 months slowing in the rate of progression from mild to moderate Alzheimer’s disease, but this is just not enough benefit to justify the additional cost to the NHS.”
Prior to being given the drugs patients will need to have a baseline MRI scan and then either a Pet-CT scan or lumbar puncture to confirm Alzheimer’s.
An audit published earlier this month showed only 2.1% of people with dementia undergo the specialist investigations that would be required.
Experts have repeatedly warned the NHS does not have anywhere near enough diagnosis capacity to catch the disease early enough for the drugs to have benefit in most cases.
The MHRA set some exclusions including patients who carry the APOE gene, which is carried by about one quarter of people, because they are at greater risk of dangerous side effects. In a small number of trial participants who experienced bleeds and swelling on the brain the side effects were fatal.
According to Nice, clinical trials showed lecanemab can slow cognitive decline by four to six months, however there is little evidence on its long-term effects. A public consultation on Nice’s draft guidance will close on September 20.
NHS England said it has a “dedicated national team” in place to plan for the roll out of new disease-modifying treatments for Alzheimer’s. Medical director Prof Sir Stephen Powis said: “The NHS now awaits a final decision from the NICE that will look at the clinical benefits and cost-effectiveness of lecanemab and determine if it should be routinely offered by the NHS in England.
“Lecanemab is the first disease modifying treatment for Alzheimer’s disease with a market approval in the UK, and to ensure the health system is prepared for future advances in treatments, a dedicated NHS team is also looking ahead to 27 other drugs which are currently in advanced clinical trials that could be potentially approved by 2030.”
Hilary Evans-Newton, chief executive of Alzheimer’s Research UK, said: “Further negotiations between Nice, Eisai and the NHS may offer a way forward. But the heartbreaking reality is that those who could benefit from drugs like lecanemab don’t have time to wait. We’ve written to the Health Secretary, Wes Streeting, urging him to act, and to find a solution so that people with dementia in the UK don’t continue to miss out on innovative treatments.
“As well as considering how to make drugs like lecanemab available, there also needs to be a real focus on improving the way dementia is diagnosed within the NHS. The shocking reality is that one in three people in England with dementia never get a formal diagnosis, leaving them unable to access the care and support they need.”
Ms Evans-Newton said it is “deeply disappointing” that 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”. She added: “Lecanemab represents the beginning of a sea change in how diseases like Alzheimer’s will be treated in future.
“There are now more than 160 trials under way, testing over 125 experimental treatments for Alzheimer’s across the globe, including 30 in late-stage trials. Despite today’s frustrating news, it really is a matter of when, not if, new treatments become available. What people living with Alzheimer’s disease and other forms of dementia desperately need are new medicines that improve their lives.”