A new Alzheimer’s drug has been rejected for widespread use in the health service in England after the health spending watchdog said it “does not currently demonstrate value for the NHS”.
The news comes as the UK’s medicines regulator said donanemab could be licensed for use in the UK.
It is the second disease-modifying Alzheimer’s drug to be rejected by the National Institute for Health and Care Excellence (Nice) in a matter of months.
One charity said the news is a “frustrating setback” for people with Alzheimer’s disease.
Alzheimer’s Research UK said the announcement “also risks signalling that the UK is no longer a good place to launch new dementia treatments”.
Donanemab, manufactured by pharmaceutical giant Eli Lilly, is a targeted antibody drug which slows the early stages of Alzheimer’s.
The drug, and another new drug for Alzheimer’s called lecanemab, have been billed as a huge step forward in research because they target a known cause of the disease, rather than just treating the symptoms.
Nice’s director of medicines evaluation, Helen Knight, said: “For Nice to be able to approve a medicine for use in the NHS it must provide additional benefits to patients, and it must also represent a good use of NHS resources and taxpayers’ money.
“Our independent committee looked at all the available evidence, including the benefits for carers. This shows donanemab could slow down cognitive decline by four to seven months, but this is just not enough benefit to justify the additional cost to the NHS. The cost-effectiveness estimate for donanemab is five to six times above what Nice normally considers an acceptable use of NHS resources.
“I know this will be disappointing news, but this is an emerging field of medicine and there are other treatments being developed.”
It has been estimated that around 70,000 adults in England would have been eligible for treatment with donanemab.
Nice’s new draft guidance says that more evidence is needed on the clinical and cost-effectiveness of the drug.
“The costs of providing donanemab, including the monthly infusions and intensive monitoring for serious side effects, balanced against the relatively small benefit it provides to patients, means it cannot currently be considered good value for the taxpayer,” Nice said.
It said that it did not recommend donanemab for use on the NHS “as the benefits it offers patients are too small to justify the additional costs”.
Nice added that there are “significant uncertainties to how much benefit donanemab provides, and how long this lasts for after stopping treatment”.
In August, lecanemab was approved by the medicines regulator, the Medicines and Healthcare products Regulatory Agency (MHRA), making it the first drug of its kind to be licensed for use in the UK.
But Nice quickly said the benefits of lecanemab, made by Eisai and sold under the brand name Leqembi, are “just too small to justify the significant cost to the NHS”, and in draft guidance it said the drug should not be rolled out for widespread use across the NHS.
Concerns were raised that the decision would lead to a two-tier system for Alzheimer’s patients – with those who can afford the drug able to access it privately while others who rely on NHS care were left without.
Both donanemab and lecanemab bind to amyloid, a protein which builds up in the brains of people living with Alzheimer’s disease.
By binding to amyloid, the drugs are designed to help clear the build-up and slow down cognitive decline.
Evidence suggests people get the most benefit if they are given the treatment at an earlier stage of the disease.
Clinical trials testing the safety and efficacy of donanemab, also known as Kisunla, found it could slow the rate at which memory and thinking get worse by more than 20%.
Results also suggest the drug leads to a 40% reduction in the decline of everyday activities such as driving, enjoying hobbies and managing money.
The drug, which is given to patients in an intravenous drip once every four weeks, does carry a risk of side-effects – some of them serious, including brain swelling and micro brain bleeds.
Both donanemab and lecanemab have been approved for use in the US, though the European medicines regulator rejected lecanemab earlier this year.
Julian Beach, from the MHRA, said the drug’s safety will be kept under “close review”, adding: “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.”
Hilary Evans-Newton, chief executive at Alzheimer’s Research UK, said: “Today’s announcement marks another frustrating setback for people affected by Alzheimer’s disease.
“We finally have two new treatments licensed in Britain for Alzheimer’s, but it’s incredibly disappointing that NHS patients in England and Wales won’t receive them.
“While these drugs are not cures and come with risk of side-effects, trials show they are the first treatments to slow the decline in memory and thinking skills linked to Alzheimer’s, rather than just alleviating symptoms.
“We’ve written to the Health Secretary again, calling for his leadership to bring together Nice, NHS England and industry so that people with dementia in the UK aren’t left behind.
“Today’s decision also risks signalling that the UK is no longer a good place to launch new dementia treatments.”
Professor Fiona Carragher, chief policy and research officer at Alzheimer’s Society, said: “Disease-modifying therapies like donanemab and lecanemab offer a new horizon of hope in the fight against dementia.
“MHRA’s approval of donanemab marks another milestone in this journey, but it comes alongside a draft Nice decision not to recommend donanemab for use on the NHS. While this is disheartening, we respect the decision of the regulator.”
Manufacturer Lilly welcomed the news that the drug has been licensed in the UK and said it “remains confident in the clinical and cost-effectiveness of donanemab and the value that it can bring to patients and to the NHS”.
Chris Stokes, president and general manager of UK and northern Europe at Lilly, said: “After more than 35 years of commitment to scientific progress in Alzheimer’s disease, we’re delighted that donanemab has been authorised in Great Britain for eligible adults who need effective treatment options.
“Donanemab is part of a new class of medication and has demonstrated meaningful results for people with mild cognitive impairment and mild dementia due to Alzheimer’s disease.”
The MHRA licensed donanemab for the early stages of Alzheimer’s disease in adults who have “one or no copies of the apolipoprotein E4 gene (ApoE4)”.
Around 15% of people diagnosed with Alzheimer’s disease have two copies of this gene – and would therefore not be eligible.
The MHRA said those with two ApoE4 genes did not demonstrate a significant slowing in decline in clinical trials and they were also at higher risk of side effects.
