When could aducanumab reach the UK?

Today is a historic day for dementia research with the US Food and Drug Administration (FDA) announcing the first licence of a new drug for Alzheimer’s disease in a generation.

David Thomas, Head of Policy at Alzheimer’s Research UK, explains more about the drug aducanumab – known commerically as Aduhelm – and when it might reach patients in the UK.

“Today marks an important turning point for people with  Alzheimer’s disease in the US who now have the potential to be prescribed the first ever drug treatment to targets the underlying disease itself. The FDA ruling could pave the way for a new generation of drug treatments, but aducanumab still has hurdles to overcome before it could be made available to people in the UK.

The drug has been approved under the FDA’s Accelerated Approval pathway, which includes a requirement for Biogen, the pharmaceutical company that makes the drug, to conduct a further stage four clinical trial.

Aducanumab was tested in people who have mild cognitive impairment (MCI) and very early stage of Alzheimer’s who have had amyloid detected in their brains using a PET scan. However, early information suggests the FDA’s licence appears to be broad enough to cover anyone with the disease, with the regulator highlighting uncertainties around the benefits it may bring.

UK regulators are assessing the data right now

The UK and European regulators are currently completing their own rigorous assessment of the data around aducanumab and are expected to announce their response in the autumn.

Regulators decide whether a drug is safe and effective. However, this is only the first step towards the drug reaching people in the NHS. The health services then needs to decide if it will fund a medicine. Aducanumab would need to be assessed by NICE and the Scottish Medicines Consortium to determine its cost-effectiveness for the health service and therefore whether it is funded for routine use in the NHS. This process looks at the cost of the medicine and also the financial impact it would have on NHS services against the clinical benefit of the medicine, For Aducanumab we don’t expect the process to finish until the middle of next year at the earliest.

Until the outcome of those decisions, the drug won’t be available to NHS patients.

Watch our animation to find out more about this process.

We know from recent experience with COVID-19 vaccines that it’s possible to speed up this process without compromising on the quality of the assessment. So today, our Chief Executive Hilary Evans has written to the Health Secretary Matt Hancock to ask the government to prioritise and accelerate this process, so that people with Alzheimer’s in the UK can be given the answers they need as quickly as possible.

How does aducanumab work?

Aducanumab is designed to target amyloid, one of the hallmark proteins that builds up in the brains of people with Alzheimer’s at an early stage in the disease process. The drug is given through an infusion into a person’s arm.

Existing Alzheimer’s drugs like Aricept can help people day to day by stabilising memory loss but unfortunately they don’t work for everyone and over time their effects wear off. While these drugs have shown real benefit for many people, they do not stop the underlying disease in the brain.

Aducanumab is the first drug to be approved in the US that acts on the disease itself, rather than helping with symptoms.

The drug has been approved using the FDA’s Accelerated Approval pathway, where a licence can be granted based on a drug’s effect on what’s known as a ‘surrogate endpoint’ – a biological marker of disease – rather than its proven effect on people’s symptoms. In Aducanumab’s case, both trials showed evidence that the drug was able to clear amyloid from the brain. Further trials will need to verify that it has the desired effect in terms of slowing decline in people’s thinking, memory and ability to carry out day to day tasks.

Over recent months, the clinical trial results from aducanumab have sparked debate about how regulators judge the effectiveness of any new Alzheimer’s treatment, and how meaningful the impact of a drug on someone’s day-to-day life would need to be to meet that threshold.

The benefits of aducanumab are modest and it will be vital to see how these play out when the drug starts being used in the real-world and what long-term impact it has on people’s lives.

Reason for hope

People with dementia and their families have been waiting almost 20 years for a new drug treatment for Alzheimer’s disease and Alzheimer’s Research UK has been there throughout each high and low.

Aducanumab has not had a straightforward route to approval in the US and regulators across the world have to go through a robust process to be sure that any new drug meets the threshold for both safety and effectiveness. However, today’s announcement has the potential to drive a major step change in the level of priority and investment on drug development for Alzheimer’s and other dementias.

There are currently 126 drugs across more than 150 clinical trials being trialled for Alzheimer’s disease worldwide with many more in the pipeline thanks to initiatives like our Drug Discovery Alliance and Dementia Consortium.

Aducanumab is only designed to help certain eligible people with mild cognitive impairment and early Alzheimer’s disease, and so we must continue to lead the search for life-changing treatments for all dementias, including people with later-stage disease.

Preparing the NHS for new breakthroughs

Delivering any life-changing new Alzheimer’s treatment to patients not only relies on an effective drug but also our ability to identify those in the early stages of disease and the appropriate infrastructure and training to deliver the treatment.

We will continue to work tirelessly to prepare the UK health system so that when that day comes, a new drug can reach those who need it most without delay. We’re already driving this conversation through our Dementia Access Taskforce, developing collaborative solutions to prepare for future dementia treatments.

Research has the potential to transform the lives of everyone affected by dementia and with your support, we will keep working to make more breakthroughs possible.