Holding on to what matters and risking a lot to do so – what do people want from new dementia treatments?

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By Isolde Radford | Thursday 28 September 2023

Today marks the one-year anniversary of a historic moment in dementia research. The pharmaceutical company Eisai announced that their drug, lecanemab, had shown small but clear benefits for people with early Alzheimer’s in a rigorous clinical trial. In the year that has followed, we’ve seen this drug be approved for use in the United States, and most recently, Japan – with UK regulators due to make a ruling early next year. We’ve also witnessed a second Alzheimer’s drug, donanemab, successfully slow memory decline, by a similar degree, in another well-conducted clinical trial.

These long-awaited milestones, albeit exciting, have prompted discussions on safety, with some participants in the trials having experienced significant side effects, including infusion-related reactions and brain swelling. And in rare cases, some participants sadly died.

So as research continues to progress, there has never been a more pressing need to understand what outcomes people want from new dementia treatments, and the level of risk they’d be willing to take in order to benefit from such drugs.

This is especially true for people in their mid-life (aged 40-60), as drugs are increasingly aimed at the earlier stages of disease progression and a younger population.

To learn more, Alzheimer’s Research UK commissioned two research studies from leading universities, together representing the views of nearly 9,500 people across the UK from all walks of life, including people living with mild cognitive impairment or a form of dementia.

 

What outcomes are worth the risk?

In the first study, carried out in collaboration with the University of Edinburgh, we asked 5,800 people what is important to them about their brain health, and the outcomes that would matter most from potential new dementia treatments.

The findings, published in the Journal of Prevention of Alzheimer’s Disease (JPAD), found that the five most important things people would want treatments to help them keep in their everyday lives were family connections, driving, socialising, reading, and friendships.

Yet we know that collecting information about these is not standard across the work that drug regulators, industry, and clinicians do:

  • None of these outcomes are included in the Clinical Dementia Rating (CDR) scale, a tool which is commonly used to determine the stage of a person’s dementia in clinical practice.
  • In research, the pharmaceutical industry often focuses more on clinical endpoints rather than improvements on the overall quality of life of those living with dementia.
  • Regulators and health technology assessors who, together, are responsible for judging the efficacy and cost-effectiveness of new drugs, often do not consider these sorts of outcomes.

 

“Living rurally, driving is a major issue, my parents were three miles from the nearest town and 45 minutes from the nearest hospital. Both of my parents had to stop driving, my father due to Parkinson’s dementia and then my mother who went on to display symptoms of Alzheimer’s disease. Suddenly they were completely isolated. They were really reluctant to let go of the car as it signifies independence.” Shelle Luscombe, who cared for both of her parents with dementia.

 

“I couldn’t do without my reading. I’ve always read… I would really miss my reading if I couldn’t do it.’’ Martin Robertson, who is living with a rare form of dementia called Posterior Cortical Atrophy.

 

 

 

 

 

What level of risk is acceptable?

In a separate piece of work, we funded researchers at University Medical Centre Groningen to survey over 3,600 people to determine the highest level of risk people would be willing to accept in order to delay by two years their dementia from advancing to a more severe stage.

More than half of the people surveyed were willing to accept very high risks of serious side effects.

Their findings, also published in JPAD, showed that people are willing to accept greater risks from dementia treatments compared to what is seen for other chronic diseases. This is perhaps due to the irreversible nature of dementia as it progresses, and the current lack of any effective treatments to slow the disease. And notably, respondents’ backgrounds, whether having memory problems and/or being a caregiver or not, had no effect on their risk acceptance.

Attitudes to risk were also influenced by whether people lived alone and their beliefs about the benefits and potential overuse of medicines. For example, being younger, male, more highly educated, living alone, or having positive views about the general benefit of medicines, were all associated with higher levels of risk acceptance.

Prioritising the perspectives and preferences of individuals living with dementia

It’s clear that people want to hold on to the things that matter most to them in life, like their friendships and independence. The things we take for granted in everyday life quickly become priceless when faced with the prospect of developing dementia.

We believe that the results of these studies provide useful insights for regulatory and clinical trials discussions, as well as helping to inform the conversations between people with dementia and their clinicians about potential treatments, should these drugs get approved for routine use. This should enable doctors to make more informed recommendations, and support individuals in making decisions that align with their values and priorities.

We don’t have all the answers, and it’s important to note that in our research only a relatively small percentage of respondents reported having mild cognitive impairment, early-stage dementia, and/or memory problems. Nevertheless, these insights are crucial as we know many of the people surveyed are likely to be affected by dementia in the future – whether developing it themselves, caring for someone with it, or both.

We need more collaboration among stakeholders to agree how the outcomes that matter most to people with dementia and their attitudes to risk are appropriately measured and incorporated throughout the research and healthcare process.

If you’d like to know more about this research, or to share your views on outcome preferences and risks, email policy@alzheimersresearchuk.org.

 

Related reading:

  • We’ll be discussing this in more detail at our Clinical Conference on 10th Nov, join us!
  • Read our full position statement on this work here.

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About the author

Isolde Radford

Policy Manager