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Dementia treatments: what matters most to people

Almost one million people are living with dementia today. If nothing changes, one in two of us will be directly affected by the condition in our lifetime – either by developing it ourselves, caring for someone who has it, or both. As the UK’s leading dementia research charity, Alzheimer’s Research UK exists to change that.  

We’re striving for a cure, and for the first time in over two decades, there is hope. The first disease-modifying treatments for Alzheimer’s disease, the most common cause of dementia, could be available in the UK as early as 2024. Other disease-modifying treatments will follow. This means it is all the more urgent that those developing, assessing and prescribing new treatments for dementia must understand what people who might take them really want.  

We commissioned two pieces of research to understand the outcomes from new treatments that matter most to people and the side-effects they might be willing to risk. The two studies together represent the views of nearly 9,500 people across the UK from all walks of life, with a mix of ages, genders, levels of educational attainment. A small number of people had mild cognitive impairment (MCI) or dementia.  

The research

The researchers from the Edinburgh University collected and analysed free text responses from over 5,800 people on what is important to them about their brain health. People who responded to the survey represent those from all walks of life, including those who may be in the earliest stages of dementia before they have any noticeable symptoms. 3% of respondents had a diagnosis of MCI or dementia.  

The research by University Medical Center Groningen surveyed over 3,600 people in the UK to determine the trade-offs they were willing to make between the benefits and risks of hypothetical treatments for Alzheimer’s disease (AD). They explored the highest level of risk people would accept in exchange for delaying the progression of Alzheimer's Disease to a more severe stage by two years. 15% of respondents reported living with memory problems.   

What we learned

  • Among all demographics, family connections, driving, socialising, reading, and friendships rank as the highest priority outcomes for new treatments. These are not included in the Clinical Dementia Rating Scale (CDR) which clinicians employ to evaluate the severity of dementia.
     

“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, cared for both her parents with dementia and is a member of the charity’s Policy Insights and Experience Panel.
 

“I love my reading, 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, lives with posterior cortical atrophy and is a member of the charity’s Policy Insights and Experience Panel.  

 

  • More than half of the respondents were willing to accept very high risks of severe side-effects of a new treatment – this might be due to the irreversible consequences of the progression of dementia, which will lead to less independence and poorer quality of life.  
  • What matters most to people about their brain health and the side-effects they are willing to tolerate can depend on their individual characteristics and health. Many of the disease-modifying treatments in development now are increasingly aimed at the earlier stages of disease progression and a younger population, so it is important outcomes are meaningful and relevant to them. The evidence shows that:  
  • Priorities change as people progress from mild cognitive impairment through to diagnosed dementia, and between Alzheimer’s and other forms of dementia.  
  • People were more likely to accept higher levels of risk in a new treatment if they were young, male, highly educated and lived alone. 
  • Individuals who hold positive beliefs regarding the benefits of medicines are more willing to accept higher risks. On the other hand, those who have negative views about the potential harm and overuse of medicines tend to be more cautious and prefer lower risks.  

Recommendations

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 clinicians about potential treatments.  

Regulators:  

  • The Medicines and Healthcare Products Regulatory Agency (MHRA) should continue in their commitment to developing their use of Patient Reported Outcome Measures to make patient outcomes more central to clinical trials. This would promote the development of dementia treatments that align with patients' needs and preferences. 
  • Patient preference research may highlight situations where the regulators’ value judgments differ from those of patients and where there is greater need for transparent and accessible communication about the rationale behind regulators’ decisions. 

Health technology assessors:  

  • Patient preference data should be used as supplementary evidence in relevant technology appraisals for dementia. Given the limitations of the quality-adjusted life year (QALY) approach and its unintentional prejudice against older people, without patient-reported outcome measures the true value of a drug may not be captured by existing methodologies.  
  • Health technology assessors such as the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) should consider the varying impact and perceived significance of certain outcomes among different subgroups within the dementia population, considering sociodemographic factors and disease stages, when making recommendations.  

Clinicians: 

  • When new disease-modifying treatments for dementia are approved, clinicians will need to actively engage people living with dementia in discussions about the risks and potential benefits of these treatments, striving for more patient-centred and informed decisions. More work is needed to help support clinicians potentially prescribing new treatments with these sorts of conversations, learning from other specialities.  

Industry: 

  • Pharmaceutical companies should ensure clinical trials protocols reflect the outcomes that matter most to people with dementia. This can be achieved by recognising the shifting priorities across different stages of dementia and ensuring that interventions not only focus on clinical endpoints but also improve the overall quality of life for individuals. 

Next steps

We don't have all the answers, but we hope this policy paper sparks more discussions about how we embed what matters to people living with dementia in the wider discussion about new treatments. We need more collaboration across a range of stakeholders to agree how patient reported outcomes are measured and incorporated across the lifecycle of a new treatment, from research through to use in healthcare. We believe that collaborative effort will ultimately result in improved outcomes for individuals living with dementia and their families.