From 2023 to 2024 Us Against Alzheimer’s published a number of publications and abstracts covering their “Alzheimer’s Disease Patient and Caregiver Engagement (AD PACE)” What Matters Most (WMM) research program. AD PACE was a collaborative effort that included not only people from Us Against Alzheimer’s, but also representatives from Biogen, Lundbeck, Otsuka, BI, Takeda and more.

The objective of AD PACE was to identify symptoms, impacts and outcomes of AD that most mattered to patients and their caregivers. In recent years the FDA, and pharmaceutical companies, have placed more emphasis on picking measures that matter to patients and their families, hence the importance of this type of work.

Article 1 - “Assessing What Matters to People Affected by Alzheimer’s Disease: A Quantitative Analysis” Link to heading

In previous research the authors distilled 83 potential concepts of interests down to 42 concepts of interest. The initial 83 concepts came from 60 in-person interviews, literature and an instrument that measures AD.

In this study a web-based survey was sent out to five groups of individuals, 3 groups were at risk of having AD, or had it and the other 2 groups were caregivers of those with severe AD. The respondents rated 42 symptoms, impacts and outcomes on a scale from 1 (not at all important) to 5 (extremely important). Out of the 42 items half were very important or extremely important. Some of the most important items were: taking medications correctly, being able to stay safe and not feeling down or depressed as the most important items.

274 respondents completed the questionnaire. People living with AD (PLWAD) rated independence highly and not being a burden on their family highly. While caregivers may have also rated independence highly it was driven by “the desire to maintain a patient at home”.

The authors concluded “Our findings complement this prior work, and further demonstrate that symptoms and impacts meaningful to patients with AD and their care partners extend beyond issues with memory, or indeed clinical definitions of ‘‘cognition’’ or ‘‘function.” Specifically, emotional well-being and a desire to maintain independence, overall physical and mental health and safety were rated as important and not typically measured by conventional endpoints in AD trials.

Thus, in addition COA measures like the CDR, ADAS-cog and ADLs pharmaceutical companies should also consider measures that tap into these complementary symptoms/impacts of the disease.

Abstract 1 - “Measuring “What Matters Most” to People Living with Alzheimer’s Disease and Care Partners: A Next Generation Conceptual Model of Alzheimer’s Disease” Link to heading

Their next publication was an abstract presented at the Alzheimer’s Association International Conference in 2023. In this abstract their objective was to contextualize the findings through a conceptual model of disease, verify previous results and identify/missing new concepts.

Methods:

  • Develop a draft conceptual model of the disease
  • Conduct in-depth qualitative interviews with PLWAD to refine the conceptual model

Results:

  • 42 WMM were categorized into 6 domains: 1) thought processing, 2) communications, 3) mood/emotions, 4) daily activities, 5) social life/activities and 6) independence.
  • These 6 domains formed a draft conceptual model.
  • Study participants rated all WMM concepts as important.

Abstract 2 - “Measuring What Matters Most to People Living with Alzheimer’s Disease and Care Partners: What Matters Most Quantitative Research Development” Link to heading

The objective of this abstract was to “evaluate meaningful treatment goals and the impact of the lived experience of WMM concepts.” This will be done via ranking and verification and finally quantitative evaluation of the construct validity and concept domain structure. This abstract was more about the methodology of the ongoing study, rather than presenting results.