At the Alzheimer’s Association International Conference in July, UsAgainstAlzheimer’s (UsA2) released “Preparing Us for a Cure,” three research products on key areas of Alzheimer’s disease that impact the lives of patients, caregivers, and communities.
For the Alzheimer’s Disease Patient and Caregiver Engagement (AD PACE) Initiative What Matters Most research product, RTI Health Solutions partnered with UsA2 to look at preferences and priorities for treatment outcomes for those across various stages of Alzheimer’s disease and their caregivers.
“There are almost 100 drugs currently in the Alzheimer’s pharmacological pipeline. We must ensure that as these new therapies are being developed, patients are able to know whether the drug is delivering an outcome that is most meaningful to them and their caregivers and whether the tools used to measure those outcomes are sufficiently sensitive to detect the changes viewed by them as meaningful,” said George Vradenburg, chairman and co-founder of UsA2.
With that in mind, AD PACE has been initiated by patients and caregivers — working with industry, researchers, patient and care partner representatives, non-profit groups, the FDA, and other government agencies — to conduct deep and scientifically rigorous research to understand what matters most to patients and caregivers and to translate that understanding into new or modified measurement tools of utility to inform regulatory authorities. “In the future, information ascertained from AD-PACE will ultimately help to inform trial design, regulatory endpoints and outcomes measures, and prepare us for a cure. Further, AD PACE intends that this research will inform payers’ coverage and reimbursement decisions so families can obtain access to treatments when they become available,” said Vradenburg.
- While all participants reported short-term memory issues, the majority of those with underlying Alzheimer’s pathology – but no clinical diagnosis of Alzheimer’s – reported specific problems like misplacing common objects and increased reliance on reminders and lists, and all reported symptoms related to memory and forgetfulness. Individuals in this “pre-symptomatic” subset were not expected to report such memory complaints, which are more correlated with a formal Alzheimer’s diagnosis, such as Mild Cognitive Impairment, than with general memory concerns expected in the population.
- Participants reported specific issues such as forgetting dates or appointments, or forgetting to take medications with increasing frequency in relation to disease severity.
- Participants diagnosed with mild Alzheimer’s disease (middle-stage) reported the most varied symptoms of any subject group, compared to those with unimpaired cognition but evidence of Alzheimer’s pathology and late-stage participants whose symptoms were highly convergent within their respective groups. The broad heterogeneity in the group diagnosed with mild Alzheimer’s may suggest differences in how clinicians view the diagnosis of “mild Alzheimer’s disease,” a finding of significance for drug development aimed at this population.
- “Improving and restoring memory” and “stopping disease progression” were the most important treatment outcomes across all participant groups – underscoring the need to better understand how these two very different patient outcomes are weighted by individuals across the multiple stages of the disease. Caregivers of people with advanced disease also reported “stopping the progression” as second only to restoring memory.
- Traditional neuropsychological assessments may measure various aspects of cognitive functioning often impaired by Alzheimer’s disease, but it is unclear whether they can measure the often subtle changes that are important to individuals with Alzheimer’s and care partners (e.g., decreased socialization due to concerns about forgetting names or losing track when speaking to others; depressed or anxious thoughts due to changes in memory; increased dependence on others for even basic chores).
View the research here:
Findings from the Alzheimer's Disease Patient and Caregiver Engagement (AD PACE) initiative's what matters most qualitative study. Vradenburg G, DiBenedetti DB, Hauber B, Slota C, Wronski SL, Comer M, Callahan LF, Winfield J, Rubino I, Krasa HB, Hartry A, Wieberg BA, Kremer IN, Lappin D, Martin AD, Frangiosa T, Biggar V.