Alzheimer's Drug Development Study

The devastating effects that Alzheimer’s Disease (AD) has on both patients and families are sadly well established. As researchers in the pharmaceutical space, we are acutely aware that the current drug development landscape consists of a roller coaster of high hopes juxtaposed with the dashing heartbreak of failed trials. As the key to unlocking a cure continues to elude researchers, drug makers forge ahead in searching for effective treatments to slow progression and relieve symptoms for patients and their families managing the disease today.

At the forefront of this cause are groups ­dedicated to keeping patients and the people who care for them at the center of the conversation. UsAgainstAlzheimer’s (UsA2)’s central vision is to ensure that we understand what is most meaningful to those whose lives are impacted by AD when we make decisions about care and treatment.

In a groundbreaking approach, the Alzheimer’s Disease Patient and Caregiver Engagement (AD PACE) was designed as a precompetitive, cross-sponsor initiative to develop data on treatment outcomes that matter most to those affected by AD with the goal of informing research, approval, access, and care. The team at RTI-Health Solutions was tapped to conduct the What Matters Most (WMM) study to inform drug development from the perspectives of patients and care partners across the AD spectrum. When companies follow a patient-focused drug-development model, their potential treatments are designed to meet the needs of patients and the clinical trials are conducted to demonstrate not only clinical outcomes, but also the importance of those outcomes to patients.

The research was divided into two phases. The first part of the project—characterized as the “What Matters” phase—was designed as a qualitative study to understand which potential treatment-related outcomes matter to people with AD and their care partners. This was followed by the “How Much it Matters” quantitative phase to estimate how much each potential treatment-related outcome matters and which potential treatment-related outcomes matter most.

What we discovered from talking to patients (or their caregivers in the case of moderate or severe disease) is that everyone across the diagnosis spectrum—including people who were at risk for AD or had pathology but did not have impaired cognition, all the way to people with severe AD—experienced impacts related to AD symptoms. This included affecting their emotions and mood or their social lives. Similarly, all patients or caregivers reported issues with cognitive functioning, particularly memory and communication/language, including individuals with no formal diagnosis.

We also observed patterns for when symptoms and impacts appear and the differences across the stages of disease. Patients with AD risk or who have pathology with or without impairment were similar in self-reported symptoms, impacts, and treatment priorities. As patients went from mild cognitive impairment to mild or moderate AD, we began to see changes starting to emerge. Care partners of patients with moderate or severe AD reported a different set of symptoms, impacts, and treatment priorities from the early groups.

Armed with this data, we developed a survey instrument designed to elicit quantitative assessments of the importance of symptoms, impacts, and outcomes of AD and dementia. Respondents at all stages of disease rated many of the survey items as “Very important” or “Extremely important,” including items related to memory, orientation, function, and emotional well-being. This suggests that emotional well-being is likely as important as cognition and function to patients and care partners across the AD spectrum.

Ultimately, this work has been instrumental in increasing our understanding of the experiences and concerns of AD patients and their caregivers. In addition to helping inform the types of therapies that have the potential to provide meaningful benefit to these patients, it also provides a base for additional research that puts AD patients’ needs and preferences at the center of the investigation.