At the ISCTM 20th Annual Scientific Meeting, Senior Research Scientist Jen Blankenship, PhD, presented our ongoing work to understand patient perspectives on how mild AD impacts their daily life and function and identify the meaningful aspects of health that are measurable with low-burden wearable actigraphy sensors. Download the poster and read the abstract below.
The Methodological Issue Being Addressed
Most Alzheimer’s disease (AD) clinical research has focused on developing drugs that support early intervention and delay progression beyond mild forms of the disease. Because maintaining functional independence is of high importance to patients with AD, measures of function are frequently included as endpoints in drug development trials.
However, current approaches to assess functioning in AD are captured episodically, burdensome to patients, and fail to capture the complex challenges that patients must navigate daily.
Introduction
FDA’s Patient-Focused Drug Development initiatives require that new evidence-generation tools are rooted in meaningful aspects of the patient experience. Measures of physical behavior, like walking, derived from wearable actigraphy sensors have the potential to measure how patients function in real-world environments and support AD drug development efforts.
Furthermore, these measures can capture aspects of mobility and everyday functioning that patients find meaningful and want to preserve. However, before digital endpoints can be implemented, a foundational understanding of the measurable concepts of functioning that are meaningful to those with mild AD, the target population of many AD drug development clinical trials, is needed.
Therefore, the objectives of this study are to (1) understand patient perspectives on how mild AD impacts their daily life and function and (2) identify the meaningful aspects of health that are measurable with low-burden wearable actigraphy sensors.
Methods
Informational interviews will be conducted with 8 older adults (≥65 years) who self-report a mild AD diagnosis. Interviews will explore patients’ perspectives on the impact of AD on their daily routines and their ability to function independently and specific measurable concepts related to functioning. Participants will be asked about concerns about their future ability to function independently.
Interviews will conclude by eliciting participant feedback on aspects of life that should be considered in future clinical trials to more wholistically capture their ability to function in real-world environments. Interview transcripts will be analyzed using grounded theory. Transcripts will be coded to identify themes reflecting patients’ experiences with AD and their ability to function independently.
Based on the themes that emerge, a conceptual framework will be developed that maps specific patient experiences to health concepts and outcome measures.
Anticipated Results
This study is ongoing. This poster will discuss the benefits, challenges, and strategies to defining the meaningful aspects of health and identifying measurable concepts of function for patients with mild AD. We will present our preliminary qualitative interview findings in this poster (n=5 anticipated).
Our working hypothesis is that maintaining the ability to perform walking behaviors and mobility will emerge as meaningful aspects of health that are critical to maintaining independence. The precise measurable outcomes will be dependent on patient experiences. Data collected in this study will inform future research exploring the validity and generalizability of our concept model, which will be the basis for future outcome development and regulatory qualification.
Finally, this poster will comment on how our approach to collecting patient experience data prior to initiating a clinical trial can inform other aspects of drug development and ultimately improve patient outcomes.
Conclusions
Data collected from this study will provide foundational evidence to inform the development of novel patient-centric digital endpoints of functioning for use in AD clinical trials. Systematically evaluating the aspects of daily living that are important to patients and measurable with digital health technologies is a critical first step supporting our patient-centered approach to developing and implementing ecologically relevant tools for AD clinical development.
Jen Blankenship, PhD
Jen Blankenship, PhD, is a clinical and translational scientist with a deep interest in wearable technology (e.g., continuous glucose monitors and accelerometers).
LinkedIn Profile for Patient-Centric Digital Endpoints of Function for AD Research