Development of a Qualification Plan for an Activity Monitor-Based Endpoint Measure in Chronic Heart Failure (DDT COA #000114) - ABSTRACT Chronic heart failure (CHF) results when the heart cannot pump enough blood to meet the body’s needs. Approximately 6.5 million people in the United States and 26 million people worldwide have heart failure. Specific CHF symptoms, such as shortness of breath, chest pain, and fatigue, are contributing factors to reduced activity and limitations in ability to perform daily activities. Hence, in conjunction with the assessment of patient-reported symptoms and impacts of CHF, regulators and sponsors have expressed interest in novel measures of daily activity that leverage wearable devices. To help address this measurement opportunity, the CHF Working Group’s proposed activity monitor-based endpoint measure was accepted into the Center for Drug Evaluation and Research’s COA Qualification Program (DDT #000114) on May 3, 2019. It is intended to assess aspects of objectively measured physical activity that reflect a person with CHF’s ability to perform meaningful daily activities. To further this qualification effort, the proposed project will result in the development and submission of a Qualification Plan (QP). A concept elicitation study was conducted to generate qualitative evidence regarding the day-to- day physical activities that bring purpose and meaning to the lives of persons with CHF. Activities frequently reported involved light to moderate physical activity (e.g., cleaning, cooking, doing laundry, and gardening) and walking (e.g., shopping and going to appointments). The next step will be to form an advisory panel, composed of individuals with expertise in the identification of clinically meaningful activity monitor-based outcomes, to review the qualitative study findings and gain consensus on the existing (or proposed novel) activity monitor metric(s) that best reflect/capture the meaningful aspect of health represented by these activities. Once there is agreement on the specific activity monitor-based metric(s) to be evaluated further, we will proceed with development of the QP. The compiled QP will address the CHF Working Group’s research plan for generating the quantitative evidence necessary to support qualification of the activity monitor-based endpoint measure for use in CHF drug development. Our approach includes two aims: (1) convene an advisory panel to provide critical input regarding the results of the qualitative study and gain consensus on the specific activity monitor metric(s) that will be the focus of the QP, and (2) prepare and submit a QP for the activity monitor-based endpoint measure. The long-term result of this project will be a qualified, publicly available activity monitor-based endpoint measure, which will fill a critical gap in the assessment of physical activity in CHF treatment trials for emerging innovative therapies.
