Combining ePRO and Fitbit™ data to better characterize the patient experience during treatment for rare disease
Background and Challenge
- PNH is a rare, chronic, and potentially life-threatening blood disorder
- Clinical trials offer a limited picture of quality of life and fatigue among PNH patients through patient-reported outcomes (PROs)
- Without daily activity data and a correlation to PROs, our understanding of the patient experience will be limited
- We used digital measures and ePROs collected from a digital wearable device to characterize the patient experience during treatment for PNH
Approach
- Enrolled 33 adult patients into a 32-week prospective observational cohort study
- Passively collected continuous activity data for physical activity, sleep pattern, and heart rate
- Weekly ePRO data gathered through online surveys
- Analyzed the relationship between activity data and ePROs
Results
High engagement and high retention throughout 32-week study:
- Achieved 82% survey completion
- Achieved 81% Fitbit collection
Receiving ravulizumab treatment was positively correlated with activity data. This suggests that activity data from wearables devices (i.e., steps, sleep, heart rate) shows promise as a digital marker for treatment response for PNH.
More results available in poster
Evidation Differentiators
- Fast access to a cohort of people with a rare disease who are eager to share their health experience
- Wearable data provided a real-world look at how PNH affects people's sleep and daily life
- By sending out timely surveys to the right individuals, we saw high engagement and were able to activate individuals in clinically meaningful ways