Mobile Health Outcomes
FED is providing support to Roxanna Bendixen, an expert occupational therapist at the University of Pittsburgh, to develop mobile health outcomes using emerging network-enabled personal health devices (specifically the Microsoft Band).
Roxanna and FED realize that there are many, many clinical trials for DMD (thanks in large part to the previous efforts of FED). This creates many opportunities for DMD boys and their families to participate in critical research studies, and helping to define how well a drug works for DMD.
But participation in clinical trials can also be a substantial burden to the patient and family. Loss of time in school, loss of work hours by the parents, travel to remote clinical trial sites, arranging care of other children, and additional expenses that may not be fully covered by the clinical trial funding.
There are also concerns regarding the clinic-based measurement of patient strength and function (called ‘outcome measures’). Typically a drug is determined to work or not based on just a few visits to a clinic, using tests that may take just seconds to perform (time to climb four stairs, time to run 10 meters, etc.). Do these short and isolated tests really reflect how the drug is positively (or negatively) influencing the daily quality of life and daily functioning of the patient in their home and community?
The development of mobile health outcomes may be a solution to these problems. Newly emerging mobile health devices are exquisitely sensitive and reliable – capable of measuring the speed of each step a patient takes in their home and school (not just 4 steps in a clinic). The ability to store and move large amounts of the data collected by mobile health devices is advancing rapidly. The end result is measurements of patient function in the community setting – lots of data in a setting highly relevant to the patient and his family.
Roxanna is recruiting DMD boys into a natural history study using the Band, as well as implementing the Band as an exploratory outcome measure in the vamorolone clinical trials.