Study uses mobile app to collect data on PWS caregiver burden
Folia Health says real-world experiences are key to treatment development
Written by |
A new study uses a mobile app to track the experiences of caregivers for people with Prader-Willi syndrome (PWS), with the aim of giving researchers insight into day-to-day life that could inform treatment development.
“Pragmatic measurement is an important key to drug development that can provide insight into the needs of individuals living with rare diseases, such as PWS,” Nell Meosky Luo, CEO and founder of Folia Health, which is conducting the study using its app-based platform, said in a company press release. “PWS impacts the entire family, including caregivers.”
The BRAVE-PWS (Burden and Real‑world Assessment of caregiver Voice and Experiences in PWS) study “will give us first-hand information on caregivers’ experiences that may inform future research, clinical care strategies, and the evaluation of emerging treatments for PWS,” Luo said.
The study is enrolling adults in the U.S. who are the main caregivers of PWS patients aged 4 or older who are not taking Vykat XR (diazoxide choline), an oral therapy for PWS-related hyperphagia (insatiable hunger).
The study is sponsored by Soleno Therapeutics, the original developer of Vykat XR. The company recently agreed to be acquired by Neurocrine Biosciences.
Weekly reporting to focus on caregivers and patients
PWS is caused by the loss of function of genes located in a region of chromosome 15. PWS symptoms include impaired growth, cognitive and behavioral problems, and hyperphagia (a persistent sensation of hunger that leads to extreme food-seeking behaviors).
PWS caregivers face a substantial burden managing hyperphagia and associated behavioral issues, which require continuous supervision and consistent routines. This level of care can significantly impair the quality of life for patients and their caregivers.
In the BRAVE-PWS study, caregivers will use Folia’s mobile app to collect weekly home-reported outcomes, or health observations reported outside the clinic, focused on their own well-being and household functioning, as well as symptoms and behaviors of the PWS patients they care for.
Participants will also respond to short monthly check-in prompts in the app. Data will be collected for six months, and caregivers will be compensated for their participation.
“In addition to serving as a research tool, the platform gives users visual feedback on their tracked data over time,” helping families notice patterns in symptoms, prepare for medical appointments, and identify environmental factors that may influence both their and the patient’s health, such as hunger and behavior, Folia said.
