Feng Xie part of international team assessing the use of digital technology in the treatment and care of people with a rare disease

Feng Xie is part of an international team of researchers who have received more than $1 million in funding from the European Joint Program for Rare Diseases for a three-year-project entitled “Patient-reported, health economic, and psychosocial outcomes in Friedreich Ataxia (PROFA).”

Friedreich ataxia (also called FA) is a rare inherited disease that causes progressive nervous system damage and movement problems. It usually begins in childhood and leads to impaired muscle coordination (ataxia) that worsens over time.

Xie will work with Bernhard Michalowsky and Thomas Klockgether from the German National Center for Neurodengerative Diseases (DZNE), Stephanie Borel and Alex Durre from Paris Brain Institute and Sorbonne Universite in France to understand the economic and humanistic burden of FA. The team will seek to identify determinants of patient health and social life and the efficient use of health care resources using a patient-centric, remote monitoring e-health app within a large European cohort of patients with FA.

Specific objectives include:

1. To assess the acceptability, feasibility and usability of the e-health app, collecting real-time health-related quality of life, economic and psychosocial data from patients with FA.
2. To determine the healthcare resource utilization and costs for patients with FA from a societal perspective and to analyze the associations between these costs and socio-demographic and clinical variables as well as the impact of evidence-based treatment on costs.
3. To assess patients’ HRQoL and identify socio-demographic and clinical factors associated with health improvement and deterioration over time.
4. To develop and validate a new measure of hearing and speech disabilities’ impact on patients’ psychosocial health.
5. To evaluate interaction effects between healthcare resource utilization patterns (evidence-based treatment and care), HRQoL and psychosocial health.