The Impact of Charcot-Marie-Tooth Disease in the Real World
Charcot-Marie-Tooth Disease / Posted 1 year ago
An observational, non-interventional registry study to collect real-world data from people living with Charcot-Marie-Tooth disease (CMT) and its treatment, which will be available to researchers to further the knowledge of Charcot-Marie-Tooth disease and improve patient care.
The registry uses Vitaccess’ MyRealWorld™ digital real-world evidence platform and has been developed in collaboration with CMT experts, Patient Advocacy Organizations (PAOs) and the biopharmaceutical company Pharnext.
Eligible participants install a study app on their smartphone. Researchers access aggregated, anonymised data via a cloud-based research portal.
The platform provides benefits to participants, which it is hoped will encourage persistence with data submission; these include options to upload electronic documents such as scans, a clinically-validated Knowledge feature, and access an online CMT community.
The aggregated data are available in close to real time via “dashboards” and can be analysed according to a number of pre-set criteria (e.g. disease stage, age, geographic location).
State-of-the-art technologies and security policies are used in the platform to ensure industry-standard data storage and privacy for all users. Participants’ personally identifiable information will remain confidential at all times, and researchers will not be able to identify individuals.
The study is ethics-approved in all scope countries and led by a Scientific Advisory Board comprising representatives from international patient associations, patients and key opinion leaders from each country, and study leads from both Pharnext Société Anonyme (SA) and Vitaccess Ltd.