Innovative patient-centric digital observational study in rare disease
Client success story
Innovative research organization, Vitaccess, has leveraged its flexible platform to connect a European biopharmaceutical company in real time to Charcot-Marie-Tooth disease (CMT) patients worldwide.
Case study by the numbers
1st conference presentation after only 1 month
participants recruited in 23 days
adults recruited since October 2018
consent rate for data to be used in future research
A European biopharmaceutical company was researching and developing treatments for the most common variant of CMT – a group of inherited, chronic, progressive motor and sensory neuropathies – called CMT1A.
The company approached us about conducting an observational study to collect real-world evidence from international patients in an accessible, fast, and secure manner.
Our client wished to generate real-world data that could complement their phase III clinical trial and be used in pharmacoeconomic modelling.
The observational study was to be developed quickly and flexibly in a number of countries.
We were to support the client in driving patient recruitment and engagement.
Global digital longitudinal observational study, collecting patient-reported data on the impact of CMT across the US, UK, Germany, France, Italy, and Spain.
Co-creation of digital study app, CMT&Me, with scientific experts, patient advocacy groups, and people living with CMT.
Study design and app localized and ethically approved across all countries.
Over 2,500 participants recruited since October 2018.
Demographic and patient-reported outcome data collected regularly and made accessible via interactive real-time dashboards.
Participant engagement and retention driven by quarterly study newsletters, patient-centric “data nuggets”, rapid peer-reviewed publications, country-specific webinars, and competitions.
Real-world evidence gathered from international patient cohort quickly and at a low cost.
Patient-reported data to complement the client’s phase III clinical trial.
Availability of data in near real-time for use in pharmacoeconomic modelling.
The CMT&Me app is an international, observational, non-interventional, longitudinal study that gathers data from study participants living with Charcot-Marie-Tooth (CMT) disease across Europe and the USA. It explores the impact of CMT and drug treatment in the real world. The dataset is available to researchers to further knowledge of CMT and improve care.
Researchers can access interactive dashboards via Vitaccess Real™ to export the dataset, and various selected cohorts. Dashboards include interactive figures and summary tables for demographics, treatment patterns and patient-reported outcome (PRO) instruments that measure the health-related quality of life and symptoms of study participants.
Applications to access the anonymized, aggregated dataset can be made to the study’s scientific advisory board via the data request form (below). Once applications have been approved, researchers can access the dataset.
For commercial subscriptions, please contact firstname.lastname@example.org.