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 participant 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 3,200 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 conference presentations and pharmacoeconomic modelling.