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.

  Key deliverables

  • 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.

  Client benefit

  • 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.


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 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.  

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