OBJECTIVES

: To evaluate:

• co-creation of a digital app with patients and patient advocacy organizations (PAOs) for use in a 2.5-year observational study of Charcot Marie Tooth disease (CMT) in the US and Europe
• recruitment via a range of channels (including social media and word of mouth) and response rates.

METHODS

: The ‘bring your own device’ app – ‘CMT&Me’ – was co-created with patients and PAOs in the USA, France, Italy, Spain, and the UK, to evaluate the impact of CMT in the real world. Individual and group feedback was obtained on the data capture plan, user interface and experience, and recruitment and communications strategy.

The study was implemented according to best practice for observational studies. Participants’ interests and access to the data are safeguarded by a scientific advisory board comprising a PAO and a clinician from each country, and representatives from Pharnext and Vitaccess.

The recruitment strategy was tailored to each country using social media (Facebook, Twitter, Instagram), multi-media resources (video vignettes and images), email, and PAOs’ networks of patients. The CMT&Me app also enables participants to refer other patients. CMT&Me is available in Germany, Belgium, Netherlands, and Portugal but recruitment in these countries is by word of mouth (i.e. not in collaboration with PAOs).

CMT&Me was localized according to industry standards. App text was translated and culturally adapted, and linguistically validated PRO instruments are used. Local language is used for all patient communications.

RESULTS

: The co-creation methodology and innovative recruitment strategy enabled faster recruitment than traditional multi-site studies, in terms of numbers of participants and time to achieve coverage across a country. This approach also provided strong representation of subgroups (e.g. by age, sex and CMT subtype).

CONCLUSIONS

: For international observational studies, using digital technologies co-created with patients and PAOs yields significantly quicker and more extensive recruitment than traditional methods.