Optimizing CFTR modulator therapy management for cystic fibrosis through the ReX platform

<bold>Background:</bold> CF is a chronic multi-systemic disease that requires a complex daily treatment regimen. Therefore, there is sub-optimal adherence to therapies, and it was shown to impact its clinical and economic burden. Cystic fibrosis transmembrane conductance regulator modulators (CFTRm) are high-cost medications that demonstrated significant benefit in clinical trials. The aim of this study was to evaluate the safety, usability, and efficacy of the ReX platform in medication management of CFTRm for the treatment of people with CF (pwCF). <bold>Methods:</bold> ReX is a patient engagement platform consisting of a cloud-based management system and a cell-enabled handheld device intended to dispense oral medication into the patient's mouth. It provides real-time adherence data to caregivers and timely, personalized reminders to patients. This is a prospective open study for pwCF>12 years, who were prescribed elexacaftor/tezacaftor/ivacaftor (ETI) or tezacaftor/ivacaftor (TI), and provided consent to use ReX platform. <bold>Results:</bold> Ten pwCFs were included, mean age 31.5 years, eight were taking ETI and two TI. Median adherence was 97.5% (range 70–100%) in the 1st year and 94% (range 84–99%) in the 2nd year, which is higher than the previously reported CFTRm adherence of ∼80%. No adverse events related to the use of the platform were reported. Patients reported ReX to be valuable and user friendly. Estimated mean FEV1% increased from 74.4% to 80.8% (p = 0.004) and estimated BMI% increased from 53.5 to 59.0 (p < 0.001) over 2 years. <bold>Conclusions:</bold> Using the ReX platform in medication management of pwCF treated by CFTRm is safe, easy to use, and effective in improving the adherence to treatment and the clinical outcomes.