Direct-to-Patient Model for Fragile X Syndrome Trials

This case study presents the implementation of a direct-to-patient model for clinical trials focused on Fragile X Syndrome, a rare neurological condition. It outlines the challenges faced by families of Fragile X patients, including caregiving responsibilities and the difficulties associated with traditional clinical visits. The study highlights the burden of complex protocols that require frequent specimen collection and caregiver-administered dosing, which can lead to increased irritability and high attrition rates. The solution involved utilizing Science 37's Direct-to-Patient Site, allowing patients to participate in trials from home. Pediatric-trained mobile nurses provided in-home support, enhancing patient comfort and adherence to protocols. The study also emphasizes the importance of collaboration with advocacy groups and provider networks to improve recruitment and retention rates. Overall, the findings demonstrate the effectiveness of the direct-to-patient model in improving the clinical trial experience for pediatric patients with rare conditions.