Evaluate real-world effectiveness and burden of disease in Cystic Fibrosis patients

Challenge

• CF is associated with significantly lower quality of life and a high disease burden.
• Evaluate the change in patient-reported and caregiver-reported burden of disease after initiating CF treatment.

Solution

• Conducted an observational study to evaluate the real-world effectiveness of treatment on patient and caregivers’ burden of illness
• PROs and Caregiver Reporter Outcomes were collected every 2 months
• Described select clinical outcomes