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


  • 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.


  • 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

Targeting ~300 patients for enrollment

Retrospective & Prospective

2 year study