995-19 | An Observational Study to Evaluate the Real-world Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor on the Burden of Illness in Cystic Fibrosis Patients

Overview

“An Observational Study to Evaluate the Real-world Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor on the Burden of Illness in Cystic Fibrosis Patients”

This is a multicenter, observational study with prospective data collection designed to evaluate the real-world burden of illness before and after initiation of treatment with ELX/TEZ/IVA from the CF patient and caregiver-reported perspective. A triple combination (TC) regimen with elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) is being developed for the treatment of patients with CF. The pivotal Phase 3 program demonstrated that ELX/TEZ/IVA provides substantial clinical benefit, including improvements in lung function and CF-related quality of life measures in F/MF patients. However, the effectiveness on burden of illness in F/MF patients and the burden on their caregivers in a real-world setting over time is not known. This observational study will evaluate the real-world effectiveness of treatment with ELX/TEZ/IVA on patient burden of illness and caregiver burden, as well as clinical outcomes in the real-world setting.