PRS77 Application of the Cfq-R-8D to Estimate Utility Benefit of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in People with Cystic Fibrosis (CF)

CF is a rare, multi-systemic genetic disease characterized by impaired survival and reduced health-related quality-of-life (HRQoL). The CFQ-R-8D (Acaster NACFC 2019) is a disease-specific preference-based scoring algorithm that estimates utilities based on a standard, well-validated HRQoL instrument in CF (CFQ-R). The CFTR modulator ELX/TEZ/IVA provided substantial improvements in lung function (measured by ppFEV1) and CFQ-R respiratory symptom domains in a registrational trial enrolling people with CF (pwCF) aged ≥12 years with F508del/minimal function (F/MF) genotypes (NCT03525444).