SOLAR: AN OBSERVATIONAL STUDY OF THE LONG-TERM EFFECTIVENESS OF ELEXACAFTOR/TEZACAFTOR/IVACAFTOR IN PEOPLE WITH CYSTIC FIBROSIS USING DATA FROM THE FRENCH CYSTIC FIBROSIS REGISTRY—IMPACT ON QUALITY OF LIFE AND HEALTH UTILITY VALUES
Author(s)
Conor Daly, MSc1, Pierre-Regis Burgel, PhD2, Isabelle Sermet-Gaudelus, MD3, Olivier Giraudier, MD1, Pia Clara Pafundi, MSc, PhD1, Carl Baxter, PhD1, Heike Wöhling, PhD1, Catherine Payen-Champenois, MD1, Antoine Bessou, MSc4, Gabriela Vega-Hernandez, BSc, MSc1.
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Université Paris Cité, Paris, France, 3Institut Necker -Enfants Malades, Paris, France, 4French CF Registry, Vaincre La Mucoviscidose, PARIS, France.
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Université Paris Cité, Paris, France, 3Institut Necker -Enfants Malades, Paris, France, 4French CF Registry, Vaincre La Mucoviscidose, PARIS, France.
OBJECTIVES: To assess the impact of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) treatment initiation on quality of life (QoL) using the Cystic Fibrosis Questionnaire-Revised (CFQ-R) adult instrument and Cystic Fibrosis Questionnaire-Revised-8 Domains (CFQ-R-8D) utility values.
METHODS: This registry-based cohort study included people with CF (PwCF) and ≥1 F508del allele, initiating ELX/TEZ/IVA in France on or after 1st July 2021, with follow up until 31st December 2023. Participants were required to have ≥1 CFQ-R item-level record at baseline and follow-up within 12 months. CFQ-R domain level values were calculated at baseline and follow-up. Overall utility values were calculated using the CFQ-R-8D scoring algorithm1. Baseline QoL and utility values were obtained from the most recent record prior to ELX/TEZ/IVA initiation, follow-up values were calculated as a mean of all available scores in the 12-month follow-up period following ELX/TEZ/IVA initiation. A subgroup analysis of pwCF with advanced lung disease (ALD) [i.e., percent predicted forced expiratory volume (ppFEV1) <40%] was also included.
RESULTS: 627 pwCF were included, with a mean (SD) age at initiation of 31.2 (10.5) years; 52.6% were male; 53.1% had prior CF transmembrane conductance regulator modulator (CFTRm) treatment; mean (SD) ppFEV1 at baseline was 67.7 (20.7) percentage points. Absolute changes from baseline to end of follow-up demonstrated improvements across all 12 CFQ-R domains. Mean changes from baseline ranged from +2.1 (95%CI: 0.5,3.7) in digestive symptoms, to +25.7 (95%CI: 23.9,27.5) in respiratory symptoms domain (respiratory domain minimal clinically important difference =4.0).2 Overall utility scores also showed improvements, with mean changes from baseline of +0.096 (95%CI: 0.086, 0.106) in the main cohort, and +0.154 (95%CI: 0.121,0.187) in the ALD subgroup (n=61).
CONCLUSIONS: Amongst pwCF who initiated ELX/TEZ/IVA in France, substantial improvements in QoL and utilities were observed, consistent with clinical studies and real-world practice. These findings highlight the transformative impact of CFTRms in delivering meaningful patient benefit.
METHODS: This registry-based cohort study included people with CF (PwCF) and ≥1 F508del allele, initiating ELX/TEZ/IVA in France on or after 1st July 2021, with follow up until 31st December 2023. Participants were required to have ≥1 CFQ-R item-level record at baseline and follow-up within 12 months. CFQ-R domain level values were calculated at baseline and follow-up. Overall utility values were calculated using the CFQ-R-8D scoring algorithm1. Baseline QoL and utility values were obtained from the most recent record prior to ELX/TEZ/IVA initiation, follow-up values were calculated as a mean of all available scores in the 12-month follow-up period following ELX/TEZ/IVA initiation. A subgroup analysis of pwCF with advanced lung disease (ALD) [i.e., percent predicted forced expiratory volume (ppFEV1) <40%] was also included.
RESULTS: 627 pwCF were included, with a mean (SD) age at initiation of 31.2 (10.5) years; 52.6% were male; 53.1% had prior CF transmembrane conductance regulator modulator (CFTRm) treatment; mean (SD) ppFEV1 at baseline was 67.7 (20.7) percentage points. Absolute changes from baseline to end of follow-up demonstrated improvements across all 12 CFQ-R domains. Mean changes from baseline ranged from +2.1 (95%CI: 0.5,3.7) in digestive symptoms, to +25.7 (95%CI: 23.9,27.5) in respiratory symptoms domain (respiratory domain minimal clinically important difference =4.0).2 Overall utility scores also showed improvements, with mean changes from baseline of +0.096 (95%CI: 0.086, 0.106) in the main cohort, and +0.154 (95%CI: 0.121,0.187) in the ALD subgroup (n=61).
CONCLUSIONS: Amongst pwCF who initiated ELX/TEZ/IVA in France, substantial improvements in QoL and utilities were observed, consistent with clinical studies and real-world practice. These findings highlight the transformative impact of CFTRms in delivering meaningful patient benefit.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD26
Topic
Patient-Centered Research, Real World Data & Information Systems
Disease
Rare & Orphan Diseases, Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)