(396) Long-term Impact of Elexacaftor/Tezacaftor/Ivacaftor on Healthcare Resource Utilization and Costs Among People with Cystic Fibrosis Aged ≥6 Years in the US: A Retrospective Analysis of Commercial Claims in the Forsyth HealthSM Proprietary Database

Background: : TRIKAFTA or elexacaftor/tezacaftor/ivacaftor (ETI) is a cystic fibrosis (CF) transmembrane conductance regulator modulator (CFTRm) first approved for the treatment of people with cystic fibrosis (pwCF) aged ≥12 years in the US in October 2019; the label was expanded to include pwCF aged 6-11 years in June 2021. Real-world studies have demonstrated the significant impact of ETI on healthcare resource utilization (HCRU) and economic benefits but have been limited to short-term ( < 1 year) trajectories.

Objective: : The goal of the study was to investigate the long-term impact of ETI therapy on pulmonary exacerbations (PEx), HCRU, concomitant medication use, and associated medical costs using Forsyth Health’s proprietary claims database, which represents the lives of a large US Commercial national payer. Vertex Pharmaceuticals (a manufacturer) and Forsyth Health (a Real-World Data & Analytics company affiliated with Evernorth) collaborated on the design of the study protocol and interpreting results.

Methods: : We identified pwCF aged ≥6 years who had their first-ever recorded outpatient (OP) prescription for ETI (date of first fill for ETI = index date) on or after October 21, 2019 and were continuously enrolled for 1 year pre- and ≥2 years post-index. We compared annualized rates of PEx (inpatient or IV antibiotics), HCRU, and associated costs pre- and post-index. Exploratory analyses were conducted for a subset of patients continuously enrolled for ≥3 years post-index.

Results: : 645 patients aged ≥12 years (mean age 27.4, 43.3% female) were identified in the Forsyth commercial database. 48.2% were CFTRm-treatment naïve. The annualized rate of PEx was 0.49 (95% CI 0.41, 0.57) in the 1-year pre-index period and 0.19 (0.15, 0.24) over 2 years post-index—a 61.2% reduction. Similarly, the annualized rate of inpatient hospitalizations was 0.43 (0.35, 0.51) pre-index vs. 0.18 (0.13, 0.23) over 2 years post—a 58.1% reduction. The annualized rate of OP visits (inclusive of ED, office, home health, and other outpatient visits) was 30.9 (28.6, 33.2) pre-index vs. 23.0 (21.2, 24.8) over 2 years post—a 25.6% reduction. These reductions were maintained in the subset of n=466 pwCF continuously insured for 3 years (reductions of 64.2% in PEx, 59.1% in hospitalizations, and 30.4% in OP visits vs. pre-index). Additional analyses of concomitant medication use, costs, and outcomes for the 6-11 population will be available at time of presentation.

Conclusions: : ETI demonstrates durable reductions in disease burden associated with CF over time and meaningful reductions in HCRU within a large US payer.

Sponsorship:: Vertex Pharmaceuticals Inc.