OBJECTIVES: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is available in Portugal to treat people with cystic fibrosis (CF) ≥6 years of age who have at least one F508del variant. We report results from a non-interventional, retrospective study evaluating hospital healthcare resource utilization among people with CF who are treated with ELX/TEZ/IVA in Portugal.

METHODS: Data on people initiating ELX/TEZ/IVA between 21 August 2020 and 30 June 2022 (index date period) with 12 months of data available prior to and post-index date were extracted from Health Market Research database, a hospital consumption database. Outcomes included hospital medication consumption (episodes) according to outpatient, day hospital, emergency room, inpatient (surgery, intensive care unit or inpatient-other), as well as prescription medication usage and pulmonary exacerbation (PEx) proxied by intravenous antibiotic episodes.

RESULTS: The study included 127 people, with 76.4% aged 15 to 39 years. The total number of PEx episodes decreased (73%), from 37 prior to 10 post-index date (rate ratio 0.27 [95% CI: 0.13, 0.54]). Decreases in prescription medication consumption of anti-fungals (87%), anti-inflammatories (33%), vitamins (22%), and antibiotics (14%) were observed post-index date, with small increases in digestive and pancreatic enzymes (11%) and antacids (9%). For inpatient-other, the number of participants with at least one episode decreased from 26 (20.5%) prior to 12 (9.4%) post-index date, with a corresponding decrease in the total number of episodes from 44 to 21 (rate ratio 0.48 [95% CI: 0.28, 0.80]). For outpatient, the total number of episodes increased from 1,243 prior to 1,518 post-index date. This increase is due to monthly prescription collections from the hospital pharmacy, which increased post-ELX/TEZ/IVA. Other hospital types had too few numbers to analyze.

CONCLUSIONS: After a year of treatment, ELX/TEZ/IVA led to decreases in hospital pharmacy consumption with reductions in inpatient hospital admissions, prescription medication usage, and PEx in people with CF in Portugal.