CFTR-Modulators (CFTRM) Impact on Real-World Healthcare Resource Utilisation (HCRU) in Cystic Fibrosis (CF) in Wales: Retrospective, Observational, Database Study Using Linked Data from the UK CF Registry (UKCFR) and Sail-Databank

Author(s)

Vega-Hernandez G1, Duran A1, Akbari A2, Griffiths R2, Wöhling H1
1Vertex Pharmaceuticals Incorporated, London, LON, UK, 2Swansea University, Swansea, UK

OBJECTIVES: This real-world study assessed changes in HCRU in patients with CF before and after initiating treatment with any CFTRm: ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor, or ivacaftor/tezacaftor/elexacaftor (IVA/TEZ/ELX).

METHODS: The study estimated differences in HCRU during 12-months before and after CFTRm initiation in patients with a record of CFTRm-initiation and linked data available in the Secure Anonymised Information Linkage (SAIL)-Databank (CFTRm cohort). The SAIL-Databank includes patient data from electronic health records and UKCFR to provide demographic and clinical characteristics and HCRU from primary and secondary care between April 2009 and April 2021. An IVA/TEZ/ELX cohort was identified to estimate differences in HCRU following initiation of IVA/TEZ/ELX as their first CFTRm (IVA/TEZ/ELX cohort); given its license/availability after August 2020, people in this cohort were required to have ≥6-months linked data.

RESULTS: The CFTRm cohort included 166 patients, while 36 patients made up the IVA/TEZ/ELX cohort. The CFTRm cohort had lower mean annual hospitalisation rates after CFTRm-initiation vs before (0.89 vs 1.78). Reductions were also observed in mean hospital length of stay (20 vs 25.8 days), mean time on IV-antibiotics at home (5.0 vs 5.7 days) and in hospital (9.4 vs 9.5 days). In the IVA/TEZ/ELX cohort, the mean hospital event rates were lower after CFTRm vs before CFTRm (0.6 vs 1.99).

CONCLUSIONS: In patients with CF in Wales, a notable decline in the number of hospitalisations was observed after CFTRm initiation, which appeared more pronounced (pre vs post initiation) in patients whose first CFTRm was IVA/TEZ/ELX.

Conference/Value in Health Info

2023-05, ISPOR 2023, Boston, MA, USA

Value in Health, Volume 26, Issue 6, S2 (June 2023)

Code

EE182

Topic

Economic Evaluation

Disease

Rare & Orphan Diseases

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