Real-World Evidence of Healthcare Resource Utilization in Cystic Fibrosis (CF) Patients Treated With Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in France Using Long-term Data From the SNDS Database Linked to the French CF Registry: Findings From...
Author(s)
Conor Daly, MSc1, Erika Guyot, PhD2, Isabelle Durieu, MD3, Manon Belhassen, MPH, PhD2, Maeva Nolin, MSc2, Melanie Nee, PhD2, Olivier Giraudier, MD1, Pia Clara Pafundi, PhD1, Gabriela Vega-Hernandez, BSc, MSc1.
1Vertex Pharmaceuticals, Inc., Boston, MA, USA, 2Pharmaco-Epidémiologie Lyon (PELyon), Lyon, France, 3Hospices Civils de Lyon, Lyon, France.
1Vertex Pharmaceuticals, Inc., Boston, MA, USA, 2Pharmaco-Epidémiologie Lyon (PELyon), Lyon, France, 3Hospices Civils de Lyon, Lyon, France.
OBJECTIVES: To describe real-world healthcare resource utilization (HCRU) in people with Cystic Fibrosis (pwCF) before and after ELX/TEZ/IVA initiation in France.
METHODS: This non-interventional, observational, retrospective cohort-study was based on data linkage between the French claims database (SNDS) and demographic and clinical characteristics from the French CF Registry. Inclusion criteria were age ≥12 years and ELX/TEZ/IVA initiation between 12/01/2019, and 12/31/2021. Data censoring was upon death, loss of follow-up or switch to another CFTRm. Assessment of HCRU notably included records of health care professional (HCP) consultations and unique identification codes of reimbursed prescription medications available in the SNDS; medical therapies include, inhaled, oral (tablets), and intravenous (vials) medications. HCP consultations include nurse, physiotherapists and physician visits and can take place during the same visit to a medical facility but are counted as unique events. Differences across HCRU between 12-months pre (baseline period) and 12-months post ELX/TEZ/IVA initiation (follow-up period) were assessed.
RESULTS: 2,316 pwCF met the inclusion criteria. At index date, mean age was 25.9 (SD: 10.3) years; 54.7% male; 59.9% were homozygous for F508del mutation; 46.6% previously received a CFTRm and mean ppFEV1 was 76.5% (SD: 25.0). Mean annual use of medical therapies reduced by 40% following ELX/TEZ/IVA initiation, from 269.4 (SD: 235.3) unique prescriptions of reimbursed medication during baseline to 161.7 (SD: 139.2) during follow-up. Mean annual IV antibiotics use reduced by 85.6% after ELX/TEZ/IVA initiation from 55.4 (SD: 121.0) prescriptions during baseline to 8.0 (SD: 39.3) during follow-up, consistent with the decrease in the proportion of patients receiving IV antibiotics [33.0% (baseline) vs. 8.2% (follow-up)]. Mean annual HCP consultations decreased by 38.1% after treatment initiation, from 135.0 (SD: 167.6) during baseline to 83.6 (SD: 165.8) during follow-up.
CONCLUSIONS: The findings demonstrated the substantial positive impact of ELX/TEZ/IVA reducing HCRU and were consistent with research from other countries.
METHODS: This non-interventional, observational, retrospective cohort-study was based on data linkage between the French claims database (SNDS) and demographic and clinical characteristics from the French CF Registry. Inclusion criteria were age ≥12 years and ELX/TEZ/IVA initiation between 12/01/2019, and 12/31/2021. Data censoring was upon death, loss of follow-up or switch to another CFTRm. Assessment of HCRU notably included records of health care professional (HCP) consultations and unique identification codes of reimbursed prescription medications available in the SNDS; medical therapies include, inhaled, oral (tablets), and intravenous (vials) medications. HCP consultations include nurse, physiotherapists and physician visits and can take place during the same visit to a medical facility but are counted as unique events. Differences across HCRU between 12-months pre (baseline period) and 12-months post ELX/TEZ/IVA initiation (follow-up period) were assessed.
RESULTS: 2,316 pwCF met the inclusion criteria. At index date, mean age was 25.9 (SD: 10.3) years; 54.7% male; 59.9% were homozygous for F508del mutation; 46.6% previously received a CFTRm and mean ppFEV1 was 76.5% (SD: 25.0). Mean annual use of medical therapies reduced by 40% following ELX/TEZ/IVA initiation, from 269.4 (SD: 235.3) unique prescriptions of reimbursed medication during baseline to 161.7 (SD: 139.2) during follow-up. Mean annual IV antibiotics use reduced by 85.6% after ELX/TEZ/IVA initiation from 55.4 (SD: 121.0) prescriptions during baseline to 8.0 (SD: 39.3) during follow-up, consistent with the decrease in the proportion of patients receiving IV antibiotics [33.0% (baseline) vs. 8.2% (follow-up)]. Mean annual HCP consultations decreased by 38.1% after treatment initiation, from 135.0 (SD: 167.6) during baseline to 83.6 (SD: 165.8) during follow-up.
CONCLUSIONS: The findings demonstrated the substantial positive impact of ELX/TEZ/IVA reducing HCRU and were consistent with research from other countries.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
P21
Topic
Study Approaches
Topic Subcategory
Registries
Disease
Rare & Orphan Diseases, Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)