Health state utilities associated with treatment burden in cystic fibrosis: A patient valuation study

Cameron, Rory A., Matthews, Jessie, Office, Daniel, Rowley, Mark, Abbott, Janice, Simmonds, Nicholas J., Whitty, Jennifer A. and Carr, Siobhán B. (2025) Health state utilities associated with treatment burden in cystic fibrosis: A patient valuation study. CHEST Pulmonary, 3 (1). ISSN 2949-7892

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Abstract

Background: Although recent advancements in the treatment of cystic fibrosis (CF) have improved survival, reducing high levels of treatment burden remains a priority issue for many people with cystic fibrosis (pwCF). However, economic evaluations of novel interventions may fail to capture their impact on treatment burden due to a lack of suitable outcome measures. This study aimed to estimate health state utilities (HSUs) for changes in treatment burden associated with different CF treatments. Research Question: What value do pwCF place on changes in treatment burden associated with IV antibiotic treatment of pulmonary exacerbations, use of inhaled medicines, and physiotherapy? Study Design and Methods: Adults attending a specialist CF center were invited to participate in a web-based time trade-off interview. Participants valued their own health and five health state vignettes describing varying levels of intensity of physiotherapy, use of inhaled medicines, and IV antibiotic treatment. HSUs for additional instances of each treatment type were estimated using mixed effect linear regression models. Results: Fifty one pwCF completed the interview (median age, 30 years; range, 19-66); 53% were female; mean FEV 1 % predicted was 65% (SD, 20%). Mean utility scores for own health were very similar between the EQ-5D index value (0.81; SD, 0.20) and the time trade-off value (0.82; SD, 0.20); however, limited concordance was observed at the individual level. Adjusted utility decrements associated with treatment burden were −0.037 (SE, 0.008) for an additional annual IV antibiotic treatment, −0.029 (SE, 0.014) for an additional daily physiotherapy session, and −0.019 (SE, 0.013) for an additional daily inhaled medicine. Interpretation: In this study, increasing treatment burden was associated with decreasing HSU values. The utility decrements associated with treatment burden changes suggest meaningful differences in health-related quality of life for pwCF. These findings align with existing literature on the impact of treatment burden on health-related quality of life, and highlight the importance of considering treatment burden in economic evaluations of interventions in CF.

Item Type: Article
Additional Information: Funding/Support: This study was funded by an NIHR Research for Patient Benefit Grant [Grant PB-PG-1217-20018]. J. A. W. and R. A. C. were also supported by the National Institute of Health Research (NIHR) Applied Research Collaboration East of England program. Views expressed are those of the authors and not necessarily those of the NHS, the NIHR, or the Department of Health.
Uncontrolled Keywords: cystic fibrosis,health state utility,health-related quality of life,patient and public involvement,time trade-off,treatment burden,medicine(all),ophthalmology,otorhinolaryngology ,/dk/atira/pure/subjectarea/asjc/2700
Faculty \ School: Faculty of Medicine and Health Sciences > Norwich Medical School
UEA Research Groups: Faculty of Medicine and Health Sciences > Research Groups > Health Economics
Faculty of Medicine and Health Sciences > Research Centres > Norwich Institute for Healthy Aging
Faculty of Medicine and Health Sciences > Research Groups > Health Services and Primary Care
Faculty of Medicine and Health Sciences > Research Groups > Respiratory and Airways Group
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Depositing User: LivePure Connector
Date Deposited: 29 Aug 2024 11:30
Last Modified: 28 Mar 2025 12:52
URI: https://ueaeprints.uea.ac.uk/id/eprint/96393
DOI: 10.1016/j.chpulm.2024.100097

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