TY - JOUR
T1 - Health state utilities associated with treatment burden in cystic fibrosis: A patient valuation study
AU - Cameron, Rory A.
AU - Matthews, Jessie
AU - Office, Daniel
AU - Rowley, Mark
AU - Abbott, Janice
AU - Simmonds, Nicholas J.
AU - Whitty, Jennifer A.
AU - Carr, Siobhán B.
N1 - 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.
PY - 2025/3
Y1 - 2025/3
N2 - 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.
AB - 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.
KW - cystic fibrosis
KW - health state utility
KW - health-related quality of life
KW - patient and public involvement
KW - time trade-off
KW - treatment burden
UR - http://www.scopus.com/inward/record.url?scp=86000727703&partnerID=8YFLogxK
U2 - 10.1016/j.chpulm.2024.100097
DO - 10.1016/j.chpulm.2024.100097
M3 - Article
SN - 2949-7892
VL - 3
JO - CHEST Pulmonary
JF - CHEST Pulmonary
IS - 1
M1 - 100097
ER -