Health State Utilities Associated With Treatment Burden in Cystic Fibrosis: A Patient Valuation Study
Rory A Cameron1,2, Jessie Matthews3, Daniel Office3
1Norwich Medical School, University of East Anglia, Norwich, England.
CHEST Pulmonary
|August 4, 2026
Summary
People with cystic fibrosis (pwCF) value reduced treatment burden, with significant health state utility decrements observed for IV antibiotics, physiotherapy, and inhaled medicines. These findings emphasize the importance of considering treatment burden in CF economic evaluations.
Area of Science:
- Health Economics
- Patient-Reported Outcomes
- Cystic Fibrosis Research
Background:
- Advancements in cystic fibrosis (CF) treatment improve survival but increase treatment burden for many people with CF (pwCF).
- Economic evaluations of novel CF interventions often lack suitable outcome measures for treatment burden.
- Estimating health state utilities (HSUs) for treatment burden is crucial for comprehensive economic assessments.
Purpose of the Study:
- To estimate the value (health state utilities) pwCF place on changes in treatment burden.
- To quantify the impact of IV antibiotic treatments, inhaled medicines, and physiotherapy on HSUs.
- To inform economic evaluations by incorporating patient-valued treatment burden.
Main Methods:
- Adults with CF participated in web-based time trade-off interviews.
- Participants valued their own health and health state vignettes representing varying treatment intensities.
- Mixed-effect linear regression models estimated HSUs for additional treatment instances.
Main Results:
- Fifty-one pwCF (median age 30) completed the study.
- Adjusted utility decrements were -0.037 for annual IV antibiotics, -0.029 for daily physiotherapy, and -0.019 for daily inhaled medicines.
- Increasing treatment burden was associated with decreasing HSUs.
Conclusions:
- Treatment burden significantly impacts health-related quality of life for pwCF.
- Utility decrements associated with treatment burden changes are meaningful.
- Incorporating treatment burden into economic evaluations is essential for CF interventions.
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