Acaster, S., Mukuria, C. orcid.org/0000-0003-4318-1481, Rowen, D. orcid.org/0000-0003-3018-5109 et al. (8 more authors) (2023) Development of the CFQ-R-8D: estimating utilities from the cystic fibrosis questionnaire-revised. Value in Health, 26 (4). pp. 567-578. ISSN 1098-3015
Abstract
Objective
Cystic fibrosis (CF) limits survival and negatively affects health-related quality of life (HRQOL). Cost-effectiveness analysis (CEA) may be used to make reimbursement decisions for new CF treatments; however, generic utility measures used in CEA, such as EQ-5D, are insensitive to meaningful changes in lung function and HRQOL in CF. Here we develop a new, CF disease–specific, preference-based utility measure based on the adolescent/adult version of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely used, CF-specific, patient-reported measure of HRQOL.
Methods
Blinded CFQ-R data from 4 clinical trials (NCT02347657, NCT02392234, NCT01807923, and NCT01807949) were used to identify discriminating items for a classification system using psychometric (eg, factor and Rasch) analyses. Thirty-two health states were selected for a time-trade-off (TTO) exercise with a representative sample of the UK general population. TTO utilities were used to estimate a preference-based scoring algorithm by regression analysis (Tobit models with robust standard errors clustered on participants with censoring at −1).
Results
A classification system with 8 dimensions (CFQ-R-8D; Physical Functioning, Vitality, Emotion, Role Functioning, Breathing Difficulty, Cough, Abdominal Pain, and Body Image) was generated. TTO was completed by 400 participants (mean age, 47.3 years; 49.8% female). Among the regression models evaluated, the Tobit heteroscedastic–ordered model was preferred, with a predicted utility range from 0.236 to 1, no logical inconsistencies, and a mean absolute error of 0.032.
Conclusion
The CFQ-R-8D is the first disease-specific, preference-based scoring algorithm for CF, enabling estimation of disease-specific utilities for CEA based on the well-validated and widely used CFQ-R.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2023, International Society for Pharmacoeconomics and Outcomes Research, Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
Keywords: | cost utility; cystic fibrosis; Cystic Fibrosis Questionnaire-Revised; patient-reported outcomes; quality of life |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Health and Related Research (Sheffield) > ScHARR - Sheffield Centre for Health and Related Research |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 13 Dec 2022 12:42 |
Last Modified: | 04 Apr 2023 11:40 |
Status: | Published |
Publisher: | Elsevier |
Refereed: | Yes |
Identification Number: | 10.1016/j.jval.2022.12.002 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:194247 |