Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised

Sarah Acaster; Clara Mukuria; Donna Rowen; John E Brazier; Claire E Wainwright; Bradley S Quon; Jamie Duckers; Alexandra L Quittner; Yiyue Lou; Patrick R Sosnay; Lisa J McGarry

doi:10.1016/j.jval.2022.12.002

Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised

Value Health. 2023 Apr;26(4):567-578. doi: 10.1016/j.jval.2022.12.002. Epub 2022 Dec 9.

Authors

Affiliations

¹ Acaster Lloyd Consulting Ltd, London, England, UK. Electronic address: sarah.acaster@acasterlloyd.com.
² School of Health and Related Research, The University of Sheffield, Regent Court, Sheffield, England, UK.
³ Child Health Research Centre, University of Queensland, Brisbane, QLD, Australia.
⁴ Centre for Heart Lung Innovation, St. Paul's Hospital, and Division of Respiratory Medicine, Department of Medicine, University of British Columbia, Vancouver, BC, Canada.
⁵ Department of Respiratory Medicine, Cardiff and Vale University Health Board, NHS Wales, Cardiff, Wales, UK.
⁶ Research, Behavioral Health Systems Research, Miami, FL, USA.
⁷ Biostatistics, Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
⁸ Clincal Development, Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
⁹ Health Economics and Outcomes Research, Vertex Pharmaceuticals Incorporated, Boston, MA, USA.

PMID: 36509366
DOI: 10.1016/j.jval.2022.12.002

Abstract

Objectives: 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; nevertheless, 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-8 dimensions; 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-8 dimensions 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.

Keywords: Cystic Fibrosis Questionnaire-Revised; cost utility; cystic fibrosis; patient-reported outcomes; quality of life.

Publication types

Clinical Trial
Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Adult
Algorithms
Cystic Fibrosis* / diagnosis
Female
Humans
Male
Middle Aged
Psychometrics
Quality of Life*
Surveys and Questionnaires

Associated data

ClinicalTrials.gov/NCT01807923
ClinicalTrials.gov/NCT01807949
ClinicalTrials.gov/NCT02347657
ClinicalTrials.gov/NCT02392234