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

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
Pages (from-to)567-578
Number of pages12
JournalValue in Health
Volume26
Issue number4
DOIs
StatePublished - Apr 2023
Externally publishedYes

Keywords

  • Cystic Fibrosis Questionnaire-Revised
  • cost utility
  • cystic fibrosis
  • patient-reported outcomes
  • quality of life

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health
  • Health Policy

Fingerprint

Dive into the research topics of 'Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised'. Together they form a unique fingerprint.

Cite this