TY - JOUR
T1 - Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions
T2 - Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised
AU - Acaster, Sarah
AU - Mukuria, Clara
AU - Rowen, Donna
AU - Brazier, John E.
AU - Wainwright, Claire E.
AU - Quon, Bradley S.
AU - Duckers, Jamie
AU - Quittner, Alexandra L.
AU - Lou, Yiyue
AU - Sosnay, Patrick R.
AU - McGarry, Lisa J.
N1 - Funding Information:
Conflict of Interest Disclosures: All authors reported receiving nonfinancial support (assistance with manuscript preparation) from ArticulateScience LLC, which received funding from Vertex Pharmaceuticals Incorporated. Ms Acaster reported employment with Acaster Lloyd Consulting Ltd, which received payment from Vertex Pharmaceuticals Incorporated for their contribution to the design, management, and undertaking of all aspects of the study reported in this manuscript; in addition, Dr Acaster is an associate editor for Value in Health and had no role in the peer-review process of this article. Dr Mukuria reported receiving grants from Vertex Pharmaceuticals and membership in the EuroQol Group Association. Dr Rowen reported receiving grants from Vertex Pharmaceuticals Incorporated. Dr Wainwright reported advisory board membership for Vertex Pharmaceuticals Incorporated; consulting fees from Vertex Pharmaceuticals Incorporated; editor duties for Respirology (associate editor) and Thorax (deputy editor); honoraria from BMJ, DKBmed, Gilead, In Vivo Academy, Novartis, Thorax, University of Miami, and Vertex Pharmaceuticals Incorporated; research grant from Novo Nordisk; being a study investigator for Boehringer Ingelheim and Vertex Pharmaceuticals Incorporated; and travel expenses from Vertex Pharmaceuticals Incorporated. Dr Quon reported receiving grants and personal fees from Vertex Pharmaceuticals Incorporated. Dr Duckers reported receiving personal fees from Vertex Pharmaceuticals Incorporated, Chiesi Pharmaceuticals, and Insmed. Dr Quittner reported research grants from the NIH, CF Foundation, and American Cochlear Implant Alliance and consulting fees from Insmed and Vertex Pharmaceuticals Incorporated. Drs Lou, Sosnay, and McGarry reported employment with Vertex Pharmaceuticals Incorporated and may own stock or stock options in that company. Dr Quittner was an employee of Behavioral Health Systems Research at the time the study was completed but is currently an employee of the Cystic Fibrosis and Pulmonary Center at Joe DiMaggio Children’s Hospital (Hollywood, FL). No other disclosures were reported.
Publisher Copyright:
© 2023
PY - 2023/4
Y1 - 2023/4
N2 - 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.
AB - 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.
KW - Cystic Fibrosis Questionnaire-Revised
KW - cost utility
KW - cystic fibrosis
KW - patient-reported outcomes
KW - quality of life
UR - http://www.scopus.com/inward/record.url?scp=85146457399&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85146457399&partnerID=8YFLogxK
U2 - 10.1016/j.jval.2022.12.002
DO - 10.1016/j.jval.2022.12.002
M3 - Article
C2 - 36509366
AN - SCOPUS:85146457399
SN - 1098-3015
VL - 26
SP - 567
EP - 578
JO - Value in Health
JF - Value in Health
IS - 4
ER -