Health state utility estimates for value assessments of novel treatments in Huntington's disease: a systematic literature review.
Cost-effectiveness analysis
Huntington disease
Neurodegenerative diseases
Quality of life
Quality-adjusted life years
Systematic review
Journal
Health and quality of life outcomes
ISSN: 1477-7525
Titre abrégé: Health Qual Life Outcomes
Pays: England
ID NLM: 101153626
Informations de publication
Date de publication:
16 Apr 2024
16 Apr 2024
Historique:
received:
09
08
2023
accepted:
18
03
2024
medline:
17
4
2024
pubmed:
17
4
2024
entrez:
16
4
2024
Statut:
epublish
Résumé
Huntington's disease (HD) is a progressive neurodegenerative disease with a devastating impact on patients and their families. Quantifying how treatments affect patient outcomes is critical for informing reimbursement decisions. Many countries mandate a formal value assessment in which the treatment benefit is measured as quality-adjusted life-years, calculated with the use of utility estimates that reflect respondents' preferences for health states. To summarize published health state utility data in HD and identify gaps and uncertainties in the data available that could be used to inform value assessments. We conducted a systematic literature review of studies that used preference-based instruments (e.g., EQ-5D and SF-6D) to estimate utility values for people with HD. The studies were published between January 2012 and December 2022. Of 383 articles screened, 16 articles reported utility values estimated in 11 distinct studies. The utility measure most frequently reported was EQ-5D (9/11 studies). Two studies reported SF-6D data; one used time trade-off methods to value health state descriptions (vignettes). Although utility scores generally worsened to a lower value with increased HD severity, the estimates varied considerably across studies. The EQ-5D index range was 0.89 - 0.72 for mild/prodromal HD and 0.71 - 0.37 for severe/late-stage disease. This study uncovered high variability in published utility estimates, indicating substantial uncertainty in existing data. Further research is needed to better understand preferences and valuation across all stages and domains of HD symptoms and the degree to which generic utility measures capture the impact of cognitive changes on quality of life.
Sections du résumé
BACKGROUND
BACKGROUND
Huntington's disease (HD) is a progressive neurodegenerative disease with a devastating impact on patients and their families. Quantifying how treatments affect patient outcomes is critical for informing reimbursement decisions. Many countries mandate a formal value assessment in which the treatment benefit is measured as quality-adjusted life-years, calculated with the use of utility estimates that reflect respondents' preferences for health states.
OBJECTIVE
OBJECTIVE
To summarize published health state utility data in HD and identify gaps and uncertainties in the data available that could be used to inform value assessments.
METHODS
METHODS
We conducted a systematic literature review of studies that used preference-based instruments (e.g., EQ-5D and SF-6D) to estimate utility values for people with HD. The studies were published between January 2012 and December 2022.
RESULTS
RESULTS
Of 383 articles screened, 16 articles reported utility values estimated in 11 distinct studies. The utility measure most frequently reported was EQ-5D (9/11 studies). Two studies reported SF-6D data; one used time trade-off methods to value health state descriptions (vignettes). Although utility scores generally worsened to a lower value with increased HD severity, the estimates varied considerably across studies. The EQ-5D index range was 0.89 - 0.72 for mild/prodromal HD and 0.71 - 0.37 for severe/late-stage disease.
CONCLUSIONS
CONCLUSIONS
This study uncovered high variability in published utility estimates, indicating substantial uncertainty in existing data. Further research is needed to better understand preferences and valuation across all stages and domains of HD symptoms and the degree to which generic utility measures capture the impact of cognitive changes on quality of life.
Identifiants
pubmed: 38627749
doi: 10.1186/s12955-024-02242-1
pii: 10.1186/s12955-024-02242-1
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
33Informations de copyright
© 2024. The Author(s).
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