Bitte verwenden Sie diesen Link, um diese Publikation zu zitieren, oder auf sie als Internetquelle zu verweisen: https://hdl.handle.net/10419/315210 
Erscheinungsjahr: 
2024
Quellenangabe: 
[Journal:] The European Journal of Health Economics [ISSN:] 1618-7601 [Volume:] 25 [Issue:] 8 [Publisher:] Springer [Place:] Berlin, Heidelberg [Year:] 2024 [Pages:] 1437-1448
Verlag: 
Springer, Berlin, Heidelberg
Zusammenfassung: 
Introduction: Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B. Methods: Seven adult and seven child health state vignettes describing ASMD were developed based on published literature, clinical trial results, and interviews with clinicians, patients with ASMD, and parents of children with ASMD. The health states were valued in time trade-off interviews with adult general population respondents in the UK. Results: Interviews were completed with 202 participants (50.0% female; mean age = 41.3 years). The health state representing ASMD without impairment had the highest mean utility for both the adult and child health states (0.92/0.94), and severe ASMD had the lowest mean utility (0.33/0.45). Every child health state had a significantly greater utility than the corresponding adult health state. Differences between adult/child paired states ranged from 0.02 to 0.13. Subgroup analyses explored the impact of parenting status on valuation of child health states. Discussion: Greater severity of ASMD was associated with lower mean utility. Results have implications for valuation of pediatric health states. The resulting utilities may be useful in cost-utility modeling estimating the value of treatment for ASMD.
Schlagwörter: 
Utility
Acid sphingomyelinase deficiency
Rare disease
Time trade-off
TTO
Pediatric utility
JEL: 
I
I00
I01
I10
Persistent Identifier der Erstveröffentlichung: 
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Dokumentart: 
Article
Dokumentversion: 
Published Version

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