Abstract
ObjectiveObtain utilities (preferences) for a generalizable set of health states experienced by older children and adolescents who receive therapy for chronic health conditions.MethodsA health state classification system, the Adolescent Health Utility Measure (AHUM), was developed based on generic health status measures and input from children with Hunter syndrome and their caregivers. The AHUM contains six dimensions with 4–7 severity levels: self-care, pain, mobility, strenuous activities, self-image, and health perceptions. Using the time trade off (TTO) approach, a UK population sample provided utilities for 62 of 16,800 AHUM states. A mixed effects model was used to estimate utilities for the AHUM states. The AHUM was applied to trial NCT00069641 of idursulfase for Hunter syndrome and its extension (NCT00630747).ResultsObservations (i.e., utilities) totaled 3,744 (12*312 participants), with between 43 to 60 for each health state except for the best and worst states which had 312 observations. The mean utilities for the best and worst AHUM states were 0.99 and 0.41, respectively. The random effects model was statistically significant (p < 0.0001; adjusted R2 = 0.361; RMSE = 0.194). When AHUM utilities were applied to the idursulfase trial, mean utilities in the idursulfase weekly and placebo groups improved +0.087 and +0.006, respectively, from baseline to week 53. In the extension, when all patients received idursulfase, the utilities in the treatment group remained stable and the placebo group improved +0.039.DiscussionThe AHUM health state classification system may be used in future research to enable calculation of quality-adjust life expectancy for applicable health conditions.
Highlights
An important innovation in cost effectiveness analysis in health care has been the development of the quality adjusted life year (QALY) that combines longevity with quality of life
Selection of Adolescent Health Utility Measure (AHUM) attributes In selecting the attributes for the AHUM, we considered the concepts captured by common generic measures such as the EQ-5D and the SF-6D
All participants who provided illogical ratings were included in the modeling exercise; one participant was excluded from the analysis given that, because of religious reasons, the person did not want to make choices with respect to trading time off to avoid being in hypothetical health states
Summary
An important innovation in cost effectiveness analysis in health care has been the development of the quality adjusted life year (QALY) that combines longevity with quality of life. The last decade has seen increasing use made of generic utility measures that are assigned preference weights based on the general population perspective, as recommended by the US Panel on Cost-Effectiveness in Health and Medicine and used by the National Institute for Health and Clinical Excellence (NICE) [4,5] Examples of such measures include the EQ-5D and the SF-6D [6,7]. These are multi-attribute scales based on a generic health state classification where health is described across multi-level dimensions of health applicable across different health conditions These measures come with a set of preference weights obtained from members of the general public using a preference elicitation technique like the time trade off (TTO) approach. All such multiattribute measures have descriptive systems that are derived from adult populations with the exception of the Heath Utility Index Mark 2, and additional attributes that are relevant to child health, including autonomy and body image, may not be captured [2]
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