• Gaëlle Bégo-Le Bagousse, Xiaoying Jia, Sorrel Wolowacz, Laurent Eckert, Jules Tavi, and Richard Hudson.
• Health Economics and Value Assessment, Sanofi, Chilly-Mazarin, France.
• Curr Med Res Opin. 2020 Jul 1; 36 (7): 1209-1224.

AbstractObjective: Health utility estimates for children and adolescents are critical for cost-utility analyses informing health technology assessment (HTA) authorities' decisions governing access to pediatric treatments. However, in a recent review, only 29% of published pediatric cost-utility models used a utility measure validated for children. We examined utility estimates used in pediatric HTAs.Methods: A targeted review of pediatric HTAs was performed, focusing on agencies reporting utility estimate sources and methods.Results: Searches identified 11 HTAs in pediatric indications and five in mixed populations with separate analyses for adults and children. Among 13 appraisals reporting methodological detail, five used pediatric utility estimates (based on the Health Utilities Index [HUI], n = 3; Atopic Dermatitis Quality of Life [ADQoL], n = 1; or mapping, n = 1). Issues were identified with mapping, use of adult data for some health states, and assumptions about ADQoL responses. In the remaining eight appraisals, adult utility estimates were applied. Caregiver utility was included in two of 16 appraisals.Conclusions: Only 38% of pediatric HTAs reviewed used pediatric utility estimates, and HTA authorities raised concerns about these data in many cases; only 12% of HTAs included caregiver utility. Although several preference-based utility measures are available for pediatric populations, limited data and guidance on selection of measures are available. When estimating pediatric utility weights, alternative measures should be reviewed for suitability given the model population and health condition. Pediatric and adult utility estimates should be applied appropriately as patients age over time, and caregiver and/or family member utility should be included, where relevant. Gaps exist in utility measures for children aged <4 years and caregivers.

16 keywords...

hide…