OBJECTIVES: Health utility estimation for sensory losses and pediatric populations is methodologically challenging, further compounded in congenital cytomegalovirus disease (cCMVd), where infants and children may sustain hearing, visual, and cognitive losses and a variety of other sequalae. These sequelae may occur alone or in combination, range in severity and age of onset, and persist into adulthood. We developed a conceptual study design to measure utility for cCMVd health states and spillover effects for parents and siblings to inform future economic evaluation of new prevention programmes.
METHODS: Systematic and targeted reviews were performed to identify sequelae associated with cCMVd and existing utility estimates. Economic model health states were defined for hearing impairment, other moderate sequelae, and severe sequelae (transient and long-term) by age group (neonate, infant, child, adolescent, and adult). The following preference-based measures were reviewed for age-range suitability, alignment of the descriptive system with key sequelae, and empirical responsiveness to key sequelae: EQ-5D, EQ-5D-Y, HUI, SF-6D, CHU-9D, 15/16/17D, AQoL, QWB, and mapping from the PedsQL. Alternative study designs for utility measurement in cCMVd were evaluated.
RESULTS: The systematic review identified no utility estimates for cCMVd. The HUI was determined to be the most appropriate utility measure overall. An observational study with a sampling frame capturing the spectrum of age, sequela type, and severity was recommended, using proxy respondents for young children and patients with cognitive impairment. Estimation of spillover effects was recommended by valuation of health state descriptions (vignettes), as existing utility measures were not designed for and may not adequately capture spillover effects.
CONCLUSIONS: An observational study using the HUI and a vignette valuation study is recommended to address the lack of utility data for cCMVd. Additional research is recommended to further evaluate the responsiveness of the HUI in cCMVd.
