Global Assessment of Health Utilities Associated with Pneumococcal Disease in Children-Targeted Literature Reviews.

Abstract

Background: Pneumococcal disease can significantly impact the quality of life (QoL) of children. Health utilities are used to measure the disease burden and calculate quality-adjusted life year (QALY) estimates. These estimates provide critical inputs in economic evaluations of pneumococcal vaccines in children.

Objectives: This study aimed to synthesize utility values used in cost-utility analyses (CUAs) of pediatric pneumococcal vaccines and to summarize published utility studies on pneumococcal disease and post-meningitis sequelae (PMS) in children on a global scale.

Methods: Two targeted literature reviews were conducted to identify CUAs of pediatric pneumococcal vaccines and original studies on health utilities of pneumococcal disease and PMS. Both literature reviews identified relevant studies using published reviews, supplemented by de novo searches conducted in MEDLINE in June 2024 to cover periods not included in those reviews. References from published literature reviews on QoL of pneumococcal disease and CUAs were screened to identify additional original utility studies. Health utility values applied in the CUAs were summarized and the source studies for these utilities were reviewed. For original utility studies, methods and utility estimates were summarized for each condition.

Results: The study identified 45 CUAs of pediatric pneumococcal vaccines in North America and Europe published from 2004 to 2024, and 21 original utility studies on pneumococcal disease or PMS in children published globally from 1994 to 2017. QALY decrement was the most common utility input in CUAs. Most CUAs referenced an earlier CUA for utility inputs, which were often sourced from one or two original utility studies for each health state. Most source studies were published more than two decades ago; some common source studies were conducted in adults. Utility estimates from original studies showed considerable variability, with ranges of -0.330 to 0.6882 for meningitis, -0.331 to 0.93 for non-meningitis invasive pneumococcal disease (IPD), -0.054 to 0.71 for inpatient pneumonia, 0.412-0.82 for outpatient pneumonia, 0.389-0.97 for acute otitis media (AOM)/simple AOM, 0.434-0.540 for recurrent AOM, -0.33 to 0.89 for neurological deficits, and 0.217-0.97 for hearing loss. Variability in methods, including in the surveyed population, utility elicitation method, and use of different country-specific preference weights, substantially impacted utility values. Overall, the methods were not suitable for temporary health states. Additionally, many studies used instruments that have not been validated in children.

Conclusions: Original utility studies demonstrated that pneumococcal disease and PMS are associated with impaired QoL in children; however, there was considerable variability in utility estimates across studies, reflecting the inherent methodological challenges in estimating utilities for acute diseases in children. Most CUAs referenced previous CUAs for health utility values, which were sourced from a limited number of outdated original utility studies. Contemporary data and methods adapted for acute diseases in children are needed for future studies. Given the significance of health utilities in the economic valuations of new pneumococcal vaccines, utility values should be carefully selected in CUAs, considering alternative sources and assumptions.