Pediatrics
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Cost-utility analysis in which health benefits are quantified in terms of quality-adjusted life-years (QALYs) has now become the standard type of cost-effectiveness analysis. These studies are potentially influential in determining the extent of funding for particular pediatric interventions, and so their methodologic quality is extremely important. The objective of this study was twofold: first, to critically appraise published cost-utility analyses of interventions in child and adolescent health care in terms of the methods used to derive QALYs and, second, to discuss unresolved methodologic issues that are pertinent to the measurement of QALYs in pediatric populations. ⋯ Although variations from standard recommendations may be attributable to poor practice among researchers who are either unaware of these recommendations or choose not to follow them, they could equally be the result of attempts to make research more rigorous and more defensible than it might be if the standard recommendations were followed. There are 4 potential approaches to conducting cost-utility analysis in pediatric populations: (1) the explicit development of a generic instrument designed to be applicable across both child and adult populations (likely to be difficult in practice), (2) insistence on use of a generic instrument developed for adults, (3) the use of generic instruments specifically developed for children without being concerned about comparability with interventions aimed at adults, and (4) abandoning attempts to use single outcome measures that combine mortality with quality weights. In the absence of a clear way forward, it is suggested that an expert panel be convened to debate and further consider these potential solutions and recommendations for best practice and future research. In the interim, comparisons of the relative cost-effectiveness reported as cost per QALY gained across interventions for different diseases and populations should be treated with extreme caution.