Sources of the utilities used in paediatric cost-utility analyses in children aged under 5 years, and the methods used in their elicitation: a systematic review.

OBJECTIVES

Few multi-attribute utility instruments (MAUIs) have been developed and validated for children under five years, limiting the evidence on health-related quality of life used to inform resource allocation decisions. This systematic review aimed to characterize utilities used in cost-utility analyses (CUAs) involving children aged 0-5 years published between 2012 and 2023, their sources, and the methods used to obtain them.

METHODS

Studies were identified from the Paediatric Economic Database Evaluation (PEDE) Project. Eligible studies were CUAs reporting quality-adjusted life years (QALYs) with at least one health state for children aged 0-5 years. Descriptive analyses were conducted to summarise study characteristics, sources of utilities, and methods used to obtain utilities. Changes over time were explored by comparing studies published in 2012-2017 with 2018-2023.

RESULTS

Of the 1,098 studies screened, 162 met inclusion criteria. Of these, 87 (53.7%) had health states exclusively in the 0-5 years age-group, 16 (9.9%) included states also in the 5-18 years age-group, and 59 (36.4%) included states also in adulthood. Two-thirds of CUAs evaluated were preventative interventions. Seventy percent of studies clearly reported their utility sources. Among studies that obtained utilities from the literature (82.2%), most used MAUIs, with the adult EQ-5D-3L valued using the UK tariff being most common (33%).

CONCLUSIONS

CUAs involving children under five years have increased; but the methods for obtaining utilities remain varied and underreported. The frequent reliance on adult MAUIs and utilities underscores the need for validated age-appropriate instruments to improve the validity of QALY estimates in this age group.