OBJECTIVES : Methods for valuing health-related quality-of-life (HRQoL) are accepted for use in the general population but use of these methods is more challenging in children (aged under eighteen). In this study, we investigated the use of HRQoL measures for medicines used by children by reviewing Public Summary Documents released by the Australian Pharmaceutical Benefits Advisory Committee (PBAC), where a cost-utility analysis was performed.

METHODS : We systematically searched the PBAC Public Summary Documents for medicines used in children for the management of childhood diseases using a comprehensive framing list. Medicines from our sample frame where Public Summary Documents were available were searched for whether children were mentioned, and whether utilities, QALYs or a cost-utility analysis were used. Most medicines had multiple documents representing multiple submissions. Where documents met these inclusion criteria, we extracted relevant data.

RESULTS : From a sample of 1889 available Public Summary Documents, we found that 62 documents, (29 medicines) met the inclusion criteria (referred to children and used utility values). Four of these documents (2 medicines) included child-specific HRQoL measures (the Health Utility Index Mark 2). Sixteen documents (8 medicines) used adult-specific HRQoL, most of which were the EuroQol-5D. In 11 documents (8 medicines), direct elicitation was used; predominantly the time trade-off method. In 31 documents (16 medicines), it was not possible to determine the source of the utilities. We then investigated whether the use of child-specific HRQOL measures may have reduced decision-making uncertainty. We found that in 85% of medicines in our sample (where child-specific utilities had not been used) decision-making uncertainty may have been reduced through the use of child-specific utilities.

CONCLUSIONS : Child-specific utilities are not frequently used in decision-making for the subsidization of medicines used by children in Australia, but increased use may reduce decision-making uncertainty.