OBJECTIVES: A systematic review was conducted to identify utilities associated with adolescents and adults with haemophilia A (HA) and B (HB) (either with or without inhibitors or mixed population of with and without inhibitors) in the US and EU5 (France, Germany, Italy, Spain, UK), to use these findings for future economic evaluations in these territories.

METHODS: Nine publication databases and five other information sources were searched in June 2022. Two reviewers independently assessed records against the eligibility criteria in the protocol. One reviewer extracted data from included studies and a second checked each data point. The review was limited to studies published since 2011 to identify current data.

RESULTS: Twenty-two publications from twenty studies were included: eleven primary studies and nine cost-effectiveness analyses. Data were derived using: EQ-5D-3L (10 studies), EQ-5D-5L (5), SF-6D (2), TTO (1) or were not reported (2). Five studies reported data in patients with inhibitors: four in the US (0.63 to 0.82); one in France (0.57 to 0.77). Eleven studies reported data in patients without inhibitors: six in the US (0.47 to 0.94); seven in the EU5 (0.44 to 0.87) (including two in both territories). Seven studies reported data in mixed populations: four in the US (0.52 to 0.86); two in the EU5 (0.68 to 0.78). More studies reported data for HA (15) than for HB (10). One study that compared utilities in HA with HB found no significant difference.

CONCLUSIONS: The SR shows substantial evidence on the burden of haemophilia in the US and EU5. Baseline values for HA and HB were similar, in the region of 0.7 to 0.8. Evidence on the impact of the presence of inhibitors is limited. The distinction between haemophilia types may not be critical for utilities and studies of mixed populations may be useful for economic models.