Systematic Review of Cost-Effectiveness Modelling Studies for Haemophilia

OBJECTIVES: Haemophilia is a rare genetic disease that hinders blood clotting. Novel treatment approaches have significantly improved clinical outcomes and relieved the treatment burden on patients but also incurred substantial costs to health care systems. We aimed to review model-based cost-effectiveness analyses (CEAs), determine the cost-effectiveness of different treatment strategies, summarize the cited clinical effectiveness literature used by the included CEAs, and assess the quality and risk of bias of the included CEAs.

METHODS: We conducted a systematic literature review of model-based CEAs on haemophilia treatments by searching MEDLINE, Embase, the Tufts Medical Center CEA registry, and the grey literature. We summarized the results of the included CEAs based on the specifications of our protocol, and we performed a qualitative synthesis.

RESULTS: We identified 32 eligible studies that were performed in 12 countries and reported 53 pairwise comparisons. Most studies analysed patients with haemophilia A rather than patients with haemophilia B. Some comparisons of prophylactic versus on-demand treatment indicated that prophylactic treatment may be too expensive compared to improvements in clinical outcomes, but there was no clear consensus. Emicizumab was found to be more effective and less costly than prophylactic treatment with coagulation factors. Gene therapies as well as treatment with extended half-life coagulation factors were always more cost-effective than their comparators.

CONCLUSIONS: Differences in results between previous CEAs may have been driven by heterogeneity in modelling approaches, clinical input data, and potential funding biases. A more consistent evidence base and modelling approach would enhance the comparability between CEAs.