OBJECTIVES: Whilst lifelong high-dose prophylaxis (Px) is considered the optimal standard of care (SOC) for hemophilia A (HA), the perceived high cost and limited access and availability of factor FVIII could limit adoption of high-dose Px. Thus, many developing countries adopt less costly Px management alternatives, including low- and intermediate-dose Px treatment. This study estimates the potential clinical and economic benefits of optimizing Px treatment strategies to improve the SOC in patients with severe HA.

METHODS: A burden-of-illness model compared optimization of Px regimens, including low-, intermediate- and high-dose Px in two developing countries – Malaysia and Chile – over a five-year period. Annualized bleeding rates (ABRs) were calculated for each treatment strategy, based on relative risk reductions taken directly, or based on data, from published international, long-term clinical studies. Epidemiology, unit costs, and healthcare resource utilization (HCRU) data were obtained via local publications or expert interviews. Regimens were compared in terms of clinical outcomes – including bleeds, target joints, and disabilities – as well as direct and indirect costs.

RESULTS: Optimizing Px treatment strategy in patients could lead to a 15% or greater improvement in clinical outcomes, including bleeds, target joints and disabilities over a five-year period, depending on the level of Px adopted. Whilst Px-related factor cost is expected to increase with higher Px dosing, this cost is partially offset by reduced HCRU costs, bleed-related treatment factor costs and indirect costs due to productivity loss and school/work absenteeism. Results varied according to baseline SOC and the level of Px adopted.

CONCLUSIONS: A flexible model considering baseline SOC and comparing various Px regimens can provide insights into long-term benefits of different treatment strategies and inform health policy decisions to improve the SOC for HA patients, especially for countries such as Malaysia and Chile, where resources and budgets are likely to be more limited.