PB1247 - The societal and economic Impact of severe haemophilia A in paediatric patients without inhibitors treated with Emicizumab

Background: Congenital Haemophilia A present challenges for paediatric patients and caregivers. Conventional treatment involves frequent intravenous prophylactic injections, impacting patients’ lives. Emicizumab is a subcutaneous bispecific monoclonal antibody that has proven efficacy preventing bleeding complications

Aims: This study evaluates the societal and economic impact of emicizumab on severe haemophilia A paediatric patients without inhibitors in Malaysia.

Methods: The study used surveys and economic modeling to evaluate emicizumab's inclusion into clinical practice. Interviews utilising Kid-KINDL and WHOQOL-BREF conducted on 5 patients and their caregivers . Three haematologist were interviewed to give current haemophilia scenarios in the country. The economic impact from the societal perspective, covering direct medical cost, transportation, and productivity loss, was modelled over five years from year 2025. The model estimated the emicizumab-eligible population based on haemophilia A prevalence and , prophylaxis rates and assumed weight of 25kg for paediatric patients. The analysis projected market shares by comparing the treatment scenarios with and without emicizumab.

Results: The study revealed improved QoL quality of life for emicizumab users, with both patients and caregivers reporting higher scores. The economic impact analysis demonstrated a reduction in direct non-medical and indirect costs with emicizumab inclusion in the clinical practice. When comparing the direct medical costs, the economic impact of introducing emicizumab was RM 12.98 million over 5 years. The economic impact of introducing emicizumab was reduced to RM 11.8 million over 5 years when considered from the societal perspective. The reduction was caused by lesser travels for treatments and improved productivity (work/ school days) due to reduction in treatment administration and monitoring, outpatient visits, and bleeding complications.

Conclusion(s): Emicizumab's inclusion in the clinical practice improves both clinical outcomes and economic efficiency in Malaysia. This shift towards a more patient-friendly and cost-effective treatment approach signifies a positive step in managing haemophilia A, enhancing the overall well-being of patients.