ECONOMIC IMPACT OF RECOMBINANT ACTIVATED FACTOR VII PROPHYLAXIS IN SEVERE GLANZMANN THROMBASTHENIA: A PROSPECTIVE COHORT ANALYSIS FROM SAUDI ARABIA
Author(s)
George Morgan, MSc1, Sarah Brighton, MSc1, Emily Back, MSc1, Samuel Bristow, BSc, MSc1, Ahmad Tarawah, Prof.2.
1DHT.health, Clitheroe, United Kingdom, 2Madinah Hereditary Blood Disorders Center, King Salman Medical City, Madinah, Saudi Arabia.
1DHT.health, Clitheroe, United Kingdom, 2Madinah Hereditary Blood Disorders Center, King Salman Medical City, Madinah, Saudi Arabia.
OBJECTIVES: Glanzmann thrombasthenia (GT) is an ultra-rare inherited platelet disorder associated with recurrent bleeding, frequent hospitalization, and substantial healthcare resource utilization. Recombinant activated factor VII (rFVIIa) prophylaxis has demonstrated clinical benefit in patients with severe GT, however evidence on its economic impact remains limited. This study aimed to evaluate the direct medical cost impact of rFVIIa prophylaxis compared with on-demand treatment in a cohort of pediatric patients with severe GT.
METHODS: A prospective cohort study of pediatric patients with severe GT receiving rFVIIa prophylaxis was conducted at a specialized hereditary bleeding disorders center in Saudi Arabia between September 2022 and August 2025. Direct medical costs during prophylaxis were compared with an equal-duration pre-prophylaxis period within each patient. Cost categories included rFVIIa acquisition, hospitalization, and platelet transfusion costs. Median annual costs were calculated from a healthcare system perspective. Deterministic sensitivity analyses varying key unit costs by ±20% were conducted.
RESULTS: Eleven pediatric patients with severe GT were included. Prophylaxis was associated with reductions in treated bleeding episodes, hospital days, and platelet transfusion requirements. Although annual rFVIIa acquisition costs increased during prophylaxis, these were offset by reductions in hospitalization and platelet transfusion costs. Median net annual cost impact was -1,305 SAR per patient. Sensitivity analyses estimated median net annual cost impact ranging from -93,339 SAR to 90,729 SAR across scenarios. Based on an estimated GT prevalence of 1:200,000 in Saudi Arabia, findings suggest limited overall budget impact if similar outcomes were observed more broadly.
CONCLUSIONS: In this cohort of pediatric patients with severe GT, reductions in hospitalization and platelet transfusion requirements offset much of the additional expenditure associated with prophylactic rFVIIa treatment. These findings provide the first evidence that prophylactic rFVIIa may reduce clinical burden while maintaining broadly comparable direct medical costs to on-demand treatment in patients with severe GT.
METHODS: A prospective cohort study of pediatric patients with severe GT receiving rFVIIa prophylaxis was conducted at a specialized hereditary bleeding disorders center in Saudi Arabia between September 2022 and August 2025. Direct medical costs during prophylaxis were compared with an equal-duration pre-prophylaxis period within each patient. Cost categories included rFVIIa acquisition, hospitalization, and platelet transfusion costs. Median annual costs were calculated from a healthcare system perspective. Deterministic sensitivity analyses varying key unit costs by ±20% were conducted.
RESULTS: Eleven pediatric patients with severe GT were included. Prophylaxis was associated with reductions in treated bleeding episodes, hospital days, and platelet transfusion requirements. Although annual rFVIIa acquisition costs increased during prophylaxis, these were offset by reductions in hospitalization and platelet transfusion costs. Median net annual cost impact was -1,305 SAR per patient. Sensitivity analyses estimated median net annual cost impact ranging from -93,339 SAR to 90,729 SAR across scenarios. Based on an estimated GT prevalence of 1:200,000 in Saudi Arabia, findings suggest limited overall budget impact if similar outcomes were observed more broadly.
CONCLUSIONS: In this cohort of pediatric patients with severe GT, reductions in hospitalization and platelet transfusion requirements offset much of the additional expenditure associated with prophylactic rFVIIa treatment. These findings provide the first evidence that prophylactic rFVIIa may reduce clinical burden while maintaining broadly comparable direct medical costs to on-demand treatment in patients with severe GT.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE716
Topic
Economic Evaluation, Health Service Delivery & Process of Care, Study Approaches
Topic Subcategory
Budget Impact Analysis, Cost/Cost of Illness/Resource Use Studies
Disease
Pediatrics, Rare & Orphan Diseases