Cost-Savings Analysis of Fitusiran Prophylaxis: Reducing Breakthrough Bleeding Treatment Expenditure in the Kingdom of Saudi Arabia
Author(s)
Daisy Ng-Mak, PhD1, Marion Afonso, MSc2, Yuqian Shen, PhD3, Shariq Ali, PhD4, Amy Chen, PhD4, Ahmed Mekky, PharmD5, Wedad Alnemari, MSc, PharmD6, Ion Agirrezabal, PhD7.
1HEVA, Rare Disease Business Partner, Sanofi, Hopkinton, MA, USA, 2Sanofi, Gentilly, France, 3Sanofi, Bridgewater, NJ, USA, 4Sanofi, Cambridge, MA, USA, 5Sanofi, Dubai, United Arab Emirates, 6Sanofi, Riyadh, Saudi Arabia, 7Sanofi, Barcelona, Spain.
1HEVA, Rare Disease Business Partner, Sanofi, Hopkinton, MA, USA, 2Sanofi, Gentilly, France, 3Sanofi, Bridgewater, NJ, USA, 4Sanofi, Cambridge, MA, USA, 5Sanofi, Dubai, United Arab Emirates, 6Sanofi, Riyadh, Saudi Arabia, 7Sanofi, Barcelona, Spain.
OBJECTIVES: The results from ATLAS-OLE (NCT03754790) trial demonstrated that fitusiran’s antithrombin-based dosing regimen (AT-DR) could reduce the use of clotting factor concentrates (CFCs)/bypassing agents (BPAs) in breakthrough bleed management compared with CFCs/BPAs prophylaxis in haemophilia. This analysis utilised the ATLAS-OLE data to estimate cost-savings resulting from this reduction in the Kingdom of Saudi Arabia (KSA).
METHODS: A cost calculator was developed in MS Excel incorporating CFC/BPA dosing for breakthrough treatment with CFC/BPA prophylaxis, mean body weight in the KSA, local treatment acquisition and administration costs for CFC/BPA and the decrease in CFC/BPA episodic usage with fitusiran AT-DR. For people with haemophilia (PwH) A without inhibitors, episodic treatments comprised octocog alfa, efmoroctocog alfa and rurioctocog alfa pegol for PwH A and nonacog alfa, albutrepenonacog alfa and eftrenonacog alfa for PwH B. Treatments included for PwH with inhibitors were factor VIII inhibitor bypassing activity (FEIBA) and eptacog alfa. The base case considered no vial sharing. Drug, administration and combined total costs per bleed were computed for CFC/BPA prophylaxis and fitusiran AT-DR. The cost difference between the two regimens were calculated. A scenario analysis assessed the impact of vial sharing.
RESULTS: Among PwH without inhibitors, fitusiran AT-DR demonstrated cost savings per bleed between Saudi riyal (SAR) 3,947 (efmoroctocog alfa) and SAR 6,269 (rurioctocog alfa pegol) in PwH A and between SAR 7,019 (nonacog alfa) and SAR 23,586 (albutrepenonacog alfa) in PwH B. Among PwH with inhibitors, fitusiran AT-DR resulted in the cost savings of SAR 60,581 (FEIBA) to SAR 77,961 (eptacog alfa) per bleed. The results of the scenario analysis assuming vial sharing were consistent with those of the base case.
CONCLUSIONS: In the KSA, fitusiran AT-DR prophylaxis may considerably reduce breakthrough bleed management costs in PwH versus CFC/BPA prophylaxis. Cost savings are predicted to be more substantial in PwH with inhibitors than in those without inhibitors.
METHODS: A cost calculator was developed in MS Excel incorporating CFC/BPA dosing for breakthrough treatment with CFC/BPA prophylaxis, mean body weight in the KSA, local treatment acquisition and administration costs for CFC/BPA and the decrease in CFC/BPA episodic usage with fitusiran AT-DR. For people with haemophilia (PwH) A without inhibitors, episodic treatments comprised octocog alfa, efmoroctocog alfa and rurioctocog alfa pegol for PwH A and nonacog alfa, albutrepenonacog alfa and eftrenonacog alfa for PwH B. Treatments included for PwH with inhibitors were factor VIII inhibitor bypassing activity (FEIBA) and eptacog alfa. The base case considered no vial sharing. Drug, administration and combined total costs per bleed were computed for CFC/BPA prophylaxis and fitusiran AT-DR. The cost difference between the two regimens were calculated. A scenario analysis assessed the impact of vial sharing.
RESULTS: Among PwH without inhibitors, fitusiran AT-DR demonstrated cost savings per bleed between Saudi riyal (SAR) 3,947 (efmoroctocog alfa) and SAR 6,269 (rurioctocog alfa pegol) in PwH A and between SAR 7,019 (nonacog alfa) and SAR 23,586 (albutrepenonacog alfa) in PwH B. Among PwH with inhibitors, fitusiran AT-DR resulted in the cost savings of SAR 60,581 (FEIBA) to SAR 77,961 (eptacog alfa) per bleed. The results of the scenario analysis assuming vial sharing were consistent with those of the base case.
CONCLUSIONS: In the KSA, fitusiran AT-DR prophylaxis may considerably reduce breakthrough bleed management costs in PwH versus CFC/BPA prophylaxis. Cost savings are predicted to be more substantial in PwH with inhibitors than in those without inhibitors.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE292
Topic
Clinical Outcomes, Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)