Cost-Savings Assessment of Fitusiran Prophylaxis in Minimizing Breakthrough Bleeding Treatment Expenses in the United Arab Emirates
Author(s)
Marion Afonso, MSc1, Daisy Ng-Mak, PhD2, Yuqian Shen, PhD3, Shariq Ali, PhD4, Amy Chen, PhD4, Ahmed Mekky, PharmD5, Wedad Alnemari, MSc, PharmD6, Ion Agirrezabal, PhD7.
1Sanofi, Gentilly, France, 2Sanofi, Hopkinton, MA, USA, 3Sanofi, Bridgewater, NJ, USA, 4Sanofi, Cambridge, MA, USA, 5Sanofi, Dubai, United Arab Emirates, 6Sanofi, Riyadh, Saudi Arabia, 7Sanofi, Barcelona, Spain.
1Sanofi, Gentilly, France, 2Sanofi, Hopkinton, MA, USA, 3Sanofi, Bridgewater, NJ, USA, 4Sanofi, Cambridge, MA, USA, 5Sanofi, Dubai, United Arab Emirates, 6Sanofi, Riyadh, Saudi Arabia, 7Sanofi, Barcelona, Spain.
OBJECTIVES: In the ATLAS-OLE (NCT03754790) trial, fitusiran’s antithrombin-based dosing regimen (AT-DR) was associated with a meaningful reduction in clotting factor concentrates (CFCs)/bypassing agents (BPAs) utilisation for managing breakthrough bleeding episodes versus CFC/BPA prophylaxis. This analysis evaluated cost-savings resulting from this reduction in the United Arab Emirates (UAE).
METHODS: A cost calculator was developed in MS Excel, incorporating episodic CFC/BPA doses, the number of CFC/BPA doses required to treat a bleed during CFC/BPA prophylaxis, mean body weight in the UAE, treatment acquisition and administration costs for CFC/BPA in the UAE, and the decrease in CFC/BPA episodic usage with fitusiran AT-DR. The episodic treatments included were efmoroctocog alfa, octocog alfa and rurioctocog alfa pegol for people with haemophilia (PwH) A without inhibitors and albutrepenonacog alfa, nonacog alfa and eftrenonacog alfa for PwH B without inhibitors. Treatments for PwH with inhibitors included factor VIII inhibitor bypassing activity (FEIBA) and eptacog alfa. The base case analysis assumed no vial sharing. Drug, administration and combined total costs per bleed were calculated for CFC/BPA prophylaxis and fitusiran AT-DR. The cost difference between the two prophylactic treatments was calculated. A scenario analysis examined the impact of vial sharing.
RESULTS: Among PwH without inhibitors, fitusiran AT-DR enabled per-bleed savings ranging from UAE Dirham (AED) 4,625 (efmoroctocog alfa) to AED 11,521 (rurioctocog alfa pegol) in PwH A and from AED 8,935 (nonacog alfa) to AED 30,053 (albutrepenonacog alfa) in PwH B. In PwH with inhibitors, fitusiran AT-DR usage generated per-bleed savings of AED 71,846 (FEIBA) to AED 90,761 (eptacog alfa). Results of the scenario analysis assuming vial sharing supported the base-case findings.
CONCLUSIONS: In the UAE, fitusiran AT-DR prophylaxis may considerably reduce costs associated with episodic treatments for breakthrough bleeds in PwH compared with CFC/BPA prophylaxis. PwH with inhibitors might have larger cost savings than those without inhibitors.
METHODS: A cost calculator was developed in MS Excel, incorporating episodic CFC/BPA doses, the number of CFC/BPA doses required to treat a bleed during CFC/BPA prophylaxis, mean body weight in the UAE, treatment acquisition and administration costs for CFC/BPA in the UAE, and the decrease in CFC/BPA episodic usage with fitusiran AT-DR. The episodic treatments included were efmoroctocog alfa, octocog alfa and rurioctocog alfa pegol for people with haemophilia (PwH) A without inhibitors and albutrepenonacog alfa, nonacog alfa and eftrenonacog alfa for PwH B without inhibitors. Treatments for PwH with inhibitors included factor VIII inhibitor bypassing activity (FEIBA) and eptacog alfa. The base case analysis assumed no vial sharing. Drug, administration and combined total costs per bleed were calculated for CFC/BPA prophylaxis and fitusiran AT-DR. The cost difference between the two prophylactic treatments was calculated. A scenario analysis examined the impact of vial sharing.
RESULTS: Among PwH without inhibitors, fitusiran AT-DR enabled per-bleed savings ranging from UAE Dirham (AED) 4,625 (efmoroctocog alfa) to AED 11,521 (rurioctocog alfa pegol) in PwH A and from AED 8,935 (nonacog alfa) to AED 30,053 (albutrepenonacog alfa) in PwH B. In PwH with inhibitors, fitusiran AT-DR usage generated per-bleed savings of AED 71,846 (FEIBA) to AED 90,761 (eptacog alfa). Results of the scenario analysis assuming vial sharing supported the base-case findings.
CONCLUSIONS: In the UAE, fitusiran AT-DR prophylaxis may considerably reduce costs associated with episodic treatments for breakthrough bleeds in PwH compared with CFC/BPA prophylaxis. PwH with inhibitors might have larger cost savings than those without inhibitors.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE293
Topic
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)