Is It Possible to Reduce the Costs of Care for Pediatric Patients With Hemophilia A and Inhibitors in Trinidad & Tobago?

Author(s)

Munoz Bedoya A, Ragoobar K, Quesada J
Roche Central America & The Caribbean, Heredia, Costa Rica

OBJECTIVES: To compare the annual cost of prophylaxis in a pediatric patient with Hemophilia A and inhibitors on therapy with a bypassing agent versus the annual cost of prophylaxis with Emicizumab in Trinidad & Tobago.

METHODS: Assuming a 20 kg pediatric patient with a diagnosis of Hemophilia A (HA) and inhibitors, scenario A, estimated the use of aPCC in prophylaxis at a dose of 50 IU/Kg three times a week (156 intravenous applications per year) versus scenario B, estimated the use of emicizumab at a maintenance dose of 6 mg/kg every 4 weeks (13 subcutaneous applications per year). In the two scenarios, bleeding events were included according to the literature, where emicizumab presents a reduction in ABR compared to the use of aPCC and the bleeding event management doses were used according to the guidelines of the World Federation of Hemophilia. The time horizon of the analysis was one year from the perspective of the Trinidad & Tobago healthcare system. Indirect costs were not included in the analysis.

RESULTS: The estimated cost of one year of aPCC prophylaxis treatment for a pediatric patient with HA and inhibitors was $382,477 USD. The estimated cost of one year of emicizumab prophylaxis treatment for a pediatric patient with HA and inhibitors was $163,977 USD.

CONCLUSIONS: When evaluating direct drug costs, Emicizumab could generate annual savings of $218.550 USD (57%) when compared to prophylaxis with aPCC in a pediatric patient with Hemophilia A and Inhibitors in Trinidad & Tobago. It is recommended in future analyzes to include costs associated with the reduction in hospitalizations, reduction of complications such as hemophilic arthropathy, disability, and improvement in quality of life that the literature shows with the use of emicizumab in patients with Hemophilia A.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

EE246

Disease

Rare & Orphan Diseases

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