Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian study

OBJECTIVE: The aim of the study was to compare the treatment with plasma-derived activated prothrombin complex concentrate (APCC) and recombinant activated FVII (rFVIIa) in patients with Hemophilia A with inhibitors, analyzing the costs and budget impact of prophylactic and on-demand treatment in t...

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Main Authors: Christiane Maria da Silva Pinto, André Luis Souza Silveira, Fábio Carvalho, Janaina da Silva, Tatiane Bomfim Ribeiro, Gabriela Vilela de Brito
Format: Article
Language:English
Published: Instituto Nacional de Assistência Farmacêutica e Farmacoeconomia 2025-08-01
Series:Jornal de Assistência Farmacêutica e Farmacoeconomia
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Online Access:https://ojs.jaff.org.br/ojs/index.php/jaff/article/view/1055
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author Christiane Maria da Silva Pinto
André Luis Souza Silveira
Fábio Carvalho
Janaina da Silva
Tatiane Bomfim Ribeiro
Gabriela Vilela de Brito
author_facet Christiane Maria da Silva Pinto
André Luis Souza Silveira
Fábio Carvalho
Janaina da Silva
Tatiane Bomfim Ribeiro
Gabriela Vilela de Brito
author_sort Christiane Maria da Silva Pinto
collection DOAJ
description OBJECTIVE: The aim of the study was to compare the treatment with plasma-derived activated prothrombin complex concentrate (APCC) and recombinant activated FVII (rFVIIa) in patients with Hemophilia A with inhibitors, analyzing the costs and budget impact of prophylactic and on-demand treatment in the Brazilian Unified Health System (In Portuguese, Sistema Único de Saúde [SUS]). METHODS: This study performs a Cost-Minimization Analysis (CMA) and a Budget Impact Model) BIM for APCC versus rFVIIa in the prophylaxis and on-demand treatment in SUS perspective, based on analysis of guidelines and public databases from Brazil. RESULTS: The cost per patient for prophylaxis is estimated to be lower with APCC than in rFVIIa for the maximum dose with an incremental cost difference of BRL 2,730,798 (APCC BRL 3,325,894 versus rFVIIa BRL 6,056,692). The BIM estimated that 5 years after the partial substitution of rFVIIa for APCC (30% and 70%, respectively) brings the potential saving of BRL 201,622,888 considering prophylaxis in adults and BRL 83,095,850 in children. For on-demand usage, the amount saved could be BRL 42,696,097 in adults and BRL 17,596,556 in children. CONCLUSION: This analysis suggests a potential cost saving from payer’s perspective (SUS) using APCC instead rFVIIa for Hemophilia A patients with inhibitors, mainly for prophylaxis. The results could potentially bring value to patients and payers as resources could be reallocated to provide more care with the same budget, considering patient clinic response.
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publishDate 2025-08-01
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spelling doaj-art-0ea37dc2387c4a77b350ec5032ee953d2025-08-20T04:02:51ZengInstituto Nacional de Assistência Farmacêutica e FarmacoeconomiaJornal de Assistência Farmacêutica e Farmacoeconomia2525-50102525-73232025-08-0110310.22563/2525-7323.2025.v10.e00251Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian studyChristiane Maria da Silva PintoAndré Luis Souza SilveiraFábio CarvalhoJanaina da SilvaTatiane Bomfim RibeiroGabriela Vilela de Brito OBJECTIVE: The aim of the study was to compare the treatment with plasma-derived activated prothrombin complex concentrate (APCC) and recombinant activated FVII (rFVIIa) in patients with Hemophilia A with inhibitors, analyzing the costs and budget impact of prophylactic and on-demand treatment in the Brazilian Unified Health System (In Portuguese, Sistema Único de Saúde [SUS]). METHODS: This study performs a Cost-Minimization Analysis (CMA) and a Budget Impact Model) BIM for APCC versus rFVIIa in the prophylaxis and on-demand treatment in SUS perspective, based on analysis of guidelines and public databases from Brazil. RESULTS: The cost per patient for prophylaxis is estimated to be lower with APCC than in rFVIIa for the maximum dose with an incremental cost difference of BRL 2,730,798 (APCC BRL 3,325,894 versus rFVIIa BRL 6,056,692). The BIM estimated that 5 years after the partial substitution of rFVIIa for APCC (30% and 70%, respectively) brings the potential saving of BRL 201,622,888 considering prophylaxis in adults and BRL 83,095,850 in children. For on-demand usage, the amount saved could be BRL 42,696,097 in adults and BRL 17,596,556 in children. CONCLUSION: This analysis suggests a potential cost saving from payer’s perspective (SUS) using APCC instead rFVIIa for Hemophilia A patients with inhibitors, mainly for prophylaxis. The results could potentially bring value to patients and payers as resources could be reallocated to provide more care with the same budget, considering patient clinic response. https://ojs.jaff.org.br/ojs/index.php/jaff/article/view/1055Blood Coagulation FactorsHemophilia ABlood Coagulation Factor InhibitorsHematologic AgentsFactor VIIaHealth Care Economics and Organizations
spellingShingle Christiane Maria da Silva Pinto
André Luis Souza Silveira
Fábio Carvalho
Janaina da Silva
Tatiane Bomfim Ribeiro
Gabriela Vilela de Brito
Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian study
Jornal de Assistência Farmacêutica e Farmacoeconomia
Blood Coagulation Factors
Hemophilia A
Blood Coagulation Factor Inhibitors
Hematologic Agents
Factor VIIa
Health Care Economics and Organizations
title Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian study
title_full Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian study
title_fullStr Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian study
title_full_unstemmed Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian study
title_short Cost-minimization and budget impact analysis of bypassing agents in hemophilia A with inhibitors: a Brazilian study
title_sort cost minimization and budget impact analysis of bypassing agents in hemophilia a with inhibitors a brazilian study
topic Blood Coagulation Factors
Hemophilia A
Blood Coagulation Factor Inhibitors
Hematologic Agents
Factor VIIa
Health Care Economics and Organizations
url https://ojs.jaff.org.br/ojs/index.php/jaff/article/view/1055
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