A cost minimization model for the treatment of minor bleeding episodes in patients with haemophilia A and high‐titre inhibitors

Abstract

Treatment of acute bleeding episodes in patients with haemophilia A and inhibitory antibodies to factor VIII (FVIII) most often involves the use of bypassing haemostatic agents, such as activated prothrombin complex concentrates (aPCC) or recombinant factor VIIa (rFVIIa). We constructed a cost minimization model to compare the costs of initial treatment with aPCC vs. rFVIIa in the home treatment of minor bleeding episodes. We developed a clinical scenario describing such a case and presented it to a panel of US haemophilia specialists. For each product class, we asked panellists to provide dosing regimens required to achieve complete resolution of a minor haemarthrosis in a child with high-titre inhibitors, and for the probabilities of success at two time points (8-12 and 24 h). Consensus among the panellists was refined by a second round of the process, and the median values resulting were used as inputs to a decision analysis model. Sensitivity analyses were conducted to determine threshold values for key variables. The base case model found that initial treatment with aPCC would result in a mean cost per episode of 21 000 dollars, compared with 33 400 dollars for initial treatment with rFVIIa. Sensitivity analyses over a range of clinically plausible values for cost, dosing, and efficacy did not change the selection of aPCC as the dominant strategy.

Pharmacoeconomic analysis of recombinant factor VIIa versus APCC in the treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors

“…Two previous US economic analyses were identified, both adopting a short-term perspective 24,25 . These studies concluded that APCC was less expensive in the initial line of treatment; however, both studies failed to account for treatment failures and re-bleeding and its associated costs in subsequent lines of treatment, thus underestimating the true costs of managing a bleed.…”

Utilization of recombinant activated factor VII for intracranial hematoma evacuation in coagulopathic nonhemophilic neurosurgical patients with normal international normalized ratios

“…The administration of rFVIIa may be met with opposition from the hematology services due to the high cost of the intervention in the setting of normal INR values [20]. In our first instance, although the use of rFVIIa was denied for the patient's second and third SDH evacuations, the prospect of coagulopathic bleeding during the patient's EDH enabled him to receive approval for rFVIIa despite his normal INR.…”

Custo-efetividade do fator VII ativado recombinante (rFVIIa) e complexo protrombínico parcialmente ativado (CCPa) no tratamento sob demanda do sangramento leve/moderado de pacientes com hemofilia congênita complicada por inibidores sob a perspectiva do sistema público de saúde do Brasil

“…Buscando validar os resultados encontrados e minimizar o impacto das limitações acima expostas, foi realizada uma busca por estudos farmacoeconômicos que avaliaram o rFVIIa em diferentes cenários e países. Sete estudos foram avaliados visando demonstrar e comparar os principais aspectos econô-micos do tratamento em outras populações e comprovaram os resultados encontrados neste estudo (Dundar et al, 2005;Ozelo et al, 2007;Putnam et al, 2005;Seremetis et al, 2006;Lyseng & Plosker, 2007;Treur et al, 2009;Knight et al, 2003). De maneira geral, os estudos concluíram que o rFVIIa foi mais eficaz e mais economicamente viável comparado ao CCPa, quando utilizado como tratamento de primeira linha dos episódios hemorrágicos leves a moderados.…”

“…Assim, para a estimativa dos custos e desfechos dos tratamentos, foram reproduzidas as estruturas das árvores de decisão de dois modelos internacionais já publicados, que acompanharam pacientes com hemofilia complicada por inibidores no tratamento de episódios de sangramento leve a moderado. Esses modelos foram extraídos de uma busca na literatura por estudos econômicos (You et al, 2009;Dundar et al, 2005;Ozelo et al, 2007;Putnam et al, 2005;Seremetis et al, 2006;Lyseng & Plosker, 2007;Treur et al, 2009;Knight et al, 2003;Jiménez-Yuste et al, 2013).…”

“…Uma busca na literatura sobre estudos econômicos avaliando a relação de custo-efetividade do uso de rFVIIa em comparação ao CCPa no mundo mostrou resultados favorá-veis para rFVIIa, motivando a elaboração de uma análise de custo-efetividade para a realidade brasileira (You et al, 2009;Dundar et al, 2005;Ozelo et al, 2007;Putnam et al, 2005;Seremetis et al, 2006;Lyseng & Plosker, 2007;Treur et al, 2009;Knight et al, 2003;Jiménez-Yuste et al, 2013).…”