Cost-effectiveness of recombinant activated factor VII vs. plasma-derived activated prothrombin complex concentrate in the treatment of mild-to-moderate bleeding episodes in patients with severe haemophilia A and inhibitors in Spain

Abstract

Several analyses have shown that recombinant activated factor VII (rFVIIa) is a cost-effective intervention compared with plasma-derived activated prothrombin complex concentrate (pd-aPCC) for the on-demand treatment of mild-to-moderate bleeds in haemophilia patients with inhibitors. The aim of the study was to assess the cost-effectiveness of rFVIIa vs. pd-aPCC in the treatment of bleeding episodes in severe haemophilia A patients with inhibitors in Spain. A decision analytic model was designed to evaluate the costs and clinical outcomes of using rFVIIa or pd-aPCC to treat mild-to-moderate joint bleeds in children (≤14 years old) and adults with inhibitors. Data were obtained from a published meta-analysis and a panel of haemophilia experts. The analysis was conducted from the perspective of the Spanish National Healthcare System. One-way sensitivity analyses were performed to assess the impact of model assumptions on study results. In the Treur meta-analysis, rFVIIa resulted in cumulative joint bleed resolution of 88% and 95% after 24 and 36 h, respectively, compared with 62% and 76%, respectively, with pd-aPCC (Treur et al. Haemophilia 2009; 15: 420-36). Here, the mean cost per bleed was estimated at €8473 and €15 579 in children and adults treated with rFVIIa, vs. €8627 and €15 677 in children and adults treated with pd-aPCC. rFVIIa treatment was found to be the dominating option (cheaper and more effective). The one-way sensitivity analysis also confirmed that rFVIIa was less costly than pd-aPCC. The model suggests that rFVIIa is a cost-effective option compared with pd-aPCC for the treatment of mild-to-moderate bleeding episodes in a Spanish setting.

Making economic evaluations more helpful for treatment choices in haemophilia

“…Twenty‐one economic evaluations met our inclusion criteria and were grouped under the following topics: prophylaxis vs. treatment on‐demand (five studies) ; bypassing therapy use (six studies) ; immune tolerance induction (four studies) ; and other topics within haemophilia (six studies) . Details of the CHEERS assessments for the 15 studies discussing the three main topics are given in Appendix S2 (available online) and described below.…”

“…All six studies reviewed examined the comparative cost or cost‐effectiveness of the two available bypassing agents, recombinant activated factor VII (rFVIIa) and plasma‐derived activated prothrombin complex concentrate (pd‐aPCC). One of the main weaknesses in these published economic evaluations stems from the lack of adequate comparative clinical trials.…”

Strategies for inhibitor treatment and costs in the short and long term: a critical evaluation of recent clinical studies

“…Four used data from observational studies or clinical trials; the remainder extracted data from the literature and used expert opinion. In a study to assess the cost‐effectiveness of rFVIIa vs. plasma‐derived activated prothrombin complex concentrates (pd‐aPCC) in the treatment of bleeding episodes in severe haemophilia A patients with inhibitors in Spain, it was found that the mean cost per bleed was estimated at €8473 and €15 579 in children and adults treated with rFVIIa, respectively, vs. €8627 and €15 677 in children and adults treated with pd–aPCC, respectively . Efficacy of rFVIIa was 88.95% compared to 62.7% for aPCC.…”

“…The keywords that we used for our search were inhibitor, haemophilia A, costs, cost analysis and ITI. We found a total of 63 studies of which 40 were excluded at title screening, six were excluded after a review of the abstracts, and five were excluded after full text review, leaving 12 [eight for bypassing agents and four for ITI ] that were suitable for our research (Tables and ).…”

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

“…Com relação ao modelo baseado no estudo de Jiménez-- Yuste et al (2013), observa-se que os únicos cenários em que o rFVIIa teve custo superior ao CCPa foram aumentando a dose de rFVIIa para 350 µg/kg, reduzindo a eficácia de rFVIIa de 88% para 68% ou aumentando a eficácia de CCPa de 62% para 82%. Todas as demais variações avaliadas mantiveram o custo de tratamento com rFVIIa inferior ao com CCPa.…”