Economic modelling of different treatment strategies for haemophilia A with high‐responding inhibitors

Abstract

This paper reports a systematic review of the cost-effectiveness of treatment options in patients with haemophilia A with inhibitors. As very little relevant published evidence was identified, an economic modelling exercise was undertaken to calculate the cost-effectiveness of different strategies in the treatment of high-responding haemophilia A patients with inhibitors. A decision analysis approach was used to model the expected lifetime clinical outcomes and costs of the more common regimens currently used in UK in treating severe haemophiliacs with inhibitors. The model attempts to reflect the outcomes of clinical events, costs and life expectancy for each different treatment regimen for haemophilic boys with inhibitors who are high responders (defined as inhibitor level >/=10 BU) throughout their life. The basic model structure is centred on a Markov decision process, which was used to simulate, at quarter-yearly intervals, the movement through discrete health states and their complications. The model allows a comparison of cost-effectiveness between three immune tolerance induction (ITI) regimens (Bonn, Mälmo and Low-Dose protocols) and against a relevant 'on-demand' (OD) regimen. It also shows the cost-effectiveness of different OD regimens using different bypassing agents. The results of the economic modelling indicate that treating haemophilia A patients who have high-responding inhibitors OD with recombinant activated factor VII is cost-effective compared to treatment with activated prothrombin complex concentrates. However, when OD treatment regimens are compared with the three ITI protocols, the Malmö ITI protocol is the preferred treatment strategy, generating more quality adjusted life-years (QALYs) and less cost than either an OD regimen or the Bonn or Low-Dose ITI protocols.

Epidemiological survey of haemophiliacs with inhibitors in France: orthopaedic status, quality of life and cost – the ‘Statut Orthopédique des Patients Hémophiles’ avec Inhibiteur study

“…French survey of haemophiliacs with inhibitors Stieltjes et al 9 It is well known that management of haemophilia patients with inhibitors is very costly [25][26][27]. The average monthly cost in our study is similar to that found in the COCIS study (s22 417 in SOPHI versus s20 893 in COCIS).…”

Prevention of bleeds in hemophilia patients with inhibitors: Emerging data and clinical direction

“…Because it directly targets the production of inhibitors, ITT is the only way to modify the natural course of this serious complication of hemophilia. Successful treatment can improve quality of life and potentially increase life expectancy [4,31]. Nonetheless, the procedure is time-consuming and expensive, approaching $1 million annually for the average 5-year-old child [4].…”

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

“…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).…”