Cost of care of haemophilia with inhibitors

Abstract

In Western countries, the treatment of patients with inhibitors is presently the most challenging and serious issue in haemophilia management, direct costs of clotting factor concentrates accounting for >98% of the highest economic burden absorbed for the healthcare of patients in this setting. Being designed to address questions of resource allocation and effectiveness, decision models are the golden standard to reliably assess the overall economic implications of haemophilia with inhibitors in terms of mortality, bleeding-related morbidity, and severity of arthropathy. However, presently, most data analyses stem from retrospective short-term evaluations, that only allow for the analysis of direct health costs. In the setting of chronic diseases, the cost-utility analysis, that takes into account the beneficial effects of a given treatment/healthcare intervention in terms of health-related quality of life, is likely to be the most appropriate approach. To calculate net benefits, the quality adjusted life year, that significantly reflects such health gain, has to be compared with specific economic impacts. Differences in data sources, in medical practice and/or in healthcare systems and costs, imply that most current pharmacoeconomic analyses are confined to a narrow healthcare payer perspective. Long-term/lifetime prospective or observational studies, devoted to a careful definition of when to start a treatment; of regimens (dose and type of product) to employ, and of inhibitor population (children/adults, low-responding/high responding inhibitors) to study, are thus urgently needed to allow for newer insights, based on reliable data sources into resource allocation, effectiveness and cost-utility analysis in the treatment of haemophiliacs with inhibitors.

Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A

“…3 Although inhibitor development is less frequently observed in patients with nonsevere hemophilia A (baseline factor VIII activity of 2-40 IU/dL), the clinical impact can be profound. In these patients, inhibitors may also interact with their endogenous factor VIII, resulting in a decrease of the factor VIII plasma level below 1 IU/dL 1 and major bleeding complications.…”

Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A

“…These so-called inhibitors may jeopardize the patient's life 3 and make therapeutic management more complex 4,5 and costly. 6 Inhibitors arise in 15% to 35% of children with severe HA. 7,8 Several genetic and nongenetic risk factors for inhibitor development have been described.…”

Optimizing management of immune tolerance induction in patients with severe haemophilia A and inhibitors: towards evidence‐based approaches

“…In the lifetime perspective, this economic burden, further multiplying the strikingly high costs of the management of inhibitor patients (Gringeri et al, 2003), is limited to 1-3 years, and the high success rate and the low number of inhibitor recurrence after ITI predict a considerable reduction of costs in the majority of treated patients (Colowick et al, 2000). However, the variability of dose regimens and success rates, may significantly affect cost-effectiveness and costutility analysis (Knight, 2005;Di Minno et al, 2010). A recent European survey highlighted this issue, showing major differences in the management of ITI (Astermark et al, 2006c).…”