Cost of care of haemophilia with inhibitors.
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.
Reference Centre for Hemophilia and Thrombosis, Department of Clinical and Experimental Medicine, Federico II University, Via S.Pansini 5, Naples, Italy. diminno@unina.it
M N D
MN
Di Minno
G
G
Di Minno
M
M
Di Capua
A M
AM
Cerbone
A
A
Coppola
D019774:Blood Coagulation Factor Inhibitors / Q000191:economics*; D001779:Blood Coagulation Factors / Q000627:therapeutic use; D003362:Cost-Benefit Analysis; D003661:Decision Support Techniques; D015942:Factor VIIa / Q000627:therapeutic use; D017048:Health Care Costs; D006467:Hemophilia A / Q000188:drug therapy* / Q000191:economics*; D002836:Hemophilia B / Q000188:drug therapy* / Q000191:economics*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D011788:Quality of Life; D011994:Recombinant Proteins / Q000627:therapeutic use
D019774:Blood Coagulation Factor Inhibitors; D001779:Blood Coagulation Factors; D011994:Recombinant Proteins; C025667:prothrombin complex concentrates; C103587:recombinant FVIIa; D015942:Factor VIIa