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Medico-economic assessment of haemophilia A at the dawn of the new millennium Volume 7, issue 6, Novembre - Décembre 2001

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Annie Chicoye Economics ACE, 10, rue Beffroy, 92200 Neuilly-sur-Seine, France

Cost and medical evaluations are difficult to achieve. The reasons are: the great variability in the way patients with hemophilia are taken charge of: the evolutivity of the illness; as well as the difficulties of rendering clinical trials on a sufficient number of patients and the external validity of the studies. Objectives. The aim of this study is to bring forward the medico-economic approaches of the different studies over the past 15 years. We studied to identify the kind of studies, strategies taken into account, methodologies and the results. Methods. A systematic search was carried out through the electronic databases MEDLINE, MEDSCAPE, British Medical Library. We identified papers through references cited by "Pharmaco-economics and outcomes news" and by authors in identified papers. They covered the period from January 1985 to January 2000. The key words were hemophilia, severe, cost, cohorts, efficacy, effectiveness, inhibitors. Articles were classified according to subjects, methods and clinical relevance. Results. We considered that only eight studies have a medical and economic approach of hemophilia. For six of these studies, the main objectives are the comparison of the prophylactic treatment with the "on demand" treatment. For the rest the objective was to identify the cost related to the presence of inhibitors. For the first ones, all authors concluded at the superiority of the prophylactic treatment with a decrease of bleedings per patient per year, but its cost is higher than with the "on demand" treatment. It seems that the prophylactic treatment is related with less consumption resources, but the main cost determinant is the cost of medications. Conclusion. Medico-economic analysis in hemophilia is scarce. This is possibly explained partly by the complexity of the taking in charge and the prevalence of the illness, the absence of uniformed data but also, by the fact that in the 80s, the evaluation was complicated by the HIV contamination. At present, published works cannot meet neither the national nor international health economics evaluation recommendations.