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Master Thesis

Les impacts médicaux, économiques et sociétaux de la thérapie génique : focus sur l’hémophilie B

Abstract : Hemophilia B is an inherited bleeding disorder related to the absence or decrease in plasma levels of one or more clotting factors. For more than 20 years, therapeutic management has been based on the intravenous administration of substitute coagulation factors to replace the defective factor IX in the coagulation cascade. Current clinical development of gene therapy in hemophilia B brings real hope to patients. As a breakthrough innovation, the marketing of this new therapy may lead to a substantial change in the patient's care pathway. The implementation of specific therapeutic education programmes along with the coordination of the various health care professionals within the framework of multidisciplinary care seem to be essential. The arrival of gene therapy also poses many technological challenges (such as transposition of production to industrial scale) and must fit into a complex ethical and regulatory framework. From a health economics point of view, the current evaluation systems seem outdated and not adapted to breakthrough innovations. New economic models are required to ensure on the one hand access to treatment for patients and on the other hand the sustainability of the French health insurance.
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Master Thesis
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Submitted on : Tuesday, February 25, 2020 - 11:41:56 AM
Last modification on : Tuesday, November 24, 2020 - 4:00:16 PM
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  • HAL Id : dumas-02490535, version 1


Robin Henocque. Les impacts médicaux, économiques et sociétaux de la thérapie génique : focus sur l’hémophilie B. Sciences pharmaceutiques. 2020. ⟨dumas-02490535⟩



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