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Master Thesis

L'autorisation temporaire d’utilisation de cohorte : les enjeux du dispositif français d’accès précoce à l’innovation thérapeutique illustré à travers l’évaluation de la demande d’ATU de cohorte de l’emicizumab, immunothérapie à destination des patients atteints d’hémophilie A

Abstract : France was a pioneer in early access to treatment, with the creation of “ATU” system use since more than 20 years ago. The objective of this thesis is to expose the new challenges, represented by the early availability of innovative treatments, for patients and doctors but also for the pharmaceutical industries and the French government. The ATU represents a strong point of our health system but with the arrival of new innovative and very expensive therapies that want to reach the market faster than their competitors, is this device still as robust as ever? In addition, we will show that the French administrative system of market access, perceived as more complex and severe than in the United States, China or other European countries, is the subject of several reform projects aimed at restoring France's attractiveness for the pharmaceutical industries. The multiple issues of early access will be illustrate through the example of French health and safety agency's evaluation of the request for ATUc of the emicizumab, an innovative immunotherapy used in patients with hemophilia A to prevent bleeding or reduce the frequency of bleeding episodes.
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-02436656
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Léa Gaillard. L'autorisation temporaire d’utilisation de cohorte : les enjeux du dispositif français d’accès précoce à l’innovation thérapeutique illustré à travers l’évaluation de la demande d’ATU de cohorte de l’emicizumab, immunothérapie à destination des patients atteints d’hémophilie A. Sciences pharmaceutiques. 2019. ⟨dumas-02436656⟩

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