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Master Thesis

Mise sur le marché d’un médicament destiné au traitement d’une maladie ultra-rare : retour d’expérience sur la commercialisation de Strensiq (asfotase alfa)

Abstract : Strensiq, the first and the only one drug product indicated in the treatment of hypophosphatasia has been marketed in France in 2018. It is the final outcome of a long journey marked by many administrative, regulatory and health technology assessment steps. This treatment and the way it has been developed and assessed represent a particular interest due to the ultra-are nature of the disease (hypophosphatasia) and his particularly high cost of the treatment. Upon commercialization in France, all severe patients have had the opportunity to benefit from the treatment in consistence with inclusion criteria from clinical trials, proven efficacity and the labelling of the marketing authorization. Assessors have indeed recognized the important efficacy of the treatment. Authorities have accepted the high price requested for the treatment which highlights their interest for patients suffering from rare diseases. Also, pharmacists have demonstrated their high commitment to affected patients by delivering the product despite a regulatory and legal framework not adapted to such exceptional products.
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-03481340
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Submitted on : Wednesday, December 15, 2021 - 11:52:56 AM
Last modification on : Saturday, December 18, 2021 - 3:53:36 AM

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  • HAL Id : dumas-03481340, version 1

Citation

Harold Le Monnier. Mise sur le marché d’un médicament destiné au traitement d’une maladie ultra-rare : retour d’expérience sur la commercialisation de Strensiq (asfotase alfa). Sciences pharmaceutiques. 2020. ⟨dumas-03481340⟩

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