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Master Thesis

Les législations des médicaments orphelins : de la conception à la dispensation

Abstract : Orphan drugs - treatments for rare diseases, have become an emerging trend in pharmaceutical industry since the last several decades. Thank to investments from pharmaceutical companies and incentives from health authorities, research and development in rare diseases has achieved significant ground-breaking outcomes. Many rare diseases now can be treated. Patient’s condition and life quality have been significantly improved. As the legislation background for Orphan Drugs was first created 36 years ago and is still evolving these days, developing a new orphan drug encounters numerous difficulties in different phases, especially in early drug discovery, clinical trials, and patient access. On the other side, the knowledge about genetic sequence and biotechnologies have significantly increased, which facilitates the research and development of orphan drugs. Due to a rapid growth in orphan drugs market, it is necessary to have a comprehensive understanding on regulatory requirement worldwide from early research to patient distribution, in order to identify challenges and also opportunities for concerned healthcare players.
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-02338530
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Submitted on : Wednesday, October 30, 2019 - 9:16:56 AM
Last modification on : Wednesday, November 13, 2019 - 1:42:23 AM

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  • HAL Id : dumas-02338530, version 1

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Thi-Thuy Linh Nguyen. Les législations des médicaments orphelins : de la conception à la dispensation. Sciences pharmaceutiques. 2019. ⟨dumas-02338530⟩

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