This article describes the regulatory frameworks and incentives associated with orphan drug designation (ODD) in the EU and the US, as well as capturing key strategic considerations needed to achieve successful orphan drug product development and registration in both regions.
Marie Deneux, Senior Director Regulatory Science, Drugs and Biologics; Pauline Messina, Regulatory Scientist; Voisin Consulting Life Sciences.
July/August 2016 - Regulatory Rapporteur-TOPRA
As Senior Director, Regulatory Science, Drugs and Biologics, Marie assists international clients in the design and implementation of global product development plans and associated regulatory strategy, in particular in the area of rare diseases. She is involved in the development of biotech products, new chemical entities, cell and gene therapy medicinal products and repurposed drugs.View Profile