Orphan Drugs: Understanding the Rare Disease Market and its Dynamics
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About this ebook
- A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients
- A source of updated information, news and trends for those who are already active in this fast-evolving field
- Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe
Elizabeth Hernberg-Ståhl
Elizabeth Hernberg-Stahl, M.Sc, is founder of Late Phase Solutions Europe AB, based in Sweden; it is an independent consultancy focusing on providing strategic and operational guidance on late phase drug development as well as guidance on processes and activities related to orphan drug development and market access. The author has more than thirty years experience from the international biotech and pharmaceutical industry (20 of which were in the orphan drugs area). During her 10 years at TKT/Shire HGT Elizabeth was responsible for developing and establishing the Global Outcome Survey Department, which manages global patient registries. Elizabeth was also member of the Shire HGT European Management Team and the Global Medical Affairs Leadership Team. Before joining Shire HGT/TKT Europe-5S, Elizabeth held a similar position for 8 years at Pharmacia/Pfizer where she established and managed a global patient registry KIMS on the rare condition adult GHD and the outcome of growth hormone treatment. Elizabeth has published numerous papers and is a frequent speaker at several congresses on topics related to orphan rare diseases.
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