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When Small Becomes Big: The New Challenges of Orphan Drugs in Europe

Over the past few years there has been a shift in the European pharmaceutical market landscape, with a rise in specialty growth and more of a focus on specialty medicines and diseases with narrow patient population. IMS Health forecasts that between 2013 and 2018, 94 percent of list price value growth will be in specialty in Europe. Orphan drugs are on the leading edge of this specialty trend. They are also a commercially attractive area due to vigorous patient advocacy, medical breakthroughs, regulatory incentives and venture capital investment. 

This white paper examines the challenges and opportunities this success brings, with a focus on Europe.

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