Rare diseases and orphan drugs: patient values, market trends and misconceptions. KU Leuven
Rare disease drug research and development has intensified over the past two decades and has been translating into an ever growing number of ‘orphan drugs’ which, in turn, has led to the emergence of an amplifying public policy debate over: the place of the rare diseases patient voice into drug development and approval; the rise of a niche market characterised by perceived stronger financial performance; concerns over the affordability of ...