In 2013 Vertex Pharmaceuticals introduced Kalydeco® (ivacaftor), the first orphan drug to successfully treat the underlying cause of cystic fibrosis in a well-defined patient subgroup, into the Irish market. The annual cost was €234,804 per patient. With this price tag the new drug had the potential to deplete over 40% of the funding available for all new drug treatments in Ireland. A cost-effective analysis conducted by the Irish National Centre for Pharmacoeconomics showed that....