Cystic Fibrosis: Breakthrough Drugs at Break-the-Bank Prices

Progress in cystic fibrosis (CF) over the past few decades has been nothing short of miraculous on several fronts: median survival in the United States has increased from 15 years in 1975 to 41 years in 2013, with predictions into the 50s for children born this century.1 Our understanding of the genetic and cellular underpinnings of CF have similarly grown exponentially, beginning in the 1980s with the identification of the cellular ion transport defect2 and the discovery of the gene, dubbed CFTR (cystic fibrosis transmembrane conductance regulator).3 Now in the past 3 years, with the advent of mutation-specific therapies, we have entered a new era that promises even greater improvement in the quality and the length of lives. The first of these small molecule therapies to come to fruition (and to market) was Vertex Pharmaceuticals' (Boston, Massachusetts) ivacaftor (sold as Kalydeco), which received FDA approval in 2012. In a clinical trial, ivacaftor brought about dramatic improvements in pulmonary funct...