Author(s) :

Ian Andrefski, Mehul Mehezabin.

Volume/Issue :

Abstract :

Cystic fibrosis (CF) is a genetic disease caused by mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which causes an inability in affected cells to properly move chloride (Cl-) and bicarbonate (HCO3 -) across epithelial tissue, leading to symptoms such as abdominal distress and the presence of thick mucus in the lungs and airways that collect bacteria and contribute significantly to CF patient mortality. Healthcare providers have developed treatment plans designed to detect CF early and treat its symptoms. CFTR modulators such as ivacaftor, elizacaftor, and tezacaftor offer patients the ability to correct the mutations in the CFTR protein and remove symptoms entirely. However, these medications are expensive, contributing to healthcare inequality and outcomes for CF patients. To promote healthcare accessibility, Vertex Pharmaceuticals, either voluntarily or through government-mandated compulsory licensing in affected countries, could allow other companies to produce generic options at a fraction of the existing price, thereby removing economic barriers and promoting healthcare accessibility for CF patients worldwide. This review analyzes the effectiveness of CFTR modulators and how that effectiveness is counterbalanced by economic and ethical disparities, which make them inaccessible to much of the general population worldwide.