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Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapies In Cystic Fibrosis: A Retrospective Evaluation Of A Nationwide Specialty Pharmacy Database, Zumi Mehta

Electronic Theses and Dissertations

Background: Cystic fibrosis (CF) is a progressive, genetic disorder caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients with CF experience excessive mucus build up and infections leading to complications in the pancreas, lungs, and other organs. There has been a paradigm shift in the treatment of CF with the introduction of CFTR modulator therapies and given their high acquisition cost, assessing their economic burden along with the adherence rate of patients is imperative. Objective: (i) To assess the utilization of CFTR modulator therapies in a specialty pharmacy CF patient population and (ii) to calculate …