Pharmacoeconomics and Pharmaceutical Economics Commons^™

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 …

Burden Of Illness And Pharmacoeconomic Evaluation Of Lumacaftor/Ivacaftor In Cystic Fibrosis, Pratyusha Vadagam

Electronic Theses and Dissertations

Objectives: (1) To estimate the burden of illness in cystic fibrosis (CF) using 2010-2014 Medical Expenditure Panel Survey (MEPS) data, and (2) to conduct a pharmacoeconomic evaluation of lumacaftor/ivacaftor in the treatment of CF.

Methods: The study was conducted in two parts. Part 1 involved a retrospective analysis using individuals in MEPS database with an International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) diagnosis code of 277.00 for a principal diagnosis of CF. Dependent variables were cumulative and out-of-pocket (OOP) expenditures and independent variables included patient demographics and clinical characteristics. Unweighted and weighted estimates of expenditure in CF group, …