Economic Evaluation of Cystic Fibrosis Transmembrane Receptor (CFTR) Modulators

OBJECTIVES: The cost of cystic fibrosis (CF) transmembrane receptor (CFTR) modulating therapies can be a financial struggle for patients and payors, especially when they are priced over $400’000 USD yearly. With the advent of enhanced CFTR modulator therapies gaining approval, it becomes imperative to assess their cost-effectiveness in comparison to their predecessors. The utilization of Elexacaftor/Tezacaftor/ivacaftor (E/T/I) has rapidly increased and total healthcare utilization in the CF population has significantly decreased using E/T/I. However, there can be barriers to for patients given the cost of E/T/I. An economic evaluation comparing E/T/I to other modulators has not been conducted.

METHODS: A group cohort model was developed using real-world data sourced from the CF registry for a single adult center, Indiana University Health. The goal of this model was to determine the incremental cost over 10 years with a population of 200 patients in the model. Transition states probabilities were found using the registry dataset. WAC inputs for inhaled antibiotics, CFTR modulators, and pancreatic enzymes were gathered from the red book. Inpatient costs were derived from the Indiana diagnosis related group (DRG) payment. Utilities associated with the nodes were gathered using a similar study to ours by Sharma et al.

RESULTS: The base case analysis revealed an incremental cost of $9’426’608.28/QALY. A probabilistic sensitivity analysis of 10’000 iterations revealed that the average incremental cost converged to $9’337’603.12/QALY. E/T/I was cost-effective when it was priced approximately $20 to $30 less than non-E/T/I medications after simulation.

CONCLUSIONS: A cost effectiveness study comparing E/T/I to other CFTR modulators has not been reported in the literature. Although E/T/I reduces exacerbations and lung transplants compared to non-E/T/I modulators, this medication is not cost-effective based on a U.S. cost acceptability of $100’000/QALY.