Cost-effectiveness of the anti-fibrotics for the treatment of idiopathic pulmonary fibrosis in the United States

Abstract

Background: The anti-fibrotic medications nintedanib and pirfenidone were approved in the United States for use in patients with idiopathic pulmonary fibrosis several years ago. While there is a growing body of evidence surrounding their clinical effectiveness, these medications are quite expensive and no prior cost-effectiveness analysis has been performed in the United States.

Methods: A previously published Markov model performed in the United Kingdom was replicated using United States data to project the lifetime costs and health benefits of treating idiopathic pulmonary fibrosis with: (1) symptom management; (2) pirfenidone; or (3) nintedanib. For the cost-effectiveness analysis, strategies were ranked by increasing costs and then checked for dominating treatment strategies. Then an incremental cost-effectiveness ratio was calculated for the dominant therapy.

Results: The anti-fibrotic medications were found to cost more than $110,000 per year compared to $12,291 annually for symptom management. While pirfenidone was slightly more expensive than nintedanib and provided the same amount of benefit, neither medication was found to be cost-effective in this U.S.-based analysis, with an average cost of $1.6 million to gain one additional quality-adjusted life year over symptom management.

Conclusions: Though the anti-fibrotics remain the only effective treatment option for patients with idiopathic pulmonary fibrosis and the data surrounding their clinical effectiveness continues to grow, they are not considered cost-effective treatment strategies in the United States due to their high price.

Keywords: Cost-effectiveness analysis; Idiopathic pulmonary fibrosis; Nintedanib; Pirfenidone.

Fig. 1

Markov model of lung function for idiopathic pulmonary fibrosis. The Markov model breaks up a disease into health states (in circles, i.e. 100% FVC, death) and health events. When a health event happens (e.g. worsening lung function), it is denoted as a transition (signified by arrows) to another health state. FVC forced vital capacity

Fig. 2

Schematic of threshold analysis demonstrating the price at which pirfenidone and nintedanib are cost-effective treatment strategies compared to symptom management

Fig. 3

Probabilistic sensitivity analysis of symptom management (red) compared to pirfenidone (green) and nintedanib (blue). X-axis is our WTP. Y-axis is the proportion of times a specific treatment strategy was cost effective among 10,000 simulations