Background: Currently, there are no pharmacological options available for the treatment of non-alcoholic steatohepatitis (NASH). In the 18-month interim analysis of an ongoing randomized, placebo-controlled phase 3 trial (REGENERATE), early results demonstrated that obeticholic acid (OCA) 25 mg significantly improved fibrosis with no worsening of NASH among patients with NASH and fibrosis compared to placebo (PBO). This study aimed to assess the potential cost-effectiveness of OCA compared to PBO in NASH patients. Methods: A state-transition model was developed to perform a cost-utility analysis comparing two treatment strategies, PBO and OCA 25 mg, from a Canadian public payer perspective. The model time horizon was lifetime with annual cycle lengths. Cost and utility parameters were discounted at 1.5% annually. The efficacy data were obtained from the REGENERATE trial, and costs and utilities were derived from other published literature. Probabilistic and deterministic sensitivity analyses were performed to test the robustness of the model. Results: Treatment with OCA led to reductions of 3.58% in decompensated cirrhosis cases, 3.95% in hepatocellular carcinoma, 7.88% in liver transplant, and 6.01% in liver-related death. However, at an annual price of CDN$36,000, OCA failed to be cost-effective compared to PBO at an incremental cost-effectiveness ratio of $815,514 per quality-adjusted life year (QALY). An 88% reduction in drug price to an annual cost of $4,300 would make OCA cost-effective at a willingness-to-pay threshold of $50,000/QALY. Conclusions: OCA failed to be cost-effective compared to PBO, despite demonstrating clinical benefits due to a high drug cost. A significant price reduction would be needed to make the drug cost-effective.
Farnesoid X nuclear receptor ligand obeticholic acid for non-cirrhotic, non-alcoholic steatohepatitis (FLINT): a multicentre, randomised, placebo-controlled trial