Cost-effectiveness of obeticholic acid for the treatment of non-alcoholic steatohepatitis: An early economic evaluation

Abstract

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.

The effect and safety of obeticholic acid for patients with nonalcoholic steatohepatitis: A systematic review and meta-analysis of randomized controlled trials

“…The cost-effectiveness of treating nonalcoholic steatohepatitis with OCA also demands further investigation. A study by Wong et al, [ 30 ] which employed a state-transition model, determined that OCA was not cost-effective compared with placebo despite its clinical benefits due to the drug’s high cost. Importantly, the model used in their study only considered OCA at the 25 mg dose in comparison with a placebo.…”

MASLD treatment—a shift in the paradigm is imminent

“…A cost-effectiveness study suggested that treatment with OCA would only be cost-effective if the price of the drug would decrease by 78% ( 188 ).…”

Fibrates and Fibrate-induced Liver Injury in Primary Biliary Cholangitis

“…Even with an annual price reduction from over $69,000 in 2017 to an estimated $36,000 currently, the price would still need to decrease by about half to be cost-effective at a willingness-to-pay threshold of $100,000 per quality-adjusted life year in PBC patients. 26,27 Although OCA is generally safe for patients with mild to moderate disease, there is evidence that those in the advanced stages of cirrhosis may experience clinical worsening and even death associated with the drug. 28 The FDA issued its first warning on the use of OCA in these patients in 2017, followed by a recommended dose reduction for cirrhotics in 2018.…”