OBJECTIVES: Non-alcoholic fatty liver disease (NAFLD) is a leading cause of liver disease and hepatocellular carcinoma worldwide. No NAFLD-specific therapy is approved, although there are several agents advancing to Phase 2 and 3 clinical trials. The objective was to estimate the cost-effectiveness of a hypothetical cell-based therapy compared to standard of care, i.e., lifestyle intervention added to pioglitazone, used in NAFLD.

METHODS: A Markov cohort model was developed to predict costs and health gains associated with alternative therapies over a lifetime horizon. The analysis was conducted from a US third-party payer perspective. A series of assumptions on the efficacy of cell-based therapy were made based on a NASH pre-clinical model. The effectiveness of standard of care was derived from a randomized controlled trials and a prospective cohort study. Utilities and costs were retrieved from data reported by the Institute for Clinical and Economic Review and previously published cost-utility analyses. Costs were adjusted to 2021 US dollars.

RESULTS: The cell-therapy was predicted to provide added life years (LY) and quality-adjusted life-years (QALY) gains in adults with NAFLD. The break-even prices of cell-based therapy corresponding to the ICER equal to a series of hypothetical willingness-to-pay thresholds of $50,000, $100,000, and $150,000 were calculated. The cost of standard of care, effectiveness of compared therapies and health-state utilities in liver disease were found the key factors driving cost-effectiveness of a new agent.

CONCLUSIONS: Clinical data suggest that cell-based therapy can be highly effective in producing long-term NASH resolution and slowing down of fibrosis progression. This study provides estimates for the economically justifiable prices of this therapy compared to the current standard of care.