Value-Based Pricing of Resmetirom for Metabolic Dysfunction-Associated Steatotic Liver Disease.

IMPORTANCE

Resmetirom can slow fibrosis progression and improve resolution of metabolic dysfunction-associated steatohepatitis (MASH). As the first Food and Drug Administration-approved pharmacologic treatment for MASH and fibrosis stages F2 and F3, resmetirom is very expensive, potentially creating a financial barrier to patient access.

OBJECTIVE

To estimate the cost-effectiveness of resmetirom vs standard of care (SoC) over a lifetime and to determine a price threshold at which resmetirom would be cost-effective.

DESIGN, SETTING, AND

PARTICIPANTS

This economic evaluation used an agent-based state-transition microsimulation model with a yearly cycle and 14 distinct histologically defined health states: metabolic dysfunction-associated steatotic liver disease (MALSD) or F0, MASH, fibrosis stages F1 to F3 with or without MASH, cirrhosis, decompensated cirrhosis, liver cancer, liver transplant, and liver-related and other-cause death. The simulated cohort included US adults who entered the model with MASH and fibrosis stage F2 or F3. The study was conducted from March to December 2024.

EXPOSURES: Resmetirom vs SoC.

MAIN OUTCOMES AND MEASURES: Costs (in 2023 US dollars), quality-adjusted life-years (QALYs), and the incremental cost-effectiveness ratio (ICER) (calculated as incremental costs divided by incremental QALYs).

RESULTS

A cohort of 200 000 patients (mean [SD] age, 57.1 [10.5] years; 89 000 [44.5%] male; 132 600 [66.2%] with MASH-F3) was simulated. Compared with SoC, resmetirom was associated with a gain of 0.26 QALYs/patient. Lifetime drug cost was $54 754, and treatment saved $18 499 in MASLD-related medical costs for a total incremental cost of $36 255 per patient. The ICER was $140 134/QALY. At willingness-to-pay (WTP) thresholds of $50 000/QALY, $100 000/QALY, and $150 000/QALY, the maximal annual prices of resmetirom were $10 914, $15 406, and $19 879, respectively. The lower the discontinuation rate, the greater the cost-effectiveness and medical cost savings, but the higher the total treatment costs. Without discontinuation, the ICER would be $318 740/QALY, supporting a price between $5645 and $10 619 per year at WTP thresholds of $50 000/QALY and $150 000/QALY, respectively.

CONCLUSIONS AND RELEVANCE: In this economic evaluation, resmetirom was not cost-effective at a $100 000/QALY WTP threshold. The conclusion was sensitive to model assumptions, especially the discontinuation rate. More data on the long-term effectiveness of resmetirom on MASLD progression and non-MASLD complications would be necessary to accurately determine the economic value of resmetirom.