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.