Impact of Resmetirom on Liver Transplant Demand and Outcomes in US Patients With MASH
Author(s)
Daniel Kim, PhD1, Yestle Kim, MSc, PharmD2, Michael Charlton, MBBS., FRCP.2, John O'Donnell, MPP, PhD2, Nipun Atreja, MS, PhD2, Mert Sahinkoc, PhD1, Gizem Sultan Nemutlu, PhD3, Jag Chhatwal, PhD1.
1Harvard Medical School / Massachusetts General Hospital, Boston, MA, USA, 2Madrigal Pharmaceuticals, West Conshohocken, PA, USA, 3Brandeis University, Waltham, MA, USA.
1Harvard Medical School / Massachusetts General Hospital, Boston, MA, USA, 2Madrigal Pharmaceuticals, West Conshohocken, PA, USA, 3Brandeis University, Waltham, MA, USA.
OBJECTIVES: Metabolic dysfunction-associated steatohepatitis (MASH) affects ~5% of US adults and is one of the leading indications for liver transplantation (LT), with costs >$800,000 per LT. In 2024, resmetirom was conditionally FDA approved for the treatment of noncirrhotic MASH with moderate-to-advanced liver fibrosis. We evaluated the potential population-level impact of resmetirom on LT demand and allocation outcomes in the US.
METHODS: We developed an individual-level model simulating natural history of MASH, including progression to advanced fibrosis, decompensated cirrhosis, and hepatocellular carcinoma. Liver allocation dynamics were modeled using United Network for Organ Sharing (UNOS) policies. Two scenarios were simulated over a 30-year horizon (2024-2054): (1) No-Resmetirom Scenario, (2) Resmetirom Scenario, where 2.3% of MASH patients with moderate-to-advanced fibrosis received resmetirom. Treatment outcomes were derived from the MAESTRO-NASH trial. Model outputs were validated against the Organ Procurement and Transplantation Network (OPTN) database.
RESULTS: Over the 30-year simulation time horizon, ~1.3 million MASH patients were estimated to be on resmetirom. 1,689 would be placed on LT waitlist if they were not on resmetirom; however, if all 1,689 patients were on resmetirom, 92.6% would have been prevented from being put on the waitlist, allowing 1,366 livers to be re-allocated to patients who are on the waitlist. Due to this impact, patients on the waitlist, regardless of etiology, waited on average 2.4 fewer days. The number of deaths on LT waitlist would reduce by 1.7%.
CONCLUSIONS: Resmetirom has potential to reduce the burden of MASH and alleviate pressure on the LT system. Improvements in these events were observed with a modest 2.3% resmetirom utilization in the indicated population, demonstrating that wider use may lead to larger improvements. By reducing the number of patients requiring LT, resmetirom may improve overall access to donor organs and lower waitlist mortality, thus demonstrating broader population-level benefits of early MASH treatment.
METHODS: We developed an individual-level model simulating natural history of MASH, including progression to advanced fibrosis, decompensated cirrhosis, and hepatocellular carcinoma. Liver allocation dynamics were modeled using United Network for Organ Sharing (UNOS) policies. Two scenarios were simulated over a 30-year horizon (2024-2054): (1) No-Resmetirom Scenario, (2) Resmetirom Scenario, where 2.3% of MASH patients with moderate-to-advanced fibrosis received resmetirom. Treatment outcomes were derived from the MAESTRO-NASH trial. Model outputs were validated against the Organ Procurement and Transplantation Network (OPTN) database.
RESULTS: Over the 30-year simulation time horizon, ~1.3 million MASH patients were estimated to be on resmetirom. 1,689 would be placed on LT waitlist if they were not on resmetirom; however, if all 1,689 patients were on resmetirom, 92.6% would have been prevented from being put on the waitlist, allowing 1,366 livers to be re-allocated to patients who are on the waitlist. Due to this impact, patients on the waitlist, regardless of etiology, waited on average 2.4 fewer days. The number of deaths on LT waitlist would reduce by 1.7%.
CONCLUSIONS: Resmetirom has potential to reduce the burden of MASH and alleviate pressure on the LT system. Improvements in these events were observed with a modest 2.3% resmetirom utilization in the indicated population, demonstrating that wider use may lead to larger improvements. By reducing the number of patients requiring LT, resmetirom may improve overall access to donor organs and lower waitlist mortality, thus demonstrating broader population-level benefits of early MASH treatment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EPH135
Topic
Clinical Outcomes, Epidemiology & Public Health, Study Approaches
Topic Subcategory
Public Health
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Gastrointestinal Disorders