Non-Invasive Tests (NITs) to Risk-Stratify Patients with Noncirrhotic Metabolic-Associated Steatohepatitis (MASH) in a Specialty Setting Eligible for Resmetirom: A Cost-Effectiveness Analysis from a U.S. Payer Perspective
Author(s)
John O'Donnell, MPP, PhD1, Yestle Kim, MSc, PharmD2, Melissa Gomez Montero, MSc3, Mehdi Javanbakht, MSc, PhD4, Suneil Hosmane, PhD1, Amir Ansaripour, PharmD, PhD3;
1Madrigal Pharmaceuticals, Inc., West Conshohocken, PA, USA, 2Madrigal Pharmaceuticals, HEOR, Jersey City, NJ, USA, 3Optimax Access, Rotterdam, Netherlands, 4Optimax Access, Hampshire, United Kingdom
1Madrigal Pharmaceuticals, Inc., West Conshohocken, PA, USA, 2Madrigal Pharmaceuticals, HEOR, Jersey City, NJ, USA, 3Optimax Access, Rotterdam, Netherlands, 4Optimax Access, Hampshire, United Kingdom
OBJECTIVES: NITs are crucial for identifying patients eligible for resmetirom. Accurate diagnosis of patients with F2-F3 non-cirrhotic MASH is essential for optimizing patient outcomes and resource allocation. This study assessed and compared the cost-effectiveness and accuracy of two diagnostic strategies: (1) two sequential NITs, and (2) the same NIT sequence followed by liver biopsy (LB) for discordant cases, reflecting clinical practice in specialty care.
METHODS: A cost-utility analysis combined a decision tree for diagnostic pathways with a Markov model to evaluate long-term cost-effectiveness of resmetirom treatment compared with placebo over a lifetime horizon, using a willingness-to-pay threshold of $100,000 per quality-adjusted life-year. Two-step NIT sequences, defined by pre-specified cut-off thresholds, were modeled: the first NIT identified significant MASH fibrosis (F≥2), and the second NIT ruled out cirrhosis (F4). Three categories of NITs (blood-based, imaging-based, and combined modalities) were assessed, yielding 57 combinations. The model simulated patients referred to specialty care for suspected non-alcoholic fatty liver disease, with or without fibrosis.
RESULTS: The most accurate NIT combinations in each category were: enhanced liver fibrosis (ELF) using two cutoffs of ≥8.8 for NIT1 and ≥11.3 for NIT2 (blood-based); transient elastography (TE) ≥8kPa followed by magnetic resonance elastography (MRE) ≥4.7kPa (imaging-based); and ELF ≥8.8, followed by MRE ≥4.7kPa (blood-imaging). Among these strategies, the imaging-based combination using TE ≥8kPa and MRE ≥4.7kPa demonstrated the highest net monetary benefit of $2,358, making it the most cost-effective approach, compared with the same respective sequence followed by LB for discordant cases.
CONCLUSIONS: This modeling study demonstrates that imaging-based NITs without LB offer the most cost-effective approach for diagnosing F2-F3 MASH in the US, while enhancing diagnostic efficiency and reducing reliance on invasive and costly LBs. These findings highlight the value of integrating NIT-only strategies to enhance access to emerging therapies, support payer value, and optimize healthcare resources.
METHODS: A cost-utility analysis combined a decision tree for diagnostic pathways with a Markov model to evaluate long-term cost-effectiveness of resmetirom treatment compared with placebo over a lifetime horizon, using a willingness-to-pay threshold of $100,000 per quality-adjusted life-year. Two-step NIT sequences, defined by pre-specified cut-off thresholds, were modeled: the first NIT identified significant MASH fibrosis (F≥2), and the second NIT ruled out cirrhosis (F4). Three categories of NITs (blood-based, imaging-based, and combined modalities) were assessed, yielding 57 combinations. The model simulated patients referred to specialty care for suspected non-alcoholic fatty liver disease, with or without fibrosis.
RESULTS: The most accurate NIT combinations in each category were: enhanced liver fibrosis (ELF) using two cutoffs of ≥8.8 for NIT1 and ≥11.3 for NIT2 (blood-based); transient elastography (TE) ≥8kPa followed by magnetic resonance elastography (MRE) ≥4.7kPa (imaging-based); and ELF ≥8.8, followed by MRE ≥4.7kPa (blood-imaging). Among these strategies, the imaging-based combination using TE ≥8kPa and MRE ≥4.7kPa demonstrated the highest net monetary benefit of $2,358, making it the most cost-effective approach, compared with the same respective sequence followed by LB for discordant cases.
CONCLUSIONS: This modeling study demonstrates that imaging-based NITs without LB offer the most cost-effective approach for diagnosing F2-F3 MASH in the US, while enhancing diagnostic efficiency and reducing reliance on invasive and costly LBs. These findings highlight the value of integrating NIT-only strategies to enhance access to emerging therapies, support payer value, and optimize healthcare resources.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE209
Topic
Economic Evaluation
Disease
SDC: Gastrointestinal Disorders