Dataset Information

Early Cost-Effectiveness and Price Threshold Analyses of Resmetirom: An Investigational Treatment for Management of Nonalcoholic Steatohepatitis.

ABSTRACT:

Background

Nonalcoholic steatohepatitis (NASH) is characterized by inflammation and hepatocellular damage caused by accumulation of fat in the liver. Resmetirom (MGL-3196) is an orally administered, small-molecule, liver-targeted, selective thyroid hormone receptor-β agonist. This early analysis explored the potential cost effectiveness of resmetirom for the treatment of NASH from a US commercial payer perspective.

Methods

An early economic model was developed to reflect the clinical pathways typically followed by patients with NASH and liver fibrosis. Use of resmetirom, compared with placebo, was assessed. The Markov model structure was informed by a previous modeling study and a randomized, double-blind, placebo-controlled, phase II trial of resmetirom. Costs and outcomes were assessed over a lifetime time horizon with results presented in terms of cost per quality-adjusted life-year (QALY) gained.

Results

Resmetirom treatment resulted in increased costs of US$66,764 per patient, while increasing QALYs by 1.24. The incremental cost-effectiveness ratio was US$53,929 per QALY gained, indicating resmetirom treatment would potentially be cost effective at a willingness-to-pay (WTP) threshold of US$100,000. Results indicated that resmetirom would reduce the lifetime number of cases of decompensated cirrhosis (- 87), hepatocellular carcinoma (- 59), and liver transplants (- 30) per 1,000 patients compared with placebo. Resmetirom treatment remained cost effective at a US$100,000 WTP threshold up to a daily price point of US$72.00.

Conclusion

Resmetirom is a potentially cost-effective treatment option for patients with NASH and liver fibrosis based on an analysis performed from a US commercial payer perspective. Future economic analyses of the technology should, however, focus on overcoming the limitations of existing modeling methodology.

SUBMITTER: Javanbakht M

PROVIDER: S-EPMC9929016 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature

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Publications

Early Cost-Effectiveness and Price Threshold Analyses of Resmetirom: An Investigational Treatment for Management of Nonalcoholic Steatohepatitis.

Javanbakht Mehdi M Fishman Jesse J Moloney Eoin E Rydqvist Peter P Ansaripour Amir A

PharmacoEconomics - open 20220914 1

<h4>Background</h4>Nonalcoholic steatohepatitis (NASH) is characterized by inflammation and hepatocellular damage caused by accumulation of fat in the liver. Resmetirom (MGL-3196) is an orally administered, small-molecule, liver-targeted, selective thyroid hormone receptor-β agonist. This early analysis explored the potential cost effectiveness of resmetirom for the treatment of NASH from a US commercial payer perspective.<h4>Methods</h4>An early economic model was developed to reflect the clini ...[more]

PMID: 36104546

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Project description:ImportanceObesity is the most common risk factor for nonalcoholic steatohepatitis (NASH), the progressive form of nonalcoholic fatty liver disease that can lead to cirrhosis and hepatocellular carcinoma. Weight loss can be an effective treatment for obesity and may slow the progression of advanced liver disease.ObjectiveTo assess the cost-effectiveness of bariatric surgery in patients with NASH and compensated cirrhosis.Design, setting, and participantsThis economic evaluation study used a Markov-based state-transition model to simulate the benefits and risks of laparoscopic sleeve gastrectomy (SG), laparoscopic Roux-en-Y gastric bypass (GB), and intensive lifestyle intervention (ILI) compared with usual care in patients with NASH and compensated cirrhosis and varying baseline weight (overweight, mild obesity, moderate obesity, and severe obesity). Patients faced varied risks of perioperative mortality and complications depending on the type of surgery they underwent. Data were collected on March 22, 2017.Main outcomes and measuresLife-years, quality-adjusted life-years (QALYs), costs (in 2017 $US), and incremental cost-effectiveness ratios (ICERs) were calculated.ResultsDemographic characteristics of the patient population were based on a previously published prospective study (n = 161). Patients in the model were 41.0% female, and the base case age was 54 years. Compared with usual care, SG was associated with an increase in QALYs of 0.263 to 1.180 (bounds of ranges represent overweight to severe obesity); GB, 0.263 to 1.207; and ILI, 0.004 to 0.216. Sleeve gastrectomy was also associated with an increase in life-years of 0.693 to 1.930; GB, 0.694 to 1.947; and ILI, 0.012 to 0.114. With usual care, expected life-years in overweight, mild obesity, moderate obesity, and severe obesity were 12.939, 11.949, 10.976, and 10.095, respectively. With usual care, QALY in overweight was 6.418; mild obesity, 5.790; moderate obesity, 5.186; and severe obesity, 4.577. Sleeve gastrectomy was the most cost-effective option for patients across all weight classes assessed: ICER for SG in patients with overweight was $66 119 per QALY; mild obesity, $18 716 per QALY; moderate obesity, $10 274 per QALY; and severe obesity, $6563 per QALY. A threshold analysis on the procedure cost of GB found that for GB to be cost-effective, the cost of the surgery must be decreased from its baseline value of $28 734 by $4889 for mild obesity, by $3189 for moderate obesity, and by $2289 for severe obesity. In overweight patients, GB involved fewer QALYs than SG, and thus decreasing the cost of surgery would not result in cost-effectiveness.Conclusions and relevanceBariatric surgery could be highly cost-effective in patients with NASH compensated cirrhosis and obesity or overweight. The findings from this analysis suggest that it can inform clinical trials evaluating the effect of bariatric procedures in patients with NASH cirrhosis, including those with a lower body mass index.

Project description:BackgroundNonalcoholic steatohepatitis (NASH) is a chronic liver disease associated with hepatic morbidity and mortality and extra-hepatic comorbidities. Published NASH cost-effectiveness models (CEMs) are heterogeneous and consistently omit comorbid conditions that frequently co-exist alongside NASH. We aimed to develop a de novo CEM framework that incorporates extra-hepatic disease states and outcomes alongside hepatic components to enable future estimation of the cost-effectiveness of NASH interventions.MethodsPatient-level simulation and cohort-level Markov models were implemented in the same framework. Model inputs included fibrosis progression; late-stage liver disease outcomes; comorbidity outcomes for cardiovascular disease, type 2 diabetes, and obesity; mortality; health-related quality of life; and direct medical costs. The prototype analysis assessed the cost-effectiveness of obeticholic acid versus standard of care from a US payer perspective over a lifetime horizon with costs and effects discounted at 3% per annum. However, the CEM was designed for easy adaptation to other countries, time horizons, and other considerations. Efficacy and adverse event parameters were obtained from the 18-month interim analysis of the REGENERATE trial. Outputs include total and incremental costs, total life years, and quality-adjusted life years.ResultsIn this model, total costs, total life years, and quality-adjusted life years were all higher with obeticholic acid compared with standard of care. Cross-validation of this model with the 2016 and 2020 Institute for Clinical and Economic Review models revealed marked differences, mainly driven by mortality inputs, transition probability estimates, and incorporation of the effect of treatment and comorbidities.ConclusionThis is the first CEM in NASH to incorporate the clinical consequences of several comorbidities. The flexible yet standardized framework permits estimation of the cost-effectiveness of NASH interventions in a variety of settings. The model currently includes several assumptions and will be further developed as more relevant data become available.

Project description:ObjectivesNonalcoholic fatty liver disease (NAFLD) is the most common liver disease in Western countries. No studies have examined the cost-effectiveness of screening its advanced form, nonalcoholic steatohepatitis (NASH).MethodsWe performed a cost-utility analysis of annual noninvasive screening strategies using third-party payer perspective in a general population in comparison to screening a high-risk obese or diabetic population. Screening algorithms involved well-studied techniques, including NAFLD fibrosis score, transient elastography (TE), and acoustic radiation force impulse (ARFI) imaging for detecting advanced fibrosis (≥ F3); and plasma cytokeratin (CK)-18 for NASH detection. Liver biopsy and magnetic resonance elastography (MRE) were compared as confirmation methods. Canadian dollar (CAD or C$) costs were adjusted for inflation and discounted at 5%. Incremental cost-effectiveness ratio (ICER) of ≤C$ 50,000 was considered cost-effective.ResultsCompared with no screening, screening with NAFLD fibrosis score/TE/CK-18 algorithm with MRE as confirmation for advanced fibrosis had an ICER of C$ 26,143 per quality-adjusted life year (QALY) gained. Screening in high-risk obese or diabetic populations was more cost-effective, with an ICER of C$ 9,051 and C$ 7,991 per quality-adjusted life-year (QALY) gained, respectively. Liver biopsy confirmation was not found to be cost-effective.ConclusionsOur model suggests that annual NASH screening in high-risk obese or diabetic populations can be cost-effective.Key points• This cost-utility analysis suggests that screening for nonalcoholic steatohepatitis may be cost-effective. • In particular, screening of high-risk obese or diabetic populations is more cost-effective. • Magnetic resonance elastography was more cost-effective to confirm disease compared to biopsy. • More studies are needed to determine quality of life in nonalcoholic steatohepatitis. • More management strategies for nonalcoholic steatohepatitis are also needed.

Project description:BackgroundInjectable lenacapavir administered every 6 months is a promising product for HIV pre-exposure prophylaxis (PrEP). We aimed to estimate the health and budget impacts and threshold price at which lenacapavir could be cost-effective in eastern and southern Africa.MethodsWe adapted an agent-based network model, EMOD-HIV, to simulate lenacapavir scale-up in Zimbabwe, South Africa, and western Kenya from 2026 to 2035. Uptake assumptions were informed by a literature review of PrEP product preferences. In the main analysis, we varied lenacapavir coverage by subgroup: female sex workers (40% coverage); male clients of female sex workers (40%); adolescent girls and young women aged 15-24 years with more than one sexual partner (32%); women aged 25 years and older with more than one sexual partner (36%); and males with more than one sexual partner (32%). We also assessed a higher coverage scenario (64-76% across subgroups) and scenarios of expanding lenacapavir use, varying from concentrated among those at highest HIV risk to broader coverage including those at medium HIV risk. We estimated the maximum per-dose lenacapavir price that achieved cost-effectiveness (<US$500 per disability-adjusted life-year averted), infections averted, and 5-year budget impact, compared with daily oral PrEP only.FindingsIn the main analysis, lenacapavir was projected to achieve from 1·6% (95% uncertainty interval [UI] 1·5-1·8) to 4·0% (3·4-5·1) population coverage across settings and to avert from 12·3% (5·4-19·5) to 18·0% (11·0-22·9) of infections over 10 years. The maximum price per dose was highest in South Africa ($106·28 [95% UI 95·72-115·87]), followed by Zimbabwe ($21·15 [17·70-24·89]), and lowest in western Kenya ($16·58 [15·44-17·70]). The 5-year budget impact was US$507·25 million (95% UI 436·14-585·42) in South Africa, $16·80 million (13·95-22·64) in Zimbabwe, and $4·09 million (3·86-4·30) in western Kenya. In the higher coverage scenario, lenacapavir distribution was projected to reach from 3·2% (95% UI 2·9-3·6) to 8·1% (6·8-10·5) population coverage and to avert from 21·2% (95% UI 14·7-18·5) to 33·3% (28·5-36·9) of HIV infections across settings over 10 years. Price thresholds were lower than in the main analysis: $88·34 (95% UI 83·02-94·19) in South Africa, $17·71 (15·61-20·05) in Zimbabwe, and $14·78 (14·33-15·30) in western Kenya. The 5-year budget impact was higher than the main analysis: $835·29 million (95% UI 736·98-962·98) in South Africa, $29·50 million (24·62-39·52) in Zimbabwe, and $7·45 million (7·11-7·85) in western Kenya. Expanding lenacapavir coverage resulted in higher HIV infections averted but lower price thresholds than scenarios of concentrated use among those with highest HIV risk.InterpretationOur findings suggest that lenacapavir could avert substantial HIV incidence and that price thresholds and budget impacts vary by setting and coverage. These results could inform policy deliberations regarding lenacapavir pricing and resource planning.FundingThe Bill & Melinda Gates Foundation.

Dataset Information

Early Cost-Effectiveness and Price Threshold Analyses of Resmetirom: An Investigational Treatment for Management of Nonalcoholic Steatohepatitis.

Background

Methods

Results

Conclusion

Publications

Early Cost-Effectiveness and Price Threshold Analyses of Resmetirom: An Investigational Treatment for Management of Nonalcoholic Steatohepatitis.

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