Disease Progression Modeling for Economic Evaluation in Nonalcoholic Fatty Liver Disease—A Systematic Review

Gruneau, Lina; Ekstedt, Mattias; Kechagias, Stergios; Henriksson, Martin

doi:10.1016/j.cgh.2021.10.040

Cited by 8 publications

(7 citation statements)

References 58 publications

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“…Numerous studies have investigated the relationship between NAFLD and extrahepatic disease outcomes in patients with NAFLD. 15,[27][28][29] However, no study has analysed the costeffectiveness of each extrahepatic disease outcome in patients with NAFLD. Remarkably, the estimated total medical cost for liver and CVD combined model was approximately 2.35 times higher than that for liver; CVD and extrahepatic malignancy combined model was approximately 6.7 times higher than that for liver-related outcomes in the no screening setting (Table 1 and Figure S3a).…”

Section: Discussionmentioning

confidence: 99%

“…Our study aimed to estimate the socio‐economic burden and benefits of ILI in the at‐risk population, with regards to hepatic disease, CVD and extrahepatic malignancies. Numerous studies have investigated the relationship between NAFLD and extrahepatic disease outcomes in patients with NAFLD 15,27–29 . However, no study has analysed the cost‐effectiveness of each extrahepatic disease outcome in patients with NAFLD.…”

Section: Discussionmentioning

confidence: 99%

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Cost‐effectiveness study of FIB‐4 followed by transient elastography screening strategy for advanced hepatic fibrosis in a NAFLD at‐risk population

Park,

Yoon,

Kim

et al. 2024

Liver International

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Background & AimsThe cost‐effectiveness to screen hepatic fibrosis in at‐risk population as recommended by several professional societies has been limited. This study aimed to investigate the cost‐effectiveness of this screening strategy in the expanded at‐risk population recently proposed by several societies.MethodsA combined model of the decision tree and Markov models was developed to compare expected costs, quality‐adjusted life‐years (QALYs) and incremental cost‐effectiveness ratio (ICER) between screening and no screening groups. The model included liver disease‐related health states and cardiovascular disease (CVD) states as a base‐case analysis. Screening strategy consisted of fibrosis‐4 index (FIB‐4) followed by vibration‐controlled transient elastography (VCTE) and intensive lifestyle intervention (ILI) as a treatment for diagnosed patients.ResultsCost‐effectiveness analysis showed that screening the at‐risk population entailed $298 incremental costs and an additional 0.0199 QALY per patient compared to no screening (ICER $14 949/QALY). Screening was cost‐effective based on the implicit ICER threshold of $25 000/QALY in Korea. When the effects of ILI on CVD and extrahepatic malignancy were incorporated into the cost‐effectiveness model, the ICER decreased by 0.85 times from the base‐case analysis (ICER $12 749/QALY). In contrast, when only the effects of liver disease were considered in the model, excluding cardiovascular disease effects, ICER increased from the baseline case analysis to $16 305. Even when replacing with medical costs in Japan and U.S., it remained cost‐effective with the estimate below the countries' ICER threshold.ConclusionsOur study provides compelling evidence supporting the cost‐effectiveness of FIB‐4‐based screening the at‐risk population for advanced hepatic fibrosis.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Cost‐effectiveness study of FIB‐4 followed by transient elastography screening strategy for advanced hepatic fibrosis in a NAFLD at‐risk population

Park,

Yoon,

Kim

et al. 2024

Liver International

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“…Quantitative models serve the purpose of informing such prospective investigations based on scenario testing that evaluates and designs sampling times and frequency and sample size considerations ( Mould, 2007 ; Cook and Bies, 2016 ). A disease progression model can also describe patient phenotypes and inform enrollment criteria as well as the timing of proposed interventions and treatments relative to the current standard of care ( Cook and Bies, 2016 ; Gruneau et al, 2021 ). Hence, they have tremendous value for both the sponsors of such proposed interventions and regulators who must evaluate their safety and efficacy.…”

Section: Discussionmentioning

confidence: 99%

Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data

et al. 2022

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The 21st Century Cures Act requires FDA to expand its use of real-world evidence (RWE) to support approval of previously approved drugs for new disease indications and post-marketing study requirements. To address this need in neonates, the FDA and the Critical Path Institute (C-Path) established the International Neonatal Consortium (INC) to advance regulatory science and expedite neonatal drug development. FDA recently provided funding for INC to generate RWE to support regulatory decision making in neonatal drug development. One study is focused on developing a validated definition of bronchopulmonary dysplasia (BPD) in neonates. BPD is difficult to diagnose with diverse disease trajectories and few viable treatment options. Despite intense research efforts, limited understanding of the underlying disease pathobiology and disease projection continues in the context of a computable phenotype. It will be important to determine if: 1) a large, multisource aggregation of real-world data (RWD) will allow identification of validated risk factors and surrogate endpoints for BPD, and 2) the inclusion of these simulations will identify risk factors and surrogate endpoints for studies to prevent or treat BPD and its related long-term complications. The overall goal is to develop qualified, fit-for-purpose disease progression models which facilitate credible trial simulations while quantitatively capturing mechanistic relationships relevant for disease progression and the development of future treatments. The extent to which neonatal RWD can inform these models is unknown and its appropriateness cannot be guaranteed. A component of this approach is the critical evaluation of the various RWD sources for context-of use (COU)-driven models. The present manuscript defines a landscape of the data including targeted literature searches and solicitation of neonatal RWD sources from international stakeholders; analysis plans to develop a family of models of BPD in neonates, leveraging previous clinical trial experience and real-world patient data is also described.

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“…The authors conducted a systematic review of published NAFLD disease progression models, including 28 articles; 13 projected NAFLD disease and economic burden without investigating a particular intervention, 8 studies evaluated NAFLD treatment interventions, and 7 studies evaluated the cost-effectiveness of NAFLD screening or diagnosis. Gruneau et al 8 find substantial heterogeneity in the simulated natural history of NAFLD across the included studies. Although all models defined natural history using NAFLD fibrosis stages, annual rate of progression from F0 to F1 varied between studies by 2-fold (0.059-0.095), from F1 to F2 varied 6-fold (0.023-0.140), from F2 to F3 varied 4-fold (0.018-0.070), and F3 to F4 (0.040-0.118) varied 3-fold.…”

Section: Heterogeneity In Simulating Nonalcoholic Fatty Liver Disease...mentioning

confidence: 99%

Nonalcoholic Fatty Liver Disease Natural History: Role of Mathematical Modeling

Chhatwal¹,

Tapper²

2023

Clinical Gastroenterology and Hepatology

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Disease Progression Modeling for Economic Evaluation in Nonalcoholic Fatty Liver Disease—A Systematic Review

Cited by 8 publications

References 58 publications

Cost‐effectiveness study of FIB‐4 followed by transient elastography screening strategy for advanced hepatic fibrosis in a NAFLD at‐risk population

Cost‐effectiveness study of FIB‐4 followed by transient elastography screening strategy for advanced hepatic fibrosis in a NAFLD at‐risk population

Landscape analysis for a neonatal disease progression model of bronchopulmonary dysplasia: Leveraging clinical trial experience and real-world data

Nonalcoholic Fatty Liver Disease Natural History: Role of Mathematical Modeling

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