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Next-generation sequencing (NGS) technologies constitute a range of methods that can perform genomic analysis of blood cancers, and therefore aims to establish precision therapies and enhance health outcomes in affected individuals. There is considerable enthusiasm surrounding the integration of NGS into healthcare practices and advocacy for its reimbursement as a genomic test. However, achieving routine reimbursement necessitates the development of sufficient and appropriate evidence showcasing its value for healthcare systems. This involves meeting the regulatory criteria set by health technology assessment (HTA) agencies, which stipulate that the intervention must be efficacious, safe, cost-effective, and more efficient than existing standard-of-care practices. Unfortunately, the existing evidence regarding the health economics outcomes of NGS technologies for blood cancers on healthcare systems is currently limited and may not be fit-for-purpose for the practical application of these precision medicine diagnostics for patients with blood cancer. This thesis aims to bridge the current economic evidence gap by evaluating the health economics implications of NGS technologies for the management of blood cancers at various stages of patient clinical care and learning from these assessments to develop a general framework for assessing the health economics value of NGS technologies for patients with blood cancer in Australia. To achieve these aims, this thesis encompasses four distinct studies. The first study reviewed health economics literature for molecular biomarker tests, encompassing NGS, and identified a research gap where various applications of NGS in blood cancers are yet to be explored in terms of their potential health economics outcomes. All subsequent studies from this thesis sought to remedy this lack of research. The second study evaluated the cost-effectiveness of a targeted NGS gene panel designed to identify patients who would respond optimally to a

BACKGROUND: Cancer-derived material circulating in the bloodstream and other bodily fluids, referred to as liquid biopsies (LBs), has become an appealing adjunct or alternative to tissue biopsies, showing vital promise in several clinical applications.PURPOSE: A systematic literature review was conducted to (1) summarize the current health economic evidence for LB assays and (2) identify and analyze the studies addressed or reported on the challenges of health economic modeling in precision medicine.METHODS: Relevant studies were identified in the EMBASE, MEDLINE, Cochrane Library, EconLit, and the University of Melbourne Full Text Journal databases from 1 January 2013 to 16 September 2022. Included papers were selected if they were economic evaluations and/or budget impact analyses.RESULTS: A total of 24 studies were included and analyzed, with the majority being full economic evaluations (n = 19, 79.2%). Four studies (16.7%) were health and budget impact analyses, and one study (4.1%) incorporated both an economic evaluation and a budget impact analysis. Cohort-level modeling techniques were the most common approach (n = 16; 80%). LB technologies were cost-effective in 15 studies (75%) considering different biomarkers, cancer types and stages, and economic analyses. These studies evaluated LBs for screening and early detection (66.7%), treatment selection (26.7%), and monitoring treatment response (6.6%). Budget impact analysis results were varied among included studies, with the majority of studies (n = 4; 80%) reporting either cost savings, minimal, or modest budget impact, while one study (20%) reported LBs as an efficient strategy. The reviewed studies often inadequately reported or addressed modeling challenges, such as patient-level processes, the combination of tests and treatments, preferences, and uncertainty.CONCLUSION: LBs could provide a cost-effective approach for treatment selection in lung cancer and aid in the screeni

Objectives: Molecular biomarker tests can inform the clinical management of genomic heterogeneous hematological malignancies, yet their availability in routine care largely depends on the supporting health economic evidence. This study aims to systematically review the economic evidence for recent molecular biomarker tests in hematological malignancies. Methods: We conducted a systematic search in five electronic databases for studies published between January 2010 and October 2020. Publications were independently screened by two reviewers. Clinical study characteristics, economic methodology, and results were extracted, and reporting quality was assessed. Results: Fourteen studies were identified, of which half (n = 7; 50%) were full economic evaluations examining both health and economic outcomes. Studies were predominantly conducted in a first-line treatment setting (n = 7; 50%) and adopted a non-lifetime time horizon to measure health outcomes and costs (n = 7; 50%). Five studies reported that companion diagnostics for associated therapies were likely cost-effective for acute myeloid leukemia, chronic myeloid leukemia, diffuse large B-cell lymphoma, and multiple myeloma. Four studies suggested molecular biomarker tests for treatment monitoring in chronic myeloid leukemia were likely cost-saving. Conclusions: Although there is initial confirmation of the promising health economic results, the present research for molecular biomarker tests in hematological malignancies is sparse with many applications of technological advances yet to be evaluated.

Objective: The aim of this systematic review was to provide a comprehensive and detailed review of structural and methodological assumptions in model-based cost-effectiveness analyses of systemic metastatic colorectal cancer (mCRC) treatments, and discuss their potential impact on health economic outcome estimates. Methods: Five databases (EMBASE, MEDLINE, Cochrane Library, Health Technology Assessment and National Health Service Health Economic Evaluation Database) were searched on 26 August 2019 for model-based full health economic evaluations of systemic mCRC treatment using a combination of free-text terms and subject headings. Full-text publications in English were eligible for inclusion if they were published in or after the year 2000. The Consolidated Health Economic Evaluation Reporting Standards checklist was used to assess the reporting quality of included publications. Study selection, appraisal and data extraction were performed by two reviewers independently. Results: The search yielded 1418 publications, of which 54 were included, representing 51 unique studies. Most studies focused on first-line treatment (n = 29, 57%), followed by third-line treatment (n = 13, 25%). Model structures were health-state driven (n = 27, 53%), treatment driven (n = 19, 37%), or a combination (n = 5, 10%). Cohort-level state-transition modelling (STM) was the most common technique (n = 33, 65%), followed by patient-level STM and partitioned survival analysis (both n = 6, 12%). Only 15 studies (29%) reported some sort of model validation. Health economic outcomes for specific strategies differed substantially between studies. For example, survival following first-line treatment with fluorouracil, leucovorin and oxaliplatin ranged from 1.21 to 7.33 years, with treatment costs ranging from US$8125 to US$126,606. Conclusions: Model-based cost-effectiveness analyses of systemic mCRC treatments have adopted varied modelling methods and structures, resulting in substantially d