scholarly journals How Are We Evaluating the Cost-Effectiveness of Companion Biomarkers for Targeted Cancer Therapies? A Systematic Review

Author(s):  
Mikyung Kelly Seo ◽  
John Cairns

Abstract Background Despite the increasing economic assessment of biomarker-guided therapies, no clear agreement exists whether existing methods are sufficient or whether different methods might produce different cost-effectiveness results. This study aims to examine current practices of modeling companion biomarkers when assessing the cost-effectiveness of targeted cancer therapies. It highlights the challenges in methods and data requirements faced in the evaluation of biomarker tests which do not necessarily arise with the evaluation of pharmaceutical drugs.Methods A literature search was performed using Medline, Embase, EconLit, Cochrane library. Articles published from 2014 to 2018 were searched. Economic evaluations on biomarker-guided therapies with companion diagnostics in cancer were searched. Preferred Reporting Items of Systematic Reviews and Meta-Analyses (PRISMA) guidelines followed. Studies were selected by pre-specified eligibility criteria using PICO framework. To make studies more comparable, data were synthesized under ten categories of key areas of methods where consensus deemed lacking.Results Eighteen papers were included in this review. Three out of eighteen studies found to be of good quality regarding incorporating the characteristics of companion biomarkers in economic evaluations. However, many evaluations focused on a pre-selected patient group with a specific biomarker status instead of including all patients with a disease regardless of their biomarker status. Companion biomarker characteristics captured in evaluations were often limited to the cost or the accuracy of the test. Often, only the costs of biomarker testing were modelled. Clinical outcomes or utilities were often difficult to include due to the limited data generated by clinical trials. We found that no consistency and consensus existed to the methods of existing economic evaluations of companion cancer biomarkers for targeted therapies. It was also shown that conflicting cost-effectiveness results were likely depending on what comparator arm was chosen and what comparison structure was designed in the model.Conclusion We found that there was no consistent approach applied in assessing the value of biomarkers and including the characteristics of biomarkers in an economic evaluation of targeted oncology therapies. Currently, many EEs fail to capture the full value of companion biomarkers beyond sensitivity/specificity and cost related to biomarker testing.

BMC Cancer ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Mikyung Kelly Seo ◽  
John Cairns

Abstract Background Despite the increasing economic assessment of biomarker-guided therapies, no clear agreement exists whether existing methods are sufficient or whether different methods might produce different cost-effectiveness results. This study aims to examine current practices of modeling companion biomarkers when assessing the cost-effectiveness of targeted cancer therapies. It investigates the current methods in modeling the characteristics of companion diagnostics based on existing economic evaluations of biomarker-guided therapies in cancer. Methods A literature search was performed using Medline, Embase, EconLit, Cochrane library for economic evaluations of biomarker-guided therapies with companion diagnostics in cancer. Preferred Reporting Items of Systematic Reviews and Meta-Analyses (PRISMA) guidelines were followed. Studies were selected using pre-specified eligibility criteria based on the PICO framework. To make the included studies more comparable, we qualitatively synthesized the data under nine domains of methods where consensus was deemed lacking. Results Only four of the twenty-two studies included in this review were found to be of good quality with respect to incorporating the characteristics of companion biomarkers in economic evaluations. However, many evaluations focused on a pre-selected patient group rather than including all patients regardless of their biomarker status. Companion biomarker characteristics captured in evaluations were often limited to the cost or the accuracy of the test. Often, only the costs of biomarker testing were modelled. Clinical outcomes and health state utilities were often not included due to the limited data generated by clinical trials. Methods of economic evaluation were not applied consistently in assessments of companion cancer biomarkers for targeted therapies. It was also shown that conflicting cost-effectiveness results were likely depending on what comparator arm was chosen and what comparison structure was designed in the model. Conclusion We found no consistent approach applied in assessing the value of companion biomarker tests and including the characteristics of biomarkers in an economic evaluation of targeted oncology therapies. Currently, many economic evaluations fail to capture the full value of companion biomarkers beyond sensitivity/specificity and cost related to biomarker testing.


Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4547-4547
Author(s):  
Ozlem Topaloglu ◽  
Chuck Stevens ◽  
Saurabh Aggarwal

Abstract Abstract 4547 Targeted cancer therapies for hematological malignancies have rapidly gained uptake in different types of indications, and some have become gold standard of treatment. However, the high cost of these therapies poses challenges for payers, patients and physicians. Methods To understand the cost-effectiveness of these new classes of drugs we reviewed the published cost-effectiveness studies and analyzed their cost/QALY, time horizon, comparators, indications and model approaches. Results Targeted therapies for hematological cancers have much lower and less variable cost/QALY than branded drugs indicated for solid tumors. For example, incremental cost effectiveness ratio (ICER) for imatinib ranges from $40,000 to $45,000/QALY, while ICER for bevacizumab ranges from $125,000 to $325,000/QALY. Secondly, the choice of comparator in some occasions may depend on the country of use, therefore models are required to be customized to local regimens. In one of the cost-effectiveness analysis for bortezomib for refractory multiple myeloma patients, NICE questioned the use of high dose dexamethasone (HDD) as comparator. Third, there is also some variability in methods used by different manufacturers, some used cost/QALY whereas others used cost/LYG. Conclusion: This analysis shows the range, variability and methods used for calculation of ICER values for these targeted cancer therapies and provides lessons for executives and policy makers. Disclosures: No relevant conflicts of interest to declare.


Author(s):  
Komal Shah ◽  
Somen Saha ◽  
Priya Kotwani ◽  
Malkeet Singh ◽  
Kirti Tyagi

IntroductionIndia has introduced health technology assessment (HTA) as a tool for improving the allocation of health resources. The core mandate of HTA in India (HTAIn) is to undertake critical appraisal of available technologies, identify cost-effective interventions, and help the government pursue evidence-informed decisions regarding public health expenditures. We conducted a systematic review to assess economic evaluation studies published in the last four years from India.MethodsEconomic evaluations published from September 2015 to September 2019 were identified by searching various databases, including PubMed, Scopus, Embase, The Cochrane Library, and CINAHL according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guideline. Cost-effectiveness studies and HTAs reported or conducted in India were included. Two independent reviewers performed the final selection of studies by assessing the full-text articles and conducted the data extraction. Differences of opinions were resolved through discussion and mutual consensus.ResultsAfter screening 2,837 articles, seventy met the inclusion criteria and were selected. The articles predominantly used secondary data (70%) to evaluate the cost effectiveness of an innovation. Among the technologies assessed, fifty-seven percent were curative in nature and most commonly addressed infectious diseases (27%), closely followed by non-communicable diseases, and maternal and child health. Principally, the cost effectiveness of a technology was expressed in terms of disability-adjusted or quality-adjusted life-years. Only two studies reported negative findings.ConclusionsHTA can play a pivotal role in equipping policy makers and public health payers to make appropriate decisions for healthcare budget allocations when mapped with the true disease burden of the population. It is important to highlight negative results and to create a national repository of HTA studies to facilitate faster adoption of best practices in India.


2018 ◽  
Vol 2018 ◽  
pp. 1-14 ◽  
Author(s):  
Lachaine Jean ◽  
Miron Audrey ◽  
Catherine Beauchemin ◽  
on behalf of the iGenoMed Consortium

Objective. The objective of this literature review was to evaluate the existing evidence regarding the cost-effectiveness of treatment options in IBD. Methods. A systematic review of the literature was conducted to identify economic evaluations of IBD therapy. The literature search was performed using electronic databases MEDLINE and EMBASE. Searches were limited to full economic evaluations published in English or French between 2004 and 2016. Results. A total of 5,403 potentially relevant studies were identified. After screening titles and abstracts, 48 studies were included, according to the eligibility criteria. A total of 56% and 42% of the studies were assessing treatments of UC or CD, respectively. Treatment options under evaluation included biological agents, mesalamine, immunosuppressants, and surgery. The majority of studies evaluated the cost-effectiveness of biological treatments. Biological therapies were dominant in 23% of the analyses and were cost-effective according to a $CAD50,000/QALY and $CAD100,000/QALY threshold in 41% and 62% of the analyses, respectively. Conclusion. This literature review provided a comprehensive overview of the economic evaluations for the different treatment options for IBD over the past 12 years and represents a helpful reference for future economic evaluations.


2019 ◽  
Vol 215 (01) ◽  
pp. 388-394 ◽  
Author(s):  
David Aceituno ◽  
Norha Vera ◽  
A. Matthew Prina ◽  
Paul McCrone

BackgroundEarly intervention in psychosis (EIP) has been developed as an approach to improve the prognosis of people with psychotic disorders and it has been claimed to be a more efficient model of care. However, the evidence is not definitive and doubts have spread regard to the economic outcomes of EIP services amid the usually restricted mental health budget.AimsWe aimed to review the cost-effectiveness evidence of EIP services worldwide.MethodWe systematically reviewed the economic literature about EIP following the Preferred Reporting Items for Systematic reviews and Meta-Analyses statement guidelines. Studies were selected according to previously stated criteria and analysed with standardised critical appraisal tools for trial-based economic evaluations and modelling studies.ResultsA total of 16 studies were selected after applying the eligibility criteria. Most of them were economic evaluations alongside clinical trials. The overall evidence was consistent in the cost-effectiveness of EIP compared with standard care for first episode of psychosis and the Clinical High Risk for Psychosis paradigm. Such evidence was replicated among different health systems, but mainly in high-income countries. The methodological quality of such evidence, however, was moderate and heterogeneity was significant across the studies.ConclusionsThere is consistent evidence that the implementation of EIP services might be a cost-effective alternative across different health systems. Such evidence, nevertheless, derives from heterogeneous and sometimes methodologically flawed studies, reducing the certainty of such statement. More efforts must be done to rigorously assess the value of this intervention, before expanding it among systems where mental health budgets are more constrained.Declaration of interestNone.


1996 ◽  
Vol 3 (1) ◽  
pp. 47-53 ◽  
Author(s):  
Afaf Girgis ◽  
Philip Clarke ◽  
Robert C Burton ◽  
Rob W Sanson—Fisher

Background and design— Australia has the highest rates of skin cancer in the world, and the incidence is estimated to be doubling every 10 years. Despite advances in the early detection and treatment of melanoma about 800 people still die nationally of the disease each year. A possible strategy for further reducing the mortality from melanoma is an organised programme of population screening for unsuspected lesions in asymptomatic people. Arguments against introducing melanoma screening have been based on cost and the lack of reliable data on the efficacy of any screening tests. To date, however, there has been no systematic economic assessment of the cost effectiveness of melanoma screening. The purpose of this research was to determine whether screening may be potentially cost effective and, therefore, warrants further investigation. A computer was used to simulate the effects of a hypothetical melanoma screening programme that was in operation for 20 years, using cohorts of Australians aged 50 at the start of the programme. Based on this simulation, cost—effectiveness estimates of melanoma screening were calculated. Results— Under the standard assumptions used in the model, and setting the sensitivity of the screening test (visual inspection of the skin) at 60%, cost effectiveness ranged from Aust$6853 per life year saved for men if screening was undertaken five yearly to $12137 if screening was two yearly. For women, it ranged from $11 102 for five yearly screening to $20 877 for two yearly screening. Conclusion— The analysis suggests that a melanoma screening programme could be cost effective, particularly if five yearly screening is implemented by family practitioners for men over the age of 50.


2012 ◽  
Vol 30 (4) ◽  
pp. 273-285 ◽  
Author(s):  
Song-Yi Kim ◽  
Hyangsook Lee ◽  
Younbyoung Chae ◽  
Hi-Joon Park ◽  
Hyejung Lee

Objective To summarise the evidence on the cost-effectiveness of acupuncture. Methods We identified full economic evaluations such as cost-effectiveness analysis (CEA), cost-utility analysis (CUA) and cost-benefit analysis (CBA) alongside randomised controlled trials (RCTs) that assessed the consequences and costs of acupuncture for any medical condition. Eleven electronic databases were searched up to March 2011 without language restrictions. Eligible RCTs were assessed using the Cochrane criteria for risk of bias and a modified version of the checklist for economic evaluation. The general characteristics and the results of each economic analysis such as incremental cost-effectiveness ratios (ICERs) were extracted. Results Of 17 included studies, nine were CUAs that measured quality-adjusted life years (QALYs) and eight were CEAs that assessed effectiveness of acupuncture based on improvements in clinical symptoms. All CUAs showed that acupuncture with or without usual care was cost-effective compared with waiting list control or usual care alone, with ICERs ranging from ¢3011/QALY (dysmenorrhoea) to ¢22 298/QALY (allergic rhinitis) in German studies, and from £3855/QALY (osteoarthritis) to £9951/QALY (headache) in UK studies. In the CEAs, acupuncture was beneficial at a relatively low cost in six European and Asian studies. All CUAs were well-designed with a low risk of bias, but this was not the case for CEAs. Conclusions Overall, this review demonstrates the cost-effectiveness of acupuncture. Despite such promising results, any generalisation of these results needs to be made with caution given the diversity of diseases and the different status of acupuncture in the various countries.


2021 ◽  
Vol 9 (3) ◽  
pp. 232596712098753
Author(s):  
Cammille C. Go ◽  
Cynthia Kyin ◽  
Jeffrey W. Chen ◽  
Benjamin G. Domb ◽  
David R. Maldonado

Background: Hip arthroscopy has frequently been shown to produce successful outcomes as a treatment for femoroacetabular impingement (FAI) and labral tears. However, there is less literature on whether the favorable results of hip arthroscopy can justify the costs, especially when compared with a nonoperative treatment. Purpose: To systematically review the cost-effectiveness of hip arthroscopy for treating FAI and labral tears. Study Design: Systematic review; Level of evidence, 3. Methods: PubMed/MEDLINE, Embase, and Cochrane Library databases, and the Tufts University Cost-Effectiveness Analysis Registry were searched to identify articles that reported the cost per quality-adjusted life-year (QALY) generated by hip arthroscopy. The key terms used were “hip arthroscopy,” “cost,” “utility,” and “economic evaluation.” The threshold for cost-effectiveness was set at $50,000/QALY. The Methodological Index for Non-Randomized Studies instrument and Quality of Health Economic Studies (QHES) score were used to determine the quality of the studies. This study was prospectively registered on PROSPERO (CRD42020172991). Results: Six studies that reported the cost-effectiveness of hip arthroscopy were identified, and 5 of these studies compared hip arthroscopy to a nonoperative comparator. These studies were found to have a mean QHES score of 85.2 and a mean cohort age that ranged from 33-37 years. From both a health care system perspective and a societal perspective, 4 studies reported that hip arthroscopy was more costly but resulted in far greater gains than did nonoperative treatment. The preferred treatment strategy was most sensitive to duration of benefit, preoperative osteoarthritis, cost of the arthroscopy, and the improvement in QALYs with hip arthroscopy. Conclusion: In the majority of the studies, hip arthroscopy had a higher initial cost but provided greater gain in QALYs than did a nonoperative treatment. In certain cases, hip arthroscopy can be cost-effective given a long enough duration of benefit and appropriate patient selection. However, there is further need for literature to analyze willingness-to-pay thresholds.


BMJ Open ◽  
2021 ◽  
Vol 11 (9) ◽  
pp. e048141
Author(s):  
Sara Mucherino ◽  
Valentina Lorenzoni ◽  
Valentina Orlando ◽  
Isotta Triulzi ◽  
Marzia Del Re ◽  
...  

IntroductionThe combination of biomarkers and drugs is the subject of growing interest both from regulators, physicians and companies. This study protocol of a systematic review is aimed to describe available literature evidences about the cost-effectiveness, cost-utility or net-monetary benefit of the use of biomarkers in solid tumour as tools for customising immunotherapy to identify what further research needs.Methods and analysisA systematic review of the literature will be carried out according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement guidelines. PubMed and Embase will be queried from June 2010 to June 2021. The PICOS model will be applied: target population (P) will be patients with solid tumours treated with immune checkpoint inhibitors (ICIs); the interventions (I) will be test of the immune checkpoint predictive biomarkers; the comparator (C) will be any other targeted or non-targeted therapy; outcomes (O) evaluated will be health economic and clinical implications assessed in terms of incremental cost-effectiveness ratio, net health benefit, net monetary benefit, life years gained, quality of life, etc; study (S) considered will be economic evaluations reporting cost-effectiveness analysis, cost-utility analysis, net-monetary benefit. The quality of the evidence will be graded according to Grading of Recommendations Assessment, Development and Evaluation.Ethics and disseminationThis systematic review will assess the cost-effectiveness implications of using biomarkers in the immunotherapy with ICIs, which may help to understand whether this approach is widespread in real clinical practice. This research is exempt from ethics approval because the work is carried out on published documents. We will disseminate this protocol in a related peer-reviewed journal.PROSPERO registration numberCRD42020201549.


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