Societal Perspectives in Economic Analyses and Real-World Cost-Effectiveness Studies in Cancer

A special issue of Current Oncology (ISSN 1718-7729). This special issue belongs to the section "Health Economics".

Deadline for manuscript submissions: closed (15 March 2022) | Viewed by 34160

Special Issue Editor


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Guest Editor
Health Policy and Management, DeGroote School of Business, McMaster University, Hamilton, ON, Canada
Interests: patients’ out-of-pocket costs; financial toxicity; patient and caregiver lost income; economic evaluation; health policy
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Special Issue Information

Dear Colleagues,

The economic evaluation of health technologies for cancer is frequently seen in the literature, but not all economic perspectives are seen with the same frequency. Although a health system perspective is commonly used to address the needs of the payer, these analyses tend to ignore the broader impact on patients. In order to understand these broader perspectives, economic evaluations need to include other effects, such as patient out-of-pocket costs, travel costs, lost income for patients and caregivers, and their combined impacts on quality of life.

Similarly, the majority of cost-effectiveness analyses in cancer are based on clinical trials, which typically lack generalizability. Additional analyses that examine cost-effectiveness in real-world settings by using government and private payer databases or modeling techniques allow readers to understand whether trial-based cost-effectiveness aligns with the results seen after technology approval.

In this Special Issue we encourage those with ongoing work in these areas to submit their manuscripts. The intent is to give priority to manuscripts that meet one or both of the above listed foci. We also encourage indigenous population analyses if available in your datasets

Dr. Christopher J. Longo
Guest Editor

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Keywords

  • real-world outcomes
  • societal perspective
  • economic evaluation
  • out-of-pocket costs
  • patient income loss
  • cancer

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Published Papers (8 papers)

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Editorial

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3 pages, 160 KiB  
Editorial
Societal Perspectives and Real-World Cost-Effectiveness: Expanding the Scope of Health Economics Inquiry
by Christopher J. Longo
Curr. Oncol. 2023, 30(1), 233-235; https://doi.org/10.3390/curroncol30010018 - 23 Dec 2022
Cited by 2 | Viewed by 1632
Abstract
Economic evaluations of health technologies for cancer are frequently seen in the literature, but not all economic perspectives have the same frequency [...] Full article

Research

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15 pages, 2468 KiB  
Article
Cost-Effectiveness of Pyrotinib Plus Capecitabine versus Lapatinib Plus Capecitabine for the Treatment of HER2-Positive Metastatic Breast Cancer in China: A Scenario Analysis of Health Insurance Coverage
by Yuwen Bao, Zhuolin Zhang, Xuan He, Lele Cai, Xiao Wang and Xin Li
Curr. Oncol. 2022, 29(9), 6053-6067; https://doi.org/10.3390/curroncol29090476 - 23 Aug 2022
Cited by 7 | Viewed by 3154
Abstract
Background: The overexpression of the human epidermal growth factor receptor-2 (HER2) gene is present in 20~25% of breast cancer (BC) patients, contributing to an inferior prognosis. Recent clinical trials showed that pyrotinib has promising antitumor activities and acceptable tolerability for those patients (ClinicalTrials.gov, [...] Read more.
Background: The overexpression of the human epidermal growth factor receptor-2 (HER2) gene is present in 20~25% of breast cancer (BC) patients, contributing to an inferior prognosis. Recent clinical trials showed that pyrotinib has promising antitumor activities and acceptable tolerability for those patients (ClinicalTrials.gov, NCT03080805 and NCT02422199). Therefore, this study aims to assess the cost-effectiveness of pyrotinib plus capecitabine versus lapatinib plus capecitabine for patients with HER2-positive metastatic BC after prior trastuzumab. Methods: A lifetime-partitioned survival model was established to evaluate health and economic outcomes with different treatment strategies. The primary outcome was the incremental cost-effectiveness ratio (ICER). Data were derived from the published literature, clinical trials, expert opinions, and other local charges. Sensitivity analyses were performed to assess the robustness of the findings. Scenario analyses were developed to make further evaluations. Results: The pyrotinib regimen had significant advantages over the lapatinib regimen after enrolling in the National Reimbursement Drug List (NRDL), with cost savings of USD 15,599.27 and a gain of 0.53 QALYs. Meanwhile, before enrolling in NRDL, the pyrotinib regimen afforded the same QALYs at a higher incremental cost of USD 45,400.64 versus the lapatinib regimen, producing an ICER of USD 85,944.79 per QALY. Scenario analyses yielded similar results. Sensitivity analyses suggested stability in the cost-effectiveness findings. Conclusions: Compared to lapatinib plus capecitabine, the pyrotinib plus capecitabine enrolled in NRDL is a cost-effective alternative second-line treatment for patients with HER2-positive metastatic BC in China. Full article
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17 pages, 515 KiB  
Article
The Burden of Health-Related Out-of-Pocket Cancer Costs in Canada: A Case-Control Study Using Linked Data
by Beverley M. Essue, Claire de Oliveira, Tracey Bushnik, Sharon Fung, Jeremiah Hwee, Zhuolu Sun, Elba Gomez Navas, Jean Hai Ein Yong and Rochelle Garner
Curr. Oncol. 2022, 29(7), 4541-4557; https://doi.org/10.3390/curroncol29070359 - 27 Jun 2022
Cited by 2 | Viewed by 2290
Abstract
Background: The burden of out-of-pocket costs among cancer patients/survivors in Canada is not well understood. The objective of this study was to examine the health-related out-of-pocket cost burden experienced by households with a cancer patient/survivor compared to those without, examine the components of [...] Read more.
Background: The burden of out-of-pocket costs among cancer patients/survivors in Canada is not well understood. The objective of this study was to examine the health-related out-of-pocket cost burden experienced by households with a cancer patient/survivor compared to those without, examine the components of health-related costs and determine who experiences a greater burden. Data and methods: This study used a data linkage between the Survey of Household Spending and the Canadian Cancer Registry to identify households with a cancer patient/survivor (cases) and those without (controls). The out-of-pocket burden (out-of-pocket costs measured relative to household income) and mean costs were described and regression analyses examined the characteristics associated with the household out-of-pocket burden and annual out-of-pocket costs. Results: The health-related out-of-pocket cost burden and annual costs measured in households with a cancer patient/survivor were 3.08% (95% CI: 2.55–3.62%) and CAD 1600 (95% CI: 1456–1759), respectively, compared to a burden of 2.84% (95% CI: 2.31–3.38) and annual costs of CAD 1511 (95% CI: 1377–1659) measured in control households, respectively. Households with a colorectal cancer patient/survivor had a significantly higher out-of-pocket burden compared to controls (mean difference: 1.0%, 95% CI: 0.18, 0.46). Among both cases and controls, the lowest income quintile households experienced the highest health-related out-of-pocket cost burden. Interpretation: Within a universal health care system, it is still relevant to monitor health-related out-of-pocket spending that is not covered by existing insurance mechanisms; however, this is not routinely assessed in Canada. We demonstrate the feasibility of measuring such costs in households with a cancer patient/survivor using routinely collected data. While the burden and annual health-related out-of-pocket costs of households with a cancer patient/survivor were not significantly higher than control households in this study, the routine measurement of out-of-pocket costs in Canada could be systemized, providing a novel, system-level, equity-informed performance indicator, which is relevant for monitoring inequities in the burden of out-of-pocket costs. Full article
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13 pages, 300 KiB  
Article
Cancer Premature Mortality Costs in Europe in 2020: A Comparison of the Human Capital Approach and the Friction Cost Approach
by Paul Hanly, Marta Ortega-Ortega and Isabelle Soerjomataram
Curr. Oncol. 2022, 29(5), 3552-3564; https://doi.org/10.3390/curroncol29050287 - 13 May 2022
Cited by 8 | Viewed by 2927
Abstract
The inclusion of productivity costs can affect the outcome of cost-effectiveness analyses. We estimated the value of cancer premature mortality productivity costs for Europe in 2020 using the Human Capital Approach (HCA) and compared these to the Friction Cost Approach (FCA). Cancer mortality [...] Read more.
The inclusion of productivity costs can affect the outcome of cost-effectiveness analyses. We estimated the value of cancer premature mortality productivity costs for Europe in 2020 using the Human Capital Approach (HCA) and compared these to the Friction Cost Approach (FCA). Cancer mortality data were obtained from GLOBOCAN 2020 by sex and five-year age groups. Twenty-three cancer sites for 31 European countries were included. The HCA and the FCA were valued using average annual gross wages by sex and age group and applied to Years of Potential Productive Life Lost. 2020 friction periods were calculated and all costs were in 2020 euros. Estimated cancer premature mortality costs for Europe in 2020 were EUR 54.0 billion (HCA) and EUR 1.57 billion (FCA). The HCA/FCA cost ratio for Europe was 34.4, but considerable variation arose across countries (highest in Ireland: 64.5 v lowest in Czech Republic: 11.1). Both the HCA and the FCA ranked lung, breast and colorectal as the top three most costly cancers in Europe, but cost per death altered rankings substantially. Significant cost differences were observed following sensitivity analysis. Our study provides a unique perspective of the difference between HCA and FCA estimates of productivity costs by cancer site and country in Europe. Full article
14 pages, 852 KiB  
Article
The Economic Burden of Cancer in Canada from a Societal Perspective
by Roxanne Garaszczuk, Jean H. E. Yong, Zhuolu Sun and Claire de Oliveira
Curr. Oncol. 2022, 29(4), 2735-2748; https://doi.org/10.3390/curroncol29040223 - 14 Apr 2022
Cited by 25 | Viewed by 9314
Abstract
Cancer patients and their families experience considerable financial hardship; however, the current published literature on the economic burden of cancer at the population level has typically focused on the costs from the health system’s perspective. This study aims to estimate the economic burden [...] Read more.
Cancer patients and their families experience considerable financial hardship; however, the current published literature on the economic burden of cancer at the population level has typically focused on the costs from the health system’s perspective. This study aims to estimate the economic burden of cancer in Canada from a societal perspective. The analysis was conducted using the OncoSim-All Cancers model, a Canadian cancer microsimulation model. OncoSim simulates cancer incidence and deaths using incidence and mortality data from the Canadian Cancer Registry and demography projections from Statistics Canada. Using a phase-based costing framework, we estimated the economic burden of cancer in Canada in 2021 by incorporating published direct health system costs and patients’ and families’ costs (out-of-pocket costs, time costs, indirect costs). From a societal perspective, cancer-related costs were CAD 26.2 billion in Canada in 2021; 30% of costs were borne by patients and their families. The economic burden was the highest in the first year after cancer was diagnosed (i.e., initial care). During this time, patients and families’ costs amounted to almost CAD 4.8 billion in 2021. This study provides a comprehensive estimate of the economic burden of cancer, which could inform cost–benefit analyses of proposed cancer prevention interventions. Full article
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Review

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20 pages, 991 KiB  
Review
Rapid Review of Real-World Cost-Effectiveness Analyses of Cancer Interventions in Canada
by Andrea M. Guggenbickler, Heather K. Barr, Jeffrey S. Hoch and Carolyn S. Dewa
Curr. Oncol. 2022, 29(10), 7285-7304; https://doi.org/10.3390/curroncol29100574 - 30 Sep 2022
Cited by 3 | Viewed by 3634
Abstract
Cost-effectiveness analysis (CE Analysis) provides evidence about the incremental gains in patient outcomes costs from new treatments and interventions in cancer care. The utilization of “real-world” data allows these analyses to better reflect differences in costs and effects for actual patient populations with [...] Read more.
Cost-effectiveness analysis (CE Analysis) provides evidence about the incremental gains in patient outcomes costs from new treatments and interventions in cancer care. The utilization of “real-world” data allows these analyses to better reflect differences in costs and effects for actual patient populations with comorbidities and a range of ages as opposed to randomized controlled trials, which use a restricted population. This rapid review was done through PubMed and Google Scholar in July 2022. Relevant articles were summarized and data extracted to summarize changes in costs (in 2022 CAD) and effectiveness in cancer care once funded by the Canadian government payer system. We conducted statistical analyses to examine the differences between means and medians of costs, effects, and incremental cost effectiveness ratios (ICERs). Twenty-two studies were selected for review. Of those, the majority performed a CE Analysis on cancer drugs. Real-world cancer drug studies had significantly higher costs and effects than non-drug therapies. Studies that utilized a model to project longer time-horizons saw significantly smaller ICER values for the treatments they examined. Further, differences in drug costs increased over time. This review highlights the importance of performing real-world CE Analysis on cancer treatments to better understand their costs and impacts on a general patient population. Full article
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Other

32 pages, 741 KiB  
Systematic Review
The Health Economics of Metastatic Hormone-Sensitive and Non-Metastatic Castration-Resistant Prostate Cancer—A Systematic Literature Review with Application to the Canadian Context
by Ivan Yanev, Jessy Gatete, Jr., Armen G. Aprikian, Jason Robert Guertin and Alice Dragomir
Curr. Oncol. 2022, 29(5), 3393-3424; https://doi.org/10.3390/curroncol29050275 - 7 May 2022
Cited by 4 | Viewed by 7587
Abstract
Background: Health economic evaluations are needed to assess the impact on the healthcare system of emerging treatment patterns for advanced prostate cancer. The objective of this study is to review the scientific literature identifying cost-effectiveness and cost analyses that are assessing treatments for [...] Read more.
Background: Health economic evaluations are needed to assess the impact on the healthcare system of emerging treatment patterns for advanced prostate cancer. The objective of this study is to review the scientific literature identifying cost-effectiveness and cost analyses that are assessing treatments for metastatic hormone-sensitive prostate cancer (mHSPC) and nonmetastatic castration-resistant prostate cancer (nmCRPC). Methods: On 29 June 2021, we searched the scientific (MEDLINE, Embase, and EBSCO) and grey literature for health economic studies targeting mHSPC and nmCRPC. We used the CHEC-extended checklist and the Welte checklist for risk-of-bias assessment and transferability analysis, respectively. Results: We retained 20 cost-effectiveness and 4 cost analyses in the mHSPC setting, and 14 cost-effectiveness and 6 cost analyses in the nmCRPC setting. Docetaxel in combination with androgen deprivation therapy (ADT) was the most cost-effective treatment in the mHSPC setting. Apalutamide, darolutamide, and enzalutamide presented similar results vs. ADT alone and were identified as cost-effective treatments for nmCRPC. An increase in costs as patients transitioned from nmCRPC to mCRPC was noted. Conclusions: We concluded that there is an important unmet need for health economic evaluations in the mHSPC and nmCRPC setting incorporating real-world data to support healthcare decision making. Full article
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7 pages, 527 KiB  
Commentary
Linking Intermediate to Final “Real-World” Outcomes: Is Financial Toxicity a Reliable Predictor of Poorer Outcomes in Cancer?
by Christopher J. Longo
Curr. Oncol. 2022, 29(4), 2483-2489; https://doi.org/10.3390/curroncol29040202 - 2 Apr 2022
Cited by 7 | Viewed by 2388
Abstract
Traditionally, economic evaluations are based on clinical trials with well-defined patient populations that exclude many patient types. By contrast, studies that incorporate general patient populations end up including those in lower income categories, some of whom have significant financial burdens (often described as [...] Read more.
Traditionally, economic evaluations are based on clinical trials with well-defined patient populations that exclude many patient types. By contrast, studies that incorporate general patient populations end up including those in lower income categories, some of whom have significant financial burdens (often described as financial toxicity) related to their care. Consideration of these patient burdens when examining the incremental cost-effectiveness of newer treatments from a clinical trial perspective can result in differing conclusions regarding cost-effectiveness. The challenge is to reliably assess the link between financial toxicity, quality of life and potential decisions to forego or delay care. It is also well-documented that these financial effects are not evenly distributed across populations, with those with low income and of black or Latino decent being most affected. There is a paucity of literature in this space, but some of the early work has suggested that for lung, breast, colorectal and ovarian cancers there are poorer quality-of-life scores and/or shorter overall survival for those experiencing financial toxicity. Hence, we may see both a lower quality of life and a shorter duration of life for these populations. If this is the case, additional considerations include: are the benefits of newer, more-expensive treatment strategies muted by the lack of adherence to these newer treatments due to financial concerns, and, if true, can these effects be effectively quantified as “real-world” outcomes? This rapid review examines these possibilities and the steps that may be required to examine this reliably. Full article
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