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Objectives: Accurate risk stratification of patients with stage II and III colorectal cancer (CRC) prior to treatment selection enables limited health resources to be efficiently allocated to patients who are likely to benefit from adjuvant chemotherapy. We aimed to investigate the cost-effectiveness of a recently developed deep learning-based prognostic method, Histotyping, from the perspective of the Norwegian healthcare system., Methods: Two partitioned survival models were developed to assess the cost-effectiveness of Histotyping for two treatment cohorts: patients with CRC stage II and III. For each of the two cohorts, Histotyping was used for risk stratification to assign adjuvant chemotherapy and was compared with the standard of care (SOC) (adjuvant chemotherapy to all patients). Health outcomes measured in the model were quality-adjusted life years (QALYs) and life years (LYs) gained. Deterministic and probabilistic sensitivity analyses were performed to determine the impact of uncertainty. Scenario analyses were performed to assess the impact of the parameters with the greatest uncertainty., Results: Risk-stratifying patients with CRC stage II and III using Histotyping was dominant (less costly and more effective) compared to SOC. In patients with CRC stage II, the net monetary benefit of Histotyping was 270,934 Norwegian kroners (NOK) (year of valuation is 2021), and the net health benefit of Histotyping was 0.99. In stage III, the net monetary benefit of Histotyping was 195,419 NOK, and the net health benefit of Histotyping was 0.71., Conclusions:  Risk-stratifying patients with CRC using Histotyping prior to the administration of adjuvant chemotherapy is likely to be a cost-effective strategy in Norway., (© 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

Introduction: Clinical guidelines have recommended a 1-year trastuzumab regimen as standard care for early human epidermal growth factor receptor 2 (HER2)-positive breast cancer; however, this recommendation can have a dramatic impact on total drug expenditures in middle-income countries (MICs). We performed a cost-effectiveness analysis from the Iranian healthcare perspective to find an optimum duration of trastuzumab use in Iran., Method: We compared four treatment strategies comprising chemotherapy and varying durations of trastuzumab use (no trastuzumab, 6, 9 months, and 1 year), and a Markov model and probabilistic sensitivity analysis were used to estimate the costs and effects of the strategies. We then examined the cost effectiveness of the strategies at different willingness-to-pay (WTP) thresholds and ages at onset of treatment., Results: Incremental costs (versus no trastuzumab) were €8826 (6 months), €13,808 (9 months) and €18,588 (12 months), while incremental quality-adjusted life-years (QALYs) were 0.65 (6 months), 0.87 (9 months) and 1.14 (12 months). At a threshold of 3 × gross domestic product (GDP)/capita (€21,000/QALY) and for patients younger than 59 years, the 6-month protocol was most likely to be cost effective (probability of 42%). At a threshold of 4 × GDP/capita (€28,000/QALY), the 6-month and 1-year regimens were essentially equal in cost effectiveness (37 and 35%, respectively). At this WTP threshold, the 6-month and 1-year regimens were optimal strategies only for patients up to 66 and 44 years of age, respectively., Conclusion: In contrast to clinical guidelines, 6 months of trastuzumab may be the most cost-effective option for Iran. The lower absolute WTP threshold and lower life expectancy compared with high-income countries are two crucial parameters in the cost effectiveness of interventions in MICs. It is therefore necessary to strike a balance between maximum population health and maintaining affordability in these countries.

Background: The individual risk of recurrence in hormone receptor-positive primary breast cancer patients determines whether adjuvant endocrine therapy should be combined with chemotherapy. Clinicopathological parameters and molecular tests such as EndoPredict(®) (EPclin) can support decision making in patients with estrogen receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative cancer., Objective: Using a life-long Markov state transition model, we determined the health economic impact and incremental cost effectiveness of EPclin-based risk stratification in combination with clinical guidelines [German-S3, National Comprehensive Cancer Center Network (NCCN), and St. Gallen] to decide on chemotherapy use., Methods: Information on overall and metastasis-free survival came from Austrian Breast & Colorectal Cancer Study Group clinical trials 6/8 (n = 1,619) and published literature. Effectiveness was assessed as quality-adjusted life-years (QALYs). Costs (2010) were assessed from a German third-party payer perspective., Results: Lifetime costs per patient ranged from 28,268 (St.Gallen and EPclin) to 33,756 (NCCN). Due to an imperfect prognostic value and differences in chemotherapy use, strategies achieved between 13.165 QALYs (NCCN) and 13.173 QALYs (EPclin alone) per patient. Using German-S3 as reference, three strategies showed dominant results (St. Gallen and EPclin, German-S3 and EPclin, EPclin alone). Compared to German-S3, the addition of EPclin saved 3,388 and gained 0.002 QALYs per patient. Combining guidelines with EPclin remained preferable in sensitivity analysis., Conclusion: Our study suggests that molecular markers can be sensibly combined with clinical guidelines to determine the risk profile of adjuvant breast cancer patients. Compared with the current German best practice (German-S3), combinations of EPclin with the St. Gallen, German-S3 or NCCN guideline and EPclin alone were dominant from the perspective of the German healthcare system.

Grade IV glioma (glioblastoma) is one of the most common brain/central nervous system cancers. In 2005, the standard of care for adjuvant treatment was significantly changed with the approval of temozolomide. Carmustine wafers have also gained some popularity. Phase III trials are currently evaluating bevacizumab in conjunction with the standard temozolomide regimen. Despite these recent advances in pharmacotherapy, roughly two-thirds of patients do not survive longer than 2 years after diagnosis. Meanwhile, the costs of treatment are substantial. The goal of this study is to review the clinical, cost-of-illness, and cost-effectiveness literature relevant to treating glioblastoma. Estimates of the economic burden of glioblastoma within different healthcare systems were converted to 2013 US dollars. Temozolomide has demonstrated a 2.5-month increase in overall survival and a 1.9-month increase in progression-free survival, relative to radiotherapy alone. Carmustine wafers have also been shown to increase overall survival by 2.3 months, compared with placebo wafers. Cost-effectiveness studies of temozolomide have produced incremental cost-effectiveness ratios, adjusted to 2013 US dollars, with a range from US$73,586 per quality-adjusted life-year (QALY) (UK National Health Service perspective) to US$105,234 per QALY (US societal perspective). More research is needed to quantify the full societal burden of illness.

Background: Prior economic analysis that compared the 12-gene assay to published patterns of care predicted the assay would improve outcomes while lowering medical costs for stage II, T3, mismatch-repair-proficient (MMR-P) colon cancer patients. This study assessed the validity of those findings with real-world adjuvant chemotherapy (aCT) recommendations from the US third-party payer perspective., Methods: Costs and quality-adjusted life-years (QALYs) were estimated for stage II, T3, MMR-P colon cancer patients using guideline-compliant, state-transition probability estimation methods in a Markov model. A study of 141 patients from 17 sites in the Mayo Clinic Cancer Research Consortium provided aCT recommendations before and after knowledge of the 12-gene assay results. Progression and adverse events data with aCT regimens were based on published literature. Drug and administration costs for aCT were obtained from 2014 Medicare Fee Schedule. Sensitivity analyses evaluated the drivers and robustness of the primary outcomes., Results: After receiving the 12-gene assay results, physician recommendations in favor of aCT decreased 22 %; fluoropyrimidine monotherapy and FOLFOX recommendations each declined 11 %. Average per-patient drugs, administration, and adverse events costs decreased $US2,339, $US733, and $US3,211, respectively. Average total direct medical costs decreased $US991. Average patient well-being improved by 0.114 QALYs. Savings are expected to persist even if the cost of oxaliplatin drops by >75 % due to generic substitution., Conclusions: This study provides evidence that real-world changes in aCT recommendations due to the 12-gene assay are likely to reduce direct medical costs and improve well-being for stage II, T3, MMR-P colon cancer patients.

Background: Postoperative adjuvant chemotherapy with capecitabine and oxaliplatin was first recommended for resectable gastric cancer patients in the 2011 Chinese National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology: Gastric Cancer, but the economic influence of this therapy in China is unknown., Objective: The aim of the present study was to determine the cost-effectiveness of adjuvant chemotherapy with capecitabine and oxaliplatin after a gastrectomy with extended (D2) lymph-node dissection, compared with a D2 gastrectomy alone, for patients with stage II-IIIB gastric cancer., Methods: On the basis of data from the CLASSIC trial, a Markov model was created to determine economic and clinical data for patients in the chemotherapy and surgery group (CSG) and the surgery-only group (SOG). The costs, presented in 2010 US dollars and estimated from the perspective of the Chinese health-care system, were obtained from the published literature and the local health system. The utilities were based on published literature. Costs, life years (LYs), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICER) were estimated. A lifetime horizon and a 3 % annual discount rate were used. One-way and probabilistic sensitivity analyses were performed., Results: For the base case, the CSG compared with SOG would increase LYs and QALYs in a 3-, 5-, 10- or 30-year time horizon (except the QALYs at 3 or 5 years). In the short run (such as in 3 or 5 years), the medical costs would increase owing to adjuvant chemotherapy of capecitabine plus oxaliplatin after D2 gastrectomy, but in the long run the costs would decline. The ICERs suggested that the SOG was dominant at 3 or 5 years and the CSG was dominant at 10 or 30 years. The one-way sensitivity analysis showed that the utility of disease-free survival for 1-10 years for the SOG and the cost of oxaliplatin were the most influential parameters. The probabilistic sensitivity analysis predicted a 98.6 % likelihood that the ICER for the CSG would be less than US$13,527/QALY (three times the per capita gross domestic product of China)., Conclusion: For patients in China with resectable disease, our results suggest that adjuvant chemotherapy with capecitabine plus oxaliplatin after a D2 gastrectomy is cost-saving and dominant in the long run on the basis of a current clinical trial, compared with treatment with a D2 gastrectomy alone.

Background: A 21-gene recurrence score (RS) assay provides a method of guiding treatment decisions in women with early-stage breast cancer (ESBC). We investigated the cost effectiveness of using the RS assay versus current clinical practice (CCP) in post-menopausal women with estrogen- or progesterone-receptor-positive, one to three positive axillary lymph-node ESBC from the perspective of the Canadian public healthcare system., Methods: We developed a decision analytic model to project the lifetime clinical and economic consequences of ESBC. We assumed that the RS assay would classify patients among risk levels (low, intermediate and high) and corresponding adjuvant treatment regimens. The model was parameterized using 7-year follow-up data from the Manitoba Cancer Registry, cost data from Manitoba Health administrative databases and secondary sources. Costs are presented in 2012 Canadian dollars, and future costs and benefits were discounted at 5 %., Results: In the base case analysis, the RS assay compared with CCP led to an increase of 0.08 quality-adjusted life-year (QALY) and an increase in cost of Can$36.2 per person, resulting in an incremental cost-effectiveness ratio (ICER) of Can$464/QALY gained. The ICER was most sensitive to the proportion of women classified to intermediate risk by the RS assay who received adjuvant chemotherapy, and absolute risk of relapse among patients receiving the RS assay., Conclusions: The RS assay is likely to be cost effective in the Canadian healthcare system. Field evaluations of the assay in this patient population will help reduce uncertainty in clinical guidelines for intermediate-range RS-assay values and specific disease outcomes by the RS assay, which are important drivers of ICER.

Background and Objective: The increasing cost of chemotherapy is placing greater pressures on limited healthcare budgets. A potentially important, but often overlooked, aspect of chemotherapy is the cost associated with administration. This study aims to develop a better understanding of these costs, and in doing so, develop a model to estimate the comparative cost of administering alternative chemotherapy protocols for economic evaluation or local decision making., Methods: We identified the potential tasks and choices related to administering intravenous chemotherapy, grouped tasks according to anticipated resource use, and allocated costs to each task using data from an evidence-based collection of cancer protocols or from primary data collection. The resources were costed from a healthcare system perspective using standard data sources within Australia. The model was applied to alternative protocols used in the treatment of three different cancers: locally advanced and metastatic non-small-cell lung cancer, adjuvant colorectal cancer and adjuvant breast cancer., Results: For the three cancer types examined, the cost of completed administration ranged from 1274 Australian dollars ($A) to $A3015 (year 2009 values) for 13 different protocols potentially used for the initial treatment of locally advanced and metastatic non-small-cell lung cancer; $A5175-8445 for seven protocols for adjuvant colorectal cancer treatment; and $A1494-4074 for seven protocols for adjuvant breast cancer treatment., Conclusions: The results are of practical significance to those undertaking economic evaluations and to decision makers who use this information within the area of chemotherapy. The examples used suggest that administration costs per visit varied inversely with the number of visits. The results provide information where little has previously been available and may allow decisions about costs and resource allocation to be made with more certainty. Although our model uses costs from the public health system within an Australian state (New South Wales), it can be adapted for use in other jurisdictions.

Objective: A cost-effectiveness analysis of oral capecitabine versus intravenous bolus 5-fluorouracil/l-leucovorin (FU/LV) as adjuvant therapy in patients with stage 3 colon cancer was performed from a Japanese healthcare payer perspective., Methods: Adjuvant therapy comprised 24 weeks of treatment with either oral capecitabine 1250 mg/m(2) twice daily on days 1-14 of a 21-day cycle or intravenous bolus FU 500 mg/m(2) and LV 250 mg/m(2) weekly for 6 weeks of an 8-week cycle (Roswell Park regimen). The analysis comprised short-term (1 year after initiation of adjuvant therapy) and long-term (up to 15 years) components. The long-term analysis involved a three-state (disease-free, recurrence and death) Markov model. Estimates for transition probabilities, costs and utilities were derived from the X-ACT trial, a Japanese phase II trial, and other published sources. Cost estimates were considered from the perspective of a healthcare payer. Costs were expressed in Japanese Yen (yen), year 2007 values. A discount rate of 3% was applied to costs and outcomes. Cost effectiveness was expressed as a cost per QALY. The effects of uncertainty were explored through one-way and probabilistic sensitivity analyses., Results: In the 1-year analysis, direct costs were yen440,000 ($US4000) less per patient with capecitabine than with FU/LV. In the long-term analysis, differences between treatments in direct medical costs ranged from yen470,000 ($US4300) to yen580,000 ($US5300) depending on the time horizon used. Capecitabine was also projected to increase the number of QALYs compared with FU/LV. The sensitivity analysis suggested that the model outcome was robust. The probabilistic sensitivity analysis estimate of capecitabine being the dominant regimen was 96.6% at a zero willingness to pay. Direct costs remained lower with capecitabine if the price of generic LV was >OR=50% of the branded product., Conclusion: This analysis suggests that capecitabine improves health outcomes and lowers direct costs compared with bolus FU/LV (i.e. dominant treatment strategy) when used as adjuvant therapy in patients with stage 3 colon cancer in Japan.

An oncology trial compared four cycles of doxorubicin/cyclophosphamide (AC) with four cycles of docetaxel/cyclophosphamide (TC) in operable breast cancer patients (71% were diagnosed with hormone receptor positive and 48% with node-negative breast cancer). The objective of this study was to estimate the lifetime cost effectiveness of AC versus TC, from a Chinese healthcare provider perspective, based on a clinical trial. A lifetime cost-effectiveness analysis was performed using a Markov model. Events rates and utilities in the Markov model were derived from published papers. Data on cost of breast cancer care were obtained from the Second Xiangya Hospital of Central South University, Changsha, PR China. One-way sensitivity analysis and probabilistic sensitivity analysis were undertaken. Cost estimates were valued in Chinese yuan (Y), year 2008 values. All costs and outcomes were discounted at 3% per annum. Patients receiving TC gained 14.45 QALYs, 0.41 QALYs more than patients receiving AC. The lifetime costs of patients receiving TC were Y93 511, Y10 116 more than that of AC patients. The incremental cost-effectiveness ratios were Y26 742 per life-year gained ( pound 2719.8 per year) and Y24 305 per QALY gained ( pound2471.9 per QALY). The most sensitive parameter in the model was the cost of primary cancer treatments in the TC arm. At a threshold willingness to pay of Y86 514 per QALY, the probability of TC being cost effective was 90%. Our model suggests that TC may be considered cost effective from a Chinese healthcare provider perspective, according to the threshold defined by the WHO.

Objective: To assess the cost effectiveness of the addition of folinic acid to fluorouracil plus levamisole in patients with colon cancer from the perspective of the German Social Health Insurance., Study Design and Methods: Patients with International Union Against Cancer (Union International Contre Cancer; UICC) II/T(4) or UICC III colon cancer enrolled in an open-label randomised clinical trial in Germany (Forschungsgruppe Onkologie Gastrointestinaler Tumoren-1 [FOGT-1]) received either fluorouracil plus levamisole (A, standard) or fluorouracil plus levamisole and folinic acid (B) for 12 months as adjuvant chemotherapy after curative intended surgery. Outcome measures for economic evaluation were disease-free life-years gained (df-LYG) and overall life-years gained (LYG) derived from the respective Kaplan-Meier survival curves. Direct medical costs from the perspective of the German Social Health Insurance were estimated retrospectively (2000 values) and incremental cost-effectiveness ratios (ICERs) were calculated. A Markov model was used to project the trial results beyond 5 years for the patients' remaining life expectancy., Results: Adding folinic acid to the fluorouracil/levamisole regimen results in an increase in time to progression and survival in patients with locally advanced colon cancer. Within the trial period of 5 years ICERs (B versus A) were 33,008 Euro per df-LYG and 51,225 Euro per LYG (costs and effects discounted at 5%). The Markov model yielded ICERs of 11,176 Euro per df-LYG and 11,020 Euro per LYG (costs and effects discounted at 5%). The model was robust for variations of key variables in the sensitivity analysis., Conclusions: Results of this cost-effectiveness analysis suggest that the addition of folinic acid offers clinical benefits at additional costs which are likely to be acceptable for decision makers in the long term. Cost-effectiveness ratios calculated within the clinical trial period were just above 50,000 Euro/LYG. Because treatment benefits, i.e. prolonged survival, are sustained beyond 5 years whereas incremental costs are mainly incurred in the first year, results of the Markov model yielded cost-effectiveness ratios that compare more favourably with other published ICERs.

Cancer treatment accounts for a large proportion of healthcare costs. Often, new treatment modalities provide benefits, but at high costs. The impression that cancer treatment is expensive is enhanced by publicity surrounding treatments like bone marrow transplantation. There is a need to evaluate costs of different treatment approaches and to address the cost utility of cancer treatment in general compared with therapies for other conditions. Breast cancer can serve as a good model for economic evaluation of cancer treatment because of the broad range of treatment options and objectives it encompasses, and also because well defined benefits can be achieved. The cost utility of contemporary adjuvant therapy strategies, specifically chemotherapy in premenopausal women and hormonal treatment in estrogen-receptor (ER) positive pre- as well as postmenopausal women, seems favourable. Cost-utility ratios {cost per quality-adjusted life-year (QALY) gained} range from $US4000 to $US10 000. However, hormonal treatment in ER-negative women may be associated with cost-per-QALY ratios of $US50 000 to $US200 000. So far there are no published cost-utility analyses of neo-adjuvant therapy or adjuvant bone marrow transplantation as the long term effects of these treatment options are undefined. Few data exist on cost utilities of systemic drug treatment in advanced breast cancer, although drugs may account for only a moderate part of the total treatment and caring costs. Bone marrow transplantation in patients with metastatic breast cancer costs about $US100 000 per QALY, which is expensive.

Extensive clinical experience, summarised in the recent overview of the Early Breast Cancer Trialists' Collaborative Group (EBCTCG), have confirmed that tamoxifen reduces the rate of both disease recurrence and mortality when administered as adjuvant therapy in women with early breast cancer. Tamoxifen is now established as the preferred adjuvant agent in postmenopausal women; in particular, patients with node-positive, estrogen receptor-positive breast cancer have the most to gain from tamoxifen therapy. Data from a decision-analysis model indicated that tamoxifen monotherapy had a cost-utility ration {$US6000 per additional quality-adjusted life-year (QALY), in 1989 dollars} 5 to 6 times lower than that cited as the cost-acceptability cut-off point in the US. While tamoxifen monotherapy is effective in postmenopausal women, the EBCTCG overview findings indicate that a combined regimen of tamoxifen and antineoplastic chemotherapy has superior efficacy in the same patient group. An issue of current interest is whether the added benefit offered by such a regimen can be justified in terms of added toxicity and cost. Data from a decision-analysis model indicate that combined therapy has a high incremental cost-utility ratio ($US58 000 per additional QALY, in 1989 dollars) compared with no therapy in postmenopausal women. However, the quality-of-life measures TWiST (Time Without Symptoms and Toxicity) and Q-TWiST (quality-adjusted TWiST) indicate that the early toxicity associated with a combined regimen appears to be justified given the superior long term benefits. Patient preference data from 1 study further indicate that the degree of benefit offered by a combined regimen would be acceptable to the majority (73%) of patients. Other areas where pharmacoeconomic analyses may help define more closely the optimal use of adjuvant tamoxifen is in patients at low risk of developing metastatic disease and in determining the optimal duration of therapy. Both areas require further clinical data. In conclusion, tamoxifen adjuvant monotherapy has a low cost-utility ratio in postmenopausal women with node-positive, estrogen receptor-positive breast cancer. Combined therapy in the same patient group has a high cost-utility ratio compared with no therapy but quality-of-life and patient preference data suggest that the costs may be justified. Firm conclusions relating to the use of the drug in other patient subgroups and the optimal duration of therapy await further research.