A probabilistic threshold analysis of metformin (Met) with enzalutamide (Enza) to determine the cost and added efficacy needed to make such a combination theray cost-effective (CE).

2017 ◽  
Vol 35 (6_suppl) ◽  
pp. e577-e577
Author(s):  
Jordan Hill ◽  
Peter Venner ◽  
Mike Paulden ◽  
Christopher McCabe ◽  
Scott A. North ◽  
...  

e577 Background: Enza has become known for its' effectiveness in treating patients with metastatic castrate-resistant prostate cancer. However, Enza is expensive (~$3175/month) and unlikely to be CE at any willingness to pay (WTP) threshold from $0-$125 000/quality-adjusted life years (QALYs). Met is inexpensive (~$8.00/month) and recently two large population-based studies of prostate cancer (PCa) demonstrated that diabetics taking Met had improved PCa specific and overall survival compared to those not taking Met. As a result we theorized that there must be a cost of Enza and a specific added effect Met could provide that would make Met a CE adjuvant therapy to Enza even though it is not currently used as such. Methods: We constructed a Markov-based decision analytic model and performed a probabilistic threshold analysis to narrow in on several combinations of costs of Enza and added efficacies needed to make Enza + Met a CE combination therapy at different WTP thresholds. Costs, utilities, and transition probabilities were derived from existing literature. Effectiveness was measured using QALYs. Costs and QALYs were considered over a lifetime horizon and discounted at 5% per annum. Results: At a WTP threshold of $50 000/QALY Enza + Met is unlikely to be CE no matter the cost decrement or added efficacy applied to the model. At a WTP threshold of $75 000/QALY Met is also unlikely to be CE unless the price of Enza could drop to $1934/month and Met could also add 1% to the efficacy of Enza. At Enza's current cost and a WTP threshold of $100 000, Enza + Met could be CE barring Met adds 0.621% to the number of patients still on treatment at 36 months. Conclusions: Enza + Met is unlikely to be CE at WTP thresholds of $50 000/QALY or $75 000/QALY unless there is a significant price drop for Enza; these results make sense because a therapy that is considered not CE at these WTP thresholds by itself is unlikely to be CE with a theoretical adjuvant therapy that would hold a patient on such a treatment for even longer. Our results show that the only way for Enza + Met to be CE is through the theoretical overall survival benefit of Met as the price of Enza is unlikely to drop any time soon.

2016 ◽  
Vol 34 (2_suppl) ◽  
pp. 340-340
Author(s):  
Jordan Hill ◽  
Mike Paulden ◽  
Christopher McCabe ◽  
Peter Venner ◽  
Brita Lavender Danielson ◽  
...  

340 Background: Several new therapies have changed the landscape of prostate cancer (PCa) treatment, primarily due to their effectiveness in treating patients with mCRPC. Enza has garnered much attention, but is relatively expensive (~$3175/month). Met is less expensive (~$8.00/month) and has been used for decades to treat patients with non-insulin dependent diabetes. Two recent large population-based studies of PCa have demonstrated that diabetics taking Met had improved PCa specific and overall survival compared to those not taking Met. As a result, we hypothesized that Met has the potential to be a cost-effective adjunct therapy to Enza, although it is not currently used as such. Methods: We constructed a Markov-based decision analytic model to compare the cost-effectiveness of Enza alone versus Enza combined with Met. Through expert elicitation, we assumed that adding Met to Enza increases its efficacy by 15%. All other costs, utilities, and transition probabilities were derived from existing literature or expert elicitation. Effectiveness was measured using quality-adjusted life years (QALYs). Costs and QALYs were considered over a lifetime horizon and discounted at 5% per annum. Cost-effectiveness was considered using a willingness to pay threshold of $50 000/QALY. Results: Adding Met to Enza increases expected lifetime costs per patient by $83 651, and improves the expected effectiveness of treatment by 3.74 QALYs, compared to Enza alone. The incremental cost-effectiveness ratio is $22 374/QALY. Accounting for parameter uncertainty, adding Met to Enza has a 72% probability of being cost-effective. Conclusions: Although Met is not currently used as an adjunct therapy to Enza, doing so would likely be cost-effective provided it is as effective as we have assumed in our model. Additionally, our results indicate that the combination of Enza and Met could be among the most cost effective interventions in oncology. However, given the uncertainty around the effectiveness of such an adjunct therapy, our results support the need for further clinical trials to provide more robust evidence of the effectiveness of such a combination therapy in clinical practice.


2020 ◽  
pp. neurintsurg-2020-016765
Author(s):  
Mihir Khunte ◽  
Xiao Wu ◽  
Sam Payabvash ◽  
Chengcheng Zhu ◽  
Charles Matouk ◽  
...  

BackgroundThe cost-effectiveness of endovascular thrombectomy (EVT) in patients with acute ischemic stroke due to M2 branch occlusion remains uncertain.ObjectiveTo evaluate the cost-effectiveness of EVT compared with medical management in patients with acute stroke presenting with M2 occlusion using a decision-analytic model.MethodsA decision-analytic study was performed with Markov modeling to estimate the lifetime quality-adjusted life years and associated costs of EVT-treated patients compared with no-EVT/medical management. The study was performed over a lifetime horizon with a societal perspective in the Unites States setting. Base case, one-way, two-way, and probabilistic sensitivity analyses were performed.ResultsEVT was the long-term cost-effective strategy in 93.37% of the iterations in the probabilistic sensitivity analysis, and resulted in difference in health benefit of 1.66 QALYs in the 65-year-old age groups, equivalent to 606 days in perfect health. Varying the outcomes after both strategies shows that EVT was more cost-effective when the probability of good outcome after EVT was only 4–6% higher relative to medical management in clinically likely scenarios. EVT remained cost-effective even when its cost exceeded US$200 000 (threshold was US$209 111). EVT was even more cost-effective for 55-year-olds than for 65-year-old patients.ConclusionOur study suggests that EVT is cost-effective for treatment of acute M2 branch occlusions. Faster and improved reperfusion techniques would increase the relative cost-effectiveness of EVT even further in these patients.


Author(s):  
Stan Wijn ◽  
Mayke Hentschel ◽  
Andy Beynon ◽  
Henricus Kunst ◽  
Maroeska Rovers

Objectives: To determine the cost-effectiveness of auditory brainstem response prior to MRI (ABR-MRI) compared to standalone MRI to diagnose vestibular schwannoma. Design: A state transition decision-analytic model was developed to simulate costs and effects (quality-adjusted life years) for both treatment strategies for patients suspected of a vestibular schwannoma. Model input was derived from literature, hospital databases, and expert opinions. Scenario and sensitivity analyses addressed model uncertainty. Results: Over a lifetime horizon, ABR-MRI resulted in a limited cost-saving of \euro68 or \euro98 per patient (dependent on MRI sequence) and a health loss of 0.005 QALYs over standalone MRI. ABR-MRI, however, did miss patients with other important pathology (2% of the population) that would have been detected when using standalone MRI. Calculating the incremental cost-effectiveness ratio resulted in \euro14,203 or \euro19,550 saved per lost QALY if ABR-MRI was used instead of standalone MRI. The results were sensitive to the detection rate of vestibular schwannoma and health-related quality of life of missed patients. Conclusion: The cost-saving with ABR-MRI does not seem to outweigh the number of missed patients with VS and other important pathologies that would have been detected when using standalone MRI.


2006 ◽  
Vol 24 (12) ◽  
pp. 1868-1876 ◽  
Author(s):  
Urs E. Studer ◽  
Peter Whelan ◽  
Walter Albrecht ◽  
Jacques Casselman ◽  
Theo de Reijke ◽  
...  

Purpose This study (EORTC 30891) attempted to demonstrate equivalent overall survival in patients with localized prostate cancer not suitable for local curative treatment treated with immediate or deferred androgen ablation. Patients and Methods We randomly assigned 985 patients with newly diagnosed prostate cancer T0-4 N0-2 M0 to receive androgen deprivation either immediately (n = 493) or on symptomatic disease progression or occurrence of serious complications (n = 492). Results Baseline characteristics were well balanced in the two groups. Median age was 73 years (range, 52 to 81). At a median follow-up of 7.8 years, 541 of 985 patients had died, mostly of prostate cancer (n = 193) or cardiovascular disease (n = 185). The overall survival hazard ratio was 1.25 (95% CI, 1.05 to 1.48; noninferiority P > .1) favoring immediate treatment, seemingly due to fewer deaths of nonprostatic cancer causes (P = .06). The time from randomization to progression of hormone refractory disease did not differ significantly, nor did prostate-cancer specific survival. The median time to the start of deferred treatment after study entry was 7 years. In this group 126 patients (25.6%) died without ever needing treatment (44% of the deaths in this arm). Conclusion Immediate androgen deprivation resulted in a modest but statistically significant increase in overall survival but no significant difference in prostate cancer mortality or symptom-free survival. This must be weighed on an individual basis against the adverse effects of life-long androgen deprivation, which may be avoided in a substantial number of patients with a deferred treatment policy.


2012 ◽  
Vol 30 (15_suppl) ◽  
pp. e15107-e15107
Author(s):  
Akhil Chopra ◽  
Stefan Gluck ◽  
Alberto J. Montero ◽  
Kiran Kumar Venkata Raja Avancha ◽  
Gilberto Lopes

e15107 Background: Treatment with abiraterone improves overall survival (OS), time to prostate-specific antigen progression and radiologic progression-free survival when added to prednisone and best supportive care in patients with advanced castrate-resistant prostate cancer (CRPC) who previously received docetaxel. Little is known about its cost-effectiveness in the United States. Methods: In order to raise awareness of pharmacoeconomics concepts and inform policy-makers in the US, this study aimed to assess the cost-effectiveness of abiraterone in the treatment of advanced CRPC patients, from a payer perspective. We created a decision-analytical model using clinical data from the pivotal phase III trial: COU-AA-301. Health utilities were derived from the available literature. Costs for drug acquisition, physician visits and laboratory tests were obtained from the Center for Medicare Services Drug Payment Table and Physician Fee Schedule and are represented in 2011 US dollars. Life-years saved (LY), Quality-adjusted life years (QALY) and Incremental Cost Effectiveness Ratio (ICER) were calculated using updated survival data presented at the 2011 ASCO meeting. Other main scenarios used the original median survival data published in the New England Journal of Medicine article and adjusted median OS to represent an overall survival hazard ratio of .66. Sensitivity analyses were performed using the confidence intervals for median OS in the pivotal study as well as by halving or doubling all other model inputs. No discounting was used due to the short time-horizon. Results: Abiraterone added 0.38 LY and 0.30 QALY with an incremental cost of $39,320 and therefore a cost of $102,600/LY and an ICER of $129,000/QALY. The main drivers of the model were drug acquisition cost, median OS, and health utility values. The results of the model were robust in different scenarios and sensitivity analyses. Conclusions: Using commonly accepted willingness-to-pay thresholds, such as those suggested by the World Health Organization, treatment of patients with advanced CRPC patients with abiraterone is likely to be cost-effective in the US.


2021 ◽  
Vol 11 ◽  
Author(s):  
Weiting Liao ◽  
Huiqiong Xu ◽  
David Hutton ◽  
Qiuji Wu ◽  
Kexun Zhou ◽  
...  

BackgroundThe INVICTUS trial assessed the efficacy and safety of ripretinib compared with placebo in the management of advanced gastrointestinal stromal tumors.MethodWe used a Markov model with three health states: progression-free disease, progression disease and death. We parameterized the model from time-to-event data (progression-free survival, overall survival) of ripretinib and placebo arms in the INVICTUS trial and extrapolated to a patient’s lifetime horizon. Estimates of health state utilities and costs were based on clinical trial data and the published literature. The outcomes of this model were measured in quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratios (ICERs). Uncertainty was tested via univariate and probabilistic sensitivity analyses.ResultsThe base-case model projected improved outcomes (by 0.29 QALYs) and additional costs (by $70,251) and yielded an ICER of $244,010/QALY gained for ripretinib versus placebo. The results were most sensitive to progression rates, the price of ripretinib, and health state utilities. The ICER was most sensitive to overall survival. When overall survival in the placebo group was lower, the ICER dropped to $127,399/QALY. The ICER dropped to $150,000/QALY when the monthly cost of ripretinib decreased to $14,057. Probabilistic sensitivity analyses revealed that ripretinib was the cost-effective therapy in 41.1% of simulations at the willingness-to-pay (WTP) threshold of $150,000.ConclusionAs the fourth- or further-line therapy in advanced gastrointestinal stromal tumors, ripretinib is not cost-effective in the US. Ripretinib would achieve its cost-effectiveness with a price discount of 56% given the present effectiveness.


10.36469/9808 ◽  
2017 ◽  
Vol 5 (2) ◽  
pp. 162-174
Author(s):  
María Teresa Gómez-Casares ◽  
Juan Carlos Hernández-Boluda ◽  
Antonio Jiménez-Velasco ◽  
Joaquin Martínez-López ◽  
María Giovanna Ferrario ◽  
...  

Introduction: Primary myelofibrosis (MF) is a rare hematologic disease belonging to the group of Philadelphia-negative chronic myeloproliferative neoplasms. Identification of the Janus Kinase (JAK) gene mutations inaugurated a new era in the targeted therapy of myeloproliferative diseases. Ruxolitinib is the first JAK1/JAK2 inhibitor specifically approved for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis. The objective of this study was to assess the cost-effectiveness of ruxolitinib vs best available therapy (BAT) in MF patients in Spain. Methods: A decision-tree and Markov model were adapted to the Spanish setting to assess the cost-effectiveness of ruxolitinib vs. BAT on a lifetime horizon (≤15 years) from the societal perspective, while healthcare system perspective was included in the one-way sensitivity analysis. The population was assumed to be similar to that of the COMFORT-II clinical trial (CT), which was also the source of treatment efficacy data. BAT composition was derived from the same CT and validated with Spanish experts. Utilities were derived from the COMFORT-I CT. Costs included treatment, management, hospitalizations, emergency and outpatient visits, as well as adverse events and end-of-life costs. Additionally, costs associated to productivity loss were taken into account. Resource use was validated with experts and costs were extracted from Spanish sources. A probabilistic sensitivity analysis was also performed to evaluate the consistency of the results under the uncertainty or variability of the input data. Results: Patients on ruxolitinib accumulated 6.1 life years gained (LYGs), resulting in 73% extra life-years compared to patients treated with BAT (3.5LYs gained). Ruxolitinib provided 4.4 quality-adjusted life years (QALYs), with a 99% improvement compared to BAT (2.2 QALYs). This analysis gave an incremental cost of €47 199 per LYG and an incremental cost of €55 616 per QALY gained from the societal perspective. Conclusions: Ruxolitinib would be cost-effective in Spain according to the end-of-life criteria defined by the NICE and commonly referred for Spain (cost-effectiveness threshold of €61 500/QALY), in line with results published for other European countries.


2020 ◽  
Vol 7 (Supplement_1) ◽  
pp. S57-S58
Author(s):  
Kelsey Olmack ◽  
Curtis D Collins

Abstract Background In the hospital setting, cefepime (CFP) and piperacillin/tazobactam (PTZ) are among the most commonly utilized antipseudomonal agents in the empiric treatment of nosocomial and healthcare-associated infections. Institutional preference of CFP or PTZ as the preferred antipseudomonal antibiotic varies. Recent literature suggests each may be associated with increased rates of harmful adverse effects including Clostridiodes difficile infection (CDI) and acute kidney injury (AKI). The objective of this study is to perform a pharmacoeconomic analysis comparing CFP versus PTZ for empiric antibiotic treatment in patients where Pseudomonas aeruginosa is a concern. Methods We performed a cost-utility analysis comparing CFP and PTZ for empiric utilization in the hospital setting by creating a decision analytic model from the hospital perspective. Model variables were populated utilizing published clinical and economic data including incidence of AKI and CDI, their associated costs and mortality, and the cost of antibiotic therapy. Secondary and univariate sensitivity analyses tested the impact of model uncertainties and the robustness of our model. A willingness to pay (WTP) threshold of $0 was utilized. Results Results of our base-case model predicted that the use of CFP dominated PTZ as empiric utilization was less expensive ($7690 vs. $9331) and associated with a higher quality-adjusted life-years (QALY) (0.9193 vs. 0.9191) compared to the use of PTZ. Several variables had the potential to impact base case results. PTZ became cost-effective at our WTP threshold if CFP nephrotoxicity rates increased to 17.3%, the PTZ nephrotoxicity decreased to 28.5%, or if the cost of nephrotoxicity was less than $17,457. No other model variables, including incidence of CDI, impacted base case results. Sensitivity Analysis on Cefepime Clostridioides difficile Infection Incidence and Piperacillin/tazobactam Nephrotoxicity Conclusion Results of our model showed that CFP dominated PTZ for the empiric treatment of nosocomial infections. The model was sensitive to variation in CFP and PTZ nephrotoxicity rates. Disclosures All Authors: No reported disclosures


2020 ◽  
Author(s):  
Zhi Peng ◽  
Xingduo Hou ◽  
Yangmu Huang ◽  
Tong Xie ◽  
Xinyang Hua

Abstract Background: In this study, we analyze the cost-effectiveness of fruquintinib as third-line treatment for patients with metastatic colorectal cancer in China, especially after a recent price drop suggested by the National Healthcare Security Administration. Methods: A Markov model was developed to investigate the cost-effectiveness of fruquintinib compared to placebo among patients with metastatic colorectal cancer. Effectiveness was measured in quality-adjusted life years (QALY). The Chinese healthcare payer’s perspective was considered with a lifetime horizon, including direct medical cost (2019 US dollars [USD]). A willing‐to‐pay threshold was set at USD 27,130/QALY, which is three times the gross domestic product (GDP) per capita. We examined the robustness of the model in one-way and probabilistic sensitivity analysis.Results: Fruquintinib was associated with better health outcomes than placebo (0.640 vs 0.478 QALYs) with a higher cost (USD 20750.9 vs USD 12042.2), resulting in an incremental cost-effectiveness ratio (ICER) of USD 53508.7 per QALY. This ICER is 25% lower than the one calculated before the price drop (USD 70952.6 per QALY).Conclusion: After the price negotiation, the drug becomes cheaper and the ICER is lower, but the drug is still not cost effective under the standard of 3 times GDP willing‐to‐pay threshold. For patients with metastatic colorectal cancer in China, fruquintinib is not a cost-effective option under the current circumstances in China.


BMC Cancer ◽  
2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Zhi Peng ◽  
Xingduo Hou ◽  
Yangmu Huang ◽  
Tong Xie ◽  
Xinyang Hua

Abstract Background In this study, we analyze the cost-effectiveness of fruquintinib as third-line treatment for patients with metastatic colorectal cancer in China, especially after a recent price drop suggested by the National Healthcare Security Administration. Methods A Markov model was developed to investigate the cost-effectiveness of fruquintinib compared to placebo among patients with metastatic colorectal cancer. Effectiveness was measured in quality-adjusted life years (QALY). The Chinese healthcare payer’s perspective was considered with a lifetime horizon, including direct medical cost (2019 US dollars [USD]). A willing-to-pay threshold was set at USD 27,130/QALY, which is three times the gross domestic product (GDP) per capita. We examined the robustness of the model in one-way and probabilistic sensitivity analysis. Results Fruquintinib was associated with better health outcomes than placebo (0.640 vs 0.478 QALYs) with a higher cost (USD 20750.9 vs USD 12042.2), resulting in an incremental cost-effectiveness ratio (ICER) of USD 53508.7 per QALY. This ICER is 25% lower than the one calculated before the price drop (USD 70952.6 per QALY). Conclusion After the price negotiation, the drug becomes cheaper and the ICER is lower, but the drug is still not cost effective under the standard of 3 times GDP willing-to-pay threshold. For patients with metastatic colorectal cancer in China, fruquintinib is not a cost-effective option under the current circumstances in China.


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