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Dirksen, Carmen D., André J. H. A. Ament, Eddy M. M. Adang, Geerard L. Beets, Peter M. N. Y. H. Go, Cor G. M. I. Baeten, and Gauke Kootstra. "Cost-Effectiveness of Open Versus Laparoscopic Repair for Primary Inguinal Hernia." International Journal of Technology Assessment in Health Care 14, no. 3 (1998): 472–83. http://dx.doi.org/10.1017/s0266462300011454.
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AbstractA cost-effectiveness (CE) analysis was performed of Bassini versus laparoscopic repair for primary inguinal hernia. Incremental costs per 1 -year recurrence-free patient were calculated for the societal and hospital perspective. From the hospital perspective, the incremental CE ratio of laparoscopic repair is 5.348 guilders. From the societal perspective, laparoscopic repair is both less costly and more effective than Bassini repair. Results were sensitive to assumptions about recurrence rates, laparoscopic operating time, and return to work. Laparoscopic repair should replace Bassini repair in order to benefit society. From the hospital perspective, the decision to accept laparoscopic repair depends on the willingness to pay.
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Hernández, Miguel Angel Negrín, Francisco José Vázquez-Polo, Francisco Javier Girón González-Torre, and Elías Moreno Bas. "Complementing the net benefit approach: A new framework for Bayesian cost-effectiveness analysis." International Journal of Technology Assessment in Health Care 25, no. 4 (October 2009): 537–45. http://dx.doi.org/10.1017/s0266462309990444.
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Objectives: The aim of cost-effectiveness analysis is to maximize health benefits from a given budget, taking a societal perspective. Consequently, the comparison of alternative treatments or technologies is solely based on their expected effectiveness and cost. However, the expectation, or mean, poses important limitations as it might be a poor summary of the underlying distribution, for instance when the effectiveness is a categorical variable, or when the distributions of either effectiveness or cost present a high degree of asymmetry. Clinical variables often present these characteristics.Methods: In this study, we present a framework for cost-effectiveness analysis based on the whole posterior distribution of effectiveness and cost.Results: An application with real data is included to illustrate the analysis. Decision-making measures such as the incremental cost-effectiveness ratio, incremental net-benefit, and cost-effectiveness acceptability curves, can also be defined under the new framework.Conclusions: This framework overcomes limitations of the mean and offers complementary information for the decision maker.
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Chen, Weiwei, Sanghamitra M. Misra, Fangjun Zhou, Leila C. Sahni, Julie A. Boom, and Mark Messonnier. "Evaluating Partial Series Childhood Vaccination Services in a Mobile Clinic Setting." Clinical Pediatrics 59, no. 7 (February 28, 2020): 706–15. http://dx.doi.org/10.1177/0009922820908586.
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This study aims to evaluate the cost-benefit of vaccination services, mostly partial series administration, provided by a mobile clinic program (MCP) in Houston for children of transient and low-income families. The study included 469 patients who visited the mobile clinics on regular service days in 2 study periods in 2014 and 836 patients who attended vaccination events in the summer of 2014. The benefit of partial series vaccination was estimated based on vaccine efficacy/effectiveness data. Our conservative cost-benefit estimates show that, compared with office-based settings, every dollar spent on vaccination by the MCP would result in $0.9 societal cost averted as an incremental benefit in regular service days and $3.7 during vaccination-only events. To further improve the cost-benefit of vaccination services in the MCP, decision-makers and stakeholders may consider improving work efficiency during regular service days or hosting more vaccination events.
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Smith, Kenneth, Jacques Cornuz, Mark Roberts, and Drahomir Aujesky. "Cost-effectiveness of low-molecular-weight heparin for secondary prophylaxis of cancer-related venous thromboembolism." Thrombosis and Haemostasis 93, no. 03 (2005): 592–99. http://dx.doi.org/10.1160/th04-11-0767.
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SummaryAlthough extended secondary prophylaxis with low-molecular-weight heparin was recently shown to be more effective than warfarin for cancer-related venous thromboembolism, its cost-effectiveness compared to traditional prophylaxis with warfarin is uncertain. We built a decision analytic model to evaluate the clinical and economic outcomes of a 6-month course of low-molecular-weight heparin or warfarin therapy in 65-year-old patients with cancer-related venous thromboembolism. We used probability estimates and utilities reported in the literature and published cost data. Using a US societal perspective, we compared strategies based on quality-adjusted life-years (QALYs) and lifetime costs. The incremental cost-effectiveness ratio of low-molecular-weight heparin compared with warfarin was $149, 865/QALY. Low-molecular-weight heparin yielded a quality-adjusted life expectancy of 1.097 QALYs at the cost of $15, 329. Overall, 46% ($7108) of the total costs associated with low-molecular-weight heparin were attributable to pharmacy costs. Although the low-molecular-weigh heparin strategy achieved a higher incremental quality-adjusted life expectancy than the warfarin strategy (difference of 0.051 QALYs), this clinical benefit was offset by a substantial cost increment of $7,609. Cost-effectiveness results were sensitive to variation of the early mortality risks associated with low-molecular-weight heparin and warfarin and the pharmacy costs for low-molecular-weight heparin. Based on the best available evidence, secondary prophylaxis with low-molecular-weight heparin is more effective than warfarin for cancer-related venous thromboembolism. However, because of the substantial pharmacy costs of extended low-molecular-weight heparin prophylaxis in the US, this treatment is relatively expensive compared with warfarin.
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Mattar, Andre, Guilherme Ribeiro Fonseca, Murilo Barato Agudo Romão, Jorge Yoshinori Shida, Debbie M. Jakubowski, Calvin Y. Chao, Sergio C. Oliveira, and Luiz Henrique Gebrim. "Economic evaluation of the oncotype DX test for hormone receptor positive (HR+) early-stage breast cancer (BC) from the Brazilian societal perspective." Journal of Clinical Oncology 38, no. 15_suppl (May 20, 2020): e19380-e19380. http://dx.doi.org/10.1200/jco.2020.38.15_suppl.e19380.
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e19380 Background: Selecting appropriate patients for AC (adjuvant chemotherapy) remains an important issue in BC treatment. Although AC improves clinical outcomes toxicity and economic burden is substantial. The Oncotype DX test identifies high-risk patients likely to benefit from AC who otherwise might not be identified through standard parameters (SP), and low-risk patients unlikely to benefit from AC, avoiding toxicities and inherent risks. This study estimated the incremental cost-effectiveness ratio and budget impact (BI) of Oncotype DX testing from the perspective of the Brazilian Public Health System. Methods: A Markov transitional state model was developed with 3 states: recurrence free, distant recurrence, and death. The model compared the scenario in which patients are screened by SP with a proposed scenario with Oncotype DX testing. Changes in therapeutic recommendations and cost of treatment were obtained from a prospective clinical survey at Pérola Byington Hospital. Additional data was obtained from literature. As a societal perspective analysis, medical costs (test, AC, and adverse events), costs of productivity loss, transportation and employment leave were considered. Population was estimated from BC incidence, proportion of early stage cases, and HR expression. An incremental proportion of 10% per year of patients using Oncotype DX testing was assumed. BI analysis had a 5-year horizon and cost-effectiveness a lifetime horizon (5% annual discount). Results: Oncotype DX results as identifier of a subgroup at higher risk of relapse and greater benefit with AC was dominant over SP. Oncotype DX testing resulted in clinical benefits in terms of life-years gained (0.62) and quality-adjusted life years (0.54), related to lower incidence of distant recurrence and use of AC, both of which greatly impacted quality of life. Testing resulted in economic benefits, with lower average cost per patient (−BRL 3,855). Incorporation of Oncotype DX testing resulted in potential savings reaching BRL 107 million in the 5th year stemming from the decrease in AC and consequent decrease in indirect costs. Conclusions: Patients with HR+, HER2− early stage BC may present different risks of relapse and likelihoods of benefiting from AC. With high clinical impact for patients and high economic impact for the health system, a tool that safely and accurately identifies the subgroup of patients who really needs AC is essential. Oncotype DX test incorporation in the Brazilian Public Health System should be considered.
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Gottinger, H. W. "Assessment of Social Value in the Allocation of CT Scanners – A Case Study." Methods of Information in Medicine 25, no. 02 (April 1986): 101–8. http://dx.doi.org/10.1055/s-0038-1635453.
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SummaryThis paper describes a case study on the application of decision analytic procedures to the allocation of computer tomography in a given metropolitan health service area, the Munich Metropolitan Area. The analysis could be used as a decision-aiding and decision-supporting instrument for government regulators in making rational resource allocation decisions as to locating high-cost, high-technology medical equipment. Explicit value judgments, reflecting the monetary equivalent of the different categories of benefit, are introduced to facilitate the comparison between decision options. The difference between the (incremental) benefits, measured in monetary terms, and the (incremental) costs is called the net social value and used as the overall decision criterion.Any alternative with positive net social value is judged economically justifiable, and the alternative with the greatest net social value - subject to its stability as to various sensitivity tests on input parameters - is judged to be most attractive from a societal viewpoint.
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Schulz, Claudia, Gisela Büchele, Raphael S. Peter, Dietrich Rothenbacher, Christian Brettschneider, Ulrich C. Liener, Clemens Becker, Kilian Rapp, and Hans-Helmut König. "Health-economic evaluation of collaborative orthogeriatric care for patients with a hip fracture in Germany: a retrospective cohort study using health and long-term care insurance claims data." European Journal of Health Economics 22, no. 6 (April 4, 2021): 873–85. http://dx.doi.org/10.1007/s10198-021-01295-z.
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Abstract Background Evidence suggests benefits of orthogeriatric co-management (OGCM) for hip fracture patients. Yet, evidence on cost-effectiveness is limited and based on small datasets. The aim of our study was to conduct an economic evaluation of the German OGCM for geriatric hip fracture patients. Methods This retrospective cohort study was based on German health and long-term care insurance data. Individuals were 80 years and older, sustained a hip fracture in 2014, and were treated in hospitals providing OGCM (OGCM group) or standard care (control group). Health care costs from payer and societal perspective, life years gained (LYG) and cost-effectiveness were investigated within 1 year. We applied weighted gamma and two-part models, and entropy balancing to account for the lack of randomisation. We calculated incremental cost-effectiveness ratios (ICER) and employed the net-benefit approach to construct cost-effectiveness acceptability curves. Results 14,005 patients were treated in OGCM, and 10,512 in standard care hospitals. Total average health care costs per patient were higher in the OGCM group: €1181.53 (p < 0.001) from payer perspective, and €1408.21 (p < 0.001) from societal perspective. The ICER equalled €52,378.12/ LYG from payer and €75,703.44/ LYG from societal perspective. The probability for cost-effectiveness would be 95% if the willingness-to-pay was higher than €82,000/ LYG from payer, and €95,000/ LYG from societal perspective. Conclusion Survival improved in hospitals providing OGCM. Costs were found to increase, driven by inpatient and long-term care. The cost-effectiveness depends on the willingness-to-pay. The ICER is likely to improve with a longer follow-up.
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Lenz-Alcayaga, Rony, Daniela Paredes-Fernández, Karla Hernández-Sánchez, and Juan E. Valencia-Zapata. "Cost-utility analysis: Mechanical thrombectomy plus thrombolysis in ischemic stroke due to large vessel occlusion in the public sector in Chile." Medwave 21, no. 03 (April 30, 2021): e8152-e8152. http://dx.doi.org/10.5867/medwave.2021.03.8152.
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Introduction Several studies demonstrate the therapeutic superiority of thrombolysis plus mechanical thrombectomy versus thrombolysis alone to treat stroke. Objective To analyze the cost-utility of thrombolysis plus mechanical thrombectomy versus thrombolysis in patients with ischemic stroke due to large vessel occlusion. Methods Cost-utility analysis. The model used is blended: Decision Tree (first 90 days) and Markov in the long term, of seven health states based on a disease-specific scale, from the Chilean public insurance and societal perspective. Quality-Adjusted Life-Years and costs are evaluated. Deterministic (DSA) and probabilistic (PSA) analyses were carried out. Results From the public insurance perspective, in the base case, mechanical thrombectomy is associated with lower costs in a lifetime horizon, and with higher benefits (2.63 incremental QALYs, and 1.19 discounted incremental life years), at a Net Monetary Benefit (NMB) of CLP 37,289,874, and an Incremental Cost-Utility Ratio (ICUR) of CLP 3,807,413/QALY. For the scenario that incorporates access to rehabilitation, 2.54 incremental QALYs and 1.13 discounted life years were estimated, resulting in an NMB of CLP 35,670,319 and ICUR of CLP 3,960,624/QALY. In the scenario that incorporates access to long-term care from a societal perspective, the ICUR falls to CLP 951,911/QALY, and the NMB raises to CLP 43,318,072, improving the previous scenarios. In the DSA, health states, starting age, and relative risk of dying were the variables with the greatest influence. The PSA for the base case corroborated the estimates. Conclusions Thrombolysis plus mechanical thrombectomy adds quality of life at costs acceptable for decision-makers versus thrombolysis alone. The results are consistent with international studies.
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Behan, Caragh, Brendan Kennelly, Eric Roche, Laoise Renwick, Sarah Masterson, John Lyne, Brian O'Donoghue, et al. "Early intervention in psychosis: health economic evaluation using the net benefit approach in a real-world setting." British Journal of Psychiatry 217, no. 3 (July 24, 2019): 484–90. http://dx.doi.org/10.1192/bjp.2019.126.
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BackgroundEarly intervention in psychosis is a complex intervention, usually delivered in a specialist stand-alone setting, which aims to improve outcomes for people with psychosis. Previous studies have been criticised because the control used did not accurately reflect actual practice.AimsTo evaluate the cost-effectiveness of early intervention by estimating the incremental net benefit (INB) of an early-intervention programme, delivered in a real-world setting. INB measures the difference in monetary terms between alternative interventions.MethodTwo contemporaneous incidence-based cohorts presenting with first-episode psychosis, aged 18–65 years, were compared. Costs and outcomes were measured over 1 year. The main outcome was avoidance of a relapse that required admission to hospital or home-based treatment.ResultsFrom the health sector perspective, the probability that early intervention was cost-effective was 0.77. The INB was €2465 per person (95% CI − €4418 to €9347) when society placed a value of €6000, the cost of an in-patient relapse, on preventing a relapse requiring admission or home care. Following adjustment, the probability that early intervention was cost-effective was 1, and the INB to the health sector was €3105 per person (95% CI −€8453 to €14 663). From a societal perspective, the adjusted probability that early intervention was cost-effective was 1, and the INB was €19 928 per person (95% CI − €2075 to €41 931).ConclusionsEarly intervention has a modest INB from the health sector perspective and a large INB from the societal perspective. The perspective chosen is critical when presenting results of an economic evaluation of a complex intervention.
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Sharieff, Waseem, Stanley H. Zlotkin, Wendy J. Ungar, Brian Feldman, Murray D. Krahn, and George Tomlinson. "Economics of preventing premature mortality and impaired cognitive development in children through home-fortification: A health policy perspective." International Journal of Technology Assessment in Health Care 24, no. 03 (July 2008): 303–11. http://dx.doi.org/10.1017/s0266462308080409.
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Background:Home-fortification is a new strategy of adding micronutrients including zinc and iron to home-made foods. Zinc supplementation may prevent morbidity and mortality related to diarrheal illnesses, and iron supplementation may improve cognitive development, in children.Objectives:To project clinical and economic effects of home-fortification in children in an urban slum of Karachi, Pakistan.Methods:This is a cost benefit analysis of 5,000 simulated male and female infants (6–12 months) assigned to micronutrients or placebo for 4 months and followed for 55 years. We linked the effect of zinc on longitudinal prevalence of diarrhea to mortality, and the effect of iron on hemoglobin to IQ scores and lifetime earnings. Cost estimates were based on volumes of resource utilization from the Pakistan Sprinkles Diarrhea study. Main outcome was incremental benefit defined as the gain in lifetime earnings after accounting for the incremental costs of micronutrients over placebo (societal perspective).Results:Our model projected that the reduction in diarrhea and improvement in hemoglobin concentrations through home-fortification was associated with reduced child mortality, higher IQ scores, and higher earnings. The present value of incremental benefit was $106 (95 percent probability interval = $17 to $193) U.S. dollars, which corresponds to $464.79 ($74.54 to $846.27) international dollars using a purchasing power parity exchange rate.Conclusions:Home-fortification appears to improve clinical outcomes at a reasonable cost, and may actually be cost beneficial when lifetime earnings are considered.
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Greenspoon, Jeffrey Noah, Waseem Sharieff, Anthony Whitton, Timothy Joseph Whelan, Jim R. Wright, Jonathan Sussman, and Amiram Gafni. "Robotic radiosurgery for the treatment of one to three brain metastases: A pragmatic application of cost-benefit analysis using willingness to pay." Journal of Clinical Oncology 30, no. 34_suppl (December 1, 2012): 17. http://dx.doi.org/10.1200/jco.2012.30.34_suppl.17.
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17 Background: With the emergence of radiosurgery as a new radiotheraputic technique, health care decision makers are required to incorporate community need, cost and patient preferences when allocating radiosurgery resources. Conventional patient utility measures would not reflect short term preferences and would therefore not inform decision makers when allocating radiosurgery treatment units. The goal of this article is to demonstrate the feasibility of cost-benefit analysis to elicit the yearly net monetary benefit of robotic radiosurgery. Methods: To calculate the yearly incremental cost of robotic radiosurgery as compared to fixed-gantry radiosurgery we used direct local cost data. We assumed a standard 10 year replacement and 5% amortization rate. Decision boards summarizing the clinical scenario of brain metastases and the difference between robotic and fixed-gantry radiosurgery in terms of immobilization, comfort and treatment time were then presented to a sample of 18 participants. Participants who preferred robotic radiosurgery were randomly assigned to either a low ($1) or high ($5) starting point taxation based willingness-to-pay algorithm. Results: The yearly incremental cost of providing robotic radiosurgery was $99,177. The mean community yearly willingness-to pay for robotic radiosurgery was $2,300,000, p=0.03. The calculated yearly net societal benefit for robotic radiosurgery was $2,200,823. Among participants who preferred robotic radiosurgery there was no evidence of starting point bias, p=0.8. Conclusions: We have shown through this pilot study that it is feasible to perform cost-benefit analysis to evaluate new technologies in Radiation Oncology. Cost-benefit analysis offers an analytic method to evaluate local preferences and provide accountability when allocating limited healthcare resources.
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Rongen, Jan J., Tim M. Govers, Pieter Buma, Janneke P. C. Grutters, and Gerjon Hannink. "Societal and Economic Effect of Meniscus Scaffold Procedures for Irreparable Meniscus Injuries." American Journal of Sports Medicine 44, no. 7 (April 19, 2016): 1724–34. http://dx.doi.org/10.1177/0363546516639290.
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Background: Meniscus scaffolds are currently evaluated clinically for their efficacy in preventing the development of osteoarthritis as well as for their efficacy in treating patients with chronic symptoms. Procedural costs, therapeutic consequences, clinical efficacy, and future events should all be considered to maximize the monetary value of this intervention. Purpose: To examine the socioeconomic effect of treating patients with irreparable medial meniscus injuries with a meniscus scaffold. Study Design: Economic and decision analysis; Level of evidence, 2. Methods: Two Markov simulation models for patients with an irreparable medial meniscus injury were developed. Model 1 was used to investigate the lifetime cost-effectiveness of a meniscus scaffold compared with standard partial meniscectomy by the possibility of preventing the development of osteoarthritis. Model 2 was used to investigate the short-term (5-year) cost-effectiveness of a meniscus scaffold compared with standard partial meniscectomy by alleviating clinical symptoms, specifically in chronic patients with previous meniscus surgery. For both models, probabilistic Monte Carlo simulations were applied. Treatment effectiveness was expressed as quality-adjusted life-years (QALYs), while costs (estimated in euros) were assessed from a societal perspective. We assumed €20,000 as a reference value for the willingness to pay per QALY. Next, comprehensive sensitivity analyses were performed to identify the most influential variables on the cost-effectiveness of meniscus scaffolds. Results: Model 1 demonstrated an incremental cost-effectiveness ratio of a meniscus scaffold treatment of €54,463 per QALY (€5991/0.112). A threshold analysis demonstrated that a meniscus scaffold should offer a relative risk reduction of at least 0.34 to become cost-effective, assuming a willingness to pay of €20,000. Decreasing the costs of the meniscus scaffold procedure by 33% (€10,160 instead of €15,233; an absolute change of €5073) resulted in an incremental cost-effectiveness ratio of €7876 per QALY. Model 2 demonstrated an incremental cost-effectiveness ratio of a meniscus scaffold treatment of €297,727 per QALY (€9825/0.033). On the basis of the current efficacy data, a meniscus scaffold provides a relative risk reduction of “limited benefit” postoperatively of 0.37 compared with standard treatment. A threshold analysis revealed that assuming a willingness to pay of €20,000, a meniscus scaffold would not be cost-effective within a period of 5 years. Most influential variables on the cost-effectiveness of meniscus scaffolds were the cost of the scaffold procedure, cost associated with osteoarthritis, and quality of life before and after the scaffold procedure. Conclusion: Results of the current health technology assessment emphasize that the monetary value of meniscus scaffold procedures is very much dependent on a number of influential variables. Therefore, before implementing the technology in the health care system, it is important to critically assess these variables in a relevant context. The models can be improved as additional clinical data regarding the efficacy of the meniscus scaffold become available.
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Emanuel, Kerry, Eugenia Kalnay, Craig Bishop, Russell Elsberry, Ronald Gelaro, Daniel Keyser, Steven Lord, et al. "Observations in Aid of Weather Prediction for North America: Report of Prospectus Development Team Seven." Bulletin of the American Meteorological Society 78, no. 12 (December 1, 1997): 2859–68. http://dx.doi.org/10.1175/1520-0477-78.12.2859.
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One of the most significant impediments to progress in forecasting weather over North America is the relative paucity of routine observations over data-sparse regions adjacent to the United States. Prospectus Development Team Seven was convened to consider ways to promote research that seeks to determine implementations of observing systems that are optimal for weather prediction in the United States. An “optimal” measurement system is considered to be one that maximizes the ratio of societal benefit to overall cost. The thrust of the conclusions is that existing means of estimating the value of current observing systems and the potential benefits of new or proposed observing systems are underutilized. At the same time, no rational way exists for comparing the cost of observations across the spectrum of federal agencies responsible for measuring the atmosphere and ocean. The authors suggest that a rational procedure for configuring an observation system that is optimal for weather prediction would consist of the following steps.Identify specific forecast problems arising from insufficient data. Examples might include hurricane landfall and intensity forecasts, 24-h forecasts of intense extratropical cyclones affecting the West Coast and Alaska, and medium-range forecasts of severe weather for all of North America.Use contemporary modeling techniques, such as observing system simulation experiments, ensemble forecasting, and model adjoint-derived sensitivities, to delineate measurement requirements for each specific forecasting problem and identify candidate observing systems and data assimilation techniques that could be brought to bear on each problem.Estimate the incremental forecast improvements that could plausibly result from the added or reconfigured data and the societal benefits that would accrue from such improvements.Estimate the overall cost (to the nation, not to specific federal agencies) of obtaining the data by the various candidate techniques and the benefits that are projected to result.Use standard cost–benefit analysis as a basis for deciding the optimal deployment of measuring systems. The authors believe that a rational approach to atmospheric measurement is critical to further improvements in weather prediction and that such improvements might very well be made within the current budget of routine observations, integrated across all of the responsible federal agencies. This document outlines a proposed strategy for rationalizing atmosphere observations in aid of weather prediction in the United States. The paper begins with a summary of recommendations.
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Kunz, Wolfgang G., Mohammed A. Almekhlafi, Bijoy K. Menon, Jeffrey L. Saver, Myriam G. Hunink, Diederik W. J. Dippel, Charles B. L. M. Majoie, et al. "Public Health and Cost Benefits of Successful Reperfusion After Thrombectomy for Stroke." Stroke 51, no. 3 (March 2020): 899–907. http://dx.doi.org/10.1161/strokeaha.119.027874.
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Background and Purpose— The benefit that endovascular thrombectomy offers to patients with stroke with large vessel occlusions depends strongly on reperfusion grade as defined by the expanded Thrombolysis in Cerebral Infarction (eTICI) scale. Our aim was to determine the lifetime health and cost consequences of the quality of reperfusion for patients, healthcare systems, and society. Methods— A Markov model estimated lifetime quality-adjusted life years (QALY) and lifetime costs of endovascular thrombectomy–treated patients with stroke based on eTICI grades. The analysis was performed over a lifetime horizon in a United States setting, adopting healthcare and societal perspectives. The reference case analysis was conducted for stroke at 65 years of age. National health and cost consequences of improved eTICI 2c/3 reperfusion rates were estimated. Input parameters were based on best available evidence. Results— Lifetime QALYs increased for every grade of improved reperfusion (median QALYs for eTICI 0/1: 2.62; eTICI 2a: 3.46; eTICI 2b: 5.42; eTICI 2c: 5.99; eTICI 3: 6.73). Achieving eTICI 3 over eTICI 2b reperfusion resulted on average in 1.31 incremental QALYs as well as healthcare and societal cost savings of $10 327 and $20 224 per patient. A 10% increase in the eTICI 2c/3 reperfusion rate of all annually endovascular thrombectomy–treated patients with stroke in the United States is estimated to yield additional 3656 QALYs and save $21.0 million and $36.8 million for the healthcare system and society, respectively. Conclusions— Improved reperfusion grants patients with stroke additional QALYs and leads to long-term cost savings. Procedural strategies to achieve complete reperfusion should be assessed for safety and feasibility, even when initial reperfusion seems to be adequate.
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MacEwan, Joanna P., Istvan Majer, Jacquelyn W. Chou, and Sumeet Panjabi. "The value of survival gains from therapeutic innovations for US patients with relapsed/refractory multiple myeloma." Therapeutic Advances in Hematology 12 (January 2021): 204062072110274. http://dx.doi.org/10.1177/20406207211027463.
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Aims: This study quantifies the value of survival gains attributable to novel treatments approved since 2003 for United States (US) patients with relapsed/refractory multiple myeloma (RRMM). Methods: We estimated the increase in survival attributable to lenalidomide and bortezomib for multiple myeloma (MM) patients in the 1983–2013 Surveillance, Epidemiology, and End Results (SEER) registry. To estimate the survival benefit of treatments approved since 2015 (carfilzomib, elotuzomab, daratumumab, used in combination with lenalidomide and dexamethasone) we used clinical trial data to calibrate survival estimated using the SEER data. We then conducted an economic valuation of the estimated shift in survival curves for all therapies. Finally, we estimated the share of the value accruing to patients and manufacturers using treatment costs estimated from MarketScan data. Results: The introduction of bortezomib in combination with dexamethasone (Vd) and lenalidomide in combination with dexamethasone (Rd) resulted in substantial survival gains and societal value for multiple myeloma patients, generating 1.7 additional life-years per RRMM patient. More recently, approved novel treatments have improved survival over effective treatments (i.e. Rd/Vd) by an additional 2.5 life-years – the monetary value of this incremental survival benefit far exceeds the incremental cost of treatment. At the patient level, the incremental benefit of Rd/Vd is $335,500 and with novel treatments is $565,000. Applying this benefit to all future cohorts of US RRMM patients translates into a value of at least $75 billion and $130 billion with Rd/Vd and the novel treatments, respectively. Conclusions: SEER registry data were only available through 2013. Therefore, survival gains for recently approved treatments were estimated based on clinical trials, rather than observed survival. Our valuation analysis does not capture sources of value aside from survival gains, for example, better quality of life, increased productivity, or the value of surviving until subsequent novel therapies become available. Substantial extensions in life expectancy in RRMM since 2003 translate into real economic value gained by society.
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Clarkson, Jan E., Nigel B. Pitts, Beatriz Goulao, Dwayne Boyers, Craig R. Ramsay, Ruth Floate, Hazel J. Braid, et al. "Risk-based, 6-monthly and 24-monthly dental check-ups for adults: the INTERVAL three-arm RCT." Health Technology Assessment 24, no. 60 (November 2020): 1–138. http://dx.doi.org/10.3310/hta24600.
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Background Traditionally, patients are encouraged to attend dental recall appointments at regular 6-month intervals, irrespective of their risk of developing dental disease. Stakeholders lack evidence of the relative effectiveness and cost-effectiveness of different recall strategies and the optimal recall interval for maintenance of oral health. Objectives To test effectiveness and assess the cost–benefit of different dental recall intervals over a 4-year period. Design Multicentre, parallel-group, randomised controlled trial with blinded clinical outcome assessment at 4 years and a within-trial cost–benefit analysis. NHS and participant perspective costs were combined with benefits estimated from a general population discrete choice experiment. A two-stratum trial design was used, with participants randomised to the 24-month interval if the recruiting dentist considered them clinically suitable. Participants ineligible for 24-month recall were randomised to a risk-based or 6-month recall interval. Setting UK primary care dental practices. Participants Adult, dentate, NHS patients who had visited their dentist in the previous 2 years. Interventions Participants were randomised to attend for a dental check-up at one of three dental recall intervals: 6-month, risk-based or 24-month recall. Main outcomes Clinical – gingival bleeding on probing; patient – oral health-related quality of life; economic – three analysis frameworks: (1) incremental cost per quality-adjusted life-year gained, (2) incremental net (societal) benefit and (3) incremental net (dental health) benefit. Results A total of 2372 participants were recruited from 51 dental practices; 648 participants were eligible for the 24-month recall stratum and 1724 participants were ineligible. There was no evidence of a significant difference in the mean percentage of sites with gingival bleeding between intervention arms in any comparison. For the eligible for 24-month recall stratum: the 24-month (n = 138) versus 6-month group (n = 135) had an adjusted mean difference of –0.91 (95% confidence interval –5.02 to 3.20); the risk-based (n = 143) versus 6-month group had an adjusted mean difference of –0.98 (95% confidence interval –5.05 to 3.09); the 24-month versus risk-based group had an adjusted mean difference of 0.07 (95% confidence interval –3.99 to 4.12). For the overall sample, the risk-based (n = 749) versus 6-month (n = 737) adjusted mean difference was 0.78 (95% confidence interval –1.17 to 2.72). There was no evidence of a difference in oral health-related quality of life between intervention arms in any comparison. For the economic evaluation, under framework 1 (cost per quality-adjusted life-year) the results were highly uncertain, and it was not possible to identify the optimal recall strategy. Under framework 2 (net societal benefit), 6-month recalls were the most efficient strategy with a probability of positive net benefit ranging from 78% to 100% across the eligible and combined strata, with findings driven by the high value placed on more frequent recall services in the discrete choice experiment. Under framework 3 (net dental health benefit), 24-month recalls were the most likely strategy to deliver positive net (dental health) benefit among those eligible for 24-month recall, with a probability of positive net benefit ranging from 65% to 99%. For the combined group, the optimal strategy was less clear. Risk-based recalls were more likely to be the most efficient recall strategy in scenarios where the costing perspective was widened to include participant-incurred costs, and in the Scottish subgroup. Limitations Information regarding factors considered by dentists to inform the risk-based interval and the interaction with patients to determine risk and agree the interval were not collected. Conclusions Over a 4-year period, we found no evidence of a difference in oral health for participants allocated to a 6-month or a risk-based recall interval, nor between a 24-month, 6-month or risk-based recall interval for participants eligible for a 24-month recall. However, people greatly value and are willing to pay for frequent dental check-ups; therefore, the most efficient recall strategy depends on the scope of the cost and benefit valuation that decision-makers wish to consider. Future work Assessment of the impact of risk assessment tools in informing risk-based interval decision-making and techniques for communicating a variable recall interval to patients. Trial registration Current Controlled Trials ISRCTN95933794. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme [project numbers 06/35/05 (Phase I) and 06/35/99 (Phase II)] and will be published in full in Health Technology Assessment; Vol. 24, No. 60. See the NIHR Journals Library website for further project information.
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Yellman, Merissa A., Cora Peterson, Mary A. McCoy, Shelli Stephens-Stidham, Emily Caton, Jeffrey J. Barnard, Ted O. Padgett, Curtis Florence, and Gregory R. Istre. "Preventing deaths and injuries from house fires: a cost–benefit analysis of a community-based smoke alarm installation programme." Injury Prevention 24, no. 1 (February 9, 2017): 12–18. http://dx.doi.org/10.1136/injuryprev-2016-042247.
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BackgroundOperation Installation (OI), a community-based smoke alarm installation programme in Dallas, Texas, targets houses in high-risk urban census tracts. Residents of houses that received OI installation (or programme houses) had 68% fewer medically treated house fire injuries (non-fatal and fatal) compared with residents of non-programme houses over an average of 5.2 years of follow-up during an effectiveness evaluation conducted from 2001 to 2011.ObjectiveTo estimate the cost–benefit of OI.MethodsA mathematical model incorporated programme cost and effectiveness data as directly observed in OI. The estimated cost per smoke alarm installed was based on a retrospective analysis of OI expenditures from administrative records, 2006–2011. Injury incidence assumptions for a population that had the OI programme compared with the same population without the OI programme was based on the previous OI effectiveness study, 2001–2011. Unit costs for medical care and lost productivity associated with fire injuries were from a national public database.ResultsFrom a combined payers' perspective limited to direct programme and medical costs, the estimated incremental cost per fire injury averted through the OI installation programme was $128,800 (2013 US$). When a conservative estimate of lost productivity among victims was included, the incremental cost per fire injury averted was negative, suggesting long-term cost savings from the programme. The OI programme from 2001 to 2011 resulted in an estimated net savings of $3.8 million, or a $3.21 return on investment for every dollar spent on the programme using a societal cost perspective.ConclusionsCommunity smoke alarm installation programmes could be cost-beneficial in high-fire-risk neighbourhoods.
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Grochtdreis, Thomas, Christian Brettschneider, Frederike Bjerregaard, Christiane Bleich, Sigrid Boczor, Martin Härter, Lars P. Hölzel, et al. "Cost-effectiveness analysis of collaborative treatment of late-life depression in primary care (GermanIMPACT)." European Psychiatry 57 (January 15, 2019): 10–18. http://dx.doi.org/10.1016/j.eurpsy.2018.12.007.
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AbstractBackground:Late-life depression is a highly prevalent disorder that causes a large economic burden. A stepped collaborative care program was set up in order to improve care for patients with late-life depression in primary care in Germany: GermanIMPACT is the adaption of the Improving Mood-Promoting Access to Collaborative Treatment (IMPACT) program that has already been established in primary care in the USA. The aim of this study was to determine the cost-effectiveness of GermanIMPACT compared with treatment as usual from a societal perspective.Methods:This study is part of a 12-month bi-centric cluster-randomized controlled trial aiming to assess the effectiveness of GermanIMPACT compared with treatment as usual among patients with late-life depression. A cost-effectiveness analysis using depression-free days (DFDs) was performed. Net-monetary benefit (NMB) regressions adjusted for baseline differences for different willingness-to-pay (WTP) thresholds were conducted and cost-effectiveness acceptability curves were constructed.Results:In total, n = 246 patients (intervention group: n = 139; control group: n = 107) with a mean age of 71 from 71 primary care practices were included in the analysis. After 12 months, adjusted mean differences in costs and DFDs between intervention group and control group were +€354 and +21.4, respectively. Only the difference in DFDs was significant (p = 0.022). According to the unadjusted incremental cost-effectiveness ratio, GermanIMPACT was dominant compared with treatment as usual. The probability of GermanIMPACT being cost-effective was 80%, 90% or 95% if societal WTP per DFD was ≥€70, ≥€110 or ≥€180, respectively.Conclusion:Evidence for cost-effectiveness of GermanIMPACT relative to treatment as usual is not clear. Only if societal WTP was ≥€180 for an additional DFD, GermanIMPACT could be considered cost-effective with certainty.
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Mandelblatt, Jeanne S., Clyde B. Schechter, K. Robin Yabroff, William Lawrence, James Dignam, Peter Muennig, Yoko Chavez, Jennifer Cullen, and Marianne Fahs. "Benefits and Costs of Interventions to Improve Breast Cancer Outcomes in African American Women." Journal of Clinical Oncology 22, no. 13 (July 1, 2004): 2554–66. http://dx.doi.org/10.1200/jco.2004.05.009.
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Purpose Historically, African American women have experienced higher breast cancer mortality than white women, despite lower incidence. Our objective was to evaluate whether costs of increasing rates of screening or application of intensive treatment will be off-set by survival benefits for African American women. Methods We use a stochastic simulation model of the natural history of breast cancer to evaluate the incremental societal costs and benefits of status quo versus targeted biennial screening or treatment improvements among African Americans 40 years of age and older. Main outcome measures were number of mammograms, stage, all-cause mortality, and discounted costs per life year saved (LYS). Results At the current screening rate of 76%, there is little incremental benefit associated with further increasing screening, and the costs are high: $124,053 and $124,217 per LYS for lay health worker and patient reminder interventions, respectively, compared with the status quo. Using reminders would cost $51,537 per LYS if targeted to virtually unscreened women or $78,130 per LYS if targeted to women with a two-fold increase in baseline risk. If all patients received the most intensive treatment recommended, costs increase but deaths decrease, for a cost of $52,678 per LYS. Investments of up to $6,000 per breast cancer patient could be used to enhance treatment and still yield cost-effectiveness ratios of less than $75,000 per LYS. Conclusion Except in pockets of unscreened or high-risk women, further investments in interventions to increase screening are unlikely to be an efficient use of resources. Ensuring that African American women receive intensive treatment seems to be the most cost-effective approach to decreasing the disproportionate mortality experienced by this population.
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Manchanda, Ranjit, Li Sun, Shreeya Patel, Olivia Evans, Janneke Wilschut, Ana Carolina De Freitas Lopes, Faiza Gaba, et al. "Economic Evaluation of Population-Based BRCA1/BRCA2 Mutation Testing across Multiple Countries and Health Systems." Cancers 12, no. 7 (July 17, 2020): 1929. http://dx.doi.org/10.3390/cancers12071929.
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Clinical criteria/Family history-based BRCA testing misses a large proportion of BRCA carriers who can benefit from screening/prevention. We estimate the cost-effectiveness of population-based BRCA testing in general population women across different countries/health systems. A Markov model comparing the lifetime costs and effects of BRCA1/BRCA2 testing all general population women ≥30 years compared with clinical criteria/FH-based testing. Separate analyses are undertaken for the UK/USA/Netherlands (high-income countries/HIC), China/Brazil (upper–middle income countries/UMIC) and India (low–middle income countries/LMIC) using both health system/payer and societal perspectives. BRCA carriers undergo appropriate screening/prevention interventions to reduce breast cancer (BC) and ovarian cancer (OC) risk. Outcomes include OC, BC, and additional heart disease deaths and incremental cost-effectiveness ratio (ICER)/quality-adjusted life year (QALY). Probabilistic/one-way sensitivity analyses evaluate model uncertainty. For the base case, from a societal perspective, we found that population-based BRCA testing is cost-saving in HIC (UK-ICER = $−5639/QALY; USA-ICER = $−4018/QALY; Netherlands-ICER = $−11,433/QALY), and it appears cost-effective in UMIC (China-ICER = $18,066/QALY; Brazil-ICER = $13,579/QALY), but it is not cost-effective in LMIC (India-ICER = $23,031/QALY). From a payer perspective, population-based BRCA testing is highly cost-effective in HIC (UK-ICER = $21,191/QALY, USA-ICER = $16,552/QALY, Netherlands-ICER = $25,215/QALY), and it is cost-effective in UMIC (China-ICER = $23,485/QALY, Brazil−ICER = $20,995/QALY), but it is not cost-effective in LMIC (India-ICER = $32,217/QALY). BRCA testing costs below $172/test (ICER = $19,685/QALY), which makes it cost-effective (from a societal perspective) for LMIC/India. Population-based BRCA testing can prevent an additional 2319 to 2666 BC and 327 to 449 OC cases per million women than the current clinical strategy. Findings suggest that population-based BRCA testing for countries evaluated is extremely cost-effective across HIC/UMIC health systems, is cost-saving for HIC health systems from a societal perspective, and can prevent tens of thousands more BC/OC cases.
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Barbosa, Estela Capelas, Talitha Irene Verhoef, Steve Morris, Francesca Solmi, Medina Johnson, Alex Sohal, Farah El-Shogri, et al. "Cost-effectiveness of a domestic violence and abuse training and support programme in primary care in the real world: updated modelling based on an MRC phase IV observational pragmatic implementation study." BMJ Open 8, no. 8 (August 2018): e021256. http://dx.doi.org/10.1136/bmjopen-2017-021256.
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ObjectivesTo evaluate the cost-effectiveness of the implementation of the Identification and Referral to Improve Safety (IRIS) programme using up-to-date real-world information on costs and effectiveness from routine clinical practice. A Markov model was constructed to estimate mean costs and quality-adjusted life-years (QALYs) of IRIS versus usual care per woman registered at a general practice from a societal and health service perspective with a 10-year time horizon.Design and settingCost–utility analysis in UK general practices, including data from six sites which have been running IRIS for at least 2 years across England.ParticipantsBased on the Markov model, which uses health states to represent possible outcomes of the intervention, we stipulated a hypothetical cohort of 10 000 women aged 16 years or older.InterventionsThe IRIS trial was a randomised controlled trial that tested the effectiveness of a primary care training and support intervention to improve the response to women experiencing domestic violence and abuse, and found it to be cost-effective. As a result, the IRIS programme has been implemented across the UK, generating data on costs and effectiveness outside a trial context.ResultsThe IRIS programme saved £14 per woman aged 16 years or older registered in general practice (95% uncertainty interval −£151 to £37) and produced QALY gains of 0.001 per woman (95% uncertainty interval −0.005 to 0.006). The incremental net monetary benefit was positive both from a societal and National Health Service perspective (£42 and £22, respectively) and the IRIS programme was cost-effective in 61% of simulations using real-life data when the cost-effectiveness threshold was £20 000 per QALY gained as advised by National Institute for Health and Care Excellence.ConclusionThe IRIS programme is likely to be cost-effective and cost-saving from a societal perspective in the UK and cost-effective from a health service perspective, although there is considerable uncertainty surrounding these results, reflected in the large uncertainty intervals.
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Ronaldson, Sarah J., Ada Keding, Puvan Tharmanathan, Catherine Arundel, Sarah R. Kingsbury, Philip G. Conaghan, and David J. Torgerson. "Cost-effectiveness of hydroxychloroquine versus placebo for hand osteoarthritis: economic evaluation of the HERO trial." F1000Research 10 (August 17, 2021): 821. http://dx.doi.org/10.12688/f1000research.55296.1.
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Background: An economic evaluation alongside the Hydroxychloroquine Effectiveness in Reducing symptoms of hand Osteoarthritis (HERO) trial was undertaken to assess the cost-effectiveness of hydroxychloroquine compared with placebo for symptomatic treatment of hand osteoarthritis for patients with at least moderate hand pain and inadequate response to current therapies. Methods: A trial-based cost–utility analysis was undertaken from the perspective of the UK National Health Service and Personal Social Services over a 12-month time horizon, using evidence from 248 participants included in the HERO trial, conducted in England. Patient-level data were collected prospectively over a 12-month period, using participant-completed questionnaires and investigator forms, to collect healthcare utilisation, costs and quality-adjusted life years (QALYs) using the EQ-5D-5L. The base-case analysis was conducted on an intention-to-treat basis and used multiple imputation methods to deal with missing data. Results were presented in terms of incremental cost-effectiveness ratios (incremental cost per QALY) and net health benefit, with uncertainty surrounding the findings explored using cost-effectiveness acceptability curves. Results: The base-case analysis estimated slightly lower costs on average (−£11.80; 95% confidence interval (CI) −£15.60 to −£8.00) and marginally fewer QALYs (−0.0052; 95% CI −0.0057 to −0.0047) for participants in the hydroxychloroquine group versus placebo group at 12 months. The resulting incremental cost-effectiveness ratio of £2,267 per QALY lost indicated that although costs were saved, health-related quality of life was lost. Even assuming symmetrical preferences regarding losses and gains for health benefits, the findings do not fall within the cost-effective region. Similar findings arose for analyses conducted from the societal perspective and using complete cases only. Conclusions: This economic evaluation indicates that hydroxychloroquine is unlikely to provide a cost-effective pain relief option for improving health-related quality of life in adult patients with moderate-to-severe hand osteoarthritis.
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Bentley, Tanya G., Michael Samuel Broder, Lopamudra Das, Jesse Ortendahl, Yun Su, and Samuel Wagner. "Targeted therapies for metastatic colorectal cancer (mCRC): A systematic review of cost-effectiveness (CE)." Journal of Clinical Oncology 30, no. 4_suppl (February 1, 2012): 583. http://dx.doi.org/10.1200/jco.2012.30.4_suppl.583.
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583 Background: Currently three targeted agents are available for the treatment of mCRC. Making the right choice requires balancing efficacy, safety, quality of life, and, in cost-constrained systems, cost. This study aims to determine the most cost effective treatment in each line of therapy for mCRC through systematic review of published CE analyses (CEA). Methods: Literature was searched in Medline, Cancerlit, EMBASE, Cochrane, CINAHL, BIOSIS, Web of Science, Tufts CEA registry, ASCO and ASCO GI for CEAs of the three mCRC targeted agents, cetuximab, bevacizumab, and panitumumab. Manuscripts from 2004-2011 and abstracts from 2009-2011, published in English and considering payer/societal perspectives were included. Incremental CE ratios (ICERs) were converted to US$ using 2010 purchasing power parity. Results: 15 models from six countries were identified. Mean incremental costs per quality adjusted life year (QALY) and per life year (LY) gained are shown in the Table. All four models evaluating cetuximab in 1st line therapy, and only one of the six in 2nd+ line therapy, were done among biomarker selected KRAS wild type patients. Cetuximab’s cost effectiveness in 1st line therapy was driven by its ability to convert initially unresectable liver metastasis to resectable. Conclusions: Lower ICERs appear to be associated with the use of a predictive biomarker and / or a sub patient population that can benefit the most from a treatment. With the KRAS biomarker and its potential curative benefit in patients with initially unresectable liver metastasis, cetuximab appears to be the most cost-effective targeted agent in 1st line mCRC treatment. In 2nd or later lines, direct CEA between the three agents in comparable biomarker selected patients are needed to determine the most cost effective agent. [Table: see text]
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Reeves, Penny, Christopher Doran, Mariko Carey, Emilie Cameron, Robert Sanson-Fisher, Finlay Macrae, and David Hill. "Costs and Cost-Effectiveness of Targeted, Personalized Risk Information to Increase Appropriate Screening by First-Degree Relatives of People With Colorectal Cancer." Health Education & Behavior 46, no. 5 (March 12, 2019): 798–808. http://dx.doi.org/10.1177/1090198119835294.
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Background. Economic evaluations are less commonly applied to implementation interventions compared to clinical interventions. The efficacy of an implementation strategy to improve adherence to screening guidelines among first-degree relatives of people with colorectal cancer was recently evaluated in a randomized-controlled trial. Using these trial data, we examined the costs and cost-effectiveness of the intervention from societal and health care funder perspectives. Method. In this prospective, trial-based evaluation, mean costs, and outcomes were calculated. The primary outcome of the trial was the proportion of participants who had screening tests in the year following the intervention commensurate with their risk category. Quality-adjusted life years were included as secondary outcomes. Intervention costs were determined from trial records. Standard Australian unit costs for 2016/2017 were applied. Cost-effectiveness was assessed using the net benefit framework. Nonparametric bootstrapping was used to calculate uncertainty intervals (UIs) around the costs and the incremental net monetary benefit statistic. Results. Compared with usual care, mean health sector costs were $17 (95% UI [$14, $24]) higher for those receiving the intervention. The incremental cost-effectiveness ratio for the primary trial outcome was calculated to be $258 (95% UI [$184, $441]) per additional person appropriately screened. The significant difference in adherence to screening guidelines between the usual care and intervention groups did not translate into a mean quality-adjusted life year difference. Discussion. Providing information on both the costs and outcomes of implementation interventions is important to inform public health care investment decisions. Challenges in the application of cost–utility analysis hampered the interpretation of results and potentially underestimated the value of the intervention. Further research in the form of a modeled extrapolation of the intermediate increased adherence effect and distributional cost-effectiveness to include equity requirements is warranted.
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El Ouagari, Khalid, Christopher John Knight, and Estella T. Mendelson. "Cost-Effectiveness of Ruxolitinib Versus Best-Available Therapy for Medical Treatment of Myelofibrosis: Canadian Societal Perspective." Blood 120, no. 21 (November 16, 2012): 4255. http://dx.doi.org/10.1182/blood.v120.21.4255.4255.
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Abstract Abstract 4255 Background: Ruxolitinib demonstrated significant clinical benefits for patients (pts) with myelofibrosis (MF) in two phase 3 COMFORT studies. Nevertheless, in countries where health technology assessments are conducted, cost-effectiveness is an important consideration. Economic evaluations play an increasingly important role in reimbursement decisions for new treatments. To help inform such decisions, an evaluation of the cost-effectiveness (CE) of adding ruxolitinib as a treatment for MF was conducted from a Canadian societal perspective. Methods: A Markov model was built to estimate the costs, health outcomes, and CE of ruxolitinib compared with investigator-selected best available therapy (BAT), which included conventional therapies or no treatment. Data were derived from pts enrolled in the COMFORT-II study and included high-risk or intermediate-2–risk pts according to the International Working Group for Myelofibrosis Research and Treatment risk categorization. For this analysis, response was defined as a ≥ 35% reduction in spleen volume (COMFORT-II primary endpoint) or absence of constitutional symptoms. Survival assumptions were derived from a historical comparison of patients in the phase 1/2 study (Verstovsek et al, Blood. 2012) that showed a benefit for those achieving a ≥ 50% reduction in spleen length (equivalent to 35% spleen volume reduction). The model simulated disease progression in a cohort of pts with MF through 4 different health states with a 12-week cycle and a lifetime time horizon: 1) responder; 2) nonresponder; 3) leukemic transformation (LT); and 4) death. In each time cycle, pts incurred cost and utility estimates associated with their health state. The model considered the resource and utility implications arising from treatments aimed at directly treating splenomegaly, namely splenic irradiation and splenectomy. The outcomes of the Markov model included costs over the time horizon (drug costs, costs linked to the management of adverse events, other medical costs, indirect costs), life years, quality-adjusted life years (QALYs), and time spent as a responder to treatment. Both a 1-way sensitivity analysis and a probabilistic sensitivity analysis were performed, using estimated ranges and probability distributions for each input parameter. Results: The results showed that ruxolitinib-treated pts with MF had a better clinical outcome but at an increased cost. The total average lifetime cost of treating a pt receiving ruxolitinib was $494,859 CAD, with drug costs of $205,484 and other medical costs of $217,527, the majority of which were resource costs. The total average lifetime cost of treating a pt receiving BAT was $421,755, of which $59,289 was drug costs. Indirect costs were higher for BAT pts, at $96,458, exceeding the figure of $71,848 for ruxolitinib pts (Table). The clinical results showed longer average overall survival for pts receiving ruxolitinib compared with pts receiving BAT (Verstovsek et al, Blood. 2012). The ruxolitinib-treated pts had 4.01 QALYs compared with 2.82 QALYs for those in the BAT arm. The overall incremental CE ratio (ICER) was $61,444 per QALY. The key model drivers were the resource cost for nonresponders and improved mortality in the intermediate-2–risk group. Adopting a $100,000 per QALY willingness-to-pay threshold showed that 100% of the simulations for ruxolitinib therapy would be cost-effective compared with BAT for the treatment of MF. The mean ICER from the simulations was $59,216 per QALY, which was similar to the deterministic average of $61,444. Conclusions: There has been a high, unmet need for an effective therapeutic option for pts with MF. The COMFORT-II study has shown that ruxolitinib is clinically effective and superior to BAT at reducing splenomegaly and disease-related symptoms and improving health-related quality of life. The present analysis suggests that compared with BAT, ruxolitinib is an economically acceptable treatment option. Continued long-term evaluation of survival and potential reduction in rates of LT will also impact the future CE of ruxolitinib. Disclosures: El Ouagari: Novartis Pharma: Employment. Knight:Novartis Pharmaceuticals Corporation: Consultancy; Novartis Pharmaceuticals Canada: Consultancy. Mendelson:Novartis Pharmaceuticals Corporation: Employment, Equity Ownership.
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Esnaola, Nestor F., Sophia N. Lazarides, Steven J. Mentzer, and Karen M. Kuntz. "Outcomes and Cost-Effectiveness of Alternative Staging Strategies for Non–Small-Cell Lung Cancer." Journal of Clinical Oncology 20, no. 1 (January 1, 2002): 263–73. http://dx.doi.org/10.1200/jco.2002.20.1.263.
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PURPOSE: To identify the optimal strategy for staging the mediastinum of patients with known non–small-cell lung cancer (NSCLC), stratified by tumor (T) classification. METHODS: We used a decision-analytic model to compare the health outcomes and cost-effectiveness of three staging strategies: (1) chest computed tomography alone, (2) selective mediastinoscopy, and (3) routine mediastinoscopy. The overall effectiveness and cost of each strategy was a function of the proportion of patients accurately staged and the risks, benefits, and costs of the diagnostic tests and treatments used. Probability estimates and costs were derived from primary data and the literature. We adopted a societal perspective and calculated incremental cost-effectiveness ratios (ICERs) as cost per quality-adjusted life year (QALY) gained. RESULTS: Both mediastinoscopy strategies correctly identified more patients with mediastinal involvement (N2/N3 disease) and assigned them to multimodal regimens. Routine mediastinoscopy maximized quality-adjusted life expectancy in all patients, irrespective of T classification, and this result was robust to varying the model estimates over their reported ranges. In T1 patients, selective mediastinoscopy cost $24,500 per QALY gained, compared with $78,800 per QALY gained for routine mediastinoscopy. In T2 and T3 patients, the ICER of routine mediastinoscopy was more favorable ($42,800 and $53,400 per QALY gained, respectively). CONCLUSION: Routine mediastinoscopy maximizes quality-adjusted life expectancy in patients with known NSCLC, and its ICER compares favorably with other currently accepted medical technologies. The survival benefit and cost-effectiveness of this strategy are greater in patients with T2 and T3 tumors and are likely to improve with advances in multimodal therapy.
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Gupta, Nidhi, Rohan Kumar Verma, Sudeep Gupta, and Shankar Prinja. "Cost Effectiveness of Trastuzumab for Management of Breast Cancer in India." JCO Global Oncology, no. 6 (September 2020): 205–16. http://dx.doi.org/10.1200/jgo.19.00293.
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PURPOSE We undertook this study to evaluate the incremental cost per quality-adjusted life-year (QALY) gained with use of adjuvant trastuzumab as compared with chemotherapy alone among patients with nonmetastatic breast cancer in India. METHODS We used a Markov model to estimate the incremental cost of using trastuzumab (for 1 year, 6 months, or 9 weeks) as compared with chemotherapy alone using a societal perspective, excluding indirect productivity losses. Although the outcomes (QALYs) in the standard chemotherapy arm were estimated after calibrating the model as per survival data from 2 Indian cancer registries, effectiveness estimates from the HERA trial and a joint analysis of the NSABP B-31 and NCCTG N9831 trials were used to estimate the consequences of 1-year trastuzumab use. The cost of treatment was estimated using national standard treatment guidelines and real-world use estimates for different treatment modalities as per data from Indian cancer registries. Probabilistic sensitivity analysis was undertaken to evaluate parameter uncertainty. RESULTS For 1 year of trastuzumab use, the incremental benefit per patient, incremental cost per QALY gained, and probability of being cost effective using HERA trial estimates were 1.29 QALYs, 178,877 Indian national rupees (INRs; US$2,558), and 4%, respectively, whereas the corresponding figures using joint analysis estimates were 1.69 QALYs, INR 134,413 (US$1,922), and 57.3%, respectively. CONCLUSION Use of trastuzumab for 1 year is not cost effective in India at the current price. However, trastuzumab use for 9 weeks is cost effective and should be included in clinical guidelines and reimbursement policies. A price reduction of 15% to 35% increases the probability of 1-year trastuzumab use being cost effective, to 90%.
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Wang, Huijuan, Lingfei Huang, Peng Gao, Zhengyi Zhu, Weifeng Ye, Haiying Ding, and Luo Fang. "Cost-effectiveness analysis of cetuximab combined with chemotherapy as a first-line treatment for patients with RAS wild-type metastatic colorectal cancer based on the TAILOR trial." BMJ Open 10, no. 2 (February 2020): e030738. http://dx.doi.org/10.1136/bmjopen-2019-030738.
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ObjectivesCetuximab plus leucovorin, fluorouracil and oxaliplatin (FOLFOX-4) is superior to FOLFOX-4 alone as a first-line treatment for patients with metastatic colorectal cancer with RAS wild-type (RAS wt mCRC), with significantly improved survival benefit by TAILOR, an open-label, randomised, multicentre, phase III trial. Nevertheless, the cost-effectiveness of these two regimens remains uncertain. The following study aims to determine whether cetuximab combined with FOLFOX-4 is a cost-effective regimen for patients with specific RAS wt mCRC in China.DesignA cost-effectiveness model combined decision tree and Markov model was built to simulate pateints with RAS wt mCRC based on health states of dead, progressive and stable. The health outcomes from the TAILOR trial and utilities from published data were used respectively. Costs were calculated with reference to the Chinese societal perspective. The robustness of the results was evaluated by univariate and probabilistic sensitivity analyses.ParticipantsThe included patients were newly diagnosed Chinese patients with fully RAS wt mCRC.InterventionsFirst-line treatment with either cetuximab plus FOLFOX-4 or FOLFOX-4.Main outcome measuresThe primary outcomes are costs, quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs).ResultsBaseline analysis disclosed that the QALYs was increased by 0.383 caused by additional cetuximab, while an increase of US$62 947 was observed in relation to FOLFOX-4 chemotherapy. The ICER was US$164 044 per QALY, which exceeded the willingness-to-pay threshold of US$28 106 per QALY.ConclusionsDespite the survival benefit, cetuximab combined with FOLFOX-4 is not a cost-effective treatment for the first-line regime of patients with RAS wt mCRC in China.Trial registration numberTAILOR trial (NCT01228734); Post-results.
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Whitehurst, David G. T., Stirling Bryan, Martyn Lewis, Jonathan Hill, and Elaine M. Hay. "Exploring the cost–utility of stratified primary care management for low back pain compared with current best practice within risk-defined subgroups." Annals of the Rheumatic Diseases 71, no. 11 (April 4, 2012): 1796–802. http://dx.doi.org/10.1136/annrheumdis-2011-200731.
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ObjectivesStratified management for low back pain according to patients' prognosis and matched care pathways has been shown to be an effective treatment approach in primary care. The aim of this within-trial study was to determine the economic implications of providing such an intervention, compared with non-stratified current best practice, within specific risk-defined subgroups (low-risk, medium-risk and high-risk).MethodsWithin a cost–utility framework, the base-case analysis estimated the incremental healthcare cost per additional quality-adjusted life year (QALY), using the EQ-5D to generate QALYs, for each risk-defined subgroup. Uncertainty was explored with cost–utility planes and acceptability curves. Sensitivity analyses were performed to consider alternative costing methodologies, including the assessment of societal loss relating to work absence and the incorporation of generic (ie, non-back pain) healthcare utilisation.ResultsThe stratified management approach was a cost-effective treatment strategy compared with current best practice within each risk-defined subgroup, exhibiting dominance (greater benefit and lower costs) for medium-risk patients and acceptable incremental cost to utility ratios for low-risk and high-risk patients. The likelihood that stratified care provides a cost-effective use of resources exceeds 90% at willingness-to-pay thresholds of £4000 (≈ 4500; $6500) per additional QALY for the medium-risk and high-risk groups. Patients receiving stratified care also reported fewer back pain-related days off work in all three subgroups.ConclusionsCompared with current best practice, stratified primary care management for low back pain provides a highly cost-effective use of resources across all risk-defined subgroups.
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Earle, C. C., R. H. Chapman, C. S. Baker, C. M. Bell, P. W. Stone, E. A. Sandberg, and P. J. Neumann. "Systematic Overview of Cost-Utility Assessments in Oncology." Journal of Clinical Oncology 18, no. 18 (September 18, 2000): 3302–17. http://dx.doi.org/10.1200/jco.2000.18.18.3302.
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PURPOSE: Cost-utility analyses (CUAs) present the value of an intervention as the ratio of its incremental cost divided by its incremental survival benefit, with survival weighted by utilities to produce quality-adjusted life years (QALYs). We critically reviewed the CUA literature and its role in informing clinical oncology practice, research priorities, and policy. METHODS: The English-language literature was searched between 1975 and1997 for CUAs. Two readers abstracted from each article descriptions of the clinical situation and patients, the methods used, study perspective, the measures of effectiveness, costs included, discounting, and whether sensitivity analyses were performed. The readers then made subjective quality assessments. We also extracted utility values from the reviewed papers, along with information on how and from whom utilities were measured. RESULTS: Our search yielded 40 studies, which described 263 health states and presented 89 cost-utility ratios. Both the number and quality of studies increased over time. However, many studies are at variance with current standards. Only 20% of studies took a societal perspective, more than a third failed to discount both the costs and QALYs, and utilities were often simply estimates from the investigators or other physicians. CONCLUSION: The cost-utility literature in oncology is not large but is rapidly expanding. There remains much room for improvement in the methodological rigor with which utilities are measured. Considering quality-of-life effects by incorporating utilities into economic studies is particularly important in oncology, where many therapies obtain modest improvements in response or survival at the expense of nontrivial toxicity.
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Rejas-Gutiérrez, J., E. Bruguera, and S. Cedillo. "Modelling a budgetary impact analysis for funding drug-based smoking cessation therapies for patients with major depressive disorder in Spain." European Psychiatry 45 (September 2017): 41–49. http://dx.doi.org/10.1016/j.eurpsy.2017.05.027.
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AbstractBackground:Smoking is associated with high healthcare resource utilisation and cost to society. Patients with major depressive disorder (MDD) exhibit high susceptibility to nicotine dependence. Varenicline, bupropion and nicotine replacement therapy are all indicated for smoking cessation; however funding by the Spanish national health system (SNHS) is limited. We modelled a budgetary impact analysis (BIA) to estimate the impact of the SNHS funding drug-based therapies for smoking cessation in smokers with MDD.Methods:The BIA compared the current unfunded scenario versus a funded scenario (varenicline, bupropion, nicotine replacement therapy combined with medical follow-up and counselling) using the Spanish SNHS and societal perspectives. The BIA design was a hybrid model using a decision tree algorithm (population size: smokers with MDD) and Markov chains (smoking cessation attempts) over a 5-year horizon. Smoking cessation drug efficacy was derived from clinical trials, and smoking cessation costs avoided were taken from an analysis of the Spanish National Health Survey. Results were shown as incremental cost savings. Scenarios and threshold univariate sensitivity analyses tested model robustness.Results:The funded scenario resulted in an increase of 43,478 cessation attempts and 8930 fewer smokers after 5 years compared to the unfunded scenario. The cost of funding was €25.3 million and costs avoided were €26.5 million. There was a cumulative 5-year incremental cost saving of €1.2 million to Spanish society. Results were robust using alternative scenarios.Conclusions:Funding smoking cessation drugs in patients with MDD is of economic benefit to Spain and could produce net savings from the third year of implementation.
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Teng, Monica, Hui Jun Zhou, Liang Lin, Pang Hung Lim, Doreen Yeo, Suzanne Goh, Soon Yin Tjan, and Boon Peng Lim. "Cost-effectiveness of hydrotherapy versus land-based therapy in patients with musculoskeletal disorders in Singapore." Journal of Public Health 41, no. 2 (March 9, 2018): 391–98. http://dx.doi.org/10.1093/pubmed/fdy044.
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Abstract Background The study evaluated the cost-effectiveness of hydrotherapy versus land-based therapy in patients with musculoskeletal disorders (MSDs) in Singapore. Methods A decision-analytic model was constructed to compare the cost-effectiveness of hydrotherapy to land-based therapy over 3 months from societal perspective. Target population comprised patients with low back pain (LBP), osteoarthritis (OA), rheumatoid arthritis (RA), total hip replacement (THR) and total knee replacement (TKR). Subgroup analyses were carried out to determine the cost-effectiveness of hydrotherapy in individual MSDs. Relative treatment effects were obtained through a systematic review of published data. Results Compared to land-based therapy, hydrotherapy was associated with an incremental cost-effectiveness ratio (ICER) of SGD 27 471 per quality-adjusted life-year (QALY) gained, which was below the willingness-to-pay threshold of SGD 70 000 per QALY (one gross domestic product per capita in Singapore in 2015). For the respective MSDs, hydrotherapy were dominant (more effective and less costly) in THR and TKR, cost-effective for LBP and RA, and not cost-effective for OA. Treatment adherence and cost of hydrotherapy were key drivers to the ICER values. Conclusions Hydrotherapy was a cost-effective rehabilitation compared to land-based therapy for a population with MSDs in Singapore. However, the benefit of hydrotherapy was not observed in patients with OA.
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Hayman, James A., Bruce E. Hillner, Jay R. Harris, Lori J. Pierce, and Jane C. Weeks. "Cost-Effectiveness of Adding an Electron-Beam Boost to Tangential Radiation Therapy in Patients With Negative Margins After Conservative Surgery for Early-Stage Breast Cancer." Journal of Clinical Oncology 18, no. 2 (January 1, 2000): 287. http://dx.doi.org/10.1200/jco.2000.18.2.287.
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PURPOSE: Electron-beam boosts (EBB) are routinely added after conservative surgery and tangential radiation therapy (TRT) for early-stage breast cancer. We performed an incremental cost-utility analysis to evaluate their cost-effectiveness. METHODS: A Markov model examined the impact of adding an EBB to TRT from a societal perspective. Outcomes were measured in quality-adjusted life years (QALYs). On the basis of the Lyon trial, the EBB was assumed to reduce local recurrences by approximately 2% at 10 years but to have no impact on survival. Patients’ utilities were used to adjust for quality of life. Given the small absolute benefit of the EBB, baseline utilities were assumed to be the same with or without it, an assumption evaluated by Monte Carlo simulation. Direct medical, time, and travel costs were considered. RESULTS: Adding the EBB led to an additional cost of $2,008, an increase of 0.0065 QALYs and, therefore, an incremental cost-effectiveness ratio of over $300,000/QALY. In a sensitivity analysis, the ratio was moderately sensitive to the efficacy and cost of the EBB and highly sensitive to patients’ utilities for treatment without it. Even if patients do value a small risk reduction, the mean cost-effectiveness ratio estimated by the Monte Carlo simulation remains high, at $70,859/QALY (95% confidence interval, $53,141 to $105,182/QALY). CONCLUSION: On the basis of currently available data, the cost-effectiveness ratio for the EBB is well above the commonly cited threshold for cost-effective care ($50,000/QALY). The EBB becomes cost-effective only if patients place an unexpectedly high value on the small absolute reduction in local recurrences achievable with it.
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Degli Esposti, Ezio, and Radovan Tomic. "Clinical and economic impact of aliskiren in uncontrolled hypertensive patients." Farmeconomia. Health economics and therapeutic pathways 13, no. 1 (March 14, 2012): 15–23. http://dx.doi.org/10.7175/fe.v13i1.199.
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BACKGROUND: the majority of hypertensive patients do not achieve adequate blood pressure (BP) control and thus remain at risk of cardio-cerebrovascular events. Aliskiren, a novel antihypertensive drug acting as direct renin inhibitor, was authorized in Italy for the treatment of hypertension in patients who remain uncontrolled and at risk despite the use of at least two antihypertensive drugs. It was subject to an AIFA web-based monitoring registry. Results of the registry show a decrease of 20.8/9.2 mmHg in systolic/diastolic BP, within 6 months, when aliskiren is added to current therapy.OBJECTIVE: to evaluate the clinical and economic impact of such BP reduction in terms of avoidable cardio-cerebrovascular events.METHODS: an Excel-based Markov model compared aliskiren plus current antihypertensive treatment to current antihypertensive treatment alone over a 5-year horizon. Patients’ baseline characteristics and BP-reduction were taken from the AIFA registry and literature. Using Wilson and Anderson risk equations, the model simulated patient’s transitions from Pre-Event to Post-Event and Death, calculating the number of those who experience an event. Unit costs were assigned to treatments, events and follow-up. Sensitivity analyses considered: efficacy variability and societal costs of events.RESULTS: 2.47% of patients treated with aliskiren added-on to their antihypertensive therapy were expected to avoid an event. As observed in the AIFA registry, 19.8% of patients remained treated only with aliskiren whereas others reduced the number of antihypertensive treatments, leading to a 38.6% reduction of monthly concomitant antihypertensive treatment cost. Considering events and follow-up cost reduction, the per-patient annual incremental cost of aliskiren is calculated at € 187 and generates 0.042 QALYs over 5 years. The ICER was € 22,062 per QALY (€ 16,845 to € 30,771 for an efficacy range of ± 25%). Considering societal costs ICER was € 20,094 per QALY.Conclusions: AIFA registry real-world data confirmed aliskiren’s efficacy in uncontrolled hypertensive patients. Together with reaching their BP goals, patients consumed less medication from other antihypertensive drug classes and are expected to avoid cardio-cerebrovascular events. The ICER remained within acceptable thresholds, confirming that aliskiren represents a good investment in terms of health benefit.
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Webers, C., S. Grimm, A. Van Tubergen, F. A. Van Gaalen, D. Van der Heijde, M. Joore, and A. Boonen. "THU0549 COST-EFFECTIVENESS OF DIAGNOSING AXIAL SPONDYLOARTHRITIS IN THE NETHERLANDS." Annals of the Rheumatic Diseases 79, Suppl 1 (June 2020): 514.1–515. http://dx.doi.org/10.1136/annrheumdis-2020-eular.1065.
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Background:In order to facilitate diagnosis of axial spondyloarthritis (axSpA) in clinical practice, three diagnostic algorithms have been published: the original Berlin algorithm (BER), and modifications 1 (M1) and 2 (M2). These mainly differed mainly in the position and definition of inflammatory back pain (Figure). BER was the most specific of these algorithms, while M2 was most sensitive, and the latter was accepted as the algorithm of choice. However, it is unknown to what extent it is acceptable, in terms of costs and effects, to increase sensitivity (i.e. reducing underdiagnosis and undertreatment) at the expense of specificity (i.e. increasing overdiagnosis and overtreatment).Figure.Flowchart of the Berlin algorithm (A), and Modifications 1 (B) and 2 (C)Objectives:To compare lifetime cost-effectiveness of currently available diagnostic algorithms for axSpA in the Netherlands.Methods:A cost-effectiveness model was developed to estimate costs, quality adjusted life years (QALYs) and net monetary benefit (NMB) of different diagnostic algorithms for axSpA among patients with chronic low back pain (CLBP) referred to a rheumatologist, over a 60-year time horizon. The model combined a decision-tree (diagnostic process) with a state-transition model (long-term treatment). For the diagnostic process, patient-level data from an observational cohort of referred CLBP patients (SPondyloArthritis Caught Early, SPACE) were used. In the state-transition model, both axSpA and CLBP were modelled using three health states each, based on disease activity and treatment (including up to three sequential biologicals) (for axSpA), or disease severity (for CLBP). Three available diagnostic algorithms (BER, M1, M2) were compared. To estimate the impact of uncertainty on results, probabilistic sensitivity analyses were conducted. For all analyses, both a societal perspective (including all healthcare, informal and work-related costs) and a healthcare perspective (only including healthcare costs) were adopted. Results were expressed as incremental cost-utility ratio (ICUR) and incremental NMB (iNMB).Results:From a societal perspective, algorithm M2 resulted in more QALYs and lowest costs per patient (24.23 QALY; €157,274), thus dominating BER and M1 (Table). From a healthcare perspective, BER had lowest costs, but M2 was still considered cost-effective at €3,250/QALY (Table). At a willingness-to-pay (WTP) threshold of €20,000 per one QALY gained, the probability of M2 being most cost-effective was 94%, compared to 5% for M1 and 1% for BER. Compared to the most cost-effective algorithm (M2), an additional €7,500 could be spent per patient to achieve perfect diagnosis while remaining cost-effective.Table.Base-case deterministic cost-utility results of comparisons between diagnostic algorithmsPerspectiveAlgorithmSE/SPCost, €QALYiCost, €iQALYICUR, €/QALYiNMB, €*SocietalM251% / 88%157,27424.23----M173% / 82%158,23624.15962-0.08Dominated by M2-2,583Berlin80% / 85%159,29523.962,021-0.27Dominated by M2-7,479HealthcareBerlin51% / 88%91,75523.96----M273% / 82%92,64224.238870.273,2504,571M180% / 85%92,71024.1567-0.08Dominated by M2-1,688*Calculated using a WTP threshold of €20,000/QALY. iCost, incremental cost; iQALY, incremental QALY; SE, sensitivity; SP, specificity.Conclusion:The relative increase in sensitivity of M2 at the expense of specificity when compared to the original BER algorithm is acceptable in terms of costs and effects from both societal and healthcare perspectives. A considerably more expensive diagnostic algorithm with better accuracy than M2 would still be considered good value for money. It is worthy to invest in more accurate diagnosis in axSpA.Disclosure of Interests:Casper Webers: None declared, Sabine Grimm: None declared, Astrid van Tubergen Consultant of: Novartis, Floris A. van Gaalen: None declared, Désirée van der Heijde Consultant of: AbbVie, Amgen, Astellas, AstraZeneca, BMS, Boehringer Ingelheim, Celgene, Cyxone, Daiichi, Eisai, Eli-Lilly, Galapagos, Gilead Sciences, Inc., Glaxo-Smith-Kline, Janssen, Merck, Novartis, Pfizer, Regeneron, Roche, Sanofi, Takeda, UCB Pharma; Director of Imaging Rheumatology BV, Manuela Joore: None declared, Annelies Boonen Grant/research support from: AbbVie, Consultant of: Galapagos, Lilly (all paid to the department)
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Grusenmeyer, Patrick A., and Yu-Ning Wong. "Interpreting the Economic Literature in Oncology." Journal of Clinical Oncology 25, no. 2 (January 10, 2007): 196–202. http://dx.doi.org/10.1200/jco.2006.09.0738.
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New treatment options provide hope for patients with localized and advanced cancer. However, these advances are associated with cost, both in terms of treatment-related expenditures and effects on quality of life. It is important that patients, physicians, insurers, and policymakers understand the relationship between costs and outcomes of new cancer treatments. Various methods of cost analysis can provide a structured manner to assess cost. Cost-effectiveness analysis (CEA) compares the cost of the intervention with the effect, resulting in a cost per effect (eg, cost per year of life gained) that can be compared across interventions. In this article, we review three recent CEAs in the oncology literature, including chemoprevention in breast cancer, adjuvant endocrine therapy in early-stage breast cancer, and salvage chemotherapy in advanced ovarian cancer. The important elements of CEA, including the recommendations of the US Public Health Service Panel on Cost Effectiveness in Health and Medicine as they relate to cancer treatments, are discussed. Many well-done CEAs in cancer treatment have been performed during the last decade. As with clinical trials, the rigor and methods of the analysis are critical to the reliability of the results. Therapies with high cost and small incremental improvement in survival and/or quality of life may find it difficult to meet the societal thresholds for what is considered cost effective. CEA is a method to assess the cost and effect of cancer treatments, providing important insights into the best use (ie, obtaining the most value for) of health care expenditures. As the literature indicates, one must be cognizant of the fact that there can be extraordinary costs associated with some newer cancer therapies that provide small incremental clinical benefit. Better understanding of the cancer economic literature can help lead to an informed dialogue on the health policy implications of resource allocation in cancer care.
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Gomes, Manuel, Mark Pennington, Raphael Wittenberg, Martin Knapp, Nick Black, and Sarah Smith. "Cost-effectiveness of Memory Assessment Services for the diagnosis and early support of patients with dementia in England." Journal of Health Services Research & Policy 22, no. 4 (June 16, 2017): 226–35. http://dx.doi.org/10.1177/1355819617714816.
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Background Policy makers in England advocate referral of patients with suspected dementia to Memory Assessment Services (MAS), but it is unclear how any improvement in patients’ health-related quality of life (HRQL) compares with the associated costs. Aims To evaluate the cost-effectiveness of MAS for the diagnosis and follow-up care of patients with suspected dementia. Method We analysed observational data from 1318 patients referred to 69 MAS, and their lay carers (n = 944), who completed resource use and HRQL questionnaires at baseline, three and six months. We reported mean differences in HRQL (disease-specific DEMQOL and generic EQ-5D-3L), quality-adjusted life years (QALYs) and costs between baseline and six months after referral to MAS. We also assessed the cost-effectiveness of MAS across different patient subgroups and clinic characteristics. Results Referral to MAS was associated with gains in DEMQOL (mean gain: 3.48, 95% confidence interval: 2.84 to 4.12), EQ-5D-3L (0.023, 0.008 to 0.038) and QALYs (0.006, 0.002 to 0.01). Mean total cost over six months, assuming a societal perspective, was £1899 (£1277 to £2539). This yielded a negative incremental net monetary benefit of −£1724 (−£2388 to −£1085), assuming NICE’s recommended willingness-to-pay threshold (£30,000 per QALY). These base case results were relatively robust to alternative assumptions about costs and HRQL. There was some evidence that patients aged 80 or older benefitted more from referral to MAS (p < 0.01 from adjusted mean differences in net benefits) compared to younger patients. MAS with over 75 new patients a month or cost per patient less than £2500 over six months were relatively more cost-effective (p < 0.01) than MAS with fewer new monthly patients or higher cost per patient. Conclusions Diagnosis, treatment and follow-up care provided by MAS to patients with suspected dementia appears to be effective, but not cost-effective, in the six months after diagnosis. Longer term evidence is required before drawing conclusions about the cost-effectiveness of MAS.
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Backhaus, Insa, Alice Mannocci, and Giuseppe La Torre. "A Systematic Review of Economic Evaluation Studies of Drug-Based Non-Malignant Chronic Pain Treatment." Current Pharmaceutical Biotechnology 20, no. 11 (September 30, 2019): 910–19. http://dx.doi.org/10.2174/1389201020666190717095443.
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Background and Objective: Chronic pain is a highly prevalent problem, involving high costs and seriously affecting a patient's quality of life. This review aimed to systematically review economic evaluations of pharmacological-based treatments for non-malignant chronic pain and to compare different treatment approaches with regard to their economic profile. Methods: PubMed and Scopus were systematically searched in April 2016. Studies were included if quality-adjusted life years and incremental cost-effectiveness ratios were reported. Quality assessment was carried out by using La Torre’s weighted scale on the Drummond checklist. Costs were converted into US$2014. Results: Fourteen economic evaluations met the inclusion criteria. Three treatment categories identified were: opioids, anticonvulsants, and anti-depressants. Compared to anticonvulsants and antidepressant, opioids had lower ICER. Transdermal buprenorphine showed an ICER of about US$11,000.00 while pregabalin showed an ICER of US$19,200. Studies included showed a diversity of methodological approaches, such as different modeling approaches and different perspectives (NHS and private payer). Conclusion: There are limitations to the success of making appropriate recommendations about which treatment is most cost-effective due to considerable variability between treatments, pain syndromes, and drug dosages. Opioids may have lower ICER, but the societal implications of the opioid epidemic and overdose deaths should be taken into account when coming to general conclusions about their cost-effectiveness. To ensure correct resource allocation as well as the best benefit for patients, uniform and standardized approaches of cost and outcome measurement in economic evaluations of chronic are needed.
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Ito, Kouta, Victoria S. Blinder, and Elena B. Elkin. "Cost Effectiveness of Fracture Prevention in Postmenopausal Women Who Receive Aromatase Inhibitors for Early Breast Cancer." Journal of Clinical Oncology 30, no. 13 (May 1, 2012): 1468–75. http://dx.doi.org/10.1200/jco.2011.38.7001.
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Purpose Aromatase inhibitors (AIs) increase the risk of osteoporosis and related fractures in postmenopausal women who receive adjuvant AIs for hormone receptor (HR) –positive early breast cancer (EBC). We compared the cost effectiveness of alternative screening and treatment strategies for fracture prevention. Methods We developed a Markov state transition model to simulate clinical practice and outcomes in a hypothetical cohort of women age 60 years with HR-positive EBC starting a 5-year course of AI therapy after primary surgery for breast cancer. Outcomes were quality-adjusted life-years (QALYs), lifetime cost, and incremental cost-effectiveness ratio (ICER). We compared the following strategies: no intervention; one-time bone mineral density (BMD) screening and selective bisphosphonate therapy in women with osteoporosis or osteopenia; annual BMD screening and selective bisphosphonate therapy in women with osteoporosis or osteopenia; and universal bisphosphonate therapy. Results ICERs for annual BMD screening followed by oral bisphosphonates for those with osteoporosis, annual BMD screening followed by oral bisphosphonates for those with osteopenia, and universal treatment with oral bisphosphonates were $87,300, $129,300, and $283,600 per QALY gained, respectively. One-time BMD screening followed by oral bisphosphonates for those with osteoporosis or osteopenia was dominated. Our results were sensitive to age at the initiation of AI therapy, type of bisphosphonates, post-treatment residual effect of bisphosphonates, and a potential adjuvant benefit of intravenous bisphosphonates. Conclusion In postmenopausal women receiving adjuvant AIs for HR-positive EBC, a policy of baseline and annual BMD screening followed by selective treatment with oral bisphosphonates for those diagnosed with osteoporosis is a cost-effective use of societal resources.
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Nguyen, Trang, Kim Sweeny, Thach Tran, Stanley Luchters, David B. Hipgrave, Sarah Hanieh, Tuan Tran, Ha Tran, Beverley-Ann Biggs, and Jane Fisher. "Protocol for an economic evaluation alongside a cluster randomised controlled trial: cost-effectiveness of Learning Clubs, a multicomponent intervention to improve women’s health and infant’s health and development in Vietnam." BMJ Open 9, no. 12 (December 2019): e031721. http://dx.doi.org/10.1136/bmjopen-2019-031721.
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IntroductionEconomic evaluations of complex interventions in early child development are required to guide policy and programme development, but a few are yet available.Methods and analysisAlthough significant gains have been made in maternal and child health in resource-constrained environments, this has mainly been concentrated on improving physical health. The Learning Clubs programme addresses both physical and mental child and maternal health. This study is an economic evaluation of a cluster randomised controlled trial of the impact of the Learning Clubs programme in Vietnam. It will be conducted from a societal perspective and aims to identify the cost-effectiveness and the economic and social returns of the intervention. A total of 1008 pregnant women recruited from 84 communes in a rural province in Vietnam will be included in the evaluation. Health and cost data will be gathered at three stages of the trial and used to calculate incremental cost-effectiveness ratios per percentage point improvement of infant’s development, infant’s health and maternal common mental disorders expressed in quality-adjusted life years gained. The return on investment will be calculated based on improvements in productivity, the results being expressed as benefit–cost ratios.Ethics and disseminationThe trial was approved by Monash University Human Research Ethics Committee (Certificate Number 2016–0683), Australia, and approval was extended to include the economic evaluation (Amendment Review Number 2018-0683-23806); and the Institutional Review Board of the Hanoi School of Public Health (Certificate Number 017-377IDD- YTCC), Vietnam. Results will be disseminated through academic journals and conference presentations.Trial registration numberACTRN12617000442303.
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Xu, Yifan, Joel W. Hay, Heinz-Josef Lenz, Sarmad Sadeghi, and Afsaneh Barzi. "Comparative effectiveness of panitumumab (P) and cetuximab (C) in metastatic colorectal cancer (mCRC) with wild-type KRAS (WTKRAS)." Journal of Clinical Oncology 34, no. 4_suppl (February 1, 2016): 652. http://dx.doi.org/10.1200/jco.2016.34.4_suppl.652.
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652 Background: In 2015, 140,000 patients will be diagnosed with colorectal cancer (CRC) with 20% diagnosed with mCRC and another 20% developing mCRC during their course. In recent years the outcome of mCRC has improved with survival reaching 30 months in clinical trials. P and C have similar effectiveness as reported in ASPECCT, though their administration and toxicities are different. Availability of more than one agent for one indication makes cost a variable in selection process. We aimed to evaluate the comparative effectiveness of P and C as monotherapy in chemo-refractory mCRC with WTKRAS from a societal perspective. Methods: We developed a Markov model with three states: stable disease, progressive disease and death. Probabilities of each state and transitions were extracted from the ASPECCT trial report. Costs of drug and administration were based on the Veteran Affairs Federal Supply Schedule and Medicare reimbursement rates for a base case of a 61-year-old male. We conducted a literature review to obtain data regarding the costs and utilities of toxicities. All costs are in 2015 US dollars. The model was constructed using a 1-month cycle length and 3-year time horizon, discounted at an annual rate of 3%. The model used quality-adjusted life-year (QALY) to measure the health outcomes for each treatment option. Results: P produced 9.8 months life-years gain and 0.56 QALYs at the cost of $74,827 per patient. C produced a 9.2 months life-years gain and 0.53 QALYs, at the cost of $79,916 per patient. The incremental net monetary benefit of P is $10,168 when compared to C under a societal willingness-to-pay threshold of $150,000 per QALY. The model showed robustness to the one-way sensitivity analysis and various alternative scenarios, and was found to be most sensitive to the cost of C. P and C are equivalent when cost of C drops from $1,038 to $782 per 100mL vial. Conclusions: In the US, effectiveness and toxicities are the main variables for choice of treatment regimen. The rising cost of care is forcing providers and policy makers to consider other data points in treatment selection. Our results indicate that P has a cost advantage over C and provide a strong argument to use P in lieu of C in mCRC.
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Bhaskara, Srikanta, and Kamaljit S. Bawa. "Societal Digital Platforms for Sustainability: Agriculture." Sustainability 13, no. 9 (April 30, 2021): 5048. http://dx.doi.org/10.3390/su13095048.
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During the last several decades, international and national agricultural research infrastructures have rapidly expanded, bringing the outputs of agricultural research to the world’s farmers. However, despite huge investments in agricultural research, there have been few systematic efforts to create digital platforms to meet the information requirements of farmers in a changing world. We describe an interactive information system in real time to provide agricultural information to farmers. The goals were to increase yields, reduce or optimize farm inputs, inform farmers about markets and government policies, and enable digital literacy among farmers, which (in the long run) would enhance rural incomes. Farmer clubs were created at the village level to increase engagement in the program and to access information. A call-in help center enabled farmers to get information in real time. In addition, a digital platform named eKisaan delivered relevant and contextual information in the local language, mostly in the video format via mobile and cloud technologies. The platform provided information about crop management and a variety of other parameters. The combined incremental savings and incremental earnings resulted in an estimated increase of 15% in income after 18 months, totaling INR₹26,250,000 (US$365,000), followed by an additional increase of 7% in the third year. The approximate cost of the information technology program and help center was INR₹15,000,000 (US$208,000). Over time, costs can decrease by spreading fixed costs over several years, with benefits reaching more farmers. Thus, the digital systems focused on information alone can be cost-effective, reduce inputs, increase productivity and income, and foster sustainability.
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Van Mulligen, E., A. Weel, M. Kuijper, J. Hazes, and P. De Jong. "OP0281 TWO-YEAR COST-EFFECTIVENESS BETWEEN TWO GRADUAL TAPERING STRATEGIES IN RHEUMATOID ARTHRITIS: COST-UTILITY ANALYSIS OF THE TARA TRIAL." Annals of the Rheumatic Diseases 79, Suppl 1 (June 2020): 175.2–175. http://dx.doi.org/10.1136/annrheumdis-2020-eular.740.
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Background:Current guidelines recommend to consider tapering treatment in rheumatoid arthritis (RA) patients who are in sustained remission, but the optimal approach to de-escalate conventional synthetic and biological DMARDs (respectively csDMARDs and bDMARDs) remains unknown. The benefits of tapering are a decreased risk of long-term adverse events and a reduction of health care costs, especially when bDMARDs are tapered. However, tapering treatment may lead to more transient or persistent disease flares, which have a direct impact on patients’ lives and societal costs.Objectives:To evaluate the two year cost-utility ratio between tapering the csDMARD first followed by the TNF-inhibitor, and tapering the TNF-inhibitor first followed by the csDMARD.Methods:The TARA trial is a multicenter single-blinded randomized controlled trial. RA patients that used a csDMARD(s) plus a TNF-inhibitor and who had a well-controlled disease for at least 3 months, defined as a DAS≤2.4 and a swollen joint count (SJC)≤1, were included. Patients were randomized into gradual tapering their csDMARD followed by the TNF-inhibitor or vice versa. Medication was tapered in three steps over the course of 6 months. Gradual tapering was done by cutting the dosage into half, a quarter and thereafter it was stopped. Data on QALYs (measured with the Dutch EuroQol [EQ5D]), direct and indirect costs were used to calculate the Incremental Cost Effectiveness Ratio (ICER). The incremental cost-effectiveness ratio (ICER) and the incremental net monetary benefit (iNMB) were used to assess cost-effectiveness between both tapering strategies. Direct costs comprises costs for treatment and medical consumption, while indirect costs comprises costs due to loss of productivity (i.e. sick leave and unemployment).Results:Of the 189 included patients, 94 started tapering their TNF-inhibitor first, while the other 95 tapered their csDMARD first. QALYs (sd) were, respectively, 1.64 (0.22) and 1.65 (0.22). Medication costs were significantly lower in the patients who tapered the TNF-inhibitor first, but indirect cost were higher due to more productivity loss. Therefore, total costs per QALY were similar for both tapering strategies (p=0.62). The ICER between tapering csDMARDs and the TNF-inhibitor was -€184534 (-€417314, €48245; 95% CI)(figure 1). The mean iNMB was €2831 at a willingness-to-pay (WTP) level of €80000. At all WTP levels the probability of being cost-effective was higher (62% vs. 28%) for tapering the TNF-inhibitor first (figure 2)Conclusion:Medication costs are lower when the TNF-inhibitor is tapered first, but this is counterbalanced by higher indirect costs due to loss of productivity. Therefore, overall cost savings are smilar for both tapering strategies. However, tapering the TNF-inhibitor first has a higher chance of being cost-effective at all WTP thresholds. For this reason we advise to taper the TNF-inhibitor first when tapering medication is considered.Disclosure of Interests:None declared
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Garrison, Louis, Joseph Babigumira, Anthony Masaquel, Bruce Wang, Deepa Lalla, and Melissa Brammer. "The lifetime economic burden of inaccurate HER2 testing: Comparing false positive and false negative HER2+ early breast cancer patients in the United States." Journal of Clinical Oncology 31, no. 15_suppl (May 20, 2013): 6626. http://dx.doi.org/10.1200/jco.2013.31.15_suppl.6626.
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6626 Background: Trastuzumab is administered to patients with early breast cancer (EBC) whose tumors test positive for HER2 using IHC or FISH diagnostic tests. However, due to test characteristics and testing heterogeneity, patients may be misdiagnosed as false positive (FP) or false negative (FN). This analysis estimates the lifetime economic burden of inaccurate HER2 testing in the US. Methods: We developed a national-level economic model to estimate the impact on healthcare costs and quality-adjusted life years (QALYs) in both groups in 2012. The model estimates the expected number of FP and FN patients using literature-derived estimates of each test’s sensitivity, specificity, and utilization. Based on estimates from the literature, a FP patient would generate unneeded trastuzumab costs of about $56,000 and experience a chance (2.9%) of related cardiotoxicity; an FN patient would save $56,000 in trastuzumab costs, but lose 1.7 QALYs of life expectancy and face a greater likelihood of recurrence and associated costs ($42,000) to treat metastatic disease. A net monetary benefit approach (valuing healthy life years at $100,000) is used to compare the lifetime economic burden for FP and FN. Results: The estimated overall proportions of FP and FN are 2.8% and 2.2% of 227,000 EBC patients, resulting in about 6,400 and 5,000 women in each group, respectively. Overall, approximately 8600 QALYs would be lost among FN patients who do not receive trastuzumab. We estimate the incremental per-patient lifetime burden of an FP to be about $57,000, and for an FN to be about $118,000. The implied incremental loss to society for FPs is $362 million and for FNs is $596 million. Conclusions: Current testing practices and treatment patterns for HER2+ EBC patients result in misdiagnosis and non-optimal treatment in approximately 11,500 patients each year: the combined total economic loss to society is nearly $1 billion. The greater share of the loss is among FN patients who would have benefited from trastuzumab, but did not receive it. The significant annual burden of HER2 misdiagnosis suggests that substantial societal investments to improve HER2 test accuracy should be considered.
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Moltó, A., C. López-Medina, F. Van den Bosch, A. Boonen, C. Webers, E. Dernis, F. A. Van Gaalen, et al. "THU0370 CLUSTER-RANDOMIZED PRAGMATIC CLINICAL TRIAL EVALUATING THE POTENTIAL BENEFIT OF A TIGHT-CONTROL AND TREAT-TO-TARGET STRATEGY IN AXIAL SPONDYLOARTHRITIS: THE RESULTS OF THE TICOSPA TRIAL." Annals of the Rheumatic Diseases 79, Suppl 1 (June 2020): 417.1–417. http://dx.doi.org/10.1136/annrheumdis-2020-eular.1543.
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Background:Current recommendations for axial spondyloarthritis (axSpA) management include tight control and treat-to-target (TC) strategies, but no study has evaluated its potential benefitObjectives:To evaluate the benefit of TC strategies in comparison to usual care (UC) in patients with axSpA.Methods:Study design:Pragmatic, prospective, cluster-randomized controlled (2 arms), one-year trial (NCT03043846).Centers: 18 axSpA expert centers randomly allocated (1:1) to the treatment arm: TC vs. UC.Patients: axSpA diagnosis and ASAS criteria, non-optimally treated with NSAIDs, bDMARD-naïve, and ASDAS > 2.1 at inclusion.Study treatment:a) TC arm: the strategy was pre-specified by the scientific committee based on current axSpA recommendations and aiming at a target (ASDAS <2.1); visits every 4w;b) UC arm:treatment decisions were at the rheumatologist’s discretion with visits every 12w.Outcomes:the % of patients with a significant (>30%) improvement in the ASAS-HI score over one-year follow-up was the main outcome. Other outcomes (disease activity, quality of life, treatment, …) over follow-up were evaluated (Table 1). The number/type of adverse events were collected.Statistical analysis: this was an intention-to-treat analysis. To take into account the cluster-randomization design, for all outcomes, two models were performed: first a two-level mixed model with 2 random effects was used to estimate the % of responders/the change of the outcome over follow-up (i.e. mod1); in a second step, the imbalanced variables observed at baseline were included in the model (i.e.mod2). Cost-effectiveness was assessed by estimating the (baseline- and cluster-adjusted) incremental cost per quality-adjusted life-year (QALY) gained for TC vs. UC.Estimated outcomes at week 48Cluster-adjusted (mod1)Cluster and imbalance-adjusted (mod2)TCUCASDAS LDA*76.5%59.5%<0.010.03ASDAS ID25.9%18.7%--ASDAS CII61.2%46.0%<0.010.02ASDAS MI16.5%14.9%--ASAS4052.3%34.7%<0.010.01ASAS2094.9%85.9%<0.010.03BASDAI 5079.0%43.8%0.010.03Physician Global (0-10)2.0 (0.2)1.8 (0.2)--CRP (mg/L)3.9(1.4)3.5 (1.5)--BASG (0-10)2.6 (0.5)3.4 (0.5)0.09-BASFI (0-10)1.7(0.5)2.4 (0.5)--ASAS HI SMD47.3%36.1%--EQ5D0.7(0.1)0.8(0.1)0.02-ASAS-NSAID score1.5(2.2)- 4.9 (2.9)--Results:160 patients were included (80 in TC and 80 in UC). Mean age was 37.9(11.0) years with a disease duration of 3.7(6.2) years, 51.2% were males. A radiographic damage of the SI-joints, a (ever) positive MRI sacroiliitis and HLA-B27+ were seen in 46.9%, 81.9% and 75.0% patients respectively. Mean ASDAS at inclusion was 3.0 (0.7) and mean ASASHI was 8.6 (3.7). 72 patients per group attended the one-year visit. Although 47.3% vs. 36.1% patients in the TC and UC arms achieved a significant improvement in ASASHI at the one-year visit, the difference was not statistically significant, with either model. Across all other outcomes a trend was observed in favor of the TC arm (Table 1). The number of bDMARDs was significantly higher in TC arm (56.2% vs. 27.2%). The number of infections was comparable in both groups (15 vs. 16 in the TC and UC, respectively), with only 2 severe infections occurring in the UC arm. From a societal perspective, TC resulted in an additional 0.04 QALY and saved €265 when compared to UC and a 67% probability of being cost-effective at a cost-effectiveness threshold of €20,000 per QALY.Conclusion:In this setting of SpA expert centers, UC resulted in a good outcome in a substantial number of patients but the TC was not superior for the primary outcome despite a greater number of bDMARDs prescription. Nevertheless, a general trend in favor of the tight control was observed, with a comparable safety profile and was found to be favorable from a societal health economic perspective.Acknowledgments:this trial has been conducted thanks to an unrestricted grant from UCBDisclosure of Interests:Anna Moltó Grant/research support from: Pfizer, UCB, Consultant of: Abbvie, BMS, MSD, Novartis, Pfizer, UCB, Clementina López-Medina: None declared, Filip van den Bosch Consultant of: AbbVie, Celgene Corporation, Eli Lilly, Galapagos, Janssen, Novartis, Pfizer, and UCB, Speakers bureau: AbbVie, Celgene Corporation, Eli Lilly, Galapagos, Janssen, Novartis, Pfizer, and UCB, Annelies Boonen Grant/research support from: AbbVie, Consultant of: Galapagos, Lilly (all paid to the department), Casper Webers: None declared, Emmanuelle Dernis Speakers bureau: Lilly, Novartis, Floris A. van Gaalen: None declared, Martin SOUBRIER: None declared, Pascal Claudepierre Speakers bureau: Janssen, Novartis, Lilly, Athan Baillet Consultant of: Athan BAILLET has received honorarium fees from Abbvie for his participation as the coordinator of the systematic literature review, Mirian Starmans-Kool: None declared, Désirée van der Heijde Consultant of: AbbVie, Amgen, Astellas, AstraZeneca, BMS, Boehringer Ingelheim, Celgene, Cyxone, Daiichi, Eisai, Eli-Lilly, Galapagos, Gilead Sciences, Inc., Glaxo-Smith-Kline, Janssen, Merck, Novartis, Pfizer, Regeneron, Roche, Sanofi, Takeda, UCB Pharma; Director of Imaging Rheumatology BV, Maxime Dougados Grant/research support from: AbbVie, Eli Lilly, Merck, Novartis, Pfizer and UCB Pharma, Consultant of: AbbVie, Eli Lilly, Merck, Novartis, Pfizer and UCB Pharma, Speakers bureau: AbbVie, Eli Lilly, Merck, Novartis, Pfizer and UCB Pharma
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Jongeneel, Gabrielle, Marjolein J. E. Greuter, Felice N. van Erning, Miriam Koopman, Geraldine R. Vink, Cornelis J. A. Punt, and Veerle M. H. Coupé. "Model-based evaluation of the cost effectiveness of 3 versus 6 months’ adjuvant chemotherapy in high-risk stage II colon cancer patients." Therapeutic Advances in Gastroenterology 13 (January 2020): 175628482095411. http://dx.doi.org/10.1177/1756284820954114.
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Background: Our aim was to evaluate the cost effectiveness of 3 months’ adjuvant chemotherapy versus 6 months in high-risk (T4 stage + microsatellite stable) stage II colon cancer (CC) patients. Methods: Using the validated PATTERN Markov cohort model, which simulates the disease progression of stage II CC patients from diagnosis to death, we first evaluated a reference strategy in which high-risk patients were treated with chemotherapy for 6 months. In the second strategy, treatment duration was shortened to 3 months. Both strategies were evaluated for CAPOX (capecitabine plus oxaliplatin) and FOLFOX (fluorouracil, leucovorin and oxaliplatin). Based on trial data, we assumed that shortened treatment duration compared with a 6-month regimen was equally effective for CAPOX and less effective for FOLFOX. Adverse events were highest in the 6-month strategy. Analyses were conducted from a societal perspective using a lifelong time horizon. Outcomes were number of CC deaths per 1000 patients and total discounted costs and quality-adjusted life-years (QALYs) per patient (pp). Incremental net monetary benefit (iNMB) was calculated using a willingness-to-pay value of €50,000/QALY. Results: For CAPOX, the 6-month strategy resulted in 316 CC deaths per 1000 patients, 6.71 QALYs pp and total costs of €41,257 pp. The 3-month strategy resulted in an equal number of CC deaths, but higher QALYs (6.80 pp) and lower costs (€37,645 pp), leading to a iNMB of €8454 per person for 3 months versus 6 months. For FOLFOX, the 6-month strategy resulted in 316 CC deaths per 1000 patients, 6.71 QALYs pp and total costs of €47,135 pp. The 3-month strategy resulted in more CC deaths (393), lower QALYs (6.19 pp) and lower costs (€44,389 pp). An iNMB of −€23,189 was found for 3 months versus 6 months. Conclusion: Our findings indicate that 3 months’ adjuvant chemotherapy should be considered as standard of care in high-risk stage II CC patients for CAPOX, but not for FOLFOX.
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Hyle, Emily. "Measles CEA." Open Forum Infectious Diseases 4, suppl_1 (2017): S64—S65. http://dx.doi.org/10.1093/ofid/ofx162.153.
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Abstract Background Most measles importations are due to returning US travelers infected during international travel. We projected clinical outcomes and assessed cost-effectiveness of pretravel evaluation for measles immunity and MMR vaccination among eligible adult US international travelers. Methods We designed a decision tree to investigate pretravel evaluation compared with no evaluation from the societal perspective. Data from the Global TravEpiNet Consortium and published literature informed input parameters (Figure 1). Outcomes included measles cases averted per 10 million travelers, costs, and the incremental cost-effectiveness ratio (ICER, Δcosts/Δmeasles cases averted); we considered ICERs < $100,000/measles case averted to be cost-effective. We performed sensitivity analyses to assess the impact of varying the probability of exposure based on travel destination, and the percentage of travelers with pre-existing measles immunity. Results In the base case, departure after pretravel evaluation resulted in 16 measles importations and 46 transmissions per 10 million travelers and cost $132 million, vs without pretravel evaluation (26 importations and 87 transmissions per 10 million travelers, costing $22 million). Pretravel evaluation averted 51 measles cases per 10 million travelers with an ICER of $2.2 million per case averted. Results were most sensitive to the probability of measles exposure and the traveler’s pre-existing immunity (Figure 2). Pretravel evaluation was cost-effective for travelers to Asia if pre-existing measles immunity was <80%. Evaluation was always cost-effective for travelers to Africa when pre-existing immunity was less than 100% and became cost saving when the percentage of immune travelers was lower (<70%). Travelers who were more likely to be non-immune and were visiting destinations with higher probabilities of exposure were most likely to benefit from pretravel evaluation for measles immunity at excellent economic value. Conclusion As risk of measles exposure increases and likelihood of travelers’ pre-existing immunity decreases, it can be cost-effective or cost saving to assess US international travelers’ measles immunity status and vaccinate with MMR prior to departure. Disclosures All authors: No reported disclosures.
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Booth, Christopher M., and William J. Mackillop. "Translating New Medical Therapies Into Societal Benefit." JAMA 300, no. 18 (November 12, 2008): 2177. http://dx.doi.org/10.1001/jama.300.18.2177.
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Mernagh, P., S. Campbell, M. Dietlein, M. Luster, E. Mazzaferri, and A. R. Weston. "Cost-effectiveness of using recombinant human TSH prior to radioiodine ablation for thyroid cancer, compared with treating patients in a hypothyroid state: the German perspective." European Journal of Endocrinology 155, no. 3 (September 2006): 405–14. http://dx.doi.org/10.1530/eje.1.02223.
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Objective: This investigation evaluated the cost-effectiveness of radioiodine remnant ablation following preparation with recombinant human TSH (rhTSH), compared with the standard preparation, whereby patients are rendered hypothyroid. Design: The economic evaluation relates to patients with well differentiated thyroid cancer who have undergone thyroidectomy, but have no metastases. The evaluation takes a societal perspective, considering costs and benefits to all parties. The benefits were expressed in units of quality-adjusted life years (QALY), so differences in life expectancy were captured with consideration of quality of life. Methods: A lifetime Markov model with Monte Carlo simulation of 100 000 patients was used to assess cost per QALY gained. The clinical inputs were sourced from a multi-centre, randomised controlled trial comparing remnant ablation success after rhTSH-preparation with hypothyroid preparation. The model applied German unit costs, however, the structure is generalisable to other jurisdictions. The additional cost of rhTSH procurement and administration is considered relative to the clinical benefits and cost offsets. These included avoidance of hypothyroidism, increased work productivity, earlier discharge from radioprotection and a theoretical reduction in the risk of secondary malignancy. The latter two benefits relate to faster radioiodine clearance after rhTSH preparation. Results: The additional benefits of rhTSH (0.0495 QALY) are obtained with an incremental societal cost of €47, equating to an incremental cost per QALYof €958. Sensitivity analyses had only a modest impact upon cost-effectiveness, with all one-way sensitivity results remaining under €15 000/QALY. Conclusions: The use of rhTSH prior to radioiodine ablation represents good value-for-money with the benefits to patient and society obtained at modest net cost.
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Ryan, Benjamin J., Damon Coppola, Deon V. Canyon, Mark Brickhouse, and Raymond Swienton. "COVID-19 Community Stabilization and Sustainability Framework: An Integration of the Maslow Hierarchy of Needs and Social Determinants of Health." Disaster Medicine and Public Health Preparedness 14, no. 5 (April 21, 2020): 623–29. http://dx.doi.org/10.1017/dmp.2020.109.
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ABSTRACTAll levels of government are authorized to apply coronavirus disease 2019 (COVID-19) protection measures; however, they must consider how and when to ease lockdown restrictions to limit long-term societal harm and societal instability. Leaders that use a well-considered framework with an incremental approach will be able to gradually restart society while simultaneously maintaining the public health benefits achieved through lockdown measures. Economically vulnerable populations cannot endure long-term lockdown, and most countries lack the ability to maintain a full nationwide relief operation. Decision-makers need to understand this risk and how the Maslow hierarchy of needs and the social determinants of health can guide whole of society policies. Aligning decisions with societal needs will help ensure all segments of society are catered to and met while managing the crisis. This must inform the process of incremental easing of lockdowns to facilitate the resumption of community foundations, such as commerce, education, and employment in a manner that protects those most vulnerable to COVID-19. This study proposes a framework for identifying a path forward. It reflects on baseline requirements, regulations and recommendations, triggers, and implementation. Those desiring a successful recovery from the COVID-19 pandemic need to adopt an evidence-based framework now to ensure community stabilization and sustainability.