Series Of One-way Sensitivity Analyses Research Articles

A cost-effectiveness analysis of pre-pregnancy genetic screening for deafness: an empirical study in China.

This research aims to assess the effectiveness and cost-effectiveness of pre-pregnancy deafness screening policies. Married couples from Shanghai, Beijing, and Suzhou in China were enrolled. We conducted high-throughput, pre-pregnancy genetic screenings for deafness in women and their partners. We compared the cost-effectiveness of deafness genetic screening with the status quo. The two-step screening (wife then partner) and following treatments and interventions were included in the decision tree model. We conducted a cost-effectiveness analysis based on the decrease in deaf newborns, healthy newborn births, and cost-utility analysis of pre-pregnancy deafness genetic screening separately. Cost, utility, and probability data used in the three models were collected from a survey combined with literature and expert consultants. A 5% discount rate and a series of one-way sensitivity analyses along with a Monte Carlo simulation were used to test the reliability of this research. Between Jan 1, 2019, and Dec 31, 2021, we recruited 6,200 females and 540 male spouses from community health service centers in Shanghai, Beijing, and Suzhou. The incremental cost-effectiveness ratio (ICER) for reducing deaf newborn births was USD 32,656 per case and USD 1,203,926 per case for increasing one healthy newborn birth. This gap exists because of the overall decrease of newborn births. From the perspective of the whole society, deafness genetic screening is not cost-effective for reducing the overall quality-adjusted life years (QALY) in the population. Pre-pregnancy genetic testing is effective in decreasing the occurrence of congenital deafness. It is a cost-saving measure when compared with the costs of future medical expenditure and income loss for the affected families. However, such screening and preventive avoidance of pregnancy will decrease the population size and QALY. Only post-screening ART with PGT was shown to increase the birth of healthy newborns. Focusing on key groups such as premature births or consanguineous couples may improve the societal effects of screening.

Cost-effectiveness of treatment strategies for populations from strongyloidiasis high-risk areas globally who will initiate corticosteroid treatment in the United States.

The risk of developing strongyloidiasis hyperinfection syndrome appears to be elevated among individuals who initiate corticosteroid treatment. Presumptive treatment or treatment after screening for populations from Strongyloides stercoralis-endemic areas has been suggested before initiating corticosteroids. However, potential clinical and economic impacts of preventative strategies have not been evaluated. Using a decision tree model for a hypothetical cohort of 1000 individuals from S. stercoralis-endemic areas globally initiating corticosteroid treatment, we evaluated clinical and economic impacts of two interventions, 'Screen and Treat' (i.e. screening and ivermectin treatment after a positive test), and 'Presumptively Treat,' compared to current practice (i.e. 'No Intervention'). We evaluated the cost-effectiveness (net cost per death averted) of each strategy using broad ranges of pre-intervention prevalence and hospitalization rates for chronic strongyloidiasis patients initiating corticosteroid treatment. For the baseline parameter estimates, 'Presumptively Treat' was cost-effective (i.e. clinically superior with cost per death averted less than a threshold of $10.6 million per life) compared to 'No Intervention' ($532 000 per death averted) or 'Screen and Treat' ($39 000 per death averted). The two parameters contributing the most uncertainty to the analysis were the hospitalization rate for individuals with chronic strongyloidiasis who initiate corticosteroids (baseline 0.166%) and prevalence of chronic strongyloidiasis (baseline 17.3%) according to a series of one-way sensitivity analyses. For hospitalization rates greater than 0.022%, 'Presumptively Treat' would remain cost-effective. Similarly, 'Presumptively Treat' remained preferred at prevalence rates of 4% or above; 'Screen and Treat' was preferred for prevalence between 2% and 4%, and 'No Intervention' was preferred for prevalence less than 2%. The findings support decision-making for interventions for populations from S. stercoralis endemic areas before initiating corticosteroid treatment. Although some input parameters are highly uncertain and prevalence varies across endemic countries, 'Presumptively Treat' would likely be preferred across a range for many populations given plausible parameters.

Cost-Effectiveness of Care Coordination for Children With Chronic Noncomplex Medical Conditions: Results From a Multicenter Randomized Clinical Trial

ObjectivesTo assess the cost-effectiveness of care coordination, compared with standard care, for children with chronic noncomplex medical conditions. MethodsA total of 81 children aged between 2 and 15 years newly diagnosed with a noncomplex chronic condition were randomized to either care coordination or standard care as part of a multicenter randomized controlled trial. Families receiving care coordination were provided access to an Allied Health Liaison Officer, who facilitated family-centered healthcare access across hospital, education, primary care, and community sectors. Costs were estimated over a 12-month period from the perspective of the Australian health system. Health outcomes were valued as quality-adjusted life-years (QALYs). Caregiver productivity costs were included in an alternative base-case analysis, and key assumptions were tested in a series of one-way sensitivity analyses. A probabilistic sensitivity analysis was conducted to investigate the overall impact of uncertainty in the data. ResultsChildren in the intervention arm incurred an average of $17 in additional health system costs (95% confidence interval −3861 to 1558) and gained an additional 0.031 QALYs (95% confidence interval −0.29 to 0.092) over 12 months, producing an incremental cost-effectiveness ratio of $548 per QALY. When uncertainty was considered, there was a 73% likelihood that care coordination was cost-effective from a health system perspective, assuming a willingness to pay of $50 000 per QALY. This increased to 78% when caregiver productivity costs were included. ConclusionsCare coordination is likely to be a cost-effective intervention for children with chronic noncomplex medical conditions in the Australian healthcare setting.

Cost-effectiveness of once-daily vs twice-daily tacrolimus among Hispanic and Black kidney transplant recipients.

BACKGROUND: Tacrolimus is a first-line immunosuppressive therapy to prevent rejection and graft failure in kidney transplant recipients. Once-daily extended-release tacrolimus tablets (LCPT) have been shown to be efficacious, particularly for Hispanic and Black patient subpopulations who are rapid metabolizers, but is more costly than twice-daily immediate-release tacrolimus (IR-Tac). OBJECTIVE: To evaluate the cost-effectiveness of LCPT during the first year of treatment vs IR-Tac in kidney transplant recipients who are Hispanic or Black. METHODS: A decision analytic model from a US payer perspective was developed using (1) subgroup outcomes data pooled from two phase 3 clinical trials that compared LCPT and IR-Tac, and (2) direct costs from real-world data sources (ie, costs of LCPT and IR-Tac treatments, biopsy-proven acute rejection, treatment-related serious adverse events [SAEs], graft failure, and consequent dialysis). The primary outcome was cost per successfully treated patient, defined as having a functioning graft after 1 year and without treatment-related SAEs. Probabilistic sensitivity analyses established distributions for cost and outcomes estimates, and a series of one-way sensitivity analyses identified parameters that had the most effect on results. RESULTS: Total overall cost for the Hispanic group was $14,765 for LCPT and $12,416 for IR-Tac, and total cost in the Black group was $16,626 for LCPT and $9,871 for IR-Tac. Total overall effectiveness of LCPT and IR-Tac was 88.32% and 84.75% in the Hispanic group and 93.24% and 85.78% in the Black group, respectively. The incremental cost-effectiveness ratio (ICER) for using LCPT over IR-Tac during the first year of treatment in the Hispanic group was $65,643 per additional successfully treated patient. The ICER for the Black group was $90,458. The single parameter having the most impact on results in both groups was the probability of a treatment-related SAE in IR-Tac, which accounted for 49% of variation in results in the Hispanic group and 46% in the Black group. CONCLUSIONS: Overall results for both groups show that LCPT is incrementally more costly and more effective compared with IR-Tac, indicating a trade-off scenario. LCPT is a cost-effective strategy if a decision makers' willingness to pay for 1 additional successfully treated patient exceeds the ICER and must be weighed against the costs of graft loss, continuing dialysis, and potential retransplant. This study provides a foundation for further research to update and expand inputs as more data become available to improve real-world relevance and decision making. DISCLOSURES: This study was funded by Veloxis Pharmaceuticals, Inc., which provided clinical trial file data and nonbinding feedback on the model structure, data interpretation, clinical expertise, manuscript review, and areas of publication interest (ie, managed care). Hurwitz, Grizzle, Villa Zapata, and Malone received grant funding from Veloxis Pharmaceuticals, Inc., through University of Arizona to conduct research and analysis for this study. Tyler is employed by Veloxis Pharmaceuticals, Inc. Some of the data reported and used in this research were available from the US Renal Data System, the US Bureau of Labor Statistics, and the Agency for Healthcare Research and Quality's Healthcare Cost and Utility Project. The interpretation and reporting of these data are the responsibility of the authors and in no way should be seen as an official policy or interpretation of the US government.

Cost-effectiveness analysis of cabazitaxel for metastatic castration resistant prostate cancer after docetaxel and androgen-signaling-targeted inhibitor resistance

BackgroundThe aim of our study was to evaluate the cost-effectiveness of cabazitaxel versus abiraterone or enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC) previously treated with docetaxel who had progression within 12 months while receiving an alternative inhibitor (abiraterone or enzalutamide) from a US payer’s perspective.MethodsTo conduct the cost-effectiveness analysis, a Markov decision model was established. Three health states (progression-free survival (PFS), progressive disease (PD) and death) were included, and the incremental cost-effectiveness ratio (ICER) was regarded as the primary endpoint. The willingness-to-pay (WTP) threshold was set at $100,000.00/quality-adjusted life year (QALY), and discounted rates were set at 3% annually. Efficacy data were derived from the CARD trial and Weibull distribution curves were modeled to fit the survival curves. The robustness of the analysis was tested with a series of one-way sensitivity analyses and probabilistic sensitivity analyses.ResultsOverall, the incremental effectiveness and cost of cabazitaxel versus androgen-signaling-targeted inhibitors (ASTIs) were 0.16 QALYs and $49,487.03, respectively, which yielded an ICER of $309,293.94/QALY. Our model was mostly sensitive to the duration of PFS in the cabazitaxel group, cost of cabazitaxel and utility of the PFS state. At a WTP threshold of $100,000.00/QALY, cabazitaxel was the dominant strategy in 0% of the simulations.ConclusionsCabazitaxel is unlikely to be a cost-effective treatment option compared with ASTIs in patients with mCRPC previously treated with docetaxel who had progression within 12 months while receiving ASTIs.

A cost-utility analysis of SIR-Spheres Y-90 resin microspheres versus best supportive care in the treatment of unresectable metastatic colorectal cancer refractory to chemotherapy in the UK

Background Limited treatment options are available in chemotherapy-refractory or -intolerant metastatic colorectal cancer (mCRC). The objective of the present analysis was to evaluate the cost-utility of SIR-Spheres Y-90 resin microspheres relative to best supportive care (BSC) in the treatment of chemotherapy refractory mCRC from the perspective of the UK national healthcare payer. Methods A cost-utility model was developed in Microsoft Excel to simulate transitions from progression-free survival to post-progression survival and death in patients with mCRC. Unit costs were captured in 2019 pounds sterling (GBP) based on the literature, formulary listings, and National Health Service (NHS) England reference costs. Future costs and effects were discounted at 3.5% per annum. A series of one-way sensitivity analyses, and probabilistic sensitivity analysis (PSA) were conducted. Results The base case analysis showed that SIR-Spheres Y-90 resin microspheres would result in an increase in discounted quality-adjusted life years gained from 0.69 quality-adjusted life years (QALYs) to 1.50 QALYs, with an associated increase in cost from GBP 15,268 to GBP 34,168 yielding an incremental cost-utility ratio of GBP 23,435 per QALY. PSA showed that there would be a 56% likelihood that SIR-Spheres Y-90 resin microspheres would be cost-effective relative to BSC at a willingness-to-pay threshold of GBP 30,000 per QALY gained. Conclusions This cost-utility analysis showed that, relative to BSC, SIR-Spheres Y-90 resin microspheres would be a cost-effective treatment option for patients with mCRC in the UK setting from the national healthcare payer perspective.

Cost-utility analysis of emicizumab prophylaxis in haemophilia A patients with factor VIII inhibitors in Korea.

Haemophilia A patients with factor VIII inhibitors (HAPI) experience frequent spontaneous bleeding, approximately once a week, and require expensive bypassing agent (BPA) treatments to control bleeding over their lifetime. According to the HAVEN 1 trial, weekly emicizumab (Hemlibra®) prophylaxis injection reduces annualized bleeding rates (ABR) by 87% compared with BPA on-demand treatment (BPA-OD) administered at the time of bleeding. Our study aimed to assess the cost-effectiveness of emicizumab prophylaxis in HAPI in Korea. Using a lifetime Markov model with health states of 'alive with bleeds' and 'dead', we simulated the experience of HAPI receiving emicizumab prophylaxis (treatment arm) or BPA-OD (control arm) and estimated expected clinical and economic outcomes under each treatment arm. Model parameters included comparative effectiveness, clinical and epidemiologic characteristics of Korean HAPI, costs of drug treatment and medical events and utility for 'alive with bleeds' state under each treatment. We utilized local data, including National Health Insurance claims data, national statistics, literature and expert surveys with haematologists. Base-case analysis results showed that compared with BPA-OD, lifetime emicizumab prophylaxis prevented 807 bleedings, extended 3.04 quality-adjusted life-years and reduced costs by 2.6 million US dollars. Thus, emicizumab prophylaxis is a dominant treatment option with better effectiveness and lower costs than BPA-OD. A series of one-way sensitivity analyses consistently showed dominant results, confirming that lifetime emicizumab prophylaxis is a cost-saving intervention for HAPI. Emicizumab prophylaxis is an excellent treatment choice reducing ABR, improving quality of life and reducing costs.

PMS17 COST EFFECTIVENESS ANALYSIS OF UPADACITINIB AND METHOTREXATE FOR PATIENTS WITH METHOTREXATE-RESISTANT RHEUMATOID ARTHRITIS

The purpose of this study was to determine the cost-effectiveness of upadacitinib (an oral, reversible JAK inhibitor) versus methotrexate (cDMARDs) as a first-line monotherapy in the treatment of rheumatoid arthritis (RA) in the US. A decision analysis model was constructed with a time horizon of 3 months and a US payer’s perspective was assumed. The effectiveness measure was based on probability of achieving a score of less than or equal to 3.2 on the 28 joint Disease Activity Score (DAS28). The effectiveness data was derived from published clinical trial information. Costs of the drugs were based on average wholesale price and were obtained from Redbook 2019. Cost of hospitalization, outpatient cost and treatment cost for adverse events were considered and were obtained from published literature. All costs were adjusted to 2019 US Dollars using the medical care component of the Consumer Price Index from the Bureau of Labor Statistics. TreeAge Pro 2019 was used to construct the model. A series of one-way sensitivity analyses were performed to assess the robustness of the model results. Treatment of RA with upadacitinib ($19,826) is more expensive than that with the methotrexate ($662) for a given time horizon of 3 months. The percentage for achieving a score of <= 3.2 on DAS 28 was 73% for upadacitinib and 27% for methotrexate. The incremental cost-effectiveness ratio of Drug A vs. Drug B was $ 225,687.13 per additional patient achieving a score of <= 3.2 on DAS28 over the period of 3 months. One-way sensitivity analysis findings showed the robustness of the results. The results suggest that upadacitinib appears to be cost-effective compared to methotrexate for achieving a score of <= 3.2 on DAS28. These findings were robust for plausible ranges of effectiveness and drug acquisition costs.

Cost-effectiveness Analysis of Routine Screening Using Massively Parallel Sequencing for Maturity-Onset Diabetes of the Young in a Pediatric Diabetes Cohort: Reduced Health System Costs and Improved Patient Quality of Life.

Maturity-onset diabetes of the young (MODY) is an autosomal dominant form of diabetes, with multiple causative genes. Some MODY subtypes can be treated with sulfonylureas instead of insulin, improving glycemic control, complication rates, quality of life (QoL), and costs. Using massively parallel sequencing (MPS), we recently determined the prevalence of pathogenic/likely pathogenic MODY variants in an Australian pediatric diabetes cohort. Here, these data are used to estimate cost-effectiveness of using MPS for MODY in all pediatric diabetes cases compared with standard practice (sequencing limited to individuals with specific clinical features). A Markov decision model was developed to estimate incremental costs and quality-adjusted life-years (QALYs) of MPS screening, modeled over 30 years. We used our observed prevalence of 2.14% compared with 0.7% for standard practice, based on published data. The probabilities and utility weightings of long-term diabetes complications were based on HbA1c and estimated from published data. A series of one-way sensitivity analyses were performed using the net monetary benefit framework. Routine MPS screening for MODY was more effective and less costly than standard care screening, with 26 QALYs gained and 1,016,000 AUD (782,000 USD) saved per 1,000 patients. Cost of screening was fully offset within 10 years. Routine MPS screening remained dominant until MODY prevalence fell to <1.1%. Routine MPS screening for MODY in the pediatric population with diabetes could reduce health system costs and improve patient QoL. Our results make a compelling argument for routine genetic screening in all children with presumed type 1 diabetes mellitus.

Tranexamic acid for treatment of women with post-partum haemorrhage in Nigeria and Pakistan: a cost-effectiveness analysis of data from the WOMAN trial.

SummaryBackgroundSub-Saharan Africa and southern Asia account for almost 85% of global maternal deaths from post-partum haemorrhage. Early administration of tranexamic acid, within 3 h of giving birth, was shown to reduce the risk of death due to bleeding in women with post-partum haemorrhage in the World Maternal Antifibrinolytic (WOMAN) trial. We aimed to assess the cost-effectiveness of early administration of tranexamic acid for treatment of post-partum haemorrhage.MethodsFor this economic evaluation we developed a decision model to assess the cost-effectiveness of the addition of tranexamic acid to usual care for treatment of women with post-partum haemorrhage in Nigeria and Pakistan. We used data from the WOMAN trial to inform model parameters, supplemented by estimates from the literature. We estimated costs (calculated in 2016 US$), life-years, and quality-adjusted life-years (QALYs) with and without tranexamic acid, calculated incremental cost-effectiveness ratios (ICERs), and compared these to threshold values in each country. Costs were assessed from the health-care provider perspective and discounted at 3% per year in the base case analysis. We did a series of one-way sensitivity analyses and probabilistic sensitivity analysis to assess the robustness of the results to parameter uncertainty.FindingsEarly treatment of post-partum haemorrhage with tranexamic acid generated an average gain of 0·18 QALYs at an additional cost of $37·12 per patient in Nigeria and an average gain of 0·08 QALYs at an additional cost of $6·55 per patient in Pakistan. The base case ICER results were $208 per QALY in Nigeria and $83 per QALY in Pakistan. These ICERs were below the lower bound of the cost-effectiveness threshold range in both countries. The ICERs were most sensitive to uncertainty in parameter inputs for the relative risk of death due to bleeding with tranexamic acid, the discount rate, the cost of the drug, and the baseline probability of death due to bleeding.InterpretationEarly treatment of post-partum haemorrhage with tranexamic acid is highly cost-effective in Nigeria and Pakistan, and is likely to be cost-effective in countries in sub-Saharan Africa and southern Asia with a similar baseline risk of death due to bleeding.FundingLondon School of Hygiene & Tropical Medicine, Pfizer, UK Department of Health, Wellcome Trust, and Bill & Melinda Gates Foundation.

Economic evaluation of participatory learning and action with women\u2019s groups facilitated by Accredited Social Health Activists to improve birth outcomes in rural eastern India

BackgroundNeonatal mortality remains unacceptably high in many low and middle-income countries, including India. A community mobilisation intervention using participatory learning and action with women’s groups facilitated by Accredited Social Health Activists (ASHAs) was conducted to improve maternal and newborn health. The intervention was evaluated through a cluster-randomised controlled trial conducted in Jharkhand and Odisha, eastern India. This aims to assess the cost-effectiveness this intervention.MethodsCosts were estimated from the provider’s perspective and calculated separately for the women’s group intervention and for activities to strengthen Village Health Sanitation and Nutrition Committees (VHNSC) conducted in all trial areas. Costs were estimated at 2017 prices and converted to US dollar (USD). The incremental cost-effectiveness ratio (ICER) was calculated with respect to a do-nothing alternative and compared with the WHO thresholds for cost-effective interventions. ICERs were calculated for cases of neonatal mortality and disability-adjusted life years (DALYs) averted.ResultsThe incremental cost of the intervention was USD 83 per averted DALY (USD 99 inclusive of VHSNC strengthening costs), and the incremental cost per newborn death averted was USD 2545 (USD 3046 inclusive of VHSNC strengthening costs). The intervention was highly cost-effective according to WHO threshold, as the cost per life year saved or DALY averted was less than India’s Gross Domestic Product (GDP) per capita. The robustness of the findings to assumptions was tested using a series of one-way sensitivity analyses. The sensitivity analysis does not change the conclusion that the intervention is highly cost-effective.ConclusionParticipatory learning and action with women’s groups facilitated by ASHAs was highly cost-effective to reduce neonatal mortality in rural settings with low literacy levels and high neonatal mortality rates. This approach could effectively complement facility-based care in India and can be scaled up in comparable high mortality settings.

Venoarterial extracorporeal membrane oxygenation for patients in shock or cardiac arrest secondary to cardiotoxicant poisoning: A cost-effectiveness analysis

PurposeVenoarterial extracorporeal membrane oxygenation represents an emerging and recommended option to treat life-threatening cardiotoxicant poisoning. The objective of this cost-effectiveness analysis was to estimate the incremental cost-effectiveness ratio of using venoarterial extracorporeal membrane oxygenation for adults in cardiotoxicant-induced shock or cardiac arrest compared with standard care. Materials and methodsAdults in shock or in cardiac arrest secondary to cardiotoxicant poisoning were studied with a lifetime horizon and a societal perspective. Venoarterial extracorporeal membrane oxygenation cost effectiveness was calculated using a decision analysis tree, with the effect of the intervention and the probabilities used in the model taken from an observational study representing the highest level of evidence available. The costs (2013 Canadian dollars, where $1.00 Canadian = $0.9562 US dollars) were documented with interviews, reviews of official provincial documents, or published articles. A series of one-way sensitivity analyses and a probabilistic sensitivity analysis using Monte Carlo simulation were used to evaluate uncertainty in the decision model. ResultsThe cost per life year (LY) gained in the extracorporeal membrane oxygenation group was $145 931/18 LY compared with $88 450/10 LY in the non-extracorporeal membrane oxygenation group. The incremental cost-effectiveness ratio ($7185/LY but $34 311/LY using a more pessimistic approach) was mainly influenced by the probability of survival. The probabilistic sensitivity analysis identified variability in both cost and effectiveness. ConclusionVenoarterial extracorporeal membrane oxygenation may be cost effective in treating cardiotoxicant poisonings.

Epidermal Growth Factor Receptor Mutation Testing in Thailand: A Cost-Utility Analysis

ObjectiveTo evaluate the cost utility of epidermal growth factor receptor (EGFR) testing plus first-line gefitinib treatment in patients with activating EGFR mutations in Thailand. MethodsThe study used a decision tree model considering the provider’s perspective. Direct medical costs were included and based on a local Thai database. Effectiveness was measured as quality-adjusted life-year and based on randomized controlled trials. Incremental cost-effectiveness ratio was calculated and presented in 2012. A series of one-way sensitivity analyses were conducted. ResultsWe found that the EGFR testing plus first-line gefitinib alternative gained 0.03 quality-adjusted life-year more, but 62,540 Thailand baht (US $2082.58) less total costs compared with the no-testing alternative. The results were robust when varying most variables in the model except for the duration of gefitinib treatment with activating EGFR mutation, the duration of chemotherapy treatment with activating EGFR mutation, and the utility of second-line chemotherapy. ConclusionsEGFR testing should be considered before administering EGFR tyrosine-kinase inhibitor such as gefitinib as first-line treatment in patients with non–small cell lung cancer in Thailand where the incidence of EGFR mutation is high.

An economic evaluation of intravenous versus oral iron supplementation in people on haemodialysis

Iron supplementation can be administered either intravenously or orally in patients with chronic kidney disease (CKD) and iron deficiency anaemia, but practice varies widely. The aim of this study was to estimate the health care costs and benefits of parenteral iron compared with oral iron in haemodialysis patients receiving erythropoiesis-stimulating agents (ESAs). Using broad health care funder perspective, a probabilistic Markov model was constructed to compare the cost-effectiveness and cost-utility of parenteral iron therapy versus oral iron for the management of haemodialysis patients with relative iron deficiency. A series of one-way, multi-way and probabilistic sensitivity analyses were conducted to assess the robustness of the model structure and the extent in which the model's assumptions were sensitive to the uncertainties within the input variables. Compared with oral iron, the incremental cost-effectiveness ratios (ICERs) for parenteral iron were $74,760 per life year saved and $34,660 per quality-adjusted life year (QALY) gained. A series of one-way sensitivity analyses show that the ICER is most sensitive to the probability of achieving haemoglobin (Hb) targets using supplemental iron with a consequential decrease in the standard ESA doses and the relative increased risk in all-cause mortality associated with low Hb levels (Hb < 9.0 g/dL). If the willingness-to-pay threshold was set at $50,000/QALY, the proportions of simulations that showed parenteral iron was cost-effective compared with oral iron were over 90%. Assuming that there is an overall increased mortality risk associated with very low Hb level (<9.0 g/dL), using parenteral iron to achieve an Hb target between 9.5 and 12 g/L is cost-effective compared with oral iron therapy among haemodialysis patients with relative iron deficiency.

Economic impact of prescribing error prevention with computerized physician order entry of injectable antineoplastic drugs

A cost-benefit analysis was carried out to determine the potential economic costs and benefits of pharmaceutical analysis in preventing prescribing errors for full standardized injectable antineoplastic drugs computerized physician order entry, in a pharmaceutical unit (University teaching hospital), compared with theoretical setting with no pharmaceutical analysis. The viewpoint is that of the payer or the French national Public Health Insurance system, and is limited to hospital cost (only direct medical costs related to net cost and net benefit. A decision analysis model was performed to compare two strategies: with pharmaceutical analysis (± pharmacy intervention) and without pharmaceutical analysis. are expressed in terms of benefit-to-cost ratio and total benefit. The robustness of the results was assessed through a series of one-way sensitivity analyses. Over 1 year, prescribing error incidence was estimated at 1.5% [1.3-1.7], i.e. 218 avoided prescribing errors. Potential avoidance of hospital stay was estimated at 419 days or 1.9 ± 0.3 days per prescribing error. Cost-benefit analysis could estimate a net benefit-to-cost ratio of 33.3 (€17.34/€0.52) and a total benefit at €16.82 per pharmaceutical analysis or €249,844 per year. The sensitivity analysis showed robustness of results. Our study shows a substantial economic benefit of pharmaceutical analysis and intervention in the prevention of prescribing errors. The clinical pharmacist adds both value and economic benefit, making it possible to avoid additional use of expensive antineoplastic drugs and hospitalization. Computerized physician order entry of antineoplastic drugs improves the relevance of clinical pharmacist interventions, expanding pharmaceutical analysis and also the role of the pharmacist.

Cost-effectiveness of fulvestrant 250 mg versus 500 mg in postmenopausal women with estrogen receptor-positive metastatic breast cancer and disease progression after antiestrogen therapy

To determine the cost-effectiveness of fulvestrant 250 mg compared to 500 mg in postmenopausal women with estrogen receptor-positive metastatic breast cancer and disease progression after antiestrogen therapy. A Markov model was constructed to find the incremental cost-effectiveness of fulvestrant 250 mg monthly when compared with the 500 mg monthly in patients with progression after antiestrogen therapy. The model duration was 24 months. Clinical efficacy data inputs were derived from a phase III clinical trial demonstrating a statistically significant increase in progression-free survival in patients receiving 500 mg versus 250 mg. Cost data utilized were all relevant Ambulatory Payment Classification payment rates from the 2011 Medicare Outpatient Prospective Payment System. A Monte Carlo simulation was performed to test the model at various willingness to pay thresholds. The incremental cost-effectiveness ratio as determined by the Markov model was US$10,972 per month of progression-free survival for the 500 mg dose compared with the 250 mg dose. Using a Monte Carlo simulation, it was found that 500 mg monthly was cost-effective at and above the willingness to pay threshold of US$15,000 per month. A series of one-way sensitivity analyses showed this result is robust to geographical practice variations in costs of drug administration and physician examination. From a third party payer perspective, the value of fulvestrant 500 mg monthly is dependent on the willingness to pay threshold. Despite a labeling change for fulvestrant in September 2010, fulvestrant 250 mg monthly appears to be a viable option in the target population.

PD06-06: Is Adjuvant Trastuzumab Economically Justified in Her-2/neu Positive T1bNO Breast Cancer?

Abstract Background: A number of clinical trials and cost-utility analyses of adjuvant trastuzumab (aTZ) for Her-2/neu positive breast cancer suggest favourable efficacy and safety along with acceptable cost-effectiveness. These evaluations, however, were based on patient cohorts with moderate-to-high risk disease, including node positive or high risk node negative disease with tumor sizes greater than 1cm (≥T1cN0). The role of aTZ for patients with lower risk disease, such as T1bN0, remains unclear in light of the varying 10-year relapse risks (10-30%) reported to date. Objective: To estimate the cost-utility of chemotherapy plus aTZ versus chemotherapy alone in Her-2/neu positive breast cancer patients with lower recurrence risk in terms of cost per quality-adjusted life year (QALY) gained. Methods: A state-transition economic model was developed to estimate the incremental costs and quality-adjusted life year (QALY) gains associated with a strategy of chemotherapy plus aTZ relative to chemotherapy alone over a 25-year analysis horizon. The model consisted of four broad health states, stratified with or without cardiotoxicity: 1) disease-free, 2) local recurrence, 3) distant recurrence and 4) death. Given the variability in reported risk estimates for T1bN0 disease, a range of 10-year baseline recurrence risks (10-30%) in the absence of chemotherapy or aTZ was examined. The clinical benefit of chemotherapy was assumed to differ according to age (&amp;lt;50 vs. ≥50 years). The hazard ratio of recurrence with aTZ (HR=0.64) and the rate of associated adverse cardiac events were derived primarily from the HERA clinical trial. Utility weights and the costs of local and distant cancer recurrence were derived from the literature, while the costs of adjuvant and palliative TZ were derived from our previous work. The model took a direct payer perspective, with costs reported in 2011 Canadian dollars (CDN$). Costs and QALYs were both discounted by 3% annually. The reference analysis assumed 3 years of clinical benefit from chemptherapy and aTZ. A series of one-way sensitivity analyses tested the impact of longer benefit as well as the impacts of measuring outcomes in terms life years rather than QALYs, a lifetime analysis horizon and different HRs. Results: The reference analysis observed that the cost per QALY gained was greater than CDN$100,000 across the entire range of recurrence risks tested. One-way sensitivity analyses observed that considering life years rather than QALYs, a lifetime analysis horizon, extending the duration of benefit to 5 years or improving the HR to 0.54 each did little to improve the overall economic favorability of aTZ, even at the higher range of recurrence risk. Conclusions: The cost-utility of adjuvant trastuzumab appears unfavourable in Her-2/neu positive breast cancers with a baseline 10-year relapse risk of less than 30% without treatments, such as in T1bN0 disease. Specific estimates of cost-utility await more precise estimates of the recurrence risk in patients with T1bN0 disease. Citation Information: Cancer Res 2011;71(24 Suppl):Abstract nr PD06-06.

Preventing bath water scalds: a cost-effectiveness analysis of introducing bath thermostatic mixer valves in social housing

AimsTo assess the cost-effectiveness of installing thermostatic mixer valves (TMVs) in reducing risks of bath water scalds and estimate the costs of avoiding bath water scalds.MethodsThe evaluation was undertaken from...

Long-term cost-effectiveness of clopidogrel in patients with acute coronary syndrome without ST-segment elevation in Germany

Patients with acute coronary syndrome without ST-segment elevation receiving clopidogrel in addition to acetylsalicylic acid (ASA) showed a 20% risk reduction in comparison to patients receiving ASA monotherapy (CURE trial). Economic models for assessing the impact on costs exist for several countries but not for Germany on a long-term basis. The objective of this model adaptation is to assess the long-term economic impact of clopidogrel taken in addition to ASA in Germany. A Markov model with six states [at risk, first year with stroke, following years with stroke, first year with new myocardial infarction (MI), following years with MI, and death] was adapted for Germany. Model outcome was life-years saved. Effects of 1-year treatment were calculated based on the CURE trial. Resource use for the different health states was based on published data, which included costs for drugs, outpatient care, hospitalization, rehabilitation and nursing. Risk data for MI and stroke were based on Swedish data and validated for the German adaptation. The model calculates lifetime costs and survival length. Costs were estimated from the payers' perspective. A series of one-way sensitivity analyses was conducted (follow-up costs, discount rates). The Markov analysis predicts a survival of 8.89years in the placebo treatment group and 9.02 years in the clopidogrel treatment group. The cumulated costs were euro 8,548 and euro 8,953, respectively. The incremental cost-effectiveness ratio (ICER) was euro 3,113 for each life-year saved. The model was robust regarding variations in key parameters in the sensitivity analysis, resulting in a range of ICER from euro 1,338 to euro 9,322. Our results are in line with the results for other healthcare systems. Adding clopidogrel to ASA for patients with acute coronary syndrome without ST-segment elevation generated an additional life-year saved at a comparably low value of euro 3,113. One-year treatment with clopidogrel is a cost-effective treatment option in patients with acute coronary syndrome from the perspective of a third-party payer in Germany.

Prevention of adhesion formation after radical hysterectomy using a sodium hyaluronate–carboxymethylcellulose (HA–CMC) barrier: A cost-effectiveness analysis

To evaluate the cost-effectiveness of an adhesion prevention strategy compared to routine care, in which no adhesion prevention measures are taken, through a decision analysis model in the clinical setting of patients undergoing radical hysterectomy and pelvic lymphadenectomy for Stage IB cervical cancer. A decision analysis model compared two strategies to manage the risk of adhesion-related morbidity following radical hysterectomy for Stage IB cervical cancer: (1) routine care with no adhesion prevention measures, and (2) the intervention strategy with a HA-CMC anti-adhesion barrier. The cost-effectiveness of each strategy was evaluated from the perspective of society and that of a third party payer. From the perspective of society, the HA-CMC strategy had an overall cost per patient of $1932 and effectiveness of 7.901 QALYs and dominated the routine care strategy, which had a cost per patient of $3043 and effectiveness of 7.805 QALYs. From the perspective of a third party payer, the HA-CMC strategy had an overall cost per patient of $1247 and effectiveness of 7.987 QALYs and dominated the routine care strategy, which had a cost per patient of $1629 and effectiveness of 7.970 QALYs. A series of one-way sensitivity analyses confirmed the robustness of the model. Under a conservative set of clinical and economic assumptions, an adhesion prevention strategy utilizing a HA-CMC barrier in patients undergoing radical hysterectomy for Stage IB cervical cancer is cost-effective from both the perspective of society as a whole and that of a third party payer.