Cost-effectiveness analysis for avelumab first-line maintenance treatment of advanced urothelial carcinoma in Scotland.

Aim: The cost-effectiveness of avelumab first-line maintenance treatment for locally advanced or metastatic urothelial carcinoma in Scotland was assessed. Materials & methods: A partitioned survival model was developed comparing avelumab plus best supportive care (BSC) versus BSC alone, incorporating JAVELIN Bladder 100 trial data, costs from national databases and published literature and clinical expert validation of assumptions. Incremental cost-effectiveness ratio (ICER) was estimated using lifetime costs and quality-adjusted life-years (QALY). Results: Avelumab plus BSC had incremental costs of £9446 and a QALY gain of 0.63, leading to a base-case (deterministic) ICER of £15,046 per QALY gained, supported by robust sensitivity analyses. Conclusion: Avelumab first-line maintenance is likely to be a cost-effective treatment for locally advanced or metastatic urothelial carcinoma in Scotland.

What is this article about? This study looked at the costs of avelumab when given as maintenance treatment for people in Scotland with advanced urothelial carcinoma, compared with the longer survival and other benefits that it provides. How was this done? Researchers estimated the costs and treatment benefits expected with avelumab using data from a clinical trial called JAVELIN Bladder 100, national databases, data from previously published studies and expert opinions. What were the results? Costs associated with using avelumab maintenance treatment for people with advanced urothelial carcinoma in Scotland were considered to be acceptable based on the benefits it provides. What do the results of the study mean? These results support the use of avelumab first-line maintenance as a standard treatment for people with advanced urothelial carcinoma in Scotland.

A cost-effectiveness analysis of avelumab plus best supportive care versus best supportive care alone as first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma in Taiwan.

BACKGROUND: Patients with locally advanced or metastatic urothelial carcinoma have limited treatment options and a poor prognosis. The JAVELIN Bladder 100 trial showed that avelumab as first-line maintenance plus best supportive care significantly prolonged overall survival and progression-free survival versus best supportive care alone in patients with locally advanced or metastatic urothelial carcinoma that had not progressed with first-line platinum-containing chemotherapy. AIMS: We assessed whether avelumab plus best supportive care is a cost-effective treatment option versus best supportive care alone in this patient group in Taiwan. METHODS AND RESULTS: A partitioned survival model was used to estimate the costs and effects of avelumab plus best supportive care versus best supportive care alone over a 20-year time horizon from the perspective of Taiwan's National Health Insurance Administration. Patient-level data from JAVELIN Bladder 100 on efficacy, safety, utility, and time on treatment were analyzed to provide parameters for the model. Log-normal and Weibull distributions were used for overall survival and progression-free survival, respectively. Costs of healthcare resources, drug acquisition, adverse events, and progression were identified through publicly available data sources and clinician interviews. The model estimated total costs, life years, and quality-adjusted life years. In the modeled base case, avelumab plus best supportive care increased survival versus best supportive care alone by 0.79 life years (2.93 vs. 2.14) and 0.61 quality-adjusted life years (2.15 vs. 1.54). The incremental cost-effectiveness ratio for avelumab plus best supportive care versus best supportive care alone was NT$1 827 680. Most (78%) of the probabilistic sensitivity analyses fell below three times the gross domestic product per capita. Scenario analysis indicated that life year and quality-adjusted life year gains were most sensitive to alternative survival extrapolations for both avelumab plus best supportive care and best supportive care alone. CONCLUSION: Avelumab first-line maintenance therapy combined with best supportive care was determined as a cost-effective treatment strategy for patients in Taiwan diagnosed with locally advanced or metastatic urothelial carcinoma that had not progressed with platinum-containing chemotherapy.

A Framework for Integrating Continuous Glucose Monitor-Derived Metrics into Economic Evaluations in Type 1 Diabetes.

Economic models in type 1 diabetes have relied on a change in haemoglobin A1c as the link between the blood glucose trajectory and long-term clinical outcomes, including microvascular and macrovascular disease. The landscape has changed in the past decade with the availability of regulatory approved, accurate and convenient continuous glucose monitoring devices and their ability to track patients' glucose levels over time. The data emerging from continuous glucose monitoring have enriched the clinical understanding of the disease and indirectly of patients' behaviour. This has triggered the development of new measures proposed to better define the quality of glycaemic control, beyond haemoglobin A1c. The objective of this paper is to review recent developments in clinical knowledge brought into focus with the application of continuous glucose monitoring devices, and to discuss potential approaches to incorporate the concepts into economic models in type 1 diabetes. Based on a targeted review and a series of multidisciplinary workshops, an influence diagram was developed that captures newer concepts (e.g. continuous glucose monitoring metrics) that can be integrated into economic models and illustrates their association with more established concepts. How the additional continuous glucose monitoring-based indicators of glycaemic control may contribute to economic modelling beyond haemoglobin A1c, and more accurately reflect the economic value of novel type 1 diabetes treatments, is discussed.

Prevalence of Immature Survival Data for Anti-Cancer Drugs Presented to the National Institute for Health and Care Excellence and Impact on Decision Making.

OBJECTIVES: This research aims to explore how often the National Institute for Health and Care Excellence (NICE) uses immature overall survival data to inform reimbursement decisions on cancer treatments, and the implications of this for resource allocation decisions. METHODS: NICE cancer technology appraisals published between 2015 and 2017 were reviewed to determine the prevalence of using immature survival data. A case study was used to demonstrate the potential impact of basing decisions on immature data. The economic model submitted by the company was reconstructed and was populated first using survival data available at the time of the appraisal, and then using data from an updated data cut published after the appraisal concluded. The incremental cost-effectiveness ratios (ICERs) obtained using the different data cuts were compared. Probabilistic sensitivity analysis was undertaken and expected value of perfect information estimated. RESULTS: Forty-one percent of NICE cancer technology appraisals used immature data to inform reimbursement decisions. In the case study, NICE gave a positive recommendation for a limited patient subgroup, with ICERs too high in the complete patient population. ICERs were dramatically lower when the final data cut was used, irrespective of the parametric model used to model survival. Probabilistic sensitivity analysis and expected value of perfect information may not have fully characterized uncertainty, because as they did not account for structural uncertainty. CONCLUSION: Analyses of cancer treatments using immature survival data may result in incorrect estimates of survival benefit and cost-effectiveness, potentially leading to inappropriate funding decisions. This research highlights the importance of revisiting past decisions when updated data cuts become available.

Budget Impact Of Including Avelumab As A Second-Line Treatment For Locally Advanced Or Metastatic Urothelial Cancer In The United States: Commercial And Medicare Payer Perspectives.

OBJECTIVE: To estimate the budget impact of introducing avelumab as a second-line (2L) treatment option for patients with locally advanced or metastatic urothelial cancer (mUC) from the perspective of a US third-party payer (commercial and Medicare). METHODS: A budget impact model (BIM) with a three-year time horizon was developed for avelumab. Efficacy and safety data were sourced from published literature and US package inserts. The analysis was conducted in collaboration with a specialist oncologist who validated clinical assumptions. Costs were based on the number of eligible patients, time-to-treatment failure, overall survival, adverse events (AEs), and projected market shares of various treatments. RESULTS: In a hypothetical commercial health plan of 30,000,000 members, 884 patients were estimated to be eligible for 2L treatment over a three-year time period. Without avelumab, the total cost for treating patients with mUC was estimated to be US$70,268,035. The introduction of avelumab increased total costs by $73,438 (0.10% increase). In a hypothetical Medicare health plan of 30,000,000 beneficiaries, a total of 4,705 patients were estimated to be eligible for 2L treatment. Without avelumab, the total cost for treating patients with mUC was estimated to be $292,923,098 from a Medicare perspective; however, with avelumab, there was an increase of $719,324 (0.25% increase) in total costs. Results of the sensitivity analyses demonstrated a cost-neutral impact across all tested scenarios from both perspectives. CONCLUSION: The BIM estimated that avelumab would have a cost-neutral impact within a US commercial and a Medicare health plan. Overall, avelumab can be an affordable and valuable treatment option for patients with locally advanced or mUC in the 2L setting. These findings demonstrate a consistently favorable budget impact in both populations. Further studies should be conducted to more comprehensively assess the clinical and economic implications of adding avelumab to the treatment armamentarium of 2L mUC.

Assessment-Schedule Matching in Unanchored Indirect Treatment Comparisons of Progression-Free Survival in Cancer Studies.

BACKGROUND: The timing of efficacy-related clinical events recorded at scheduled study visits in clinical trials are interval censored, with the interval duration pre-determined by the study protocol. Events may happen any time during that interval but can only be detected during a planned or unplanned visit. Disease progression in oncology is a notable example where the time to an event is affected by the schedule of visits within a study. This can become a source of bias when studies with varying assessment schedules are used in unanchored comparisons using methods such as matching-adjusted indirect comparisons. OBJECTIVE: We illustrate assessment-time bias (ATB) in a simulation study based on data from a recent study in second-line treatment for locally advanced or metastatic urothelial carcinoma, and present a method to adjust for differences in assessment schedule when comparing progression-free survival (PFS) against a competing treatment. METHODS: A multi-state model for death and progression was used to generate simulated death and progression times, from which PFS times were derived. PFS data were also generated for a hypothetical comparator treatment by applying a constant hazard ratio (HR) to the baseline treatment. Simulated PFS times for the two treatments were then aligned to different assessment schedules so that progression events were only observed at set visit times, and the data were analysed to assess the bias and standard error of estimates of HRs between two treatments with and without assessment-schedule matching (ASM). RESULTS: ATB is highly affected by the rate of the event at the first assessment time; in our examples, the bias ranged from 3 to 11% as the event rate increased. The proposed method relies on individual-level data from a study and attempts to adjust the timing of progression events to the comparator's schedule by shifting them forward or backward without altering the patients' actual follow-up time. The method removed the bias almost completely in all scenarios without affecting the precision of estimates of comparative effectiveness. CONCLUSIONS: Considering the increasing use of unanchored comparative analyses for novel cancer treatments based on single-arm studies, the proposed method offers a relatively simple means of improving the accuracy of relative benefits of treatments on progression times.

Clinical and economic outcomes associated with treatment sequences in patients with BRAF-mutant advanced melanoma.

AIM: The cost-effectiveness of treatment sequences in BRAF-mutant advanced melanoma. MATERIALS & METHODS: A discrete event simulation model was developed to estimate total costs and health outcomes over a patient's lifetime (30 years). Efficacy was based on the CheckMate 067/069 trials and a matching-adjusted-indirect comparison between immuno-oncology and targeted therapies. Safety, cost (in US dollars; US third-party payer perspective) and health-related quality-of-life inputs were based on published literature. RESULTS: Estimated survival gain was higher for sequences initiating with anti-PD-1 + anti-CTLA-4 than for anti-PD-1 monotherapy or BRAF+MEK inhibitors. The incremental cost-effectiveness ratio per QALY gained for first-line anti-PD-1 + anti-CTLA-4 was US$54,273 versus first-line anti-PD-1 and $79,124 versus first-line BRAF+MEK inhibitors. CONCLUSION: Initiating treatment with anti-PD-1 + anti-CTLA-4 was more cost-effective than initiation with anti-PD-1 monotherapy or BRAF+MEK inhibitors.

Sequential treatment approaches in the management of BRAF wild-type advanced melanoma: a cost-effectiveness analysis.

AIM: To evaluate the cost-effectiveness of treatment sequences with checkpoint inhibitors in patients with BRAF wild-type melanoma. MATERIALS & METHODS: Using a discrete event simulation model, cost and health outcomes were estimated. Pooled data from CheckMate 067/069 trials were used to calculate survival outcomes including treatment-free interval extrapolated over a patient's lifetime. Costs accounted for treatment, administration, toxicity, and disease management. RESULTS: First-line anti-PD-1 + anti-CTLA-4 initiating sequences had the highest estimated mean survival gain (7.6-7.7 years), driven by a longer estimated mean treatment-free interval (5.3 years). Incremental costs per incremental quality-adjusted life year gained for anti-PD-1 + anti-CTLA-4 followed by chemotherapy were US$30,955 versus anti-PD-1 initiating sequences, within the willingness-to-pay threshold. CONCLUSION: Anti-PD-1 + anti-CTLA-4 initiating sequences for BRAF wild-type melanoma are cost-effective versus anti-PD-1.

Cost-effectiveness of carfilzomib plus dexamethasone compared with bortezomib plus dexamethasone for patients with relapsed or refractory multiple myeloma in the United States.

BACKGROUND: We assessed the economic value of carfilzomib 56 mg/m2 and dexamethasone (Kd56) vs. bortezomib and dexamethasone (Vd) for relapsed/refractory multiple myeloma (R/RMM) using ENDEAVOR trial results. METHODS: Cost-effectiveness of Kd56 vs. Vd was assessed using a partitioned survival model by estimating progression-free survival, overall survival, and direct costs over a lifetime horizon. Surveillance Epidemiology and End Results (SEER) survival data were extrapolated after matching registry and ENDEAVOR patients. Utilities were sourced from the literature and mapped from patient-reported quality of life in ENDEAVOR to estimate quality-adjusted life-years (QALYs) from life-years (LYs). RESULTS: The model predicted an average gain of 1.66 LYs and 1.50 QALYs with Kd56 vs. Vd, and lifetime additional costs of $182,699, resulting in an incremental cost-effectiveness ratio (ICER) of $121,828/QALY gained. The ICER was $114,793/QALY in patients with 1 prior treatment; $99,263/QALY in those not transplanted, and <$150,000/QALY up to an 85% discount in bortezomib price. CONCLUSIONS: Kd56 is cost-effective for patients with R/RMM at a willingness-to-pay threshold of $150,000/QALY. Trial data in the model may limit generalizability; however, SEER registry data mitigates this challenge. Kd56 provides additional value in key subgroups, and remains cost-effective after steep comparator discounts.

Cost-effectiveness of adding carfilzomib to lenalidomide and dexamethasone in relapsed multiple myeloma from a US perspective.

OBJECTIVE: To assess the economic value of carfilzomib (Kyprolis), this study developed the Kyprolis Global Economic Model (K-GEM), which examined from a United States (US) payer perspective the cost-effectiveness of carfilzomib-lenalidomide-dexamethasone (KRd) versus lenalidomide-dexamethasone (Rd) in relapsed multiple myeloma (RMM; 1-3 prior therapies) based on results from the phase III ASPIRE trial that directly compared these regimens. METHODS: A partitioned survival model that included three health states of progression-free (on or off treatment), post-progression, and death was developed. Using ASPIRE data, the effect of treatment regimens as administered in the trial was assessed for progression-free survival and overall survival (OS). Treatment effects were estimated with parametric regression models adjusting for baseline patient characteristics and applied over a lifetime horizon. US Surveillance, Epidemiology and End Results (1984-2014) registry data were matched to ASPIRE patients to extrapolate OS beyond the trial. Estimated survival was adjusted to account for utilities across health states. The K-GEM considered the total direct costs (pharmacy/medical) of care for patients treated with KRd and Rd. RESULTS: KRd was estimated to be more effective compared to Rd, providing 1.99 life year and 1.67 quality-adjusted life year (QALY) gains over the modeled horizon. KRd-treated patients incurred $179,393 in total additional costs. The incremental cost-effectiveness ratio (ICER) was $107,520 per QALY. LIMITATIONS: Extrapolated survival functions present the greatest uncertainty in the modeled results. Utilities were derived from a combination of sources and assumed to reflect how US patients value their health state. CONCLUSIONS: The K-GEM showed KRd is cost-effective, with an ICER of $107,520 per QALY gained against Rd for the treatment of patients with RMM (1-3 prior therapies) at a willingness-to-pay threshold of $150,000. Reimbursement of KRd for patients with RMM may represent an efficient allocation of the healthcare budget.

Cost-Effectiveness of Pazopanib Versus Sunitinib for Renal Cancer in the United States.

We write to comment on a recently published study by Delea et al. in the January 2015 issue of JMCP that evaluated the cost-effectiveness (CE) of sunitinib (SU) versus pazopanib (PAZ) as first-line treatment for metastatic renal cell carcinoma (mRCC) from a U.S. third-party payer perspective.1 This analysis was based on COMPARZ and PISCES, clinical trials that compared SU and PAZ2,3 and led the authors to conclude that PAZ is cost-effective (in fact, dominant, according to the base-case results) compared with SU. Such assessment of economic value is clearly important for deciding between therapies to ensure fair access; therefore, we welcome a comparative evaluation of SU and PAZ. However, we believe that some of the key assumptions and inputs used in the model by Delea et al. render their results and conclusions invalid.  Best practice requires that results from a health economic model should reflect the most likely outcomes based on sound methodology and robust evidence for its inputs, as recommended by the International Society of Pharmacoeconomics and Outcomes Research (ISPOR).4 Here, we focus on 2 key areas (utilities and survival modeling) where, in our view, the analysis by Delea et al. falls short of this standard, and a third area (treatment costs) where the basis for the data derived is unclear.

Simulation and matching-based approaches for indirect comparison of treatments.

Estimates of the relative effects of competing treatments are rarely available from head-to-head trials. These effects must therefore be derived from indirect comparisons of results from different studies. The feasibility of comparisons relies on the network linking treatments through common comparators; the reliability of these may also be impacted when the studies are heterogeneous or when multiple intermediate comparisons are needed to link two specific treatments of interest. Simulated treatment comparison and matching-adjusted indirect comparison have been developed to address these challenges. These focus on comparisons of outcomes for two specific treatments of interest by using patient-level data for one treatment (the index) and published results for the other treatment (the comparator) from compatible studies, taking into account possible confounding due to population differences. This paper provides an overview of how and when these approaches can be used as an alternative or to complement standard MTC approaches.

Model-based cost-effectiveness analyses for the treatment of chronic myeloid leukaemia: a review and summary of challenges.

Assessing the economic value of treatments for chronic myeloid leukaemia (CML) is important but poses a number of challenges. This paper reviews economic models of CML treatment to learn lessons from this experience and support ongoing efforts to model CML. A search of databases and submissions to key health technology assessment agencies identified 12 studies that reported 22 models. Common practice included the use of cohort Markov models-most models used health states organised around the key stages in CML: chronic phase, accelerated phase and blast phase-and the use of utility estimates in the literature that correspond with the National Institute for Health and Care Excellence reference case. Two key areas of uncertainty were the extrapolation of survival outcomes beyond the period observed by the trial; and the effectiveness of second-line therapies. Further work is required to overcome these uncertainties in existing models, such as longer-term trial data collection, including trials of second-line therapies; validation of health-related quality-of-life instruments; and the testing of alternative modelling approaches. In the meantime, it is important that the impact of uncertainties is tested through the use of sensitivity and scenario analysis.

Retrospective claims analysis of best supportive care costs and survival in a US metastatic renal cell population.

INTRODUCTION: Survival and best supportive care (BSC) costs for patients with metastatic renal cell carcinoma (mRCC), after stopping therapy, are poorly characterized yet an important aspect of patient care. This study examined survival and costs associated with BSC after one or two lines of therapy (LOTs) for mRCC. METHODS: A retrospective cohort analysis used claims data from commercially insured or Medicare Advantage Prescription Drug (MAPD) plan enrollees of a large United States health plan with an index RCC diagnosis (ICD-9-CM 189.0) between January 1, 2007 and June 30, 2010; initiating any of the following therapies 30 days pre-index date through disenrollment from plan: sunitinib, temsirolimus, sorafenib, bevacizumab, everolimus, pazopanib, cytokines. LOT was identified using prescription fill and administration dates. Health care costs represent health plan- plus patient-paid amounts. RESULTS: The cohort (n = 274) was 73% male, with a mean age of 63.3 years (SD 11.1), with 80% commercially insured (20% MAPD), and 68% starting BSC following one LOT. Mean BSC duration was longer following one than two LOTs (223 [SD 260], 176 [SD 163] days). Median survival from the start of BSC was similar following one and two LOTs (126 and 118 days). Total BSC costs following one and two LOTs averaged US$50,188 (SD $96,984) and $37,295 (SD $51,102). Monthly costs for BSC following one and two LOTs ($10,151 and $10,566) were not substantially lower than costs while on treatment ($14,621 and $16,957). Inpatient hospital costs represented 47% and 49% following one and two LOTs, with ambulatory costs of approximately 36% following each LOT. CONCLUSION: Our study found similar survival and monthly costs for BSC following either one or two LOTs, with almost half of the cost reflecting inpatient care. Compared to costs on treatment ($14,621 to $16,957), BSC costs can be considerable ($10,151 to $10,566).

Overview of parametric survival analysis for health-economic applications.

Health economic models rely on data from trials to project the risk of events (e.g., death) over time beyond the span of the available data. Parametric survival analysis methods can be applied to identify an appropriate statistical model for the observed data, which can then be extrapolated to derive a complete time-to-event curve. This paper describes the properties of the most commonly used statistical distributions as a basis for these models and describes an objective process of identifying the most suitable parametric distribution in a given dataset. The approach can be applied with both individual-patient data as well as with survival probabilities derived from published Kaplan-Meier curves. Both are illustrated with analyses of overall survival from the Sorafenib Hepatocellular Carcinoma Assessment Randomised Protocol trial.

Incidence-based cost-of-illness model for metastatic breast cancer in the United States.

OBJECTIVES: This study aims to estimate the annual U.S. societal costs associated with treatment of metastatic breast cancer (MBC) patients using an incidence-based cost-of-illness (COI) framework. METHODS: An incidence-based COI model was constructed in which MBC patients were simulated from diagnosis through active treatment, palliative care, and death over 5 years. Key model parameters included: annual incidence of breast cancer in the metastatic stage, utilization of cancer therapies and other medical care resources, treatment-related adverse events, unit costs, work days missed by patient and caregiver, and wage rates. Overall survival was based on SEER data and costs were assigned to living patients monthly, according to their disease management phase. The outcomes measures were total discounted societal costs, cost/year, and cost/patient-year. RESULTS: The annual incidence of MBC in the United States in 2007 was estimated to be 49,674 patients (de novo and progressed from earlier stages). The total discounted cost to society attributable to MBC was $12.2 billion for the incident cohort, or $98,571 per patient-year. The 5-year direct medical cost of this incident cohort was $9.3 billion, or $75,415 per patient-year. Treatment-related costs (active treatment, toxicity management, and medical follow-up) contributed 44 percent of MBC expenditure, followed by palliative/best supportive care costs (31 percent). Lost productivity accounted for approximately 21 percent of the total cost ($2.6 billion over 5 years or $21,153 per patient-year). CONCLUSIONS: The societal burden of MBC in the United States is substantial. Earlier detection and effective treatment could lead to a significant decrease in costs while improving overall disease prognosis.

Economic evaluation of new targeted therapies for the first-line treatment of patients with metastatic renal cell carcinoma.

OBJECTIVE: • To assess the economic value of targeted therapies as first-line metastatic renal cell carcinoma (mRCC) treatment in the US and Sweden by indirect comparison of survival data. METHODS: • A Markov model simulated disease progression, adverse events and survival with sunitinib vs sorafenib in the US and bevacizumab plus interferon-α (IFN-α) in both countries. • Results, in life-years (LYs), progression-free LYs (PFLYs), quality-adjusted LYs (QALYs) gained and treatment costs (2008 USD) were obtained through deterministic and probabilistic analyses over the patient's lifetime. RESULTS: • Sunitinib was more effective and less costly than sorafenib (gains of 0.52 PFLYs, 0.16 LYs and 0.17 QALYs and savings/patient of $13,576 in the US) and bevacizumab plus IFN-α (gains of 0.19 PFLYs, 0.23 LYs and 0.16 QALYs in both countries and savings/patient of $67,798 and $47,264 in the US and Sweden, respectively). • Results were most influenced by hazard ratios for progression-free and overall survival and treatment costs, making results generalizable across other countries if relative costs were to fall within the ranges of those in the US and Sweden. CONCLUSION: • The present analyses suggest that first-line mRCC treatment with sunitinib is a cost-effective alternative to sorafenib and bevacizumab plus IFN-α.

Potential economic impact of increasing low dose aspirin usage on CVD in the US.

BACKGROUND: Cardiovascular disease (CVD) is a leading cause of death in the US and Western Europe, but regular use of preventive low-dose aspirin has proven effective in preventing CVD events. The purpose of this study was to explore the potential economic impact in the US if preventive aspirin usage were to be increased in line with clinical guidelines for primary and secondary prevention. METHODS: The risk profile of the US population was characterized using NHANES data, and Framingham cardiovascular risk equations were applied to calculate risk for myocardial infarction, angina and ischemic stroke according to age and gender. Primary and secondary patients were considered separately. Using publicly available unit costs, a budget impact model calculated the annual impact of increased preventive aspirin usage considering gastrointestinal bleeding and hemorrhagic stroke adverse events and diminishing aspirin adherence over a 10-year time horizon. RESULTS: In a base population of 1,000,000 patients, full implementation of clinical guidelines would potentially prevent an additional 1273 myocardial infarctions, 2184 angina episodes and 565 ischemic strokes in primary prevention patients and an additional 578 myocardial infarctions, and 607 ischemic strokes in secondary prevention patients. This represents a total savings of $79.6 million for primary prevention and $32.2 million for secondary and additional out-of-pocket expense to patients of $29.0 million for primary prevention and $2.6 million for secondary prevention for the cost of aspirin. CONCLUSIONS: This budgetary model suggests that there is a strong economic case, both for payers and society, to encourage aspirin use for patients at appropriate risk and per clinical guidelines. It also provides an example of how minimizing costs do not necessarily have to imply a rationing of care. Limitations include the exclusion of other CVD interventions in the analysis.

Cost-effectiveness analysis of anastrozole versus tamoxifen in adjuvant therapy for early-stage breast cancer - a health-economic analysis based on the 100-month analysis of the ATAC trial and the German health system.

BACKGROUND: In the 'Arimidex', Tamoxifen Alone or in Combination (ATAC) trial, the aromatase inhibitor (AI) anastrozole had a significantly better efficacy and safety profile than tamoxifen as initial adjuvant therapy for hormone receptor-positive (HR+) early breast cancer (EBC) in postmenopausal patients. To compare the combined long-term clinical and economic benefits, we carried out a cost-effectiveness analysis (CEA) of anastrozole versus tamoxifen based on the data of the 100month analysis of the ATAC trial from the perspective of the German public health insurance. PATIENTS AND METHODS: A Markov model with a 25-year time horizon was developed using the 100-month analysis of the ATAC trial as well as data obtained from published literature and expert opinion. RESULTS: Adjuvant treatment of EBC with anastrozole achieved an additional 0.32 quality-adjusted life-years (QALYs) gained per patient compared with tamoxifen, at an additional cost of D 6819 per patient. Thus, the incremental cost effectiveness of anastrozole versus tamoxifen at 25 years was D 21,069 ($30,717) per QALY gained. CONCLUSIONS: This is the first CEA of an AI that is based on extended follow-up data, taking into account the carryover effect of anastrozole, which maintains the efficacy benefits beyond therapy completion after 5 years. Adjuvant treatment with anastrozole for postmenopausal women with HR+ EBC is a cost-effective alternative to tamoxifen.