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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).

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.

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., (© 2011 PFIZER INC.)

Background: Major depressive disorders (MDD) are responsible for substantial direct and indirect health care costs. Despite the availability of numerous treatments, the need for effective pharmacotherapy remains. Duloxetine is a relatively balanced serotonin norepinephrine reuptake inhibitor (SNRI) with favourable clinical and tolerability profile. The cost-effectiveness of duloxetine versus established SSRIs, venlafaxine XR and mirtazapine was estimated in the UK., Methods: A decision analysis simulating clinical management of MDD was developed to estimate health and economic impacts of alternative treatments over one year. Patients on treatment experience remission, response without remission, no response, relapse or discontinue the initial regimen. Model outcomes were total treatment costs and quality-adjusted life years. Resource utilization data were derived from literature and practising UK psychiatrists and GPs. The robustness of findings with respect to modelling assumptions was assessed in extensive sensitivity analyses., Results: With similar efficacy to venlafaxine XR but lower drug costs, duloxetine is less costly and marginally more effective than venlafaxine XR both in the overall MDD population and in a more severe subgroup. Duloxetine has a low cost-effectiveness ratio in primary care against SSRIs and mirtazapine, and was found cost-saving against mirtazapine in more severe patients., Limitations: Cost-effectiveness results are sensitive to changes in efficacy parameters and resource use data were collected from physician panel., Conclusions: Duloxetine represents an important option in the treatment of MDD in the UK that can be recommended on economic grounds. With similar efficacy and different side-effect profile to venlafaxine XR it represents a valuable choice to MDD patients.

Lennox-Gastaut syndrome (LGS) is a catastrophic childhood form of epilepsy. The syndrome is characterized by mental impairment, frequent seizures of multiple types that are particularly resistant to treatment, and high rates of seizure-related injury. With the introduction of newer, but more costly, antiepileptic drugs (AEDs), it is important that decision makers are able to assess their value in the management of this rare and difficult-to-treat condition. To evaluate the cost effectiveness, from the UK NHS perspective, of rufinamide in patients with LGS. An individual patient-simulation model was developed to estimate the total treatment-related costs and clinical benefits of rufinamide compared with topiramate and lamotrigine over a 3-year time horizon. The model examines the treatment scenarios of adding rufinamide, lamotrigine or topiramate to older AEDs (standard therapy), or standard therapy alone within a primary-care or community setting. Three placebo-controlled clinical trials of adjunctive AED treatment for children with LGS were analysed. There are no head-to-head comparator studies. Between 98 and 139 patients were randomized in each study and the mean age in each study was 10, 11 and 14 years. A mixed-treatment comparison using a random-effects model was carried out on the number of patients in each response category, using the placebo arms of the respective trials. The primary outcome measure was the percentage of successfully treated patients, defined as >50% reduction in the frequency of total seizures and drop attacks. The hypothesis being tested was formulated after data collection. Costs ( pound, year 2006/07 values) of patient monitoring, switching treatments, hospitalization due to seizure, treatment of adverse effects, and personal and social services were included in the analysis. Results of 10,000 Monte Carlo simulations were bootstrapped to conduct probabilistic sensitivity analysis. Over 3 years, adjunctive rufinamide resulted in higher total costs than topiramate and lamotrigine; however, with more patients being treated successfully, this leads to acceptable incremental cost-effectiveness ratios. If society is prepared to pay at least 250 pounds for a 1% increase in the number of successfully treated LGS patients, in terms of a 50% reduction in the frequency of drop attacks, the probability of the treatment with rufinamide being cost effective is >80%. This cost-effectiveness analysis suggests that rufinamide results in more LGS patients being treated successfully at a reasonable cost from a UK NHS perspective.

Objective: To calculate the total cost per patient of prostate cancer treatment and the economic cost burden by stage, in the first year after diagnosis, for five European countries., Methods: Data from the Information Management Systems, Inc. database, survival data, expert opinion, published data and unit costs from various published official sources were used to calculate total costs per patient by stage for each country, from a payer's perspective. Diagnostic costs, first surgery, radio-, chemo- and hormonal therapy costs were included. Costs were aggregated for incident cases., Results: The mean direct costs per patient for initial treatment were euro3698 in Germany, euro3256 in Spain, euro3682 in the UK, euro5226 in Italy and euro5851 in France. The total costs for all diagnosed patients in the first year from diagnosis were (million euro) 116.7 (UK), 244 (Germany), 385 (France), 202 (Italy) and 114.6 (Spain)., Conclusions: The direct initial healthcare cost burden of the most common non-skin-related male cancer, prostate cancer, in France, Germany, Italy, Spain and the UK is considerable. Given the high and increasing prevalence of prostate cancer due to ageing populations in Europe, and the significant cost burden of the disease, national health policy makers should be aware of prostate cancer as a priority disease area.

Paclitaxel and docetaxel have been available for the treatment of metastatic breast cancer (MBC) since the 1990s. However, until very recently, comparisons between these two drugs have been difficult due to lack of direct comparative clinical evidence and differences in trial patient populations. To conduct a cost-effectiveness analysis comparing docetaxel with paclitaxel regimens in the treatment of MBC previously treated with an anthracycline from the perspective of the UK NHS. A cost-utility analysis was performed using a Markov model to compare taxanes in MBC patients who had progressed after treatment with an anthracycline-containing chemotherapy regimen: docetaxel 100 mg/m2 1-hour intravenous (IV) infusion every 21 days versus paclitaxel 175 mg/m2 3-hour IV infusion every 21 days (Pac3w). In parallel, additional analyses were performed versus paclitaxel administered in 1-weekly cycles (Pac1w), and a nano albumin-bound form of paclitaxel (Nab-P) given every 3 weeks. Progression-free survival (PFS), overall survival (OS) and adverse events used in the model were derived from a randomized trial directly comparing docetaxel with Pac3w; the comparisons of docetaxel versus the other two paclitaxel regimens were indirect, using patient-level data from a trial comparing Pac3w with Pac1w, and from the published literature comparing Pac3w with Nab-P. Utility values for response, progression and adverse events were derived from the literature. Direct treatment costs related to progression, best supportive care and adverse events were estimated using clinical trials data, published literature, NHS reference costs and published drug prices. The estimated costs of growth colony-stimulating factors and blood transfusion were also included in the model. The model was used to predict the expected total costs ( pound, year 2005-6 values), QALYs gained, incremental cost/life-year gained (LY) and cost/QALY over a 10-year time period. In the base-case analysis, docetaxel improved QALYs by 0.33, 0.29 and 0.22 compared with Pac3w, Pac1w and Nab-P, respectively. The incremental cost-effectiveness ratios (ICERs) for docetaxel were pound 12 032/QALY versus Pac3w, pound 4583/QALY versus Pac1w and pound 14 ,694/QALY versus Nab-P. The ICER was sensitive to the hazard ratios for PFS and OS between the comparators, the drug cost of initial treatment and the treatment costs after progression. Taking into account parameter uncertainty, and comparing all four treatments simultaneously, at a willingness to pay of pound 20,000 per QALY gained, the probability of docetaxel being the most cost-effective treatment was around 70%. In the base-case scenario, docetaxel compared with Pac3w is estimated to have a cost-effectiveness ratio that falls within the acceptable threshold in the UK. The study also suggests that docetaxel may be cost effective versus Pac1w and Nab-P, although there is more uncertainty around these findings.

Many new cancer treatments that slow progression and extend survival are emerging. These advances bring new hope to patients and physicians, but often increase health expenditures in an already cost-conscious environment. To help guide resource allocation decisions, cost-effectiveness models are constructed to compare the costs and outcomes of new treatments with current options, and to encourage the uptake of new technologies. This study focuses on the cost-effectiveness studies published since 1997 for hormonal treatment options in advanced breast cancer. This paper: i) examines the quality of studies in terms of reporting methods and transparency of the models; ii) compares the calculated cost-effectiveness ratios; and iii) makes recommendations for future cost-effectiveness models in advanced breast cancer.