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.

Use Patterns and Costs of Isolated Limb Perfusion and Infusion in the Treatment of Regional Metastatic Melanoma: A Retrospective Database Analysis.

INTRODUCTION: Isolated limb perfusion and infusion (ILP/ILI) are therapies for regional metastatic melanoma that allow high doses of anticancer drugs to be delivered directly into the circulation of an affected limb, thereby minimizing systemic drug toxicity. This procedure can lead to high response rates and is recommended in patients with Stage III unresectable metastatic melanoma. However, limited information is available on patterns of use and costs. This study examined patterns of ILP/ILI use and associated costs in patients with melanoma in the United States (US). METHODS: Retrospective, observational study, using administrative claims data from the MarketScan(®) databases, was performed in patients with a diagnosis of melanoma (ICD-9-CM: 172.xx, V10.82) who underwent ILP/ILI (CPT-4: 36823) between January 1, 2002 and March 31, 2013. Patient characteristics, use patterns, length of hospital stay, and costs (per 2014 US $) of ILP/ILI were assessed. RESULTS: One hundred and thirteen patients met the study criteria and were included in the analysis. Mean age was 61.4 years (standard deviation [SD] 13.8) and 38.9% of patients were male; the mean baseline Charlson Comorbidity Index was 0.19; 34.5% of patients were Medicare beneficiaries. The majority of patients (87.6%) had melanoma of the lower limb, 11.5% of the upper limb, and 0.9% of both limbs; 60.2% had lymph node metastasis and 56.6% had skin metastasis. Four patients (3.5%) underwent multiple ILP/ILI. The mean (± SD) length of hospital stay was 5.6 (± 3.5) days and the mean (± SD) cost was US$36,758 (± 27,124) per ILP/ILI procedure. CONCLUSIONS: Isolated limb perfusion and infusion in patients with melanoma were associated with long hospital stays and high costs. These results provide useful source data for the economic evaluation of treatment options for regional metastatic melanoma. FUNDING: This study was funded by Amgen, Inc.

Treatment patterns in patients with metastatic melanoma: a retrospective analysis.

Objective. To describe treatment patterns and factors influencing treatment in a real-world setting of US patients with metastatic melanoma (MM). Methods. This was a retrospective claims-based study among patients with MM diagnosed between 2005 and 2010 identified from MarketScan databases. Results. Of 2546 MM patients, 66.8% received surgery, 44.7% received radiation, 38.7% received systemic therapies, and 17.7% received all modalities. Patients with lung, brain, liver, or bone metastases were less likely to undergo surgery (all P < 0.0001); patients with lung (P = 0.04), brain (P < 0.001), or liver metastases (P = 0.03) were more likely to receive systemic therapies; patients with brain (P < 0.0001) or bone metastases (P < 0.0001) were more likely to receive radiation therapy. Oncologists were more likely to recommend systemic therapy (P < 0.0001) or radiation (P < 0.0001), while dermatologists were more likely to recommend surgery (P = 0.002). Monotherapy was the dominant systemic therapy (82.4% patients as first-line). Conclusions. Only 39% of MM patients received systemic therapies, perhaps reflecting efficacy and safety limitations of conventional systemic therapies for MM. Among those receiving systemic therapy, monotherapy was the most common approach. Sites of metastases and physician speciality influenced treatment patterns. This study serves as a baseline against which future treatment pattern studies, following approval of new agents, can be compared.

Costs of hospital events in patients with metastatic colorectal cancer.

BACKGROUND: In the last decade, the number of new agents, including monoclonal antibodies, being developed to treat metastatic colorectal cancer (mCRC) increased rapidly. While improving outcomes, these new treatments also have distinct and known safety profiles with toxicities that may require hospitalizations. However, patterns and costs of hospitalizations of toxicities of these new 'targeted' drugs are often unknown. OBJECTIVE: This study aimed to estimate the costs of hospital events associated with adverse events specified in the 'Special Warnings and Precautions for Use' section of the European Medicinal Agency Summary of Product Characteristics for bevacizumab, cetuximab, and panitumumab, in patients with mCRC. METHODS: From the PHARMO Record Linkage System (RLS), patients with a primary or secondary hospital discharge code for CRC and distant metastasis between 2000-2008 were selected and defined as patients with mCRC. The first discharge diagnosis defining metastases served as the index date. Patients were followed from index date until end of data collection, death, or end of study period, whichever occurred first. Hospital events during follow-up were identified through primary hospital discharge codes. Main outcomes for each event were length of stay and costs per hospital admission. RESULTS: Among 2964 mCRC patients, 271 hospital events occurred in 210 patients (mean [SD] duration of follow-up: 34 [31] months). The longest mean (SD) length of stay per hospital admission were for stroke (16 [33] days), arterial thromboembolism (ATE) (14 [21] days), wound-healing complications (WHC), acute myocardial infarction (AMI), congestive heart failure (CHF), and neutropenia (all 9 days; SD 5-15). Highest mean (SD) costs per admission were for stroke (€13,500 [€28,800]), ATE (€13,300 [€18,800]), WHC (€10,800 [€20,500]). LIMITATIONS: Although no causal link could be identified between any specific event and any specific treatment, data from this study are valuable for pharmacoeconomic evaluations of newer treatments in mCRC patients. CONCLUSIONS: Inpatient costs for events in mCRC patients are considerable and vary greatly.

The cost of treating skeletal-related events in patients with prostate cancer.

OBJECTIVES: To examine the economic burden of skeletal-related events (SREs) and to assess the frequency of different types of SREs in this population. STUDY DESIGN: Retrospective claims analysis. METHODS: Data were obtained from i3's Lab Rx Database from May 1, 2000, through March 31, 2005. Patients included had at least 2 claims with a diagnosis of prostate cancer, at least 2 subsequent claims with a diagnosis of bone metastasis, and at least 1 SRE on or after the date of the initial diagnosis of bone metastasis. Descriptive statistics for 342 patients who fit all inclusion and exclusion criteria are provided, along with Kaplan-Meier curves, which were used to estimate annual costs, adjusting for the censoring of the data. RESULTS: Patients most frequently had radiation therapy (89%), followed by pathologic fracture (23%) and bone surgery (12%). Among patients diagnosed as having at least 1 SRE, 78% experienced 1 type of SRE, 17% had 2 types of SREs, and 5% had 3 or more distinct types of SREs. The mean costs associated with SREs in the year after the initial diagnosis of an SRE, adjusted for the censoring of the data, was $12,469, with the highest costs associated with radiation therapy ($5930), followed by pathologic fracture ($3179) and bone surgery ($2218). CONCLUSION: This study of patients with prostate cancer and bone metastases revealed that the annual economic effect of medically treating SREs for these patients was $12,469.

Utilities and disutilities for type 2 diabetes treatment-related attributes.

INTRODUCTION: Although cost-utility analyses are frequently used to estimate treatment outcomes for type 2 diabetes, utilities are not available for key medication-related attributes. The purpose of this study was to identify the utility or disutility of diabetes medication-related attributes (weight change, gastrointestinal side effects, fear of hypoglycemia) that may influence patient preference. METHODS: Patients with type 2 diabetes in Scotland and England completed standard gamble (SG) interviews to assess utility of hypothetical health states and their own current health state. The EQ-5D, PGWB, and Appraisal of Diabetes Symptoms were administered. Construct validity and differences among health states were examined with correlations, t-tests, and ANOVAs. RESULTS: A total of 129 patients (51 Scotland; 78 England) completed interviews. Mean utility of diabetes without complications was 0.89. Greater body weight was associated with disutility, and lower body weight with added utility (e.g., 3% higher = -0.04; 3% lower = +0.02). Gastrointestinal side effects and fear of hypoglycemia were associated with significant disutility (p < 0.001). SG utility of current health (mean = 0.87) demonstrated construct validity through correlations with patient-reported outcome measures (r = 0.08-0.31). DISCUSSION: The vignette-based approach was feasible and useful for assessing added utility or disutility of medication-related attributes.

Economic outcomes associated with switching individuals with schizophrenia between risperidone and olanzapine: findings from a large US claims database.

OBJECTIVES: To assess the impact of switching atypical antipsychotic treatment [from (i) risperidone to olanzapine or (ii) olanzapine to risperidone] on medication use patterns and treatment costs for individuals with schizophrenia. METHODS: Using a large, integrated medical service and pharmacy claims database, 244 individuals diagnosed with schizophrenia (International Classification of Diseases [9th revision]: 295.xx) who switched treatment from risperidone to olanzapine (n = 202) or from olanzapine to risperidone (n = 42) were identified. Changes in medication use patterns and treatment costs (1999 values) per patient from the pre- to the post-switch period were evaluated. McNemar's tests were used to compare changes in use of antiparkinsonian, antidiabetic and antihyperlipidaemic agents and typical antipsychotics, while the Wilcoxon signed rank tests were applied to examine changes in treatment costs. RESULTS: After switching from risperidone to olanzapine, the percentage of patients using concomitant antiparkinsonian agents and typical antipsychotics decreased significantly from 30.20% to 21.29% (p = 0.0094) and from 30.69% to 18.32% (p = 0.0006), respectively. There was no significant change in the use of antidiabetic or antihyperlipidaemic drugs. For mental health-related treatment, annualised pharmaceutical costs increased by $US1761 (from $US1829 to $US3590, p < 0.0001) but medical service costs decreased by $US3511 (from $US11 292 to $US7781, p = 0.0036), driven primarily by significantly lower emergency room care and hospital outpatient costs. This resulted in no significant change in overall mental healthcare costs. Similar results were observed with total healthcare costs. In contrast, after switching from olanzapine to risperidone there was no significant change in treatment patterns for any of the medications assessed or in healthcare costs (mental healthcare-related or total), despite a significant decrease in mental health-related pharmaceutical costs. CONCLUSIONS: Switching from risperidone to olanzapine was associated with improved medication use patterns for antiparkinsonian and typical antipsychotic agents. While both mental health and total healthcare pharmaceutical costs increased significantly, this was not associated with a significant increase in overall mental health and total healthcare costs. These outcomes, however, were not evidenced in patients switched from olanzapine to risperidone.

Olanzapine versus risperidone in the treatment of schizophrenia : a comparison of costs among Texas Medicaid recipients.

OBJECTIVE: To examine both schizophrenia-related costs and total (schizophrenia plus non-schizophrenia) healthcare costs among Texas Medicaid recipients who had been diagnosed with a schizophrenic disorder and had been initiated on olanzapine or risperidone. METHODS: Cost data for services and prescription use were retrieved for 2,885 patients with schizophrenia who were initiated on olanzapine or risperidone between 1 January 1997 and 31 August 1998. Each patient was followed for 1 year before and 1 year after initiation of therapy. Multivariate analysis was used to control for a wide range of factors (drug choice, patient demographics, pre-utilisation costs, region, health conditions, and treatment patterns) that may influence schizophrenia-related costs and total healthcare costs. Estimation was conducted via a two-stage instrumental variables model. RESULTS: The mean unadjusted total schizophrenia-related cost per patient per year during the observation period was 4,892 US dollars, and the total unadjusted healthcare cost per patient was 7,101 US dollars. Results revealed significant regional variation in schizophrenia-related and total healthcare costs. Significantly higher total healthcare costs were found for patients with other (nonpsychiatric) diagnoses, such as HIV and diabetes mellitus. Although, on average, patients taking olanzapine stayed on therapy longer than those taking risperidone (248.2 days vs 211.1 days; p < 0.0001), multivariate analysis revealed no significant difference in schizophrenia-related costs between patients who received olanzapine and risperidone (123 US dollars lower with olanzapine; p = 0.6439). However, patients who received olanzapine compared with risperidone had significantly lower total medical costs (693 US dollars lower with olanzapine; p = 0.0311). CONCLUSION: This naturalistic study used data from a Texas Medicaid population to examine the schizophrenia-related costs and total healthcare costs for patients who received olanzapine versus risperidone. Multivariate analysis revealed no significant differences in schizophrenia-related costs for patients receiving olanzapine compared with risperidone, although total medical costs were significantly lower for patients initiated on olanzapine.