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Objective: To estimate the prevalence and economic burden of hyperkalemia in the United States (US) Medicare population.Methods: Patients were selected from a 5% random sample of Medicare beneficiaries (01 January 2010-31 December 2014) to estimate the prevalence and economic burden of hyperkalemia. The prevalence for each calendar year was calculated as the number of patients with hyperkalemia divided by the total number of eligible patients per year. To estimate the economic burden of hyperkalemia, patients with hyperkalemia (cases) were matched 1:1 to patients without hyperkalemia (controls) on age group, chronic kidney disease [CKD] stage, dialysis treatment, and heart failure. The incremental 30-day and 1-year resource utilization and costs (2016 USD) associated with hyperkalemia were estimated.Results: The estimated prevalence of hyperkalemia was 2.6-2.7% in the overall population and 8.9-9.3% among patients with CKD and/or heart failure. Patients with hyperkalemia had higher 1-year rates of inpatient admissions (1.28 vs. 0.44), outpatient visits (30.48 vs. 23.88), emergency department visits (2.01 vs. 1.17), and skilled nursing facility admissions (0.36 vs. 0.11) than the matched controls (all p < .001). Patients with hyperkalemia incurred on average $7208 higher 30-day costs ($8894 vs. $1685) and $19,348 higher 1-year costs ($34,362 vs. $15,013) than controls (both p < .001). Among patients with CKD and/or heart failure, the 30-day and 1-year total cost differences between cohorts were $7726 ($9906 vs. $2180) and $21,577 ($41,416 vs. $19,839), respectively (both p < .001).Conclusions: Hyperkalemia had an estimated prevalence of 2.6-2.7% in the Medicare population and was associated with markedly high healthcare costs. [ABSTRACT FROM AUTHOR]

Background: Hysterectomy and laparoscopy are the two most common surgical options used to treat women with endometriosis, yet the disease may still recur. This study aimed to determine the long-term retreatment rates among endometriosis patients in the United States who received either hysterectomy or laparoscopy. Materials and Methods: Patients aged 18-49 years with endometriosis who underwent hysterectomy or laparoscopy were identified in the Truven Health MarketScan claims database (2004-2013). The retreatment rate up to 8 years after the initial surgery was estimated using Kaplan-Meier survival analysis. The relative risk of retreatment among patients with hysterectomy versus laparoscopy was assessed using a Cox proportional hazard model. Results: A total of 24,915 patients with endometriosis who underwent hysterectomy and 37,308 patients with endometriosis who underwent laparoscopy were identified. The estimated retreatment rates were 3.3%, 4.7%, and 5.4% in the 2nd, 5th, and 8th year following hysterectomy, respectively, while the rates following laparoscopy were 15.8%, 27.5%, and 35.2%, respectively. The hazard ratio of retreatment was 0.157 (95% confidence interval [CI]: 0.146-0.169) comparing hysterectomy to laparoscopy. In the sensitivity analysis, which expanded the definition of retreatment by including medical treatments, the retreatment rate increased by a factor of 11-14 for the hysterectomy cohort and by a factor of 2-4 for the laparoscopy cohort, and the hazard ratio of retreatment rate for hysterectomy versus laparoscopy was 0.490 (95% CI: 0.477-0.502). Conclusions: Our study results indicated that the disease retreatment rate after laparoscopy is high among patients with endometriosis; even hysterectomy does not guarantee freedom from retreatment. [ABSTRACT FROM AUTHOR]

Introduction: The objective of this study was to analyze medical costs and healthcare resource utilization (HRU) associated with everolimus-based therapy or chemotherapy among elderly women with hormone-receptor-positive, human-epidermal-growth-factor-receptor-2-negative (HR+/HER2-) metastatic breast cancer (mBC).Methods: Elderly women (≥65 years) with HR+/HER2- mBC who failed a non-steroidal-aromatase-inhibitor and subsequently began a new line of treatment with everolimus-based therapy or chemotherapy for mBC (index therapy) during July 20, 2012 to March 31, 2014 were identified from two large commercial claims databases. All-cause, BC-, and adverse event (AE)-related medical costs (2014 USD), and all-cause and AE-related HRU per patient per month (PPPM) were compared between patients treated with everolimus-based therapy and chemotherapy across their first four lines of therapy for mBC. Adjusted costs and HRU differences were estimated by pooling all lines and using multivariable models adjusted for differences in patient characteristics.Results: In total, 925 elderly patients (mean age approximately 73 years) with HR+/HER2- mBC met the inclusion criteria; 230 received everolimus-based therapy (240 lines) and 737 received chemotherapy (939 lines). Compared with chemotherapy, everolimus-based therapy was associated with significantly lower total all-cause PPPM medical services costs (adjusted mean difference: $4007), driven by lower inpatient ($1994) and outpatient ($1402) costs; lower BC-related medical services costs ($3129), driven by both BC-related inpatient ($1883) and outpatient costs ($913); and lower AE-related medical services costs ($1873; all P < 0.01). Additionally, compared to patients treated with chemotherapy, patients treated with everolimus-based therapy had fewer all-cause outpatient visits (adjusted incidence rate ratio = 0.69), BC-related outpatient visits (0.66), other-medical-service visits (0.65), and AE-related HRU (0.59), which was driven by significantly fewer AE-related outpatient visits (0.56; all P < 0.01). Subgroup analyses comparing medical costs of everolimus-based therapy with capecitabine monotherapy showed consistent results overall.Conclusion: This retrospective claims database analysis of elderly women with HR+/HER2- mBC in the United States showed that everolimus-based therapy was associated with significantly lower all-cause, BC-related, and AE-related medical services costs and less use of healthcare resources compared with chemotherapy.Funding: Novartis. [ABSTRACT FROM AUTHOR]

Objective: This study aimed to compare time on treatment (TOT) among patients treated with everolimus and chemotherapy, two commonly used treatments for hormone-receptor-positive/human-epidermal-growth-factor-receptor-2-negative (HR+/HER2-) metastatic breast cancer (mBC).Methods: Postmenopausal women with HR+/HER2- mBC who initiated ≥1 new line of therapy for mBC during 20 July 2012-31 March 2014 after a non-steroidal aromatase inhibitor were identified from MarketScan and PharMetrics databases (2002Q1-2014Q2) using a claims-based algorithm. Patients were classified into treatment groups by regimen and line of therapy, and were followed until discontinuation of therapy, end of insurance eligibility, or data cut-off (30 June 2014). Discontinuation was defined as a treatment gap of ≥60 days; patients who did not discontinue were censored at the end of follow-up. TOT was compared between everolimus, chemotherapy, and capecitabine monotherapy using Kaplan-Meier analyses and multivariable Cox models adjusting for line of therapy, age, insurance, de novo mBC diagnosis, prior use of chemotherapy for mBC, sites of metastases, and Charlson comorbidity index.Results: Across the first four lines of therapies for mBC, a total of 940 everolimus, 3410 chemotherapy, and 721 capecitabine monotherapy regimens were included. Based on the different lines of therapies, the median TOT ranged from 5.5 to 7.2 months for everolimus, 4.3 to 4.7 months for chemotherapy, and 3.5 to 6.0 months for capecitabine monotherapy. Pooling all lines of therapies, everolimus was associated with significantly longer TOT compared to chemotherapy (multivariable-adjusted hazard ratio [HR] = 0.69, 95% confidence interval [CI]: 0.62-0.76) or capecitabine monotherapy (multivariable-adjusted HR = 0.73, 95% CI: 0.64-0.83). Longer TOT was consistently observed for everolimus for each line of therapy.Limitations: Proxies used for identifying HR + /HER2- mBC and treatment line, lack of certain clinical factors in claims data, generalizability limited to commercially insured patients in the US.Conclusions: This study found that HR+/HER2- mBC patients receiving everolimus experienced significantly longer TOT than those receiving chemotherapy overall or capecitabine monotherapy. [ABSTRACT FROM AUTHOR]

Background: Idiopathic pulmonary fibrosis (IPF) is a rare and serious disease characterized by progressive lung-function loss. Limited evidence has been published on the impact of lung-function loss on subsequent patient outcomes. This study examined change in forced vital capacity (FVC) across IPF patients in the 6 months after diagnosis and its association with clinical and healthcare resource utilization (HRU) outcomes in a real-world setting in the U.S.Methods: A retrospective chart review was conducted of patients diagnosed with IPF by U.S. pulmonologists. Patient eligibility criteria included: 1) 40 years or older with a confirmed date of first IPF diagnosis with high-resolution computed tomography and/or lung biopsy between 01/2011 and 06/2013; 2) FVC results recorded at first diagnosis (±1 month) and at 6 months (±3 months) following diagnosis. Based on relative change in FVC percent predicted (FVC%), patients were categorized as stable (decline <5%), marginal decline (decline ≥5% and <10%), or significant decline (decline ≥10%). Physician-reported clinical and HRU outcomes were assessed from ~6 months post-diagnosis until the last contact date with the physician and compared between FVC% change groups. Multivariable Cox proportional-hazards models were constructed to assess risk of mortality, suspected acute exacerbation (AEx), and hospitalization post-FVC% change. Generalized estimating equations were used to account for multiple patients contributed by individual physicians.Results: The sample included 490 IPF patients contributed by 168 pulmonologists. The mean (SD) age was 61 (11) years, 68% were male, and the mean (SD) baseline FVC% was 60% (26%). 250 (51%) patients were categorized as stable, 98 (20%) as marginal decline, and 142 (29%) as significant decline. The mean (SD) observation time was 583 (287) days. In both unadjusted analysis and multivariable models, significantly worse clinical outcomes and increased HRU were observed with greater lung-function decline.Conclusions: These findings suggest that nearly half of IPF patients experienced decline in FVC% within ~6 months following IPF diagnosis. Greater FVC% decline was associated with an increased risk of further IPF progression, suspected AEx, mortality, and higher rate of HRU. Management options that slow FVC decline may help improve future health outcomes in IPF. [ABSTRACT FROM AUTHOR]

Background: Evidence is currently equivocal on the added benefits of dual blockade of the renin-angiotensin- aldosterone system with the combination of either an ACE inhibitor (ACEI) plus an angiotensin II type 1 receptor antagonist (angiotensin receptor blocker [ARB]) or aliskiren, the first-in-class direct renin inhibitor, plus an ARB. Objective: To compare the compliance, persistence, healthcare resource utilization, and healthcare costs associated with aliskiren plus ARB versus ACEI plus ARB combination therapies among adult patients diagnosed with hypertension. Methods: Patients (aged ≥18 years) initiated on either combination therapy were identified in the Market- Scan Commercial and Medicare Supplemental Databases (1 July 2007 to 30 June 2008). The ARB components considered were candesartan, irbesartan, losartan, olmesartan, telmisartan, and valsartan. The ACEI components included benazepril, captopril, enalapril, fosinopril, lisinopril, moexipril, perindopril, quinapril, ramipril, and trandolapril. Outcomes measured during the 6-month study period included the proportion of days covered (PDC), treatment discontinuation, healthcare resource utilization, and changes in healthcare costs (study period minus 6-month baseline values). Risk-adjusted differences in outcomes between treatments and associated 95% confidence intervals (CIs) were estimated using multivariate regression models, controlling for demographics, region, co-morbidities, prescription drug use, and resource utilization during the baseline period. Results: Adjusting for baseline characteristics, aliskiren plus ARB patients (n = 1395) demonstrated a significantly higher PDC (67.0% vs 54.3%; difference 12.7%; 95% CI 10.6, 14.7) and a significantly lower discontinuation rate (50.4% vs 68.6%; odds ratio 0.46; 95% CI 0.40, 0.54) than ACEI plus ARB patients (n = 16 507). Aliskiren plus ARB patients had significantly fewer all-cause hospitalizations (adjusted incidence rate ratio [IRR] 0.73; 95%CI 0.61, 0.86) and significantly fewer all-cause emergency room (ER) visits (adjusted IRR 0.72; 95% CI 0.61, 0.85) than ACEI plus ARB patients. Compared with ACEI plus ARB therapy, aliskiren plus ARB therapy was associated with significantly larger increases in prescription costs by $US264 post therapy initiation (95% CI 153, 375), but with non-significantly greater reductions in total healthcare costs by -$583 (95% CI -2409, 1242) [2008 values]. Conclusion: In adult hypertensive patients, treatment with aliskiren plus ARB was associated with significantly better compliance/persistence and fewer hospitalizations and ER visits compared with ACEI plus ARB therapy. Reductions in total healthcare costs were non-significantly different between patients treated with aliskiren plus ARBversusACEI plusARB, despite the increased prescription costs associated with aliskiren plusARBtherapy. [ABSTRACT FROM AUTHOR]

Retrospective database analyses pose a series of methodological challenges, some of which are unique to their data sources, particularly in countries outside the US. This study aimed to qualitatively review the methodological challenges of using non-US databases to conduct retrospective economic and outcomes research studies. We conducted a MEDLINE search to obtain a sample of literature published after the year 2000 on retrospective analyses using non-US databases. We reviewed all relevant components of the selected articles in accordance with the checklist proposed for retrospective database studies by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force and identified issues found in the data sources, methods, study designs, statistics and sources of possible threats to internal and external validity. We found a wide variation in the quality of studies in terms of outcome definitions, patient selection criteria, data collection methods, sample sizes, risk adjustment methods, potential measurement errors and external validity of the studies. Few economic studies included information on indirect cost components because of a lack of relevant data. The quality of non-US retrospective database analyses varied. Future such analyses may be improved if researchers implement the checklist developed by the ISPOR Task Force on Retrospective Database Studies. [ABSTRACT FROM AUTHOR]

The article presents the research which assessed the impact of cardiometabolic risk factor clusters on health-related quality of life (HRQL) in the U.S. The study was observed by its researchers to quantify the marginal effect of cardiometabolic risk factor clusters on HRQl after controlling comorbidity and sociodemographic characteristics of the subjects.

Objective: Stimulants are recommended as a first-line treatment for attention- deficit/hyperactivity disorder (ADHD); however, a subset of the patient population augments their stimulant treatment with other medications. The objective of this study was to estimate the 1 year period prevalence of concomitant psychotropic medication use among children and adolescents with ADHD during 2009. Methods: Patients 6-17 years of age with one or more primary ADHD diagnoses between July 1, 2008 and December 31, 2009 and one or more stimulant prescription fills during 2009 were identified from a large United States commercial claims database. Concomitant psychotropic medication use, defined as 30 days of continuous medication supply overlap between the augmenting agent and stimulant, was evaluated for 14 distinct psychotropic medication categories (6 with a United States Food and Drug Administration (FDA) approved indication for ADHD, 8 without an indication for ADHD). The 1 year period prevalence of concomitant psychotropic medication use (both overall and within each medication category) was calculated and compared between patients with and without psychiatric or neurologic comorbidities. Children (6-12 years) and adolescents (13-17 years) were evaluated separately. Results: A total of 71,201 children and 49,959 adolescents met the inclusion criteria. The 1 year period prevalence of concomitant psychotropic medication use among children and adolescents was 20.3% and 23.4%, with 5.7% and 6.7% augmenting with two or more medication categories, respectively. The most common concomitant medication categories were selective serotonin reuptake inhibitors (SSRIs) (children: 6.2%; adolescents: 11.4%), atypical antipsychotics (5.8%; 6.8%) and clonidine immediate release (5.4%; 2.9%). Children and adolescents with psychiatric or neurologic comorbidities had higher rates of augmentation than did those without comorbidities (all p<0.001). Conclusions: This epidemiologic study found that the prevalence of concomitant psychotropic medication use in children and adolescents ranged from 12.6% for noncomorbid ADHD to 41.7% for comorbid ADHD, in 2009. Future research is warranted to evaluate the rationale for, and clinical benefit of, concomitant psychotropic medication usage in patients with ADHD. [ABSTRACT FROM AUTHOR]

Purpose: This study investigated the treatment patterns and outcomes for US veteran patients with chronic myeloid leukemia- chronic phase (CML-CP) initiated on imatinib (IM). Patients and Methods: Patients (age ≥ 18 years) with at least one CML diagnosis (International Classification of Diseases, Ninth Edition Clinical Modification: 205.1x) during the period January 1, 2000, to June 30, 2011, and initiated on IM as first-line therapy were identified in the VISN 16 data warehouse (N = 137). Accelerated and blastic phases (AP/BP) were identified on the basis of WHO classification using complete blood count (CBC) data. Rates of IM dose adjustment, discontinuation, and switching to another drug therapy were estimated. Time to discontinuation, progression to AP/ BP, and survival were assessed using Kaplan-Meier analysis (KM). Results: During follow-up, 19.0% of patients had at least one dose increase; of these, 19.2% switched to another therapy. Dose reductions occurred in 25.6% of patients. Among patients who discontinued IM (n_74; 54.0%), whereas 16.2% switched to other therapies, 27.0% neither restarted IM nor switched to other therapies. KM showed that 25.6% and 42.4% of patients discontinued IM treatment by year 1 and 2, and 8.1% and 16.0% demonstrated disease progression by year 1 and 2, respectively. Among patients who experienced disease progression (n = 28), 32.1% continued IM postprogression, 32.1% discontinued IM before progression, 28.6% discontinued IM postprogression without switching, and 7.1% switched to other therapies postprogression. The mortality rates were 3.0% and 9.5% after IM initiation, and 21.7% and 42.7% after disease progression by year 1 and 2, respectively. Conclusion: In this veteran population, a substantial number of IM-treated patients, including those with disease progression, either discontinued or interrupted IM use without switching to other therapies. [ABSTRACT FROM AUTHOR]

Objective: Cardiometabolic risk factors such as overweight/obesity, hyperlipidemia, diabetes, and hypertension are prone to cluster together in the same individual and result in an elevated risk of cardiovascular disease and mortality. The purpose of this study was to examine and quantify the impact of cardiometabolic risk factor clusters independent of heart disease on productivity in a nationally representative sample of US adults. Methods: The current study estimated the impact of cardiometabolic risk factor clusters on missed work days and bed days, controlling for sociodemographic characteristics, comorbidity, and smoking status in a nationally representative, pooled 2000 and 2002 Medical Expenditure Panel Survey sample. Cardiometabolic risk factor clusters included BMI ≥ 25 and two of the following three: diabetes, hyperlipidemia, and/or hypertension. All estimates were expressedin $US 2005. Sensitivity analyses were conducted to examine the impact of varying assumptions on the results. Results: After controlling for differences in sociodemographics, smoking and comorbidity, individuals with cardiometabolic risk factor clusters missed 179% more work days and spent 147% more days in bed (in addition to lost work days) than those without. Lost work days and bed days resulted in $17.3 billion annually in lost productivity attributable to cardiometabolic risk factor clusters in the United States. Sensitivity analyses resulted in a range of annual lost productivity costs from $3.2 to $23.1 billion. Conclusions: Common cardiometabolic risk factor clusters have a significant deleterious impact on the US economy, resulting in $17.3 billion in lost productivity. [ABSTRACT FROM AUTHOR]

Background: To estimate the prevalence and correlates of clinician-diagnosed DSM-IV nonaffective psychosis (NAP) in a national household survey. Methods: Data came from the United States National Comorbidity Survey Replication (NCS-R). A screen for NAP was followed by blinded sub-sample clinical reappraisal interviews. Logistic regression was used to impute clinical diagnoses to respondents who were not re-interviewed. The method of Multiple Imputation (MI) was used to estimate prevalence and correlates. Results: Clinician-diagnosed NAP was well predicted by the screen (area under the curve [AUC] = .80). The MI prevalence estimate of NAP (standard error in parentheses) is 5.0 (2.6) per 1000 population lifetime and 3.0 (2.2) per 1000 past 12 months. The vast majority (79.4%) of lifetime and 12-month (63.7%) cases met criteria for other DSM-IV hierarchy-free disorders. Fifty-eight percent of 12-month cases were in treatment, most in the mental health specialty sector. Conclusions: The screen for NAP in the NCS-R greatly improved on previous epidemiological surveys in reducing false positives, but coding of open-ended screening scale responses was still needed to achieve accurate prediction. The lower prevalence estimate than in total-population incidence studies raises concerns that systematic nonresponse bias causes downward bias in survey prevalence estimates of NAP. [Copyright &y& Elsevier]

Purpose The addition of 2 direct-acting antiviral (DAA) agents, telaprevir and boceprevir, to peginterferon and ribavirin therapy significantly improves sustained virologic response rates in patients treated for chronic hepatitis C virus (CHC) but is associated with a higher risk of adverse events (AEs), including anemia and rash. Using a large administrative claims database, this study compared the health care resource utilization and costs among CHC patients who developed anemia and/or rash while receiving DAA-based therapies (telaprevir and boceprevir) versus those who did not develop anemia or rash. Adjusted costs were compared by using regression analysis. Methods Adult patients with ≥1 CHC diagnosis and a prescription for boceprevir or telaprevir were selected from a US-based claims database. The date of the first DAA fill after May 13, 2011, was defined as the index date. Patients were required to have continuous eligibility and no claims for hepatitis B treatment during the 6 months before (baseline) and 12 months after (study period) the index date. Patients were categorized into 4 cohorts based on the development of anemia only, rash only, both anemia and rash (anemia/rash), or neither anemia nor rash (NAR) while receiving DAA-based therapies. Baseline characteristics and study period health care utilization and costs were compared by using univariate statistics between cohorts that developed anemia only, rash only, or anemia/rash and the cohort that did not develop anemia or rash. Adjusted costs were compared by using multivariable regressions. Findings A total of 2862 patients were identified and categorized into 4 cohorts: 1204 anemia only, 131 rash only, 188 anemia/rash, and 1339 NAR patients. During the study period, patients developing anemia and/or rash incurred significantly more outpatient, dermatologist, and total medical visits compared with the NAR cohort. The anemia-only and anemia/rash cohorts also had significantly more inpatient, emergency department, and hematologist visits, as well as significantly higher adjusted total medical costs ($18,285 and $21,435 vs $11,253), total drug costs ($76,723 and $79,689 vs $63,001), and non-CHC drug costs ($10,391 and $10,475 vs $2437). The rash-only cohort had comparable adjusted total medical and drug costs. Implications CHC patients who developed anemia while receiving DAA-based therapies incurred significantly higher resource utilization and costs compared with those who did not. The study highlights the need for new CHC treatment regimens that are associated with fewer and less severe AEs, particularly anemia. [ABSTRACT FROM AUTHOR]

Introduction: Eosinophilic gastritis and eosinophilic enteritis (EoG/EoN) are associated with a substantial clinical burden. However, limited information is available regarding the economic burden of EoG/EoN. This study was conducted to compare healthcare resource use (HRU) and costs among patients with EoG/EoN versus without EoG/EoN in the USA., Methods: Administrative claims data from the IBM MarketScan ® Commercial Claims and Encounters (CCAE) and Medicare Supplemental and Coordination of Benefits Databases (2009-2019) was used to identify two cohorts of patients. Patients without EoG/EoN were matched 3:1 to patients with EoG/EoN on sex, year of birth, and healthcare plan type. Study measures included demographic characteristics, select comorbidities, all-cause HRU, and costs. Comparisons were made over a 1-year period following EoG/EoN diagnosis for patients with EoG/EoN and an eligible date for patients without EoG/EoN., Results: A total of 2219 patients with EoG/EoN and 6657 patients without EoG/EoN were analyzed. Significantly higher proportions of patients with EoG/EoN versus without EoG/EoN had comorbid conditions. Rates of all-cause HRU were significantly higher among patients with EoG/EoN versus patients without EoG/EoN (adjusted rate ratio [95% confidence interval]: inpatient visits, 6.26 [5.26, 7.46]; outpatient visits, 1.17 [1.16, 1.19]; emergency department visits, 2.11 [1.98, 2.25]; all p < 0.001). Patients with EoG/EoN incurred significantly higher costs versus patients without EoG/EoN (adjusted mean cost difference $31,180; p < 0.001). Cost differences were largely due to outpatient (adjusted mean cost difference $14,018; p < 0.001) and inpatient (adjusted mean cost difference $11,224; p < 0.001) costs., Conclusion: The economic burden associated with EoG/EoN is substantial, with patients with EoG/EoN having a higher rate of HRU and incurring $31,180 more than patients without EoG/EoN on average. Most of the cost difference was attributable to outpatient and inpatient costs. Cost-saving strategies to lower the burden of illness in this patient population are needed., (© 2022. The Author(s).)

BACKGROUND: The rising prevalence and associated public health burden of obesity has led to advancements in pharmaceuticals for weight management. Semaglutide 2.4 mg, an anti-obesity medication (AOM) recently approved by the US Food and Drug Administration, has demonstrated clinically relevant weight loss in its phase 3 clinical trials. Economic evaluation comparing semaglutide 2.4 mg with other available weight management therapies is essential to inform payers for decision-making. OBJECTIVES: To assess the cost-effectiveness of semaglutide 2.4 mg in the treatment of adult patients with obesity (ie, body mass index [BMI] ≥ 30) and adult patients who are overweight (ie, BMI 27-29.9) with 1 or more weight-related comorbidities from a US third-party payer perspective. METHODS: A cohort Markov model was constructed to compare semaglutide 2.4 mg with the following comparators: no treatment, diet and exercise (D&E), and 3 branded AOMs (liraglutide 3 mg, phentermine-topiramate, and naltrexone-bupropion). All AOMs, including semaglutide 2.4 mg, were assumed to be taken in conjunction with D&E. Changes in BMI, blood pressure, cholesterol level, experience of acute and chronic obesity-related complications, costs, and quality-adjusted life years (QALYs) were simulated over 30 years based on pivotal trials of the AOMs and other relevant literature. Drug and health care prices reflect 2021 standardized values. Cost-effectiveness was examined with a willingness-to-pay (WTP) threshold of $150,000 per QALY gained. Sensitivity analyses were conducted to test the robustness of the cost-effectiveness results to plausible variation in model inputs. RESULTS: In the base-case analysis, treatment with semaglutide 2.4 mg was estimated to improve QALYs by 0.138 to 0.925 and incur higher costs by $3,254 to $25,086 over the 30-year time horizon vs comparators. Semaglutide 2.4 mg is cost-effective against all comparators at the prespecified WTP threshold, with the incremental cost per QALY gained ranging from $23,556 to $144,296 per QALY gained. In the sensitivity analysis, extended maximum treatment duration, types of subsequent treatment following therapy discontinuation, and weight-rebound rates were identified as key drivers for model results. The estimated probability of semaglutide 2.4 mg being cost-effective compared with comparators ranged from 67% to 100% when varying model parameters and assumptions. CONCLUSIONS: As a long-term weight management therapy, semaglutide 2.4 mg was estimated to be cost-effective compared with no treatment, D&E alone, and all other branded AOM comparators under a WTP threshold of $150,000 per QALY gained over a 30-year time horizon. DISCLOSURES: Financial support for this research was provided by Novo Nordisk Inc. The study sponsor was involved in several aspects of the research, including the study design, the interpretation of data, the writing of the manuscript, and the decision to submit the manuscript for publication. Dr Kim and Ms Ramasamy are employees of Novo Nordisk Inc. Ms Kumar and Dr Burudpakdee were employees of Novo Nordisk Inc at the time this study was conducted. Dr Sullivan received research support from Novo Nordisk Inc for this study. Drs Wang, Song, Wu, Ms Xie, and Ms Sun are employees of Analysis Group, Inc, who received consultancy fees from Novo Nordisk Inc in connection with this study.

To describe real-world treatment patterns and outcomes among adult patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA), patients were identified in the SEER-Medicare database (01/2006-12/2016); 3,046 patients with MDS treated with HMA were included. An algorithm was developed to categorize patients into MDS risk groups: the majority of patients were classified as Higher-risk (70.9%), 8.0% as Intermediate-risk, and 21.1% as Unknown-risk. Overall, 77.4% of patients initiated azacitidine and 22.6% decitabine; they received an average of 5.1 index-HMA cycles, of which 90.9% were complete with a median cycle duration of 28 days. Median survival was 11.6, 18.4, and 19.1 months for the Higher-risk, Intermediate-risk, and Unknown-risk groups, respectively. Median time-to-AML transformation was 19.3 months for the Higher-risk group and 50.4 months for the Intermediate-risk group (not reached for Unknown-risk). Data highlight the unmet medical needs of patients with MDS treated with HMA, particularly for the Higher-risk MDS group.

Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia B range from mild to severe forms of the disease. The objective of this study was to characterize the economic burden associated with differing clinical profiles of hemophilia B from a US health system perspective. Using the IBM MarketScan database (June 2011-February 2019), a claims-based algorithm was developed to identify 4 distinct profiles (mild, moderate, moderate-severe, and severe) in adult males with hemophilia B based on the frequency of hemorrhage events and factor IX replacement claims. Mean annual health care resource use (HRU) and costs were statistically compared between patients with hemophilia B (N = 454) and 1:1 demographic-matched controls (N = 454), both overall and with stratification by clinical profile. Compared with matched controls, patients with hemophilia B had a significantly higher comorbidity burden (Charlson Comorbidity Index, mean ± standard deviation [SD]: 0.9 ± 1.7 vs 0.3 ± 0.9, P < .001). Across all clinical profiles, patients with hemophilia B had significantly higher HRU vs matched controls (mean ± SD: 0.3 ± 0.6 vs 0.1 ± 0.3 inpatient admissions; 0.6 ± 1.2 vs 0.2 ± 0.6 emergency department visits; 17.7 ± 22.9 vs 8.0 ± 11.0 outpatient visits; all P < .001). Annual total health care costs per patient among patients with hemophilia B were more than 25-fold higher vs matched controls (mean ± SD: $201 635 ± $411 530 vs $7879 ± $29 040, respectively, P < .001). Annual total health care costs per patient increased with increasing severity (mean ± SD: mild, $80 811 ± $284 313; moderate, $137 455 ± $222 021; moderate-severe, $251 619 ± $576 886; severe, $632 088 ± $501 270). The findings of this study highlight the substantial burden of illness associated with hemophilia B., (© 2021 by The American Society of Hematology.)

Objective: To assess the economic burden of tenosynovial giant cell tumor (TGCT) among US employed workforce., Methods: Patients with TGCT medical claims (N = 1395) and matched controls (1:10) without TGCT claims (N = 13,950) were identified from the OptumHealth Care Solutions, Inc. database (January 1, 1999 to March 31, 2017). Adjusted regression models were used to compare healthcare resource utilization, time lost from work, and associated costs between cohorts., Results: In patients with TGCT, the rates of inpatient admissions, emergency room visits, outpatient visits, and work loss days were 2.8, 1.5, 2.2, and 2.6 times those of matched controls, respectively (all P < 0.001). Total annual all-cause healthcare costs and work loss-related costs were $9368 and $2708 higher for TGCT patients than for matched controls, respectively (all P < 0.001)., Conclusions: TGCT was associated with a significant healthcare and work loss burden on US employers., Competing Interests: Conflicts of Interest: F.L. and W.J.K. are employees of Daiichi Sankyo, Inc. and may own stocks/stock options; F.L. additionally declares owning stocks of Novartis. R.I.I., I.P., W.W., and S.S. are employees of Analysis Group, Inc., which provided paid consulting services to Daiichi Sankyo, Inc. for the conduct of this study. J.A.A. declares receiving research funding from Janssen Oncology, and Novartis., (Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American College of Occupational and Environmental Medicine.)

Aims: To describe healthcare resource utilization (HRU) and costs in patients with myelodysplastic syndromes (MDS) treated with hypomethylating agents (HMA) based on HMA-treatment response., Materials and Methods: SEER-Medicare data (January 2006-December 2016) were used to identify adults diagnosed with MDS (SEER: January 2009-December 2015) initiated on HMA (index date). HMA-treatment success (indicators: ≥7 HMA cycles, stem cell transplantation, and transfusion independence) or failure (indicators: acute myeloid leukemia [AML], AML-like treatment, and death) was determined using a claim-based algorithm. HRU and costs were assessed from the index date to 1-year post-index, overall and stratified by HMA-treatment success or failure. Among patients with HMA-treatment failure, HRU and costs were also assessed from failure to 1-year post-failure., Results: The study included 3,046 patients (mean age: 77.4 years; females: 36.8%). Rates of HMA-treatment success and failure were 44.4% and 76.2%, respectively (20.6% had HMA-treatment success then failure). Overall, patients had 15.2 inpatient admissions per-100-patients-per-month (median follow-up: 5.9 months). Patients with HMA-treatment success had 7.5 inpatient admissions per-100-patients-per-month (median follow-up: 12.0 months), while those with HMA-treatment failure had 20.4 and 35.3 admissions per-100-patients-per-month pre- and post-HMA-treatment failure, respectively (median follow-up: 4.3 and 1.8 months, pre- and post-HMA-treatment failure, respectively). Mean total healthcare costs were $12,494 per-patient-per-month overall, $8,069 per-patient-per-month among patients with HMA-treatment success, and $13,809 and $19,242 per-patient-per-month pre- and post-HMA-treatment failure, respectively. Outpatient costs (68.3%) were the main contributor of total healthcare costs overall, while inpatient costs (80.3%) were the main cost driver post-HMA-treatment failure., Limitations: Without available laboratory test results, clinical indicators observed in claims were used to assess HMA-treatment response., Conclusions: Over 75% of patients with MDS failed HMA-treatment within 6 months of initiation and were observed with more inpatient admissions than those with HMA-treatment success, translating into substantially higher healthcare costs. HMA-treatment failure results in an important economic burden in MDS patients.

Aims: This study estimated the total costs associated with tisagenlecleucel treatment in adult patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL) based on the JULIET trial from a United States hospital's perspective. Methods: An economic model was developed to assess the total costs associated with tisagenlecleucel treatment (from leukapheresis to two months post-infusion) in adults (aged ≥18 years) with r/r DLBCL using a fee-for-service approach. Costs were considered during the pre-treatment, tisagenlecleucel infusion, and follow-up periods, and were estimated based on the health resource utilization and safety data from the JULIET trial. Cost components included leukapheresis, lymphodepleting chemotherapy, tisagenlecleucel infusion/administration, inpatient and intensive care unit (ICU) admission, medical professional visits, lab tests/procedures, and management of adverse events (AEs). The base-case model estimated the total costs using observed hospitalization, ICU, and AE data from JULIET, while scenario analyses varied key assumptions related to AEs and hospitalization. Results: The estimated overall cost associated with tisagenlecleucel treatment from leukapheresis to two months post-infusion was $437,927/patient, of which $64,784 (14.8%) was additional to tisagenlecleucel's list price ($373,000) and the associated administration cost ($143). The top three key drivers of the additional cost were AE management ($30,594; 47.2%), inpatient/ICU not attributed to AEs ($24,285; 37.5%), and lab tests/procedures ($5,443; 8.4%). In the scenario analyses, total costs ranged from $382,702 (no AEs, no hospitalization) to $469,006 (cytokine release syndrome and B-cell aplasia, hospitalization). Limitations : This analysis was limited to two months of follow-up after tisagenlecleucel infusion, which cannot capture long-term safety outcomes associated with the treatment and may underestimate AE costs. Conclusions: The total cost of tisagenlecleucel administration from leukapheresis to two months was estimated at $437,927. In addition to tisagenlecleucel's price, the main drivers were AE management costs and inpatient/ICU costs. Future studies based on real-world, long-term use of tisagenlecleucel are warranted.

Background: Tisagenlecleucel was approved for the treatment of pediatric and young adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (ALL) based on the pivotal ELIANA trial., Objective: To comprehensively evaluate the total costs associated with tisagenlecleucel treatment, including costs from pre- to postinfusion periods of tisagenlecleucel in addition to the cost of tisagenlecleucel., Methods: An economic model was developed to estimate total costs associated with tisagenlecleucel treatment from the time of leukapheresis to 2 months postinfusion from a U.S. hospital's perspective. Costs were estimated based on resource use and safety management from the ELIANA trial and were considered during the pretreatment, tisagenlecleucel infusion, and follow-up periods of treatment. Cost components included leukapheresis, lymphodepleting chemotherapy, tisagenlecleucel infusion and hospital administration, inpatient and intensive care unit admissions, medical professional visits, laboratory tests and procedures, and management of major adverse events. Scenario analyses were conducted by varying key assumptions related to adverse events and hospitalization., Results: The total cost associated with tisagenlecleucel treatment among pediatric and young adult patients with r/r ALL was estimated to be $612,779, of which $137,636 (22.5%) was in addition to the list price of tisagenlecleucel ($475,000) and the associated administration cost of $143.08. The top 3 drivers of the additional cost were adverse event management ($70,968; 51.6%), inpatient and intensive care unit admissions not attributed to adverse events ($57,952; 42.1%), and laboratory tests and procedures ($5,209; 3.8%). The costs incurred during the pretreatment, infusion, and follow-up periods were $29,002, $476,659, and $107,118, respectively. In the scenario analyses, the total costs ranged from $483,169 (tisagenlecleucel treatment in the outpatient setting without adverse events) to $672,373 (tisagenlecleucel treatment in the inpatient setting with grade 3/4 cytokine release syndrome and B-cell aplasia)., Conclusions: In this economic model, tisagenlecleucel treatment among pediatric and young adult patients with r/r ALL was estimated to cost $612,779. The cost of care in addition to the price of tisagenlecleucel accounted for 22.5% of the total, with adverse event management and inpatient and intensive care unit admissions being main drivers. Further studies are warranted to assess the cost of tisagenlecleucel treatment in the context of current standards of care in real-world clinical practice., Disclosures: This study was supported by Novartis. The study sponsor was involved in several aspects of the research, including the study design, the interpretation of data, the writing of the manuscript, and the decision to submit the manuscript for publication. Hao is an employee of Novartis and has stock/stock options. Yang, Chai, Qi, and Wu are employees of Analysis Group, which received consulting fees from Novartis for work on this study. Part of the material in this manuscript was presented at the American Society of Hematology Annual Meeting held December 7-10, 2019, in Orlando, FL.

Background: Nocturia, characterized as waking during the main sleep period to urinate, is a common condition. Persistent nocturia results in sleep fragmentation with deleterious effects on health and well-being. Yet, there are limited data on the economic burden of nocturia in the United States., Objective: To assess the association of nocturia with health care resource utilization (HRU), work productivity, and self-rated health while estimating the societal costs of nocturia in the United States in 2017., Methods: A retrospective cross-sectional study was conducted using data from the National Health and Nutrition Examination Survey (NHANES; 2005-2006 to 2013-2014). Adults aged ≥ 18 years (excluding pregnant women) were stratified into individuals with nocturia (≥ 2 voids/night) and individuals without nocturia (< 2 voids/night), based on the threshold for clinically significant nocturia. Outcomes were self-reported and included HRU (hospitalizations, outpatient visits); work productivity (weekly hours worked, employment); and current health status. Multivariable regression analyses adjusting for age, race, sex, body mass index, insurance status, education level, alcohol use, smoking status, and self-reported comorbid conditions were used to compare the 2 cohorts, overall and stratified by age group (20-44 years, 45-64 years, and 65+ years) to distinguish the effects on different age groups including the Medicare-aged population. Excess direct health care costs and indirect productivity costs associated with nocturia in the United States were then calculated using a prevalence-based approach and available literature (i.e., nocturia prevalence estimates, aggregated unit costs by HRU type, and average hourly earnings in the United States)., Results: 22,300 individuals were identified, and 24% had nocturia (≥ 2 voids/night). Median age was 55.2 and 43.2 years among individuals with and without nocturia, respectively, and the proportion of males was 43.3% and 51.3%, respectively. Individuals with nocturia had significantly more HRU, including hospitalizations and outpatient visits, worked significantly fewer hours weekly, and were significantly less likely to be employed when compared with those without nocturia. They were also significantly less likely to report being in very good/excellent health. These comparisons remained statistically significant across age groups. Total excess direct health care costs were $62.9 billion (hospitalization: $47.6 billion; outpatient: $15.3 billion). Total excess indirect productivity costs were $151.7 billion. Altogether, costs were estimated at $214.5 billion, equivalent to $3,491 per individual with nocturia. Individuals aged 20-44 years incurred 23.5% of total excess costs, while those aged 45-64 and 65+ years incurred 48.2% and 28.3%, respectively. Sensitivity analyses based on lower prevalence estimates resulted in costs of $94.0 billion, while those based on higher prevalence estimates reached up to $231.1 billion., Conclusions: Nocturia is associated with a substantial economic burden in the United States even when evaluated based on lower prevalence estimates. This study underscores the importance of timely diagnosis and management of nocturia patients to alleviate health-related and economic consequences to patients and society., Disclosures: This work was supported by Ferring Pharmaceuticals, which contributed to and approved the study design and participated in the interpretation of data, review, and approval of the manuscript. Gauthier-Loiselle, Gagnon-Sanschagrin, and Wu are employees of Analysis Group, which received consultancy fees from Ferring Pharmaceuticals for work on this study. Jhaveri is a full-time employee of Ferring Pharmaceuticals. Parts of this work were presented as a poster presentation at AMCP Nexus 2018; October 22-25, 2018; Orlando, FL.

Introduction: The new direct acting antiviral (DAA) therapies are able to effectively treat chronic hepatitis C (CHC). This study elicited the preferences of CHC patients for treatment attributes of new DAAs., Methods: An online discrete choice experiment survey was designed to collect data from adult CHC patients in the USA, UK, France, Germany, Spain, and Italy. Patients were asked to choose from alternative hypothetical DAA options, defined by differing levels of nine attributes [i.e., treatment duration, tablet count and packaging, cure rate, required office visits when on treatment, modifications to statins or to proton pump inhibitors (PPIs), and risks of diarrhea, headache and nausea]. Logistic regression was used to assess preference for the treatment options., Results: A total of 328 patients with CHC completed the survey (USA, n = 227; European countries, n = 101), with a mean age of 47.7 years (SD = 14.4) and an average 11.2 years since CHC diagnosis; 51% of patients were female. More than half (60%) of the patients had treatment for CHC. Patients significantly preferred a DAA regimen with higher cure rate, shorter treatment duration, lower risks of diarrhea, headache, and nausea (all p < 0.001), reduced need for office visits when on treatment (p = 0.044), and without requiring dose reduction or timing change in PPIs (p = 0.032). Tablet counts were not found to be statistically significant., Conclusion: Given the overall high cure rates of new DAAs, CHC patients' preferences for therapy may be influenced by treatment attributes other than cure rates and tolerability. Treatments that are more convenient and require less disruption to their daily life (e.g., shorter treatment duration, no modification in PPI use, and fewer office visits when on treatment) are important to patients with CHC and should be considered when making treatment decisions., Funding: AbbVie.

Background: First-line treatments for cisplatin-ineligible patients with metastatic urothelial carcinoma (mUC) include carboplatin-based chemotherapy and checkpoint inhibitors such as atezolizumab (anti-PD-L1)., Objective: To compare overall survival (OS) among patients with mUC treated in the first-line setting with atezolizumab versus carboplatin-based chemotherapies (any carboplatin-based regimens or carboplatin-gemcitabine)., Design, Setting, and Participants: Cisplatin-ineligible patients with mUC from the phase 2 trial IMvigor210 (ClinicalTrials.gov NCT02951767) treated with atezolizumab and patients from the Veterans Health Administration (VHA) health care system (2006-2017, with IMvigor210 eligibility criteria applied using proxy measurements) treated according to normal clinical practice., Interventions: IMvigor210 cohort 1 patients were treated with atezolizumab, and real-world VHA cohorts were treated with carboplatin-based regimens., Outcome Measurements and Statistical Analysis: Entropy-balance weighting was applied to balance prespecified baseline patient characteristics. OS was analyzed using weighted Kaplan-Meier and Cox methods., Results and Limitations: The median OS was 15.0 mo with atezolizumab (n = 110), 12.1 mo with any carboplatin-based chemotherapy (n = 282), and 8.7 mo with carboplatin-gemcitabine (n = 120). An OS benefit occurred with atezolizumab versus carboplatin-based regimens after 9 mo (hazard ratio [HR] 0.43; p = 0.004) and with atezolizumab versus carboplatin-gemcitabine after 5 mo (HR 0.52; p = 0.005). Study limitations include a predominantly male VHA cohort and ≤24-mo follow-up. Adjustment for confounding, a potential limitation of nonrandomized studies, was limited by the availability of clinical measurements in the VHA data, which allowed for replication of IMvigor210 exclusions in the VHA cohorts., Conclusions: First-line atezolizumab for cisplatin-ineligible mUC may provide an OS benefit over carboplatin-based treatments after 5-9 mo, depending on the regimen., Patient Summary: Many patients with metastatic urothelial carcinoma are ineligible for cisplatin-based chemotherapy. This study compared patients from a clinical trial receiving the immunotherapeutic agent atezolizumab with those in Veterans Health Administration clinical practice receiving carboplatin-based chemotherapy. Atezolizumab provided a survival benefit over chemotherapy after 5-9 mo., (Published by Elsevier B.V.)

Objective: The retrospective study aimed to estimate prevalence of hyperkalemia using a large US commercial claims database., Methods: Adults with serum potassium lab data (2010 to 2014) and ≥1 calendar year of data were included from a large US commercial claims database. Hyperkalemia was defined as ≥2 serum potassium measurements >5.0 mEq/L or one hyperkalemia diagnosis code (ICD-9-CM, 276.7) or one sodium polystyrene sulfonate fill. Hyperkalemia prevalence was estimated for the overall population and subgroups with hyperkalemia-related comorbidities by calendar year. Hyperkalemia prevalence was also standardized to the US population to estimate the number of US adults with hyperkalemia., Results: The analysis included 2,270,635 patients (2010-2014). The annual prevalence of hyperkalemia in the overall population was 1.57% in 2014, with higher rates observed in patients with chronic kidney disease (CKD), heart failure, diabetes and hypertension. Among patients with CKD and/or heart failure, the 2014 annual prevalence was 6.35%. Among patients with hyperkalemia, 48.43% had CKD and/or heart failure in 2014. The prevalence of hyperkalemia was higher in patients with more severe CKD, as well as older patients and men. Extrapolating those results to the US population supports that 1.55% or 3.7 million US adults had hyperkalemia in 2014., Conclusions: An estimated 3.7 million US adults had hyperkalemia in 2014, and this prevalence rate has increased since 2010. In patients with CKD and/or heart failure, the annual prevalence of hyperkalemia was 6.35% in 2014, and about half of all patients with hyperkalemia have either CKD and/or heart failure.

Importance: Patients with resectable gastrointestinal stromal tumors (GISTs) might not receive the recommended duration of adjuvant therapy if their risk of recurrence is underestimated, which can have an impact on their recurrence-free survival (RFS)., Objective: To determine the extent of physician underestimation of risk of recurrence after complete primary GIST resection, the impact of underestimation on planned adjuvant treatment duration, and the association among high-risk patients of planned adjuvant treatment duration and RFS., Design, Setting, and Participants: This was a retrospective observational medical record review reported by participating oncologists in 2013. US patients with complete primary GIST resection after 2010 were grouped as underestimated or not if their oncologists' charted risk assessments were lower than assessments based on the Revised National Institutes of Health Consensus Criteria or not. Patients were followed by general community oncologists until death or the end of follow-up., Main Outcomes and Measures: Fisher exact tests compared planned adjuvant treatment duration between groups. Cox proportional-hazards models estimated the impact of planned adjuvant treatment duration on RFS., Results: A total of 109 oncologists reported information on 506 patients with GIST after primary resection (65.8% were high-risk and 8.7% were intermediate-risk). Physicians underestimated risk for 190 patients (37.5%); 30.1% of tumors with an intermediate-level mitotic count (6-10 per 50 high-powered fields) and an intermediate tumor size (6-10 cm) were correctly recognized as high-risk, as were 7.5% of nongastric tumors with an intermediate-level mitotic count and a tumor size of 2 to 5 cm. A smaller proportion of high-risk patients in the underestimated vs not-underestimated groups had at least 3 years of planned adjuvant therapy (36.1% vs 65.9%; P < .001). Planned adjuvant treatment of at least 3 years vs less than 3 years among high-risk patients conferred a lower hazard of recurrence and/or death (adjusted hazard ratio, 0.29; P < .001; 95% CI, 0.14-0.59)., Conclusions and Relevance: Overall, physicians tended to underestimate the risk of recurrence for many patients with GIST, especially for patients with tumors of intermediate size, intermediate-level mitotic count, and nongastric location, which had an impact on planned adjuvant therapy duration. Patients with at least 3 years of planned adjuvant treatment had longer RFS. Improved education on postresection risk assessment and risk reduction is needed.

Background: Chronic hepatitis C (CHC) is associated with substantial morbidity and mortality, with the future burden of disease predicted to significantly increase. The recent addition of 2 direct-acting antiviral (DAA) protease inhibitors, telaprevir and boceprevir, to peginterferon alfa (PEG) and ribavirin (RBV) therapy has been shown to significantly improve sustained virologic response rates and thus has become standard of care. While the efficacy and safety of DAAs has been assessed in the clinical trial setting, less is known about real-world use of these new therapies., Objectives: To (a) evaluate the treatment patterns, health care utilization, and costs of CHC patients receiving DAA-based therapies in the United States using a retrospective analysis of a large administrative claims database and (b) evaluate factors associated with therapy noncompletion using multivariable analyses., Methods: Adult patients with ≥ 1 claim for CHC and a prescription filled for boceprevir or telaprevir were selected from a de-identified U.S.-based claims database. The date of the first fill for a DAA after May 13, 2011 (date of first DAA availability) was defined as the index date, and patients were categorized into either the telaprevir or boceprevir cohort. Patients were required to have continuous eligibility and no claims for hepatitis B during the 6 months before (baseline) and 12 months following (study period) the index date. Baseline characteristics and study period treatment patterns, health care utilization, and costs were described. Factors associated with therapy noncompletion were examined using multivariable logistic regression, and adjusted health care costs were compared between the DAA cohorts using multivariable analyses., Results: A total of 871 telaprevir and 284 boceprevir patients were identified. DAA patients were aged 54 years on average and more often were male (60%, n = 688). Approximately 25% (n = 216) of telaprevir and 18% (n = 52) of boceprevir patients had cirrhosis, and 9% (n = 82) of telaprevir and 7% (n = 20) of boceprevir patients had decompensated cirrhosis at baseline. Less than 1% (n = 9) of patients were HIV co-infected. Approximately 54% (n = 470) of telaprevir and 74% (n = 210) of boceprevir patients did not complete the minimum duration of therapy as per the prescribing information (telaprevir: 12 weeks of triple + 12 weeks of dual; boceprevir: 3 weeks of lead-in + 24 weeks of triple). In multivariable analyses, females (vs. males) and patients taking boceprevir (vs. telaprevir) were more likely to not complete therapy (P = 0.011). CHC patients experienced high medical and drug-related resource utilization. Telaprevir patients had numerically higher study period unadjusted medical (boceprevir: $16,927; telaprevir: $19,519) and drug costs (boceprevir: $59,953; telaprevir: $76,497) than boceprevir patients; however, after adjusting for baseline characteristics, only drug costs remained significantly different (P  less than  0.001). , Conclusions: These results indicate that a large proportion of CHC patients receiving telaprevir or boceprevir did not complete minimum duration of therapy as per the prescribing information. CHC patients on a DAA regimen also experienced high resource utilization and high medical and drug costs.

Background: Clinical guidelines prefer endocrine therapy (ET) as initial treatment for post-menopausal women with hormone receptor positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) metastatic breast cancer (mBC). Chemotherapy (CT) should be reserved for patients who develop symptomatic visceral disease or have no clinical benefit after three sequential ET regimens. It is unclear if real-world clinical practice reflects these guidelines., Objective: To describe treatment patterns and treatment durations by lines of therapy for ET and CT among post-menopausal HR+/HER2- mBC patients., Methods: Charts were reviewed from a network of community-based oncology practices of eligible patients who had progressed after initiating adjuvant or first-line treatment for mBC between 1 January 2004 and 30 September 2010. Extracted chart data included demographics, treatment histories, and outcomes. Treatment duration was estimated using Kaplan-Meier estimators., Results: A total of 144 patients were studied. Patients received a median of two lines of ET, and <10% had three or more lines of ET before receiving CT. From first line to second line, the median treatment duration was 11.6 to 4.9 months for ET overall; 13.8 to 10.5 months for anastrozole; 18.6 to 7.0 months for letrozole; and 5.1 to 2.9 months for fulvestrant. For CT, the median duration was 5.1 months in the first line and 3.7 months and below in subsequent lines., Conclusion: During the study period (1 January 2004 - 30 September 2012), most patients received <3 lines of ET before receiving CT. The drop in median duration of ET from first to second line suggests that single agent ETs might not be as effective beyond the first line. A key limitation of this study was the small sample size. In addition, more research is needed to further investigate the short treatment duration of fulvestrant across early lines of therapy (which could indicate lack of efficacy).

Objective: The use of bevacizumab in advanced non-squamous non-small cell lung cancer (NSCLC) is controversial among elderly patients. This study aimed to compare overall survival for Medicare patients diagnosed with NSCLC and treated with either first-line bevacizumab-carboplatin-paclitaxel (BCP) or carboplatin-paclitaxel (CP)., Methods: Patients ≥ 65 years old, first diagnosed with non-squamous NSCLC stage IIIB/IV between 2006 and 2009, and treated with either first-line BCP or CP, were selected from the SEER-Medicare database that links cancer registry and US Medicare claims data. Kaplan-Meier estimates were used to evaluate survival. Multivariable Cox proportional hazards models were used to compare the effect of BCP versus CP on the hazard of death. Age-stratified analyses were conducted for patients aged 65-74 and ≥ 75 years., Results: Of 1706 patients in the study sample, 592 (34.7%) received BCP and 1114 (65.3%) received CP; 692 (40.6%) were ≥ 75 years. Adjusted median survival time in the BCP versus CP cohorts was 10.5 versus 8.5 months (p = 0.008). The difference in median survival favoring the BCP cohort was statistically significant for both patients aged ≥ 75 years (2.8 months, p = 0.019), and patients aged 65-74 years (1.5 months, p = 0.018). The adjusted hazard of death did not differ between the cohorts (HR: 0.96, 95% CI: 0.86-1.06); however, during the first year of follow-up, when most deaths (>60%) occurred, the hazard of death was 18% lower for the BCP cohort (HR: 0.82, 95% CI: 0.71-0.94). BCP patients also had 18% fewer hospital admissions than CP patients (adjusted incidence rate ratio (IRR): 0.82, 95% CI: 0.72-0.94) and 23% fewer inpatient days (IRR: 0.77, 95% CI: 0.65-0.91)., Conclusions: In this retrospective analysis of Medicare patients in the SEER database, first-line therapy with BCP was associated with longer survival and fewer hospitalizations than CP., (Copyright © 2014 Elsevier Ireland Ltd. All rights reserved.)

Objectives: To compare treatment patterns, resource utilization, and costs to US third-party payers of stimulant-treated adolescent attentiondeficit/ hyperactivity disorder (ADHD) patients who switched to or augmented with atypical antipsychotics (AAPs; not FDA-indicated for ADHD) with those who switched to or augmented with nonantipsychotic medications., Study Design: Retrospective cohort study conducted using a US commercial medical/pharmacy claims database., Methods: Adolescent patients with an ADHD diagnosis and ≥ 1 stimulant medication claim between January 2005 and December 2009 were identified. Patients were classified into the AAP or non-antipsychotic cohorts based on subsequent claims for AAPs or non-antipsychotic medications, respectively. Patients with psychiatric diagnoses for which AAPs are often prescribed were excluded. Patients were matched 1:1 from the AAP to the non-antipsychotic cohort using propensity score matching. Treatment patterns, resource utilization, and costs in the 12 months after AAP or non-antipsychotic initiation were compared using Cox models, Poisson regression, and Wilcoxon signed-rank tests, respectively., Results: After propensity score matching, a total of 849 adolescents were included in each of the matched cohorts. Patients in the AAP cohort had a significantly higher rate of medication augmentation (27.7% vs 15.5%; hazard ratio = 2.56; 95% confidence interval [CI], 1.90-3.46; P < .001) than patients in the non-antipsychotic cohort. The AAP cohort also had significantly higher incidences of inpatient admissions (0.13 vs 0.05; incidence rate ratio [IRR] = 2.45; 95% CI, 1.73-3.48; P < .001), emergency department visits (0.39 vs 0.31; IRR = 1.27; 95% CI, 1.08-1.49; P = .004), and outpatient visits (14.82 vs 13.19; IRR = 1.12; 95% CI, 1.10-1.15; P < .001), and incurred significantly higher mean annual medical ($3622 vs $3311; P = .002), drug ($4314 vs $2884; P < .001), and total healthcare ($7936 vs $6195; P < .001) costs., Conclusions: Stimulant-treated adolescents with ADHD who switched to or augmented with AAPs had significantly greater drug augmentation, healthcare resource utilization, and costs compared with the non-antipsychotic cohort.

Background: Liposomal amphotericin B (L-AMB) and amphotericin B lipid complex (ABLC) are both indicated for treating invasive fungal infections (IFIs) caused by Aspergillus, Candida and Cryptococcus spp. among patients who are refractory to or intolerant of conventional amphotericin B (CAB). Prior studies have suggested similar efficacies but differences in adverse event (AE) profiles between L-AMB and ABLC., Objective: Our objective was to conduct a cost-minimisation and budget impact analysis for the treatment of IFIs with L-AMB and ABLC in a US hospital setting., Methods: A Microsoft® Excel-based budget impact model was developed to estimate the costs associated with using L-AMB and ABLC for the treatment of adult patients with Aspergillus, Candida and Cryptococcus spp. infections, who are refractory to or intolerant of CAB, during a hospital stay. The model was built from a hospital perspective, and included drug costs of L-AMB and ABLC, and costs for treating drug-related AEs (i.e. nephrotoxicity with/without dialysis, infusion-related reactions, anaphylaxis, hypomagnesaemia and hypokalaemia). Average sales price was used as the drug cost estimate in the base-case analyses. The treatment duration and rates of AEs for L-AMB and ABLC were mainly obtained from a retrospective study of these two drugs in the target population using the Cerner Health Facts data. Treatment costs of AEs were obtained from the publicly available sources. The budget impact ($US, year 2011 values) was evaluated for a hypothetical hospital with 100 administrations where L-AMB and ABLC are used for the treatment of the target population by changing the market share of L-AMB and ABLC from 32/68% to an anticipated market share of 60/40% in the base-case analysis. Sensitivity analyses were conducted by varying drug costs, rates of AEs, costs of AEs and anticipated market shares of L-AMB and ABLC., Results: The estimated per-patient cost per hospital episode associated with L-AMB and ABLC use were $US14,563 and $US16,748, respectively. Cost of AEs accounted for 68.7% of the costs for L-AMB and 85.4% for ABLC. In a hypothetical hospital with 100 annual admissions of patients using these two drugs for IFIs, changing the market shares from 32/68% for L-AMB and ABLC, respectively, to 60/40% yielded a 3.8% cost reduction, which corresponded to an absolute cost savings of $US61,191. Sensitivity analyses indicated that the results were robust to changes in input parameter values in most cases., Conclusions: This study suggests that hospitals can realize cost savings by substituting L-AMB for ABLC in the treatment of IFIs. The cost savings are driven by the lower rates of AEs associated with L-AMB use compared with ABLC.

Objective: To assess treatment adherence in attention deficit/hyperactivity disorder (ADHD) patients initiated on Lisdexamfetamine (LDX) vs other FDA-approved stimulants and non-stimulant medications., Methods: ADHD patients initiated on an ADHD medication (index medication) were selected from a large US administrative claims database. Based on age and previous treatment status, patients were classified into treatment-naïve children and adolescents (6-17 years old), previously treated children and adolescents, treatment-naïve adults (over 18 years old), and previously treated adults. Furthermore, based on their index medication, patients were classified into seven mutually exclusive treatment groups: LDX, atomoxetine (ATX), osmotic release methylphenidate hydrochloride long acting (OROS MPH), other methylphenidate/dexmethylphenidate long acting (MPH LA) and short acting (MPH SA), and amphetamine/dextroamphetamine short acting (AMPH SA) and long acting (AMPH LA). Treatment adherence (proportion of days covered by the index medication ≥0.8) over a 12-month period was compared across treatment groups using multivariate logistic regression models., Results: In children and adolescents, LDX patients were more likely to be adherent compared to patients in each of the other treatment groups, except in treatment-naïve patients where LDX patients had a similar likelihood (p = 0.6925) and were less likely (p = 0.0004) to be adherent compared to ATX and OROS MPH patients, respectively. In adults, the LDX treatment group was also more likely to be adherent compared to each of the other treatment groups, except compared to AMPH LA, where statistically insignificant differences were observed (previously treated: p = 0.6471, treatment-naïve: p = 0.0733)., Limitations: ADHD severity information was not available in the database. Accordingly, this study did not control for ADHD severity., Conclusion: Overall, LDX-treated patients demonstrated a better treatment adherence compared to patients initiated on other ADHD medications, except for AMPH LA in adult and OROS MPH and ATX in treatment-naïve children and adolescents.

Background: Results from a phase III clinical trial showed that denosumab significantly reduced the risk of first on-study and subsequent skeletal-related events (SREs) compared with zoledronic acid. This study aims to assess the cost-effectiveness of denosumab vs. zoledronic acid in the prevention of SREs in patients with advanced breast cancer and bone metastases., Materials and Methods: A Markov model was developed with 4-week model cycles and a 1-year time horizon. The health states were defined by SRE status (no SRE, first on-study SRE, subsequent SRE, no SRE but history of SRE) and SRE type (pathologic fracture, radiation to the bone, surgery to the bone, spinal cord compression). Costs (in 2011 US dollars) included drug, SRE treatment, and adverse event (AE) costs and were assessed from a third-party payer perspective. The primary outcome was incremental total cost per SRE avoided; the secondary outcome was incremental total cost per pathologic fracture avoided. One-way and probabilistic sensitivity analyses were used to assess the robustness of the model., Results: During the 1-year treatment period, denosumab incurred $7522 higher costs ($30,033 for denosumab and $23,511 for zoledronic acid), 0.06 fewer SREs, and 0.02 fewer pathologic fractures per patient, which led to an incremental total cost per SRE and pathologic fracture avoided of $114,628 and $290,136, respectively, compared with zoledronic acid. Results were robust to 1-way and probabilistic sensitivity analyses., Conclusion: Although denosumab demonstrated superiority in preventing SREs in the phase III trial, it may not be cost-effective compared with zoledronic acid because of its high cost., (Copyright © 2012 Elsevier Inc. All rights reserved.)

Background: Attention-deficit hyperactivity disorder (ADHD) is a common psychiatric disorder in childhood, affecting 3-7% of school-age children in the US and imposing substantial economic burden. Stimulants are considered first-line pharmacological treatment and are the most prescribed treatment for ADHD. However, approximately 30% of children with ADHD do not have an optimal response to a single stimulant and may require adjunctive therapy., Objective: Our objective was to conduct a cost-effectiveness analysis (CEA) of adding a non-stimulant, guanfacine extended release (GXR), to stimulants versus maintaining existing stimulant monotherapy in the treatment of ADHD in children and adolescents with suboptimal response to stimulant monotherapy., Methods: A 1-year Markov model was developed to estimate costs and effectiveness from a US third-party payer perspective. Effectiveness was measured by the QALY. The model assumed that patients transitioned among four health states (normal, mild, moderate and severe), defined by the Clinical Global Impression-Severity (CGI-S) scale. Transition probabilities were estimated in an ordered logit model using patient-level data from a multicentre, 9-week, double-blind, placebo-controlled, dose-optimization study, where subjects (n = 461) continued their stable morning stimulant and were randomized to GXR administered in the morning, GXR administered in the evening, or placebo. The model assumed that patients in moderate/severe health states after week 8 would discontinue ADHD treatment and remain in that state for the rest of the study period. Direct costs included drug wholesale acquisition costs and health state costs, all in $US, year 2010 values. Utility associated with each health state was obtained from the literature and disutilities associated with adverse events were applied for the first 4 weeks. One-way sensitivity analyses and probabilistic sensitivity analysis (PSA) were conducted by varying costs, utilities, adverse-event duration, and transition probabilities., Results: Compared with maintaining existing stimulant monotherapy, adding GXR to existing stimulant monotherapy was associated with an incremental drug cost of $US1016 but a lower medical cost of $US124, resulting in a total incremental cost of $US892 at 1 year. The addition of GXR to stimulants led to an incremental QALY of 0.03 and an incremental cost-effectiveness ratio (ICER) of $US31,660/QALY. In one-way sensitivity analysis, ICER values ranged from $US19,723, when 100% of patients were assumed to be severe in their initial health state, to $US46,631, when the last observed states from the clinical trial were carried forward to the end of the 1-year analysis period. PSA demonstrated a 94.6% likelihood that the ICER falls below $US50,000/QALY., Conclusions: The impairment associated with residual ADHD symptoms after stimulant therapy is becoming increasingly recognized. This is the first analysis of the cost effectiveness of stimulants combined with an adjunctive medication. This study suggests that the adjunctive therapy of GXR with stimulants is a cost-effective treatment based on a willingness-to-pay threshold of $US50,000/QALY. This may address an unmet need among patients with suboptimal response to stimulant monotherapy.

Objective: To characterize treatment patterns and measure the economic burden associated with metastatic (mHNC) and recurrent, locally-advanced head and neck cancer (rHNC)., Methods: Administrative claims from Medicare- and privately-insured individuals during 2004-2008 were used in this retrospective database study of patients with advanced HNC. Patients diagnosed with HNC were matched 1:1 to cancer-free controls to measure the incremental economic burden of HNC. Outcomes of interest were measured during the 6 months following the date of a secondary tumor diagnosis for metastatic patients or the date of a diagnosis indicating rHNC. To assess treatment patterns, HNC patients were evaluated for the use frequency of treatments (radiotherapy, chemotherapy and surgery). Costs were reported in 2008 US$ from a third-party payer perspective and were analyzed using generalized linear models and two-part regression models adjusting for differences in age and baseline Charlson Comorbidity Index (excluding cancer diagnoses) between the HNC and control cohorts. Components of cost included inpatient, outpatient and other medical services as well as pharmacy costs., Results: The mHNC cohort consisted of 1042 patients and the rHNC cohort included 324 patients. The most common treatments for mHNC patients were supportive care (90.2%), radiation therapy (48.5%), surgery (41.9%) and chemotherapy (38.3%). Patients with rHNC frequently received HNC-related supportive care (71.0%), radiation therapy (67.9%) and chemotherapy (27.2%); HNC-related surgery was infrequent (12.7%) during the study period. The 6-month incremental adjusted total costs were $60,414 per patient for mHNC and $21,141 per patient for rHNC (p<0.0001). Approximately 46-58% of the incremental cost was attributable to outpatient visits, 27-37% to inpatient costs and 11-13% to pharmacy, depending on the HNC cohort., Limitations: The identification of mHNC/rHNC was based on diagnosis codes and treatment patterns with the limitation of the claims database., Conclusions: Metastatic and recurrent, locally-advanced HNC patients frequently receive cancer-related treatments and incur substantial economic burden.

Objective: Compare treatment persistence and health care costs of major depressive disorder (MDD) Medicaid patients treated with escitalopram versus citalopram., Design: Retrospective analysis of Medicaid administrative claims data., Methodology: Analyzed administrative claims data from the Florida Medicaid program (07/2002-06/2006) for patients ages 18-64 years with 21 inpatient claim or 2 independent medical claims for MDD. Outcomes included discontinuation and switching rates and prescription drug, medical, and total health care costs, all-cause and related to mental disorder. Contingency table analysis and survival analysis were used to compare outcomes between treatment groups, using both unadjusted analysis and multivariate analysis adjusting for baseline characteristics., Results: The study included 2,650 patients initiated on escitalopram and 630 patients initiated on citalopram. Patients treated with escitalopram were less likely to discontinue the index drug (63.7% vs. 68.9%, P=0.015) or to switch to another second-generation antidepressant (14.9% vs. 18.4%, P=0.029) over the six months post-index date. Patients treated with escitalopram had $1,014 lower total health care costs (P=0.032) and $519 lower health care costs related to mental disorder (P=0.023). More than half of the total cost difference was attributable to savings in inpatient hospitalizations related to mental disorder ($571, P=0.003) and to outpatient costs ($53, P<0.001). Escitalopram therapy was also associated with $736 lower medical costs related to mental disorder (P=0.009)., Conclusion: In the Florida Medicaid program, compared to adult MDD patients initiated on citalopram, escitalopram patients have better treatment persistence and lower total health care costs due to any cause and due to mental disorder, mostly driven by lower hospitalization costs related to mental disorder.

Objective: To compare persistence and adherence among patients with chronic obstructive pulmonary disease (COPD) treated with either multiple- or single- long-acting maintenance inhalers., Methods: Patients with ≥2 COPD medical claims and ≥2 prescriptions for a long-acting inhaler within 1 year were classified as single- or multiple-inhaler users based on their treatment regimen (MarketScan database; 2004-2008) and matched on demographics and statistically significant baseline characteristics. Persistence, analyzed via time to treatment discontinuation, and treatment adherence, measured by proportion of days covered (PDC), were compared between the two groups over a 12-month period. Sensitivity analyses were conducted in patients with poorly and well-controlled disease., Results: A total of 23,494 patients were grouped into 11,747 matched pairs. After adjusting for confounding factors, multiple-inhaler users had a significantly higher discontinuation rate [Hazard ratio = 1.40, p < 0.0001] compared with single-inhaler users. Multiple-inhaler users were less likely to be adherent than single-inhaler users with an average PDC of 0.51 (SD = 0.272) vs. 0.55 (SD = 0.279), respectively (p < 0.0001). These results were consistent for the poorly- and well-controlled disease groups., Conclusions: Multiple long-acting inhaler users demonstrated lower treatment persistence and adherence rates than single long-acting inhaler users. Limitations of the study are related to the retrospective, observational design and use of claims data.

Background: Exacerbations are a major contributor to the large burden of treating chronic obstructive pulmonary disease (COPD). Estimates of exacerbation costs in the United States are limited., Objective: To estimate incremental costs associated with COPD exacerbation, particularly severe exacerbation, in the United States., Methods: COPD patients with at least one exacerbation were identified in the Thomson Reuters MarketScan administrative claims database. A COPD exacerbation was defined as patient use of oral or parenteral corticosteroids on the same day or within 7 days following a claim with a COPD diagnosis. Severe exacerbation was further defined if the exacerbation was associated with hospitalization or death. Healthcare costs and exacerbations were evaluated at quarterly intervals starting from patients' first observed claim with COPD diagnostic code in the database. Incremental costs associated with exacerbation were estimated as cost differences between quarters with exacerbation and quarters without exacerbation., Results: A total of 2644,174 patient-quarters, derived from 228,978 COPD patients, were included in the analysis. The average patient was followed an average of 2.9 years. The mean total cost was $17,016 per patient-quarter with severe exacerbation, $6628 per patient-quarter with non-severe exacerbation, an average of $8726 per patient-quarters with any exacerbation compared to $4762 per patient-quarter with no exacerbation. After adjusting for patient demographics, the mean incremental total cost was $11,261 per patient-quarter with severe exacerbation, $1509 per patient-quarter for non-severe exacerbation, and $3439 per patient-quarter with any exacerbation compared with patient-quarters with no exacerbation., Limitations: The method used for defining exacerbations does not capture mild exacerbations. Additional limitations exist due to the nature of claims data., Conclusions: Exacerbations, especially severe ones, result in a significant economic burden for third-party payers. Effective management of COPD and prevention of exacerbations may lead to improved patient outcomes and reduction in total healthcare costs for long-term management of COPD.

Objective: To compare the health care costs of patients with metastatic colorectal cancer (mCRC) who received second-line treatment with Avastin (bevacizumab) versus Erbitux (cetuximab), from the third-party payer's perspective., Methods: Patients with mCRC were selected from the PharMetrics claims database if they received second-line therapy containing either bevacizumab (second-line bevacizumab cohort) or cetuximab (second-line cetuximab cohort). Six-month costs following second-line therapy start date and average monthly healthcare costs while on second-line therapy (in 2009 US$) were calculated and compared between the two groups., Results: A total of 2188 patients with mCRC who met the eligibility criteria were included in the analysis, including 1808 patients receiving bevacizumab and 380 patients receiving cetuximab in second-line treatment. Demographic and baseline characteristics were similar between the two groups. Patients' mean age was 61 years and 56% were males. In second-line treatment, bevacizumab was commonly used with oxaliplatin (43.5%) and irinotecan-based regimens (40.4%), whereas cetuximab was commonly used with irinotecan-based regimens (68.2%). Bevacizumab patients had significantly lower total all-cause healthcare costs than cetuximab patients (adjusted difference: -$10,231, p = 0.020), and lower medical costs (-$10,796, p = 0.012) during the 6 months following second-line therapy initiation. Approximately half of the difference in total all-cause healthcare costs was attributable to the lower chemotherapy and targeted therapy costs (-$5635, p = 0.032) of bevacizumab patients than those of cetuximab patients. While on second-line therapy, bevacizumab patients also had lower average monthly all-cause healthcare costs than cetuximab patients., Limitations: Second-line treatment in the current study was defined based on changes in mCRC medications, not based on disease progression due to the limited clinical information available in claims., Conclusion: The use of bevacizumab in second-line therapy was associated with significantly lower healthcare costs in mCRC patients, compared to the use of cetuximab.

Objectives: To compare compliance/persistence, health care resource utilization, and costs associated with single-pill combination (SPC) vs. free-combination (FC) therapies among adult hypertension patients at the national and state level. Combination therapies with angiotensin receptor blocker (ARB) + calcium channel blocker, ARB + hydrochlorothiazide, and angiotensin-converting enzyme inhibitor + hydrochlorothiazide were evaluated., Methods: Patients initiated on SPC or FC were identified in the MarketScan Database (2006-2008). Multivariate regression models were used to compare the health care outcomes of SPC vs. FC use during the 6-month study period. National- and state-level outcomes were analyzed and reported. Compliance was measured by medication possession ratio (MPR), and persistence was assessed based on treatment discontinuation (i.e., a lapse in therapy exceeding 30 days). Utilization and cost outcomes included frequencies of inpatient and emergency room (ER) visits and changes in health care costs from baseline., Results: Adjusting for baseline characteristics, SPC patients (N = 382,476) demonstrated significantly higher MPR than FC patients (N = 197,375) (difference = 11.6%; 95% confidence interval [CI]: 11.4%, 11.7%). SPC patients had fewer all-cause hospitalizations (adjusted incidence rate ratio [IRR] = 0.77; 95% CI: 0.75, 0.79) and ER visits (adjusted IRR = 0.87; 95% CI: 0.86, 0.89) than FC patients. Results for cardiovascular-related utilization were similar to all-cause results. Compared to FC, SPC patients showed significantly greater reductions post-therapy initiation in all-cause medical costs by -$208 (95% CI: -$302, -$114), but larger increases in hypertension-related prescription costs by $53 (95% CI: $51, $55). State-level results were generally consistent in magnitude and direction for comparisons of compliance and utilization, with greater regional variation in costs. Limitations include the possibility of residual confounding from factors not observable in claims., Conclusion: SPC use was associated with significantly better compliance/persistence and fewer hospitalizations and ER visits than FC in hypertensive patients at the national level and in almost all states. Larger reductions in medical costs with SPC use more than offset higher drug costs within most states.

Background: Proton pump inhibitors (PPIs) are the most commonly used pharmacological treatment for gastroesophageal reflux disease (GERD)., Objective: To examine the utilization patterns of PPIs and other GERD-related medications, satisfaction with PPI treatment and presence of GERD symptoms., Patients and Methods: GERD patients using prescription PPIs were identified from a mixed-model HMO health plan. Utilization patterns of PPIs and other GERD medications, satisfaction with PPI treatment and presence of GERD symptoms were assessed using questionnaires., Results: Among the 617 patients who completed the survey, 71.0% used PPIs once a day (QD), 22.2% used twice a day (BID) and 6.8% more than twice a day or on an as-needed basis. Approximately 42.1% of all patients supplemented their prescription PPIs with other GERD medications, including over-the-counter medications and H(2)-receptor antagonists. Over 85% of the patients still experienced GERD symptoms and 82.7% nighttime symptoms. Overall, 72.8% of all patients were satisfied or very satisfied with their PPI treatment., Limitations: The study used self-reported data which may have been subject to recall bias. As the study was conducted in a specific region of the US, the results may have limited generalizability to other US regions or countries., Conclusions: Patients on PPI treatment often experience GERD symptoms and supplement their prescription PPIs with other GERD medications. A substantial proportion of GERD patients receiving PPI treatment are on a BID regimen. Furthermore, more than a quarter of the patients are not completely satisfied with their PPI treatment.

Objective: We examined the association between serum uric acid (SUA) level and the frequency, risk, and cost of gout flares among the elderly., Methods: Data were extracted from the Integrated Healthcare Information Services claims database (1999-2005). Patients were included if they had gout, were aged 65 years and older and had both medical and pharmacy benefits, and electronic laboratory data. Patients with gout and gouty episodes were identified using algorithms based on ICD-9-CM codes and medications. Logistic regression and negative binomial regressions were used to study the relationship between SUA concentration and the annual frequency and one-year risk of gout episodes. Generalized linear models were used to examine the direct healthcare costs associated with gout episodes in the 30 days following each episode., Results: Elderly patients with gout (n = 2237) with high (6-8.99 mg/dl) and very high (> 9 mg/dl) SUA concentrations were more likely to develop a flare within 12 months compared to patients with normal (< 6 mg/dl) SUA levels (OR 2.1, 95% CI 1.7-2.6; OR 3.4, 95% CI 2.6-4.4, respectively). In multivariate regressions, the average annual number of flares increased by 11.9% (p < 0.001) with each unit-increase in SUA level above 6 mg/dl (p < 0.001). Among patients with very high SUA levels, average adjusted total healthcare and gout-related costs per episode were $2,555 and $356 higher, respectively, than those of patients with normal SUA levels (both p < 0.001)., Conclusion: Higher SUA levels are associated with increased frequency and risk of gout episode, and with higher total and gout-related direct healthcare costs per episode.

Objectives: To compare outcomes of induction dosing followed by continuous adalimumab treatment with those of induction dosing with reinitiation of adalimumab (in the event of clinical deterioration) for patients with moderate-to-severe Crohn's disease (CD) who participated in the Crohn's Trial of the Fully Human Antibody Adalimumab for Remission Maintenance (CHARM)., Methods: In the CHARM trial, all patients received open-label induction therapy with adalimumab 80 mg and 40 mg at weeks 0 and 2, respectively. In total, 778 patients were randomized at week 4 to one of three groups: (1) placebo after initial induction doses (followed by reinitiation of adalimumab therapy); (2) continuous maintenance treatment with adalimumab 40 mg every other week (e.o.w.); and (3) continuous maintenance treatment with adalimumab 40 mg every week. At/after week 12, patients receiving placebo with flare or non-response could reinitiate open-label adalimumab 40 mg e.o.w., and patients receiving continuous blinded adalimumab therapy could switch to open-label 40 mg e.o.w. Patients in all groups could switch to weekly therapy with continued flare/non-response. In the previously published primary analysis, results for only those patients who had responded at week 4 (decrease in Crohn's Disease Activity Index (CDAI) of > or = 70 points, referred to as "randomized responders") and remained on blinded therapy were analyzed. In this analysis, data from all randomized patients were analyzed based on original randomized treatment using an intention-to-treat analysis, regardless of whether they subsequently switched to open-label therapy. Disease activity, clinical remission, number of flares, Inflammatory Bowel Disease Questionnaire (IBDQ) score, number of CD-related surgeries, and hospitalization incidence were compared between the continuous and induction only/reinitiation adalimumab groups., Results: Results for all outcome measures were superior for both continuous groups compared with the induction only/reinitiation group. On the basis of median CDAI and IBDQ results, patients in both continuous treatment groups achieved statistically significantly greater improvements vs. the induction only/reinitiation group (P < 0.05). At week 56, a significantly greater percentage of patients who had received continuous adalimumab (51% for e.o.w. and 49% for weekly) were in clinical remission vs. the induction only/reinitiation group (38%, P < 0.05). Continuous adalimumab therapy was also associated with fewer flares and fewer CD-related surgeries (P < 0.05). Patients in both continuous adalimumab groups had significantly lower risks of CD-related and all-cause hospitalizations than did patients in the induction only/reinitiation group (P < 0.05)., Conclusions: For patients with active CD, continuous treatment with adalimumab was more effective than a strategy of induction dosing followed by reinitiation of adalimumab with clinical deterioration for maintenance of clinical remission, improved quality-of life outcomes, reduced flares, and a decrease in number of surgeries and risk of hospitalization.

Objective: To compare medical and cost profiles of patients treated for depression classified by treatment pattern groups., Methods: An analysis used de-identified 1999-2004 employer claims data (n=2.9 million beneficiaries) for employees with > or =1 diagnosis of major depressive disorder and > or =1 antidepressant prescription, following a 6-month washout period of no antidepressant prescription. Patients were classified into switcher/discontinuer/augmenter/maintainer during Healthcare Effectiveness Data and Information Set-defined initial and subsequent treatment periods, then grouped into stable, intermediate, or non-stable treatment groups, based on stability of treatment patterns. Medical/cost profiles for 6-month pre- and 12-month post-index periods were compared descriptively, and multivariate regressions were estimated, controlling for baseline characteristics/severity markers. Cost savings reflect differences between treatment pattern groups from a current US perspective., Results: Of the 5,225 patients meeting inclusion criteria, 60.8% were in stable, 24.5% in intermediate and 14.7% in non-stable treatment groups. No significant differences existed in medical profiles and costs between the three groups in the pre-index period. In the post-index period, stable group patients had lower costs compared to intermediate and non-stable groups. Stable group patients generated cost savings of $1,842 compared to intermediate and $5,231 compared to non-stable groups. Multivariate analysis confirmed these findings., Conclusion: Patients on a more stable treatment regimen yield significant cost savings compared to patients on a less stable regimen.

Objective: To describe the utilization patterns, particularly dosage-escalation patterns, and economic implications of etanercept in the treatment of moderate to severe psoriasis in a real-world setting., Methods: Patients with psoriasis receiving etanercept were identified from the Integrated Health Care Information Services database and were observed for 12 months or until etanercept discontinuation (defined as gap of >60 days between prescriptions). Patients were excluded if they had other autoimmune conditions or received TNF antagonists within 6 months of the index date. Ratios of patients with dosage increase to total sample were calculated. Among patients continuing treatment for 1 year, etanercept dosage and drug costs (measured by average wholesale price) were compared for patients with and without dosage increase using the Wilcoxon signed rank test., Results: 55.2% of patients discontinued during the study year; 51.6% of patients initiated at 100 mg/week; and 34.8% who initiated at 50 mg/week required dosage increases. Among patients continuously treated for 1 year, dosage increase resulted in incremental annual drug costs of $8,440 and $9,313 for 100 and 50 mg/week, respectively (both p < 0.0001). The annual dosage of etanercept in excess of the labeled amount translated into $2,040 and $3,032 greater etanercept costs per patient in the 100 and 50 mg/week groups, respectively., Conclusion: In this analysis, 33-50% of patients with psoriasis required dosage increases during their first year of etanercept therapy, resulting in increased annual treatment costs as compared with expected costs imputed from label indications. Because of patient selection criteria, the findings may not be representative of the entire population of patients with psoriasis.

Background: Coronary heart disease (CHD) affects 15.8 million Americans. However, data on the national impact of CHD on health-related quality of life, particularly among people of different age, sex, racial, and ethnic groups, are limited., Methods and Results: Using data from the 2000 and 2002 Medical Expenditure Panel Survey, we examined various measures of patient-reported health status, including health-related quality of life, in the CHD and non-CHD populations and differences in the measures among demographic subgroups. These measures included short-form generic measures (Short Form 12; Mental Component Summary-12 and Physical Component Summary-12) and EuroQol Group measures (EQ-5D index and EQ visual analog scale). Ordinary least-squares regressions were used to adjust for sociodemographic characteristics, risk factors, comorbidities, and proxy report. The adjusted difference between the CHD and non-CHD populations was -1.2 for Mental Component Summary-12 (2.4% of the score in the non-CHD population), -4.6 for Physical Component Summary-12 (9.2%), -0.04 for EQ-5D (4.6%), and -7.3 for EQ visual analog scale (9.0%) (all P<0.05). Differences among demographic subgroups were observed. Particularly, compared with whites, the differences between CHD and non-CHD in blacks were bigger in all measures except Physical Component Summary-12. A significantly bigger difference in Mental Component Summary-12 also was observed among Hispanics compared with non-Hispanics., Conclusions: CHD is associated with significant impairment of health-related quality of life and other patient-reported health status in the US adult population. Differences in the impairment associated with CHD exist across different age, racial, and ethnic groups. In addition to preventing CHD, effective public health interventions should be aimed at improving health-related quality of life and perceived health status in the CHD population, especially the most vulnerable groups.

Healthcare costs were determined for mild persistent asthma patients (n=796) who used inhaled corticosteroids infrequently (0 to 2 claims) or consistently (3 or more claims). Study patients, selected from a privately insured claims database (1999-2003), had at least one asthma diagnosis, no diagnosis of chronic obstructive pulmonary disease (COPD), and mild persistent asthma as defined by the 2005 Health Plan Employer Data and Information Set (HEDIS), Leidy's reliever and oral steroid methods, and the 2004 Global Initiative for Asthma (GINA) guidelines. Healthcare and asthma-specific costs were significantly higher for the infrequent inhaled corticosteroid users than the consistent users. The infrequent inhaled corticosteroid users had significantly more hospitalizations and emergency department visits compared with consistent users.

Background: Schizophrenia is a debilitating chronic mental illness. However, the annual prevalence of schizophrenia is not well understood because of under-representation of schizophrenia patients in epidemiological surveys. This study used multiple administrative claims databases to estimate the annual prevalence of diagnosed schizophrenia in the USA., Method: The annual prevalence of diagnosed schizophrenia in the USA was estimated for different health insurance coverage groups. The prevalence for privately insured individuals was calculated from an administrative claims database of approximately 3 million privately insured beneficiaries covering the period 1999-2003. The prevalence for Medicaid enrollees was calculated from California Medicaid claims covering the period 2000-2002. The prevalence for Medicare and Medicaid/Medicare dual eligibles was estimated using a combination of both databases. Published statistics were used to estimate the prevalence of schizophrenia in the uninsured and veteran populations and to weight the prevalence rates obtained to the population of the USA., Results: The 12-month prevalence of diagnosed schizophrenia in the USA in 2002 was estimated at 5.1 per 1000 lives. The Medicaid population was identified with the highest prevalence rate among the populations studied. Sensitivity analyses taking into consideration the Veterans Affairs population only changed the estimate slightly to 5.3 per 1000 lives., Conclusion: Analyses of administrative claims data contribute to the understanding of the prevalence of diagnosed schizophrenia.

Background and Purpose: Stroke is a major cause of long-term disability in the United States. This study examined the national impact of stroke on health-related quality of life (HRQoL) and disparities in HRQoL across different demographic groups., Methods: Combined 2000 and 2002 Medical Expenditure Panel Survey data were used, which include quality-of-life measures based on the short-form generic measures (SF-12) and the EuroQol Group measures (EQ-5D index and EQ VAS) for 39,680 adults aged >18 years. Stratified analysis and ordinary least square regressions were used to compare HRQoL scores between stroke and nonstroke populations., Results: The study included 1040 noninstitutionalized stroke survivors. After adjustment for sociodemographics, risk factors, and comorbidities, stroke survivors had statistically significantly lower mean scores for mental health (-4.1%), physical health (-7.9%), health utility (-6.9%), and self-rated health (-7.2%) (all P<0.01). In general, stroke did not affect differences in HRQoL among age or gender groups. However, racial and ethnic disparities in HRQoL were greater among stroke survivors than nonstroke individuals, particularly in health utility scores for black vs white participants (-0.06 in stroke survivors and -0.02 in the nonstroke population, P<0.01) and Hispanic versus non-Hispanic participants (-0.11 in stroke survivors and -0.01 in the nonstroke population)., Conclusions: Stroke significantly impairs HRQoL in the United States. The findings suggest that racial and ethnic disparities in HRQoL among stroke survivors are more pronounced than in the nonstroke population. The burden of nonfatal stroke, especially among racial and ethnic minorities, should be recognized more widely.

Objective: This study assesses the direct (medical and drugs) and indirect (work loss) annual costs associated with Atrial Fibrillation (AFIB) in a privately insured population., Background: AFIB is the most common sustained rhythm disturbance, affecting 2.3 million people in the United States., Methods: Cost and co-morbidity measures for AFIB patients were compared to matched controls using a privately insured administrative database (including medical, drug, and disability claims) for 2 million enrollees, 1999-2002 from 16 employers across the United States. Patients with an AFIB diagnosis on at least two occasions were included in the co-morbidity analysis (n = 3944). A non-AFIB control sample was randomly selected with a 1:1 ratio, with characteristics (i.e., age, gender, health plan) matched to AFIB patients. Excess medical costs (i.e., employer payments) were estimated for AFIB patients, defined as the difference in average annual costs between AFIB and control patients (n = 3944); excess work-loss costs were defined similarly for employees with available work-loss data (n = 603). Statistical significance in the descriptive analysis was measured by paired t-tests for cost, or Chi-square tests for co-morbidity comparisons. A two-part multivariate model of excess cost was further estimated to control for co-existing conditions and other patient characteristics. The excess costs of AFIB patients were estimated as the difference between the observed costs of AFIB patients and their estimated costs, assuming they did not have AFIB., Results: The multivariate analysis found that the excess annual direct cost of AFIB was $12,349 (p < 0.01), with AFIB patients approximately 5 times as costly as non-AFIB individuals ($15,553 versus $3204, respectively). The excess annual total cost was $14,875 (p < 0.01), with AFIB patients again approximately 5 times as costly as non-AFIB individuals ($18,454 versus $3,579, respectively). AFIB was associated with increased incidence of atrial flutter (p < 0.01), heart failure (Relative Risk (RR) = 29, p < 0.01), other arrhythmias/conduction disorders (RR = 16, p < 0.01), heart attack (RR = 8, p < 0.01), and stroke (RR = 6, p < 0.01)., Conclusions: AFIB is a costly disease and one for which more definitive therapies might offer some potential for reducing, not only the clinical impact, but also the economic burden of the disease.