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Routine screening is recommended for HIV detection. HIV risk estimation remains important. Our goal was to validate the Denver HIV Risk Score using a national cohort from the Centers for Disease Control and Prevention. Patients of 13 years and older were included, 4,830,941 HIV tests were performed, and 0.6% newly diagnosed infections were identified. Of all visits, 9% were very low risk (HIV prevalence = 0.20%), 27% low risk (HIV prevalence = 0.17%), 41% moderate risk (HIV prevalence = 0.39%), 17% high risk (HIV prevalence = 1.19%), and 6% very high risk (HIV prevalence = 3.57%). The Denver HIV Risk Score accurately categorized patients into different HIV risk groups.

Little is known about health outcomes in severe asthma reflected by Global Initiative for Asthma steps 4 and 5.To analyze control, risk, economic, and health resource use (HRU) outcomes associated with treatment escalation to Global Initiative for Asthma steps 4 and 5.This was a before-vs-after retrospective cohort study of patients (12-75 years old) with asthma newly initiated to omalizumab, high-intensity corticosteroids (HICS; ≥1,000 μg/day of inhaled fluticasone equivalent or oral prednisone), or high-dose inhaled corticosteroid (HDICS; ≥500 to1,000 μg/day of fluticasone equivalent) using 2002 to 2011 MarketScan data. Poisson regression was used to model HRU outcomes; Tobit regression was used to model medical expenditures.Of 19,227 patients, 856 initiated omalizumab, 6,926 initiated HICS, and 11,445 initiated HDICS. Use of β-agonist increased for the HDICS and HICS cohorts and decreased for the omalizumab cohort; acute care visits and oral corticosteroid use decreased during follow-up for the HDICS and omalizumab cohorts. Annual health care expenditures, polypharmacy burden, and outpatient visits were high for all cohorts and increased in the follow-up year (baseline to follow-up; general health care expenditures: omalizumab $14,071 to $34,887, HICS $12,030 to $15,557, HDICS $7,570 to $9,826; annual number of asthma prescriptions: omalizumab 11.74 to 19.46, HICS 7.8 to 12.44, HDICS 5.17 to 9.69; outpatient visits: omalizumab 26.79 to 34.06, HICS 18.78 to 21.37, HDICS 15.06 to 16.64).Omalizumab use was associated with improvements in risk and control accompanied by large increases in expenditures per HRU. Patients on HDICS and HICS showed improvements in risk but worsening control and increased expenditures per HRU. Innovations in disease management and available treatment options are needed to more optimally achieve treatment goals.

Cost-effectiveness models for diabetes link glycated hemoglobin (HbA1c) to diabetes-related complications. Independent of diabetes-related complications, there is little known on the association between HbA1c and health utility scores. This link can alter the cost effectiveness of interventions designed to improve HbA1c. The cross-sectional relationship between HbA1c and health utility scores in adult type 1 diabetes patients was estimated after adjusting for diabetes-related complications. The EuroQoL-5 dimension (EQ-5D) questionnaire and an ad hoc survey requesting demographic information and adherence to glucose monitoring therapies was administered to adult type 1 diabetes patients during a clinic visit and combined with clinical medical record data. Health utility scores were derived using the US time-tradeoff valuation of the EQ-5D. Linear regression was used to estimate the relationship between HbA1c and utility, adjusting for treatments, demographics, and diabetes-related complications. Among 176 patients, mean (standard deviation [SD]) age was 38 (12.2) years, duration of disease was 22 (12.1) years, and number of chronic conditions other than type 1 diabetes was 2.7 (2.0). Unadjusted mean (SD) utility was 0.94 (0.09) for those with HbA1c levels

Background The Centers for Disease Control and Prevention recommends nontargeted opt-out HIV screening in healthcare settings. Cost effectiveness is critical when considering potential screening methods. Our goal was to compare programmatic costs of nontargeted opt-out rapid HIV screening with physician-directed diagnostic rapid HIV testing in an urban emergency department (ED) as part of the Denver ED HIV Opt-Out Trial. Methods This was a prospective cohort study nested in a larger quasi-experiment. Over 16 months, nontargeted rapid HIV screening (intervention) and diagnostic rapid HIV testing (control) were alternated in 4-month time blocks. During the intervention phase, patients were offered HIV testing using an opt-out approach during registration; during the control phase, physicians used a diagnostic approach to offer HIV testing to patients. Each method was fully integrated into ED operations. Direct program costs were determined using the perspective of the ED. Time-motion methodology was used to estimate personnel activity costs. Costs per patient newly-diagnosed with HIV infection by intervention phase, and incremental cost effectiveness ratios were calculated. Results During the intervention phase, 28,043 eligible patients were included, 6,933 (25%) completed testing, and 15 (0.2%, 95% CI: 0.1%-0.4%) were newly-diagnosed with HIV infection. During the control phase, 29,925 eligible patients were included, 243 (0.8%) completed testing, and 4 (1.7%, 95% CI: 0.4%-4.2%) were newly-diagnosed with HIV infection. Total annualized costs for nontargeted screening were $148,997, whereas total annualized costs for diagnostic HIV testing were $31,355. The average costs per HIV diagnosis were $9,932 and $7,839, respectively. Nontargeted HIV screening identified 11 more HIV infections at an incremental cost of $10,693 per additional infection. Conclusions Compared to diagnostic testing, nontargeted HIV screening was more costly but identified more HIV infections. More effective and less costly testing strategies may be required to improve the identification of patients with undiagnosed HIV infection in the ED.

Background Atrial fibrillation is the most common sustained cardiac arrhythmia in adults. We hypothesized that gain-of-function KCNQ1 mutations previously associated with familial atrial fibrillation have distinct pharmacological properties that may enable targeted inhibition. Methods and Results Wild-type (WT) KCNQ1 or the familial atrial fibrillation mutation KCNQ1-S140G was heterologously coexpressed with KCNE1 to enable electrophysiological recording of the slow delayed rectifier current ( I Ks ) and investigation of pharmacological effects of the I Ks selective blocker HMR-1556. Coexpression of KCNQ1-S140G with KCNE1 generated potassium currents (S140G- I Ks ) that exhibited greater sensitivity to HMR-1556 than WT- I Ks . Enhanced HMR-1556 sensitivity was also observed for another gain-of-function atrial fibrillation mutation, KCNQ1-V141M. Heteromeric expression of KCNE1 with both KCNQ1-WT and KCNQ1-S140G generated currents (HET- I Ks ) with gain-of-function features, including larger amplitude, a constitutively active component, hyperpolarized voltage dependence of activation, and extremely slow deactivation. A low concentration of HMR-1556, which had little effect on WT- I Ks but was capable of inhibiting the mutant channel, reduced both instantaneous and steady state HET- I Ks to levels that were not significantly different from WT- I Ks and attenuated use-dependent accumulation of the current. In cultured adult rabbit left atrial myocytes, expression of S140G- I Ks shortened action potential duration compared with WT- I Ks . Application of HMR-1556 mitigated S140G- I Ks –induced action potential duration shortening and did not alter action potential duration in cells expressing WT- I Ks . Conclusions The enhanced sensitivity of KCNQ1 gain-of-function mutations for HMR-1556 suggests the possibility of selective therapeutic targeting, and, therefore, our data illustrate a potential proof of principle for genotype-specific treatment of this heritable arrhythmia.

Growing financial pressure on US dialysis providers requires economic efficiency considerations. The objective of this study was to examine short-term economic efficiencies of a cinacalcet-based treatment approach for secondary hyperparathyroidism.This study retrospectively assessed cost per biochemical response of the OPTIMA trial. OPTIMA was conducted in end-stage renal disease patients to compare biochemical control in patients receiving cinacalcet in addition to vitamin D sterols and phosphate binders vs patients receiving vitamin D sterol and phosphate binders alone. It explored three laboratory measurement response definitions from baseline to week 23: (1) decreases in parathyroid hormone (PTH) ≥30%; (2) PTH ≤ 300 pg/ml; and (3) PTH ≤ 300 pg/mL, calcium9.5 mg/dL and phosphorus5.5 mg/dL. Medication use and costs were measured to calculate average costs and incremental cost per responder. Stratification by lower and higher baseline PTH assessed cost per response by disease severity.There were 38-77% more responders with cinacalcet vs control, depending on response definition. Mean (SD) per patient total medication costs were $5423 ($3698) for cinacalcet and $2633 ($2334) for control, leading to a mean difference of $2790 over 23 weeks. When response was defined as a decrease in PTH ≥ 30% from baseline, the average cost per responder was $11,266 for control vs $7027 for cinacalcet. The incremental cost per incremental responder ranged from $5186-$9168. Across all response measures, cost per responder was lower in patients with lower baseline PTH.Representing a more efficient allocation of economic resources over the short-term, cinacalcet-based treatment algorithm led to a lower cost per biochemical response, particularly in patients with lower disease severity, vs vitamin D sterols and phosphate binders alone. These findings should be interpreted alongside the study limitation of converting international trial-based medication utilization into US costs.

To estimate the long-term comparative effectiveness of first-line treatment in patients negative for anti-JC virus (JCV) antibodies with glatiramer acetate (GA), fingolimod, or natalizumab for relapsing-remitting multiple sclerosis (RRMS).We developed a simulation model to estimate the average 20-year clinical risks and benefits of GA, fingolimod, and natalizumab for RRMS patients initially negative for anti-JCV antibodies.Model inputs included published natural history progressions of the Expanded Disability Status Scale (EDSS), treatment effects from randomized controlled trials on slowing disease progression and reducing relapse rates, risk of progressive multifocal leukoencephalopathy (PML), and utility preference scores. Outputs were long-term risks (PML risk and other non-PML risks), benefits (average relapse rate and time to disability [EDSS7]), and quality-adjusted life years (QALYs).Compared with GA, natalizumab resulted in 4.6 fewer relapses, 0.6 more years of disability free time, 0.0165 more cases of PML per treated patient, and an incremental 1.2 QALYs gained. Compared with fingolimod, natalizumab resulted in 1.7 fewer relapses, 0.1 more years of disability free time, 0.0165 more cases of PML per treated patient, and an incremental 0.4 QALYs gained. The probability that incremental QALYs favored natalizumab over GA was 0.963 and natalizumab over fingolimod was 0.720.Average QALYs, a measure that aggregates across risks and benefits, favored natalizumab, suggesting more aggressive early intervention with natalizumab in the negative anti-JCV population. For certain decision makers, more evidence may be needed to further reduce the uncertainty in these comparative projections prior to making population-based adoption decisions.

OBJECTIVE:: To provide generalizable estimates of economic burden in epilepsy and nonepilepsy populations and a comprehensive accounting for employment-based lost productivity associated with epilepsy in current US health care systems as compared with other chronic diseases. RESEARCH DESIGN:: We use the nationally representative data source (Medical Expenditure Panel Survey) from 1998 to 2009 to create a retrospective cohort of people diagnosed with epilepsy by a health professional and a comparison cohort of people with no epilepsy. MEASURES:: Health care utilization and direct costs for all components of treatment, including prescription medications, wages, employment, educational attainment, family income, and lost productivity were outcomes. RESULTS:: We observed economic disparities associated with epilepsy in the United States despite high rates of modern treatments (89% on anticonvulsant therapies). Only 42% of the people with epilepsy over age 18 reported employment compared with 70% of people with no epilepsy; among those, people with epilepsy reported missing an average of 12 days of work because of illness or injury as compared with 4 days in the nonepilepsy cohort. Holding other variables constant, people with epilepsy had a loss of productivity of $9504 in 2011 dollars compared with people with no epilepsy. In comparison, diabetes was associated with annual average lost productivity valued at $3358 and depression at $3182. CONCLUSIONS:: Lost wage-based productivity associated with epilepsy was nearly equal to combined wage losses associated with diabetes, depression, anxiety, and asthma together. To evaluate societal burden of illness, results illustrate the importance of indirect costs in addition to treated prevalence and direct medical costs. Language: en

Objective. To provide a current and comprehensive understanding of the cost-effectiveness of DMTs for the treatment of MS by quantitatively evaluating the quality of recent cost-effectiveness studies and exploring how the field has progressed from past recommendations.Methods. We assessed the quality of studies that met our systematic literature search criteria using the Quality of Health Economic Studies validated instrument.Results. Of the 82 studies that met our initial search criteria, we included 22 in this review. Four studies (18%) achieved quality category 2, three studies (14%) achieved quality category 3, and 15 studies (68%) achieved the highest quality category 4. 91% of studies were simulation models. 13 studies (59%) had quality-adjusted life years (QALYs) as the primary outcome measure, included a societal perspective in the analysis, and utilized time horizons of 10 years to lifetime.Conclusions. To continue to improve the cost-effectiveness evidence of DMTs, we recommend: lifetime horizons, societal perspectives, and QALYs; supplemental evidence with shorter horizons, payer perspectives, and clinical outcomes to inform multiple decision makers; development of modeling and input standards for comparability; head-to-head RCTs between DMTs and long-term prospective studies; and comprehensive cost-effectiveness studies that compare all appropriate DMTs.

Unintended consequences may result from changes in pharmacy benefit design. The objective was to determine the impact of increasing patient prescription copayments for guideline recommended, long-term asthma controller (LTC) medications on asthma-related medication use and healthcare services.We used 2005 MarketScan healthcare and pharmacy claims data to identify asthma (International Classification of Diseases, 9th Revision, Clinical Modification [ICD-9-CM] diagnosis code 493.xx) patients aged 12 to 64 years who were continuously enrolled through 2006 with ≥ 1 claim for an asthma medication in 2005. LTCs included: inhaled corticosteroid (ICS) (n = 10,251), ICS plus long-acting beta agonist (COMBO) (n =27,407), and leukotriene receptor antagonist (LTRA) (n = 20,664).Using multivariable models, we estimated the associations between changes in LTC copayments and LTC consumption and asthma-related outpatient and emergency department (ED) visits.Patients were dichotomized into ≥ $5 average increase in patient copayments per month of medication supplied (yes/no). The mean annual change (2005-2006) in copayments per month was $13.23 versus -$3.88 (ICS), $11.76 versus -$3.06 (COMBO), and $9.78 versus -$2.06 (LTRA). The ≥ $5 group experienced a significant decline in average annual days of medication supplied of -47.1 days of ICS (95% CI -43.5 to -50.8), -35.3 days of COMBO (-32.4 to -38.2), and -47.5 days of LTRA (-43.2 to -51.7). Among COMBO and LTRA medication users, the ≥ $5 copayment increase was associated with more asthma-related outpatient visits and ED visits compared with the$5 group.The findings suggest that even small changes in average copayment for asthma medications can result in significant reductions in medication use and unintended increases in healthcare services.

The authors used Medical Expenditure Panel Survey data for 81,097 respondents in 2004-2007, a period of economic expansion, to examine psychological distress among depressed and nondepressed persons in four categories: employed (73%), unemployed (23%), recent job loss (4%), and recent job gain (

Purpose In contrast to the United States, several European countries have health technology assessment programs for drugs, many of which assess cost effectiveness. Coverage decisions that consider cost effectiveness may lead to restrictions in access. Methods For a purposive sample of five decision-making bodies, we analyzed US and United Kingdom coverage decisions on all anticancer drugs approved by the US Food and Drug Administration (FDA) from 2004 to 2008. Data sources for the timing and outcome of licensing and coverage decisions included published and unpublished documentation, Web sites, and personal communication. Results The FDA approved 59 anticancer drugs over the study period, of which 46 were also approved by the European Medicines Agency. In the United States, 100% of drugs were covered, mostly without restriction. However, the United Kingdom bodies made positive coverage decisions for less than half of licensed drugs (National Institute for Health and Clinical Excellence [NICE]: 39%; Scottish Medicines Consortium [SMC]: 43%). Whereas the Centers for Medicare and Medicaid Services (CMS) and the Department of Veterans Affairs (VA) covered all 59 drugs from the FDA license date, delays were evident for some Regence Group decisions that were informed by cost effectiveness (median, 0 days; semi-interquartile range [SIQR], 122 days; n = 22). Relative to the European Medicines Agency license date, median time to coverage was 783 days (SIQR, 170 days) for NICE and 231 days (SIQR, 129 days) for the SMC. Conclusion Anticancer drug coverage decisions that consider cost effectiveness are associated with greater restrictions and slower time to coverage. However, this approach may represent an explicit alternative to rationing achieved through the use of patient copayments.

Background: Omalizumab, an anti-immunoglobulin E antibody, reduces exacerbations and symptoms in uncontrolled allergic asthma. The study objective was to estimate the costs and consequences of omalizumab compared to usual care from a US payer perspective. Methods: We estimated payer costs, quality-adjusted survival (QALYs), and the incremental cost-effectiveness ratio (ICER) of omalizumab compared to usual care using a state-transition simulation model that included sensitivity analyses. Every 2 weeks, patients could transition between chronic asthma and exacerbation health states. The best available evidence informed the clinical and cost input estimates. Five years of omalizumab treatment followed by usual care was assumed to estimate a lifetime horizon. Omalizumab responders (60.5% of treated) were modeled as a separate scenario where nonresponders reverted back to usual care after 16 weeks of active treatment. Results: The mean lifetime discounted costs and QALYs were $83 400 and 13.87 for usual care and $174 500 and 14.19 for omalizumab plus usual care resulting in $287 200/QALY (95% interval: $219 300, $557 900). The ICER was $172 300/ QALY when comparing omalizumab to usual care in the responder scenario. Oneway sensitivity analyses indicated that the results were sensitive to the difference in treatment-specific utilities for the chronic state, exacerbation-associated mortality, omalizumab price, exacerbation rates, and response definition. Conclusions: The results suggest that adding omalizumab to usual care improves QALYs at an increase in direct medical costs. The cost-effectiveness of omalizumab is similar to other chronic disease biologics. The value increases when omalizumab response is used to guide long-term treatment.

Many trials do not measure quality-adjusted life years (QALYs). Therefore, decision analysts often map condition-specific outcomes to preference scores. We estimated the relationship between changes in preference scores and commonly reported condition-specific outcomes in patients with urinary incontinence (UI) due to neurogenic detrusor overactivity.In 59 patients recruited to a neurogenic UI trial, clinical outcomes (UI episodes), condition-specific quality of life (Incontinence Quality of Life Instrument (I-QOL)), and SF-6D preference scores were measured at enrollment and 24 weeks. We used multiple linear regression to estimate the impact on SF-6D scores of 50; 50-99 and 100% reductions in UI episodes and a 10-point improvement in I-QOL.By 24 weeks, mean (95% CI) daily UI episodes fell by 0.85 (0.04, 1.3) and mean I-QOL scores improved by 18 (12, 24). SF-6D scores increased by 0.03 (0.003, 0.058), due to improvements in role limitations. Aor = 50% reduction in UI episodes was achieved by 49% of patients and corresponded to a 0.09 (0.02, 0.16) SF-6D increase. Aor = 10-point increase in I-QOL was attained by 65% of patients and was associated with a 0.05 (-0.02, 0.12) SF-6D increase.These estimates provide preliminary data for decision analysts wishing to map neurogenic UI outcomes to preference scores.

Asthma is the most common chronic disease of childhood and the incidence is increasing. This chapter reviews published health economic studies of asthma interventions and medical technology with a focus on pediatric and adolescent populations. It begins with descriptions of the etiology, epidemiology, and management of asthma, followed by the economic burden of illness. A detailed literature review and a quality appraisal are presented, scrutinizing asthma outcome measures, the analytic framework and handling of uncertainty. The discussion evaluates how well the current health economic recommendations in pediatric asthma have been reflected in clinical guidelines and suggests avenues for future improvement.

Switching between bioequivalent antiepileptic drugs (AEDs) in patients with epilepsy remains a concern because of possible untoward effects owing to a narrow therapeutic range. We investigated the association between switching A-rated AEDs and epilepsy-related events. We conducted a case–control study using claims from the MarketScan database. Cases were defined by an emergently treated epilepsy-related event with the primary diagnosis of epilepsy. Controls had an epilepsy outpatient office visit. Eighty-four of 757 (11.1%) cases and 147 of 2271 (6.5%) controls experienced an A-rated switch. The odds of an epilepsy-related event were 1.78-fold higher for switchers (95% CI 1.35 to 2.36) and, when adjusted for gender and total number of AED prescriptions filled, 1.57-fold higher (95% CI = 1.17–2.10). Switching between A-rated formulations of AEDs is associated with an increased risk of emergently treated epilepsy-related events. These findings suggest that care should be taken when considering a switch between A-rated AEDs in patients with epilepsy.

Article-at-a-Glance Background Prevention of pressure ulcers, one of the hospital-acquired conditions (HACs) targeted by the 2008 nonpayment policy of the Centers for Medicare & Medicaid Services (CMS), is a critical issue. This study was conducted to determine the comparative effectiveness of quality improvement (QI) interventions associated with reduced hospital-acquired pressure ulcer (HAPU) rates. Methods In an quasi-experimental design, interrupted time series analyses were conducted to determine the correlation between HAPU incidence rates and adoption of QI interventions. Among University HealthSystem Consortium hospitals, 55 academic medical centers were surveyed from September 2007 through February 2012 for adoption patterns of QI interventions for pressure ulcer prevention, and hospital-level data for 5,208 pressure ulcer cases were analyzed. Between- and within-hospital reduction significance was tested with t-tests post-CMS policy intervention. Results Fifty-three (96%) of the 55 hospitals used QI interventions for pressure ulcer prevention. The effect size analysis identified five effective interventions that each reduced pressure ulcer rates by greater than 1 case per 1,000 patient discharges per quarter: leadership initiatives, visual tools, pressure ulcer staging, skin care, and patient nutrition. The greatest reductions in rates occurred earlier in the adoption process (p Conclusions Five QI interventions had clinically meaningful associations with reduced stage III and IV HAPU incidence rates in 55 academic medical centers. These QI interventions can be used in support of an evidence-based prevention protocol for pressure ulcers. Hospitals can not only use these findings from this study as part of a QI bundle for preventing HAPUs.

High-resolution computed tomography (HRCT) has been suggested as a potential outcome surrogate for cystic fibrosis (CF) lung disease. An important attribute of a valid outcome surrogate is that the surrogate reflects true clinical outcomes.We performed this study to validate HRCT, a proposed surrogate outcome measure for CF lung disease, against a true clinical outcome, the number of respiratory tract exacerbations occurring in 2 yr, and to assess the correlation of CT scores and pulmonary function tests (PFTs) with this clinical outcome.CTs and PFTs were performed on 6- to 10-yr-old children at the beginning and end of a 2-yr study during which the number of exacerbations were recorded. Spearman correlations and Poisson models were used to assess the correlation of the number of exacerbations with baseline values and changes in PFTs and CT scores.Nine of 61 subjects had a total of 22 respiratory tract exacerbations. At baseline, PFTs and four CT scores showed significant correlation with number of exacerbations, but no variable by itself predicted exacerbations with high accuracy. For change over the 2-yr period, three CT scores showed significant correlation with exacerbations, whereas no PFTs showed significant correlation.This is the first study showing correlation between CT and a true clinical outcome. Change in CT scores correlates moderately well with the number of exacerbation. Poor correlation between change in FEV1 and exacerbations suggests that HRCT may be a more appropriate outcome surrogate for longitudinal studies of young children.

Forced expiratory volume in one second (FEV1), an important measure of pulmonary disease severity in patients with cystic fibrosis (CF), is frequently expressed as a percentage of a predicted value derived from a healthy reference population. There are limitations to comparing the lung function of a patient with CF to that of healthy control subjects, and potential advantages to comparing it to that of other patients with CF.To estimate CF-specific percentiles of FEV1 as functions of height, age, and sex.We used 287,108 FEV1 observations among more than 21,000 patients with CF in the CF Foundation National Patient Registry between 1994 and 2001. The percentiles were estimated using quantile regression methods.FEV1 percentile "growth grids" are presented, allowing comparison of an individual's FEV1 to that of patients with CF of the same sex, age, and height. Their potential uses in clinical practice and research are illustrated.CF-specific reference equations allow individual patients' FEV1 to be placed in the context of the distribution of lung function of their peers with CF, and should improve generalizability of CF clinical trials by setting entry criteria that are equitable across sex and age ranges. They may serve as a useful adjunct to conventional reference equations.

Background Treatment options for overactive bladder (OAB) with urinary urge incontinence (UUI) refractory to oral antimuscarinics include: botulinum toxin type A (BoNTA), sacral neuromodulation (SNM), and augmentation cystoplasty (AC). A standard treatment success metric that can be used in both clinical and economic evaluations of the above interventions has not emerged. Our objective was to conduct a literature review and synthesis of published measures of treatment success for OAB with UUI interventions and to identify a treatment success outcome. Methods We performed a literature review of primary studies that used a definition of treatment success in the OAB with UUI population receiving BoNTA, SNM, or AC. The recommended success outcome was compared to generic and disease-specific health-related quality-of-life (HRQoL) measures using data from a BoNTA treatment study of neurogenic incontinent patients. Results Across all interventions, success outcomes included: complete continence (n = 23, 44%), ≥ 50% improvement in incontinence episodes (n = 16, 31%), and subjective improvement (n = 13, 25%). We recommend the OAB with UUI treatment success outcome of ≥ 50% improvement in incontinence episodes from baseline. Using data from a neurogenic BoNTA treatment study, the average change in the Incontinence Quality of Life questionnaire was 8.8 (95% CI: -4.7, 22.3) higher for those that succeeded (N = 25) versus those that failed (N = 26). The average change in the SF-6D preference score was 0.07 (95% CI: 0.02, 0.12) higher for those that succeeded versus those that failed. Conclusion A treatment success definition that encompasses the many components of underlying OAB with UUI symptoms is currently not practical as a consequence of difficulties in measuring urgency. The treatment success outcome of ≥ 50% improvement in incontinence episodes was associated with a clinically meaningful improvement in disease-specific HRQoL for those with neurogenic OAB with UUI. The recommended success definition is less restrictive than a measure such as complete continence but includes patients who are satisfied with treatment and experience meaningful improvement in symptoms. A standardized measure of treatment success will be useful in clinical and health economic applications.

Background Our objective was to determine the cost-effectiveness of Continuous Glucose Monitoring (CGM) technology with intensive insulin therapy compared to self-monitoring of blood glucose (SMBG) in adults with type 1 diabetes in the United States. Methods A Markov cohort analysis was used to model the long-term disease progression of 12 different diabetes disease states, using a cycle length of 1 year with a 33-year time horizon. The analysis uses a societal perspective to model a population with a 20-year history of diabetes with mean age of 40. Costs are expressed in $US 2007, effectiveness in quality-adjusted life years (QALYs). Parameter estimates and their ranges were derived from the literature. Utility estimates were drawn from the EQ-5D catalogue. Probabilities were derived from the Diabetes Control and Complications Trial (DCCT), the United Kingdom Prospective Diabetes Study (UKPDS), and the Wisconsin Epidemiologic Study of Diabetic Retinopathy. Costs and QALYs were discounted at 3% per year. Univariate and Multivariate probabilistic sensitivity analyses were conducted using 10,000 Monte Carlo simulations. Results Compared to SMBG, use of CGM with intensive insulin treatment resulted in an expected improvement in effectiveness of 0.52 QALYs, and an expected increase in cost of $23,552, resulting in an ICER of approximately $45,033/QALY. For a willingness-to-pay (WTP) of $100,000/QALY, CGM with intensive insulin therapy was cost-effective in 70% of the Monte Carlo simulations. Conclusions CGM with intensive insulin therapy appears to be cost-effective relative to SMBG and other societal health interventions.