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To determine if children eligible for coverage by the State Children's Health Insurance Program (SCHIP) and Medicaid Programs were more likely to receive preventive dental visits after implementation of the SCHIP policy, retrospective cross-sectional analysis was done from the 1996-2000 Medical Expenditure Panel Surveys (MEPS) data. We linked the individual level data from the MEPS to state-level information on program eligibility. Using logistic regression models that adjust for the complex survey design, the association between SCHIP implementation and receipt of preventive dental care was examined for children aged 3-18 with family incomes <=200% of the Federal Poverty Line (FPL). Children who were eligible for SCHIP/Medicaid coverage in their respective states were more likely to have received preventive care three years after SCHIP implementation than children with similar eligibility profiles prior to SCHIP implementation. SCHIP has successfully increased the proportion of eligible children receiving preventive dental care among children in families with incomes less than or equal to 200% FPL. Our findings indicate, however, that SCHIP needed time to mature before detecting significant effects on national level. [ABSTRACT FROM AUTHOR]

BACKGROUND: One in 4 women is affected by intimate partner violence in her lifetime. This article reports on a cross-sectional survey to estimate community preferences for health states resulting from intimate partner violence.METHODS: A secondary analysis was conducted of data from a convenience sample of 93 abused and 138 nonabused women (231 total) recruited for in-person interviews from hospital outpatient department waiting rooms in metropolitan Boston, Massachusetts. SF-12 data were converted to utilities to describe community-perspective preferences for health states associated with intimate partner violence. Linear regression analysis was used to explore the association between violence and utility while controlling for other health and demographic factors.RESULTS: Median utility for intimate partner violence was between 0.58 and 0.63 on a scale of 0 (equivalent to death) to 1.0 (equivalent to optimal health), with a range from 0.64 to 0.66 for less severe violence to 0.53 to 0.62 for more severe violence. The data do not reveal whether violence itself is responsible for lower utility or whether a constellation of factors contributes to disutility experienced by women victims of abuse.DISCUSSION: The utility of health states experienced by women exposed to intimate partner violence is substantially diminished compared with optimal health and even other health conditions. These values quantify the substantial negative health impact of the experience of intimate partner violence in terms that allow comparison across diseases. They can be used in cost-effectiveness analyses to identify the benefits and potential returns from resources allocated to violence prevention and intervention efforts. [ABSTRACT FROM AUTHOR]

OBJECTIVES: To estimate the prevalence and correlates of unmet needs for mental health care services for children with special health care needs and their families. METHODS: We use the National Survey of Children With Special Health Care Needs to estimate the prevalence of unmet mental health care needs among children with special health care needs (1-17 years old) and their families. Using logistic-regression models, we also assess the independent impact of child and family factors on unmet needs. RESULTS: Substantial numbers of children with special health care needs and members of their families have unmet needs for mental health care services. Children with special health care needs who were poor, uninsured, and were without a usual source of care were statistically significantly more likely to report that their mental health care needs were unmet. More severely affected children and those with emotional, developmental, or behavioral conditions were also statistically significantly more likely to report that their mental health care needs went unmet. Families of severely affected children or of children with emotional, developmental, or behavioral conditions were also statistically significantly more likely to report that their mental health care needs went unmet. CONCLUSIONS: Our results indicate that children with special health care needs and their families are at risk for not receiving needed mental health care services. Furthermore, we find that children in families of lower socioeconomic status are disproportionately reporting higher rates of unmet needs. These data suggest that broader policies to identify and connect families with needed services are warranted but that child- and family-centered approaches alone will not meet the needs of these children and their families. Other interventions such as anti-poverty and insurance expansion efforts may be needed as well. [ABSTRACT FROM AUTHOR]

OBJECTIVES: This study assessed the relationship between external risks, such as personal and neighborhood danger, and smoking by using a new theoretical framework based on competing mortality risk models. METHODS: Regression analyses of self-reported data from residents of Central Harlem, New York, surveyed from 1992 through 1994 (n = 695, response rate = 72%) were used to assess the relationship between smoking and 2 measures of external health threats: levels of neighborhood danger and lifetime trauma. RESULTS: Support for the framework was mixed. At the 95% confidence level, exposure to lifetime trauma was positively related to current smoking status but was not related to the number of cigarettes smoked, conditional on being a smoker. Living in a 'somewhat unsafe neighborhood' was also statistically significantly related to current smoking status. CONCLUSIONS: Although the framework implies that policies directed at improving the physical and social environment might improve health through their indirect effects on behaviors, little supporting evidence was found. Smoking rates may decrease if exposure to violence and neighborhood danger is reduced. This framework needs to be tested on larger and more information-rich data sets. [ABSTRACT FROM AUTHOR]

OBJECTIVES: This study compared dietary risk factors among Southern-born and other Blacks in Central Harlem. METHODS: A survey of residents of Central Harlem was used to compute a 'healthy diet' score for 621 subjects. RESULTS: Southern-born respondents had the highest-risk diets. Although their numbers were small, Caribbean-born respondents, particularly those younger than 45 years, had the lowest-risk diets. CONCLUSIONS: The variation in diets in Central Harlem was considerable, with Southern-born Blacks at highest dietary risk for chronic diseases. These results remain to be tested elsewhere, as does the contribution of other chronic disease risk factors. [ABSTRACT FROM AUTHOR]

Background: Spinal muscular atrophy (SMA) is a rare, progressive, neuromuscular disorder. Recent advances in treatment require an updated assessment of burden to inform reimbursement decisions., Objectives: To quantify healthcare resource utilisation (HCRU) and cost of care for patients with SMA., Methods: Cohort study of patients with SMA identified in the Swedish National Patient Registry (2007-2018), matched to a reference cohort grouped into four SMA types (1, 2, 3, unspecified adult onset [UAO]). HCRU included inpatient admissions, outpatient visits, procedures, and dispensed medications. Direct medical costs were estimated by multiplying HCRU by respective unit costs. Average annual HCRU and medical costs were modelled for SMA versus reference cohorts to estimate differences attributable to the disease (i.e., average treatment effect estimand). The trajectory of direct costs over time were assessed using synthetic cohorts., Results: We identified 290 SMA patients. Annualised HCRU was higher in SMA patients compared with reference cohorts. Highest risk ratios were observed for inpatient overnight stays for type 1 (risk ratio [RR]: 29.2; 95% confidence interval [CI]: 16.0, 53.5) and type 2 (RR: 23.3; 95% CI: 16.4,33.1). Mean annual direct medical costs per patient for each year since first diagnosis were greatest for type 1 (€114,185 and SMA-attributable: €113,380), type 2 (€61,876 and SMA-attributable: €61,237), type 3 (€45,518 and SMA-attributable: €44,556), and UAO (€4046 and SMA-attributable: €2098). Costs were greatest in the 2-3 years after the first diagnosis for all types., Discussion and Conclusion: The economic burden attributable to SMA is significant. Further research is needed to understand the burden in other European countries and the impact of new treatments., (© 2024. The Author(s).)

Background: Electronic health records (EHRs) offer the possibility of using data entry templates to simultaneously document routine clinical care and capture disease-specific measures as discrete data elements that can be used for health services research (HSR). The objective of this study was to determine factors associated with meaningful treatment escalation (MTE) of psoriasis as a pilot study for future real-world HSR studies., Methods: We conducted a retrospective, observational cohort study of psoriasis patients by using data collected during routine clinical care from an EHR using EpiCare® SmartForms. The psoriasis SmartForm records psoriasis disease severity measures and descriptive findings to generate visit notes. These data were extracted and analyzed to identify factors associated with MTE, defined as changing or adding, phototherapy, systemic, or biologic therapy., Results: 473 psoriasis patients met study criteria; 239 underwent MTE between their first and third observed visits. Patients who experienced MTE had more severe disease at Visit 1-assessed by BSA, pPGA, oPGA, and a patient-reported disease severity measure--than patients who did not experience MTE. Other factors associated with MTE included use of topicals only or no active treatment at Visit 1, palmoplantar disease, and involvement of other difficult-to-treat body areas. Patients who underwent MTE experienced larger improvements in disease severity than those who did not., Conclusions: This study highlights how data collected during routine clinical practice can be readily used for real-world retrospective HSR when disease measures are captured as discrete elements. This approach could provide a cost-effective platform to conduct real-world HSR., Competing Interests: The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: WM and RB are employees and minor shareholders of Eli Lily and Company. JLWR has received consulting fees from Eli Lilly and Company, Boehringer Ingelheim, Genentech, and Argenx. KCD has received grants and personal fees from Eli Lilly and Company, Amgen, Janssen, Pfizer, UCB, Novartis, Celgene, AbbVie and Sienna; and has received personal fees from Ortho Dermatologica. MLG is an employee of Evidera Inc., an independent research company that received payment from Eli Lilly and Company for the conduct of this study and the development of this manuscript. MLG is a minor shareholder of Thermo Fisher Scientific., (© The Author(s) 2023.)

Purpose: Weight gain and associated negative cardiometabolic effects can occur as a result of mental illness or treatment with second-generation antipsychotics (SGAs), leading to increased rates of morbidity and mortality. In this analysis, we evaluated the effect of the SGA cariprazine on weight and metabolic parameters in a real-world, retrospective, observational dataset., Methods: Electronic health records from the Optum Humedica database (October 1, 2014-December 31, 2020) were analyzed during the 12-month period before starting cariprazine (baseline) and for up to 12 months following cariprazine initiation; approved and off-label indications were included. Body weight trajectories were estimated in the overall patient cohort and at 3-, 6-, and 12-month timepoints (primary objective). Changes in hemoglobin A1c (HbA1c), low-density lipoprotein (LDL), high-density lipoprotein (HDL), and triglycerides were also evaluated (secondary objectives). Percentages of patients with clinically relevant shifts in body weight, total cholesterol, and fasting triglycerides were also determined. Discontinuation rates for metabolic regulating medications were calculated. Average predicted values were estimated by linear mixed-effects regression models., Findings: A total of 2,301 patients were included; average duration of follow-up was 133.7 days. Average predicted weight change for patients during the cariprazine overall follow-up period was +2.4 kg, with predicted weight changes of +0.8 kg (n = 811), +1.1 kg (n = 350), and +1.4 kg (n = 107) at months 3, 6, and 12, respectively. Overall, the majority of patients did not experience clinically significant (≥7%) weight gain (82.8%) or loss (90.5%) after starting cariprazine. Average predicted HbA1c levels (n = 189) increased during baseline (0.15%/year) and decreased during cariprazine treatment (-0.2%/year). Average predicted triglyceride levels (n = 257) increased during baseline (15.0 mg/dL/year) and decreased during cariprazine treatment (-0.7 mg/dL/year). Predicted LDL (n = 247) and HDL (n = 255) values decreased during baseline (-7.3 and -1.1 mg/dL/year, respectively); during cariprazine treatment, LDL increased by 5.6 mg/dL/year and HDL decreased by -0.6 mg/dL/year. During follow-up, most patients did not shift from normal/borderline to high total cholesterol (<240 to ≥240 mg/dL; 522 [90.2%]) or fasting triglyceride (<200 to ≥200 mg/dL; 143 [88.8%] patients) levels; shifts from high to normal/borderline levels occurred in 44 (61.1%) patients for total cholesterol and 38 (57.6%) patients for fasting triglycerides. After starting cariprazine, the discontinuation rate per 100 patient-years was 60.4 for antihyperglycemic medication and 87.4 for hyperlipidemia medication., Implications: These real-world results support short-term clinical trial findings describing a neutral weight and metabolic profile associated with cariprazine treatment and they expand the dataset to include long-term follow-up., Competing Interests: Declaration of Competing Interest P. Masand has received Speaker/Consultant fees from Acadia, AbbVie, Intra-Cellular Therapies, Neumora Therapeutics, and Sunovion. R.S. McIntyre has received research grant support from CIHR/GACD/National Natural Science Foundation of China (NSFC) and the Milken Institute; speaker/consultation fees from Lundbeck, Janssen, Alkermes, Neumora Therapeutics, Boehringer Ingelheim, Sage, Biogen, Mitsubishi Tanabe, Purdue, Pfizer, Otsuka, Takeda, Neurocrine, Sunovion, Bausch Health, Axsome, Novo Nordisk, Kris, Sanofi, Eisai, Intra-Cellular, NewBridge Pharmaceuticals, Viatris, AbbVie, Atai Life Sciences. Dr. Roger McIntyre is a CEO of Braxia Scientific Corp. A.J. Cutler has been a consultant for AbbVie, Acadia Pharmaceuticals, Akili Interactive, Alfasigma, Alkermes, Avanir, Axsome, Biogen, BioXcel Therapeutics, Boehringer Ingelheim, Cerevel, Corium, Intra-Cellular Therapies, Ironshore, Janssen, Karuna Therapeutics, Lundbeck, Neumor, Neurocrine Biosciences, Noven, Otsuka, Relmada Therapeutics, Sage Therapeutics, Sunovion, Supernus Pharmaceuticals, Takeda, Teva, and Tris Pharma; has received speaker/promotional honoraria from AbbVie, Acadia Pharmaceuticals, Alfasigma, Alkermes, Avanir, Axsome, BioXcel Therapeutics, Corium, Idorsia, Intra-Cellular Therapies, Ironshore, Janssen, Lundbeck, Neurocrine Biosciences, Noven, Otsuka, Sunovion, Supernus, Takeda, Teva and Tris Pharma; and has received research grants from Akili Interactive, Alkermes, Allergan (now AbbVie), Arbor Pharmaceuticals, Biohaven, Ironshore, KemPharm, Lilly, Lundbeck, Novartis, Otsuka, Purdue Canada, Sage Therapeutics, Sunovion, Supernus Pharmaceuticals, Takeda and Tris Pharma. He is a member of DSMB (Data Safety Monitoring Board) for COMPASS Pathways, and a member of the Board for the Neuroscience Education Institute. A. Harrington and K. Kramer were employees of AbbVie at the time of the study and may hold stock. H.-B. Nguyen and K. Patel are employees of AbbVie and may hold stock. M.L. Ganz and A.L. Lorden are employees of Evidera and may hold stock in Thermo Fisher Scientific, Evidera's parent company., (Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.)

Background: No algorithms exist to identify important osteoarthritis (OA) patient subgroups (i.e., moderate-to-severe disease, inadequate response to pain treatments) in electronic healthcare data, possibly due to the complexity in defining these characteristics as well as the lack of relevant measures in these data sources. We developed and validated algorithms intended for use with claims and/or electronic medical records (EMR) to identify these patient subgroups., Methods: We obtained claims, EMR, and chart data from two integrated delivery networks. Chart data were used to identify the presence or absence of the three relevant OA-related characteristics (OA of the hip and/or knee, moderate-to-severe disease, inadequate/intolerable response to at least two pain-related medications); the resulting classification served as the benchmark for algorithm validation. We developed two sets of case-identification algorithms: one based on a literature review and clinical input (predefined algorithms), and another using machine learning (ML) methods (logistic regression, classification and regression tree, random forest). Patient classifications based on these algorithms were compared and validated against the chart data., Results: We sampled and analyzed 571 adult patients, of whom 519 had OA of hip and/or knee, 489 had moderate-to-severe OA, and 431 had inadequate response to at least two pain medications. Individual predefined algorithms had high positive predictive values (all PPVs ≥ 0.83) for identifying each of these OA characteristics, but low negative predictive values (all NPVs between 0.16-0.54) and sometimes low sensitivity; their sensitivity and specificity for identifying patients with all three characteristics was 0.95 and 0.26, respectively (NPV 0.65, PPV 0.78, accuracy 0.77). ML-derived algorithms performed better in identifying this patient subgroup (range: sensitivity 0.77-0.86, specificity 0.66-0.75, PPV 0.88-0.92, NPV 0.47-0.62, accuracy 0.75-0.83)., Conclusions: Predefined algorithms adequately identified OA characteristics of interest, but more sophisticated ML-based methods better differentiated between levels of disease severity and identified patients with inadequate response to analgesics. The ML methods performed well, yielding high PPV, NPV, sensitivity, specificity, and accuracy using either claims or EMR data. Use of these algorithms may expand the ability of real-world data to address questions of interest in this underserved patient population., (© 2023. The Author(s).)

Introduction: Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disease. Patients with ASMD type B experience multiple morbidities, potentially leading to early mortality. Before the 2022 approval of olipudase alfa for non-neuronopathic ASMD manifestations, only symptom management was offered. Data on healthcare services used by patients with ASMD type B are limited. This analysis used medical claims data to evaluate real-world healthcare service use by patients with ASMD type B in the United States of America (USA)., Methods: The IQVIA Open Claims patient-level database (2010-2019) was cross-examined. Two patient cohorts were identified: the primary analysis cohort, which included patients with at least two claims associated with ASMD type B (ICD-10 code E75.241) and more total claims with ASMD type B than any other ASMD types, and the sensitivity analysis cohort, which included patients with a high probability of having ASMD type B identified using a validated machine-learning algorithm. Claims for ASMD-associated healthcare services were recorded, including outpatient visits, emergency department (ED) visits, and inpatient hospitalizations., Results: The primary analysis cohort included 47 patients; a further 59 patients made up the sensitivity analysis cohort. Patient characteristics and healthcare service use were similar in both cohorts and were consistent with established characteristics of ASMD type B. Overall, 70% of the primary analysis cohort from this study were aged < 18 years, and the liver, spleen, and lungs were the most frequently affected organs. Cognitive, developmental, and/or emotional problems and respiratory/lung disorders caused most outpatient visits; respiratory/lung disorders accounted for most ED visits and hospitalizations., Conclusion: This retrospective analysis of medical claims data identified patients with ASMD type B who had characteristics typical of this condition. A machine-learning algorithm detected further cases with a high probability of having ASMD type B. High use of ASMD-related healthcare services and medications was observed in both cohorts., (© 2023. The Author(s).)

Limited data exist on cytomegalovirus (CMV) antiviral treatment patterns among kidney transplant recipients (KTRs). Using United States Renal Database System registry data and Medicare claims (1 January 2011-31 December 2017), we examined CMV antiviral use in 22,878 KTRs who received their first KT from 2011 to 2016. Three-quarters of KTRs started CMV prophylaxis (85.8% of high-, 82.4% of intermediate-, and 32.1% of low-risk KTRs). Median time to prophylaxis discontinuation was 98, 65, and 61 days for high-, intermediate-, and low-risk KTRs, respectively. Factors associated with receiving CMV prophylaxis were high-risk status, diabetes, receipt of a well-functioning kidney graft, greater time on dialysis before KT, panel reactive antibodies ≥80%, and use of antithymocyte globulin, alemtuzumab, and tacrolimus. KTRs were more likely to discontinue CMV prophylaxis if they developed leukopenia/neutropenia, had cardiovascular disease, or received their kidney from a deceased donor. These findings suggest that adherence to the recommended duration of CMV-prophylaxis for high and intermediate-risk patients is suboptimal, and CMV prophylaxis is overused in low-risk patients., Competing Interests: MG, KF, and AL are employed by Evidera. SS was employed by Evidera and AR and YT were employed by MSD while this study was conducted. MG is a minor shareholder of Thermo Fisher Scientific stock. CS received consulting fees from MSD for his work on this study. This work was funded by Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, United States. The funder had the following involvement with the study: study design, data acquisition, interpretation of results, critical review of manuscript, and the decision to publish., (Copyright © 2022 Raval, Ganz, Fraeman, Lorden, Saravanan, Tang and Santos.)

Objectives: Recent trials of glucose-lowering drugs (GLDs) have drawn attention to renal outcomes. Our goal was to understand how patients with diabetic kidney disease (DKD) are treated in general practices in the United States., Study Design: Retrospective cohort study using a national-level claims data set and electronic health records., Methods: Patients (≥ 18 years) with type 2 diabetes, whose estimated glomerular filtration rates (eGFRs) were between 15 and 89 mL/min/1.73 m2 between 2016 and 2018, were selected. Use of different GLDs during a 12-month period was examined across all eGFR levels., Results: Of the 25,486 sample patients, 69.2%, 18.9%, 9.6%, and 2.3% had an eGFR in the ranges of 60 to 89, 45 to 59, 30 to 44, and 15 to 29 mL/min/1.73 m2, respectively. Metformin was used by nearly 33% of patients with an eGFR of 30 to 44 mL/min/1.73 m2 and by 10% of patients with an eGFR less than 30 mL/min/1.73 m2. Less than 10% (across all eGFR levels) of patients used glucagon-like peptide-1 receptor agonists and sodium-glucose cotransporter-2 inhibitors. Use of insulin was more frequent among patients with a lower eGFR (P < .05). The findings were similar in subgroups with different hemoglobin A1c levels (< 7% and ≥ 7%)., Conclusions: Real-world treatment of DKD in the United States is suboptimal. Inappropriate use of some GLD classes, especially in advanced DKD stages, was found along with lower than expected use of modern agents that are considered safe and effective to treat glycemic outcomes. Efforts may be needed to improve understanding of safety, glycemic efficacy, and overall clinical value of GLDs across DKD stages.

Background: Patients with poor asthma control may receive oral corticosteroid (OCS) therapy despite the risk for adverse effects., Objective: We assessed OCS use frequency and treatment patterns in patients with persistent asthma in the United States (US)., Methods: We used the IBM MarketScan Commercial Claims and Encounters, Medicare Supplemental, and Medicaid Multistate Claims research databases to identify patients from January 1, 2012, to December 31, 2017, who were ≥12 years old, met the 2-year Healthcare Effectiveness Data and Information Set criteria for persistent asthma, and were continuously enrolled ≥6 months before (baseline) and ≥24 months after (follow-up) the persistent asthma index date. Patients were classified as high OCS use (defined as ≥450 mg of OCS prescribed within a 90-day period during follow-up), low use (use OCS but not meeting high use criteria), or no OCS use., Results: We identified 435,675 patients, of whom 65% used OCS and 19% were classified as high OCS users at some point during follow-up. The annual prevalence of high OCS use ranged from 5.3% in 2013 to 7.6% in 2017. During the entire follow-up, high and low OCS users filled an average of 2.2 and 0.8 OCS prescriptions and received an average daily dosage of 2.2 and 0.3 mg, respectively. Once the patients became high OCS users, the average daily OCS dosage was relatively stable (5.1-7.1 mg) over 3 years., Conclusions: Patients with persistent asthma in the US have substantial exposure to OCS. OCS therapy should be considered carefully to avoid associated adverse effects., (Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.)

Aims: To evaluate clinical and economic outcomes associated with valbenazine compared with deutetrabenazine in patients with tardive dyskinesia (TD) using a model that accounts for multiple dimensions of patient health status., Materials and Methods: A discretely integrated condition event model was developed to evaluate the cost-effectiveness of treatment with valbenazine and deutetrabenazine in a synthetic cohort of 1,000 patients with TD who were receiving antipsychotic medication to treat an underlying psychiatric disorder. Clinical inputs were derived from relevant clinical trials or from publicly available sources. Patients were assessed over 1 year using ≥50% improvement from baseline in Abnormal Involuntary Movement Scale (AIMS) total score as the primary definition of response. Response at 1 year using Clinical Global Impression of Change (CGIC) score ≤2 was also assessed. Health outcomes included quality-adjusted life years (QALYs), life years, proportion responding to treatment at 1 year, and number of psychiatric relapses., Results: Regardless of the definition used for response, patients treated with valbenazine were more likely to have responded to treatment at 1 year, lived longer, and accrued more QALYs than patients who received deutetrabenazine. Using the AIMS response criterion, the incremental cost-effectiveness ratio was $9,951/QALY for valbenazine compared with deutetrabenazine. By comparison, using the CGIC response criterion valbenazine dominated deutetrabenazine with valbenazine-treated patients accumulating more QALYs (3.4 vs 3.3 years) and incurring lower lifetime costs ($252,311 vs $283,208) than deutetrabenazine-treated patients., Limitations: There are no head-to-head trials of valbenazine and deutetrabenazine, so probabilities of response used in the model were calculated based on an indirect treatment comparison of results from individual trials with one drug or the other, using only those metrics reported across trials., Conclusions: In patients with TD, treatment with valbenazine is highly cost-effective compared with deutetrabenazine.

BACKGROUND: Approximately 5%-10% of patients with diabetes are diagnosed with type 1 diabetes mellitus (T1DM), the incidence and prevalence of which is projected to increase through 2050. Despite this, T1DM-related health care resource utilization (HCRU) and economic burden in the United States have not been adequately assessed, since previous studies used various cost definitions and underlying methods to examine these outcomes. OBJECTIVE: To assess HCRU and costs incurred by patients with T1DM in the United States. METHODS: This retrospective cohort study used IBM Watson MarketScan data from 2011 to 2015 and Optum's electronic medical record (EMR) and integrated data (i.e., linked EMR and administrative claims data) from 2011 to 2016. Included patients had ≥ 1 T1DM diagnosis (the earliest diagnosis date was designated as the index date), were continuously enrolled for ≥ 6 months during their pre-index baseline periods, and had ≥ 1 pharmacy claim for insulin or an insulin pump within ± 90 days of the index date. Baseline demographic and clinical characteristics were summarized descriptively. Average monthly HCRU and costs per patient per month (PPPM) paid by the health plan and patient were assessed. Costs were adjusted for inflation to 2018 U.S. dollars. RESULTS: We identified 181,423 patients with T1DM who met the selection criteria in MarketScan, 84,759 in the Optum EMR, and 8,948 in the Optum integrated databases. Most patients were male (range across databases: 52.6%-53.1%), relatively young (medians: 33-35 years, overall range: 0-100 years), and had a Charlson Comorbidity Index score of 1 (69.2%-73.0%) across all databases. Total all-cause and diabetes-related costs ranged from $1,482 to $1,522 and $733 to $780 PPPM, respectively, during the follow-up period. Pharmacy costs contributed most to the total cost of care, accounting for 55.3% ($431) to 61.1% ($448) of total diabetes-related costs. On an annualized basis, patients had an average of 0.2-0.9 all-cause hospitalizations and 0.1-0.3 diabetes-related hospitalizations during follow-up. The median costs per diabetes-related hospitalization ranged from $6,548 to $8,439, accounting for 4%-7% of total monthly diabetes-related costs. Patients had an average of 0.4-0.5 all-cause and 0.1-0.2 diabetes-related emergency department (ED) visits annually; the median costs of ED visits were $972-$1,499, contributing about 2% of monthly diabetes-related costs during follow-up. CONCLUSIONS: In this large, retrospective, observational study of pediatric and adult patients with T1DM, diabetes-related costs totaled nearly $800 per month. Pharmacy costs contributed to over half of diabetes-related costs, indicating the substantial economic burden associated with the treatment of T1DM. Additional research is needed to determine risk factors associated with costly events (e.g., hospitalizations and ED visits) and indirect costs associated with T1DM. DISCLOSURES: JDRF International provided funding for this project and manuscript. JDRF International also contracted with Evidera, a research and consulting firm for the biopharma industry, for its participation in the project and in the development of this manuscript. The Leona M. and Harry B. Helmsley Charitable Trust provided JDRF International with funding. Simeone, Shah, and Ganz are employed by Evidera and do not receive any payment or honoraria directly from Evidera's clients. LeGrand is an employee of JDRF International. Bushman was employed by JDRF International during the conduct of the study and development of this manuscript. Sullivan and Koralova are employees of The Leona M. and Harry B. Helmsley Charitable Trust.

Importance: Atypical antipsychotics (AAPs) are often used off-label to manage dementia-associated neuropsychiatric symptoms. In 2005, the US Food and Drug Administration (FDA) issued a boxed warning for the use of AAPs in elderly patients. The long-term association of this warning with health outcomes is unknown to date., Objective: To assess the long-term association of the 2005 FDA boxed warning on AAPs with psychiatric medication and opioid use, health events, and quality of life among elderly individuals with dementia., Design, Setting, and Participants: For this cross-sectional study, data were analyzed from the household component of the Medical Expenditure Panel Survey (MEPS), the National Ambulatory Medical Care Survey (NAMCS), and the National Hospital Ambulatory Medical Care Survey (NHAMCS) fielded between January 1, 1996, and December 31, 2014. This interrupted time-series analysis applied to 3-year moving means derived from the 1996-2014 MEPS, NAMCS, and NHAMCS. All survey respondents included in this analysis were 65 years or older and had dementia. Data analysis was performed from December 1, 2017, to March 15, 2018., Exposures: The 2005 FDA boxed warning on AAPs., Main Outcomes and Measures: Use of psychiatric medications and opioids, prevalence of cerebrovascular and cardiovascular events, prevalence of falls and/or fractures, 2-year mortality, and health-related quality of life assessed by the Medical Outcomes Study 12-Item Short-Form Health Survey scores., Results: A total of 2430 (MEPS) and 5490 (NAMCS and NHAMCS) respondents were identified, corresponding to weighted populations of 22 996 526 (MEPS) and 65 502 344 (NAMCS and NHAMCS) noninstitutionalized elderly individuals with dementia (mean [SD] age, 81.06 [1.13] years; 63.1% female). In the MEPS sample, compared with before 2005, AAP use (from an annual slope of 0.99 to -0.18 percentage points), cerebrovascular events (0.75 to -0.50 percentage points), and falls and/or fractures (-1.72 to -0.40 percentage points) decreased and opioid use (0.04 to 1.29 percentage points), antiepileptic use (-0.42 to 1.21 percentage points), cardiovascular events (-0.13 to 1.30 percentage points), and 2-year mortality risk (-0.68 to 0.18 percentage points) increased. Health-related quality of life remained relatively unchanged. The NAMCS and NHAMCS sample yielded similar findings., Conclusions and Relevance: These data suggest that the 2005 FDA boxed warning was associated with some unintended negative patient outcomes.

Objectives: In Canada, incidences of herpes zoster (HZ) and postherpetic neuralgia (PHN) are increasing, posing a significant burden on the healthcare system. This study aimed to determine the public health impact and cost effectiveness of an adjuvanted recombinant zoster vaccine (RZV) compared to no vaccination and to the live attenuated vaccine (ZVL) in Canadians aged 60 years and older., Methods: A multi-cohort Markov model has been adapted to the Canadian context using recent demographic and epidemiologic data. Simulations consisted of age-cohorts annually transitioning between health states. Health outcomes and costs were discounted at 1.5% per year. The perspective of the Canadian healthcare payer was adopted. A coverage of 80% for the first RZV and ZVL dose and a compliance of 75% for the second RZV dose were assumed., Results: RZV was estimated to be cost effective compared with no vaccination with an incremental cost-effectiveness ratio (ICER) of $28,360 (Canadian dollars) per quality-adjusted life-year (QALY) in persons aged ≥ 60 years, avoiding 554,504 HZ and 166,196 PHN cases. Compared with ZVL, RZV accrued more QALYs through the remaining lifetime and an increase in costs of approximately $50 million resulting in an average ICER of $2396. Results were robust under deterministic and probabilistic sensitivity analyses. HZ incidence rate and persistence of vaccine efficacy had the largest impact on cost effectiveness., Conclusions: The cost-utility analysis suggested that RZV would be cost effective in the Canadian population compared with no vaccination and vaccination with ZVL at a willingness-to-pay threshold of $50,000.

Objectives : We estimated the total US hospital costs associated with acute bacterial skin and skin structure infection (ABSSSI) admissions as well as the admissions that may have been potential candidates for outpatient parenteral antimicrobial therapy (OPAT). Methods : We assessed inpatient admissions for ABSSSI from the Premier database (2011-2014), focusing on all admissions of adults with length of stay (LOS) ≥ 1 days and a primary diagnosis of erysipelas, cellulitis/abscess, or wound infection. We performed a detailed analysis of 2014 admissions for patient, treatment, hospital, and economic characteristic variables. Using published selection criteria, we identified a subset of patients admitted in 2014 who may have been potential candidates for OPAT. Results : We analyzed 277,971 admissions. In 2014, most admissions were for cellulitis without major complications or comorbidities; mean ± SD LOS was 4.0 ± 3.0 days, and total hospital cost per admission was $6400 ± $6874, 54% of which was attributable to room costs. Among 2014 admissions, 14% involved patients with clinical characteristics suggesting that they were consistent with guideline recommendations for exclusive treatment with OPAT. Compared with all admissions in the year, these admissions were of younger patients (aged 50 vs. 55 years), admitted more frequently for cellulitis (90% vs. 70%), with shorter LOS (2.8 ± 1.8 days), and lower mean total hospital cost per admission ($4080 ± $3066). Conclusions : Admissions for ABSSSI impose a substantial cost to US hospitals, with half of costs attributable to room costs. When extrapolated to all US patients admitted to the hospital for ABSSSI during 2014, had OPAT guidelines been universally followed, admissions may have been reduced by 14%, thereby saving US hospitals $161 million.

Introduction: The use of antihyperglycemic agents (AHA), especially insulin and sulfonylureas (SU), is a risk factor for hypoglycemia. Despite the significant clinical and economic burdens associated with hypoglycemia and the decreasing use of SU in favor of other oral AHA, relatively little is known about hypoglycemia trends specific to the use of non-insulin AHA. We sought to estimate annual hypoglycemia event rates and costs among patients with type 2 diabetes mellitus (T2DM) who started either SU or dipeptidyl peptidase-4 inhibitors (DPP-4i) and to predict rates and costs in the absence of DPP-4i., Methods: Truven's MarketScan Commercial Claims database was used to estimate hypoglycemia event rates and costs from 2007 to 2013. Hypoglycemia, defined using diagnosis codes, was assessed during the 12 months following SU (n = 245,201) or DPP-4i (n = 176,786) initiation by adults with T2DM. Coefficients from a Poisson regression model used to estimate the impact of patient characteristics on hypoglycemia rates for patients who started SU were used to predict rates for patients who started DPP-4i had they started SU instead., Results: Hypoglycemia events per 100 patient-years (costs per event) ranged from 5.4 ($565) in 2007 to 10.4 ($1154) in 2013 for patients starting SU; rates (costs) for patients starting DPP-4i ranged from 3.2 ($308) in 2007 to 6.4 ($482) in 2013. Predicted hypoglycemia rates would have been 5.3-9.9 per 100 person-years for patients who started DPP-4i had they started SU instead. Starting DPP-4i, rather than SU, would have resulted in national savings of $750.3 million in healthcare costs due to avoided hypoglycemia events during this period., Conclusions: Hypoglycemia rates and costs were consistently higher for patients who started SU rather than DPP-4i. The overall burden of hypoglycemia could be lowered substantially in the USA if, when feasible, patients with T2DM initiate DPP-4i instead of SU., Funding: Merck & Co., Inc., Kenilworth, NJ USA.

Introduction: This study compared the clinical and economic outcomes of long-term use of liraglutide versus sitagliptin for the treatment of type 2 diabetes (T2DM) in real-world practice in the USA., Methods: We identified adult patients (≥ 18 years old) with T2DM who initiated liraglutide or sitagliptin in 2010-2014 using a large claims database. Quarterly glycemic control measures and annual healthcare costs were assessed during the 1st and 2nd years of persistent medication use. Their associations with medication use (liraglutide or sitagliptin) were estimated using multivariable regression models adjusted for patient demographic and clinical characteristics., Results: A total of 3113 patients persistently used liraglutide (N = 493) or sitagliptin (N = 2620) for ≥ 1 year [mean age (standard deviation, SD): 53 (8.5) vs. 56 (9.7) years; 48.3% vs. 62.3% males; both p < 0.05]; 911 (including 113 liraglutide users) were persistent users for ≥ 2 years. During the 1st-year follow-up, liraglutide users (versus sitagliptin users, after adjustment) experienced larger glycated hemoglobin (HbA1c) reductions from baseline (ranging from 0.34%-point in quarter 1 to 0.21%-point in quarter 4); higher likelihoods of obtaining HbA1c reductions of ≥ 1%-points or ≥ 2%-points [odds ratios (ORs) range 1.47-2.04]; and higher likelihoods of reaching HbA1c goals of < 6.5% or < 7% (ORs range 1.51-2.12) (all p < 0.05). Liraglutide users also experienced HbA1c reductions from baseline in the 2nd-year follow-up (0.53-0.33%-point, all p < 0.05). Although liraglutide users incurred higher healthcare costs than sitagliptin users during the 1st-year follow-up, they had $2674 (per patient) lower all-cause medical costs (adjusted cost ratio: 0.67, p < 0.05) and similar total costs (all-cause and diabetes-related) in the 2nd year., Conclusion: Long-term use of liraglutide for 1 or 2 years was associated with better glycemic control than using sitagliptin. Savings in medical costs were realized for liraglutide users during the 2nd year of persistent treatment, which offset differences in pharmacy costs., Funding: Novo Nordisk Inc.

Aims: Approximately 1.25 million people in the US have type 1 diabetes mellitus (T1DM), a chronic metabolic disease that develops from the body's inability to produce insulin, and requires life-long insulin therapy. Poor insulin adherence may cause severe hypoglycemia (SHO), leading to hospitalization and long-term complications; these, in turn, drive up costs of SHO and T1DM overall. This study's objective was to estimate the prevalence and costs of SHO-related hospitalizations and their additional longer-term impacts on patients with T1DM using basal-bolus insulin., Methods: Using Truven MarketScan claims, we identified adult T1DM patients using basal-bolus insulin regimens who were hospitalized for SHO (inpatient SHO patients) during 2010-2015. Two comparison groups were defined: those with outpatient SHO-related encounters only, including emergency department (ED) visits without hospitalization (outpatient SHO patients), and those with no SHO- or acute hyperglycemia-related events (comparison patients). Lengths of stay and SHO-related hospitalization costs were estimated and propensity score and inverse probability weighting methods were used to adjust for baseline differences across the groups to evaluate longer-term impacts., Results: We identified 8,734 patients, of which 4.2% experienced at least one SHO-related hospitalization. Among those who experienced SHO (i.e. of those in the inpatient and outpatient SHO groups), 31% experienced at least one SHO-related hospitalization, while 9% were treated in the ED without subsequent hospitalization. Approximately 79% of patients were admitted directly to the hospital; the remainder were first assessed or treated in the ED. The inpatient SHO patients stayed in the hospital, including time in the ED, for 1.7 days and incurred $3551 in costs. About one-third of patients were hospitalized again for SHO. Inpatient SHO patients incurred significantly higher monthly costs after their initial SHO-related hospitalization than patients in the two other groups ($2084 vs $1313 and $1372), corresponding to 59% or 52% higher monthly costs for inpatient SHO patients., Limitations: These analyses excluded patients who did not seek ED or hospital care when faced with SHO; events may have been miscoded; and we were not able to account for clinical characteristics associated with SHO, such as insulin dose and duration of diabetes, or unmeasured confounders., Conclusions: The burden associated with SHO is not negligible. About 4% of T1DM patients using basal-bolus insulin regimens are hospitalized at least once due to SHO. Not only did those patients incur the costs of their SHO hospitalization, but they also incur red at least $712 (52%) more in costs per month after their hospitalization than outpatient SHO or comparison patients. Reducing SHO events can help decrease the burden associated with SHO among patients with T1DM.

Aims: More than 29 million people in the US have type 2 diabetes mellitus (T2DM), a chronic metabolic disorder characterized by a progressive deterioration of glucose control, which eventually requires insulin. Abnormally low levels of blood glucose, a feared side-effect of insulin treatment, may cause severe hypoglycemia (SHO), leading to emergency department (ED) admission, hospitalization, and long-term complications; these, in turn, drive up the costs of T2DM. This study's objective was to estimate the prevalence and costs of SHO-related hospitalizations and their additional longer-term impacts on patients with T2DM using insulin., Methods: Using Truven MarketScan claims, we identified adult T2DM patients using basal and basal-bolus insulin regimens who were hospitalized for SHO (inpatient SHO patients) during 2010-2015. Two comparison groups were defined: those with outpatient SHO-related encounters only, including ED visits without hospitalization (outpatient SHO patients), and those with no SHO- or acute hyperglycemia-related events (comparison patients). Lengths of stay and SHO-related hospitalization costs were estimated, and propensity score and inverse probability weighting methods were used to adjust for baseline differences across the groups to evaluate longer-term impacts., Results: We identified 66,179 patients using basal and 81,876 patients using basal-bolus insulin, of which ∼1.1% (basal) to 3.2% (basal-bolus) experienced at least one SHO-related hospitalization. Among those who experienced SHO (i.e. those in the inpatient and outpatient SHO groups), 27% (basal) and 40% (basal-bolus) experienced at least one SHO-related hospitalization. One-third of basal and about one-quarter of basal-bolus patients were admitted directly to the hospital; the remainder were first assessed or treated in the ED. Inpatient SHO patients using basal insulin stayed in the hospital, including time in the ED, for 2.8 days and incurred $6896 in costs; patients using basal-bolus insulin stayed in the hospital for 2.6 days and incurred costs of $5802. Forty-to-fifty percent of inpatient SHO patients were hospitalized again for SHO. Inpatient SHO patients using basal insulin incurred significantly higher monthly costs after their initial SHO-related hospitalization than patients in the other two groups ($2935 vs $1819 and $1638), corresponding to 61% and 79% higher monthly costs; patients using basal-bolus insulin also incurred significantly higher monthly costs than patients in the other groups ($3606 vs $2731 and $2607), corresponding to 32% and 38% higher monthly costs., Limitations: These analyses excluded patients who did not seek ED or hospital care when faced with SHO; events may have been miscoded; and we were not able to account for clinical characteristics associated with SHO, such as insulin dose and duration of diabetes, or unmeasured confounders., Conclusions: The burden associated with SHO is not negligible. Nearly one in three patients using only basal insulin and one in four patients using basal-bolus regimens who experienced SHO were hospitalized at least once due to SHO. Not only did those patients incur the costs of their SHO hospitalization, but they also incurred at least $1,116 (62%) and $875 (70%) more per month than outpatient SHO or comparison patients. Reducing SHO events can help decrease the burden associated with SHO among patients with T2DM.

Background: Health plans have responded to the many treatment options for type 2 diabetes mellitus by implementing formulary restriction policies, including step therapy, to control costs. Little is known about the impact of step therapy programs on antidiabetes medication use., Objective: To assess the impact of a sitagliptin step therapy program on antidiabetes medication use among sitagliptin users., Methods: Using pharmacy claims from the Symphony Health Solutions' Integrated Dataverse, we compared the use of sitagliptin and other antidiabetes medications by patients enrolled in a health plan (Plan A) that implemented a sitagliptin step therapy program on July 1, 2013, with the use by patients who were contemporaneously enrolled in 2 comparison plans-Plans B and C-without step therapy programs. Sitagliptin-a dipeptidyl peptidase (DPP)-4 inhibitor-was in tier 3 in Plans A and B and in tier 2 in Plan C during the study period. We assessed the use of antidiabetes medications during the pre-step therapy period (January-June 2013) and the post-step therapy period (October 2013-March 2014)., Results: We identified 2995 patients enrolled in Plan A, 751 enrolled in Plan B, and 394 enrolled in Plan C who received sitagliptin during the pre-step therapy period. Patient characteristics and pre-step therapy sitagliptin use were similar across plans. During the post-step therapy period, more patients in Plan A (approximately 70%) discontinued sitagliptin than patients in Plan B (approximately 51%) and Plan C (approximately 25%). Approximately 30% of patients in Plan A switched to another DPP-4 inhibitor compared with approximately 15% and 2% of patients in Plans B and C, respectively. Seventeen percent of patients in Plan A discontinued sitagliptin without replacement but continued using other antidiabetes medications compared with approximately 13% and 8% of patients in Plans B and C, respectively. In all, 17% of patients in Plans A and B and 11% of patients in Plan C discontinued using all antidiabetes medications., Conclusion: The step therapy program changed patients' use of sitagliptin, which was the target of the step therapy program, as well as of other antidiabetes medications. Most patients stopped sitagliptin treatment after the step therapy program started. Some patients discontinued sitagliptin treatment without replacement, but others discontinued using all antidiabetes medications.

Background: A phase III trial evaluated the efficacy and safety of Daklinza (daclatasvir or DCV) in combination with sofosbuvir (SOF) for treatment of genotype (GT) 3 hepatitis C virus (HCV) patients., Aim: This study evaluated the cost-effectiveness of DCV + SOF vs SOF in combination with ribavirin (RBV) over a 20-year time horizon from the perspective of a United States (US) payer., Methods: A published Markov model was adapted to reflect US demographic characteristics, treatment patterns, costs of drug acquisition, monitoring, disease and adverse event management, and mortality risks. Clinical inputs came from the ALLY-3 and VALENCE trials. The primary outcome was the incremental cost-utility ratio. Life-years, incidence of complications, number of patients achieving sustained virological response (SVR), and the total cost per SVR were secondary outcomes. Costs (2014 USD) and quality-adjusted life years (QALYs) were discounted at 3% per year. Deterministic, probabilistic, and scenario sensitivity analyses were conducted., Results: DCV + SOF was associated with lower costs and better effectiveness than SOF + RBV in the base case and in almost all scenarios (i.e. treatment-experienced, non-cirrhotic, time horizons of 5, 10, and 80 years). DCV + SOF was less costly, but also slightly less effective than SOF + RBV in the cirrhotic and treatment-naïve population scenarios. Results were sensitive to variations in the probability of achieving SVR for both treatment arms. DCV + SOF costs less than $50,000 per QALY gained in 79% of all probabilistic iterations compared with SOF + RBV., Conclusion: DCV + SOF is a dominant option compared with SOF + RBV in the US for the overall GT 3 HCV patient population.

Aim: Amyloid positron emission tomography (aPET) measurement of Alzheimer's disease (AD) pathology could improve the accurate diagnosis of cognitive disorders. Appropriate use criteria recommend that only dementia experts order aPET., Materials & Methods: We surveyed 145 dementia experts about their current approaches to evaluation and treatment and the likely influence of aPET., Results: Experts expected aPET to alter diagnostic procedures and patient management and also increase diagnostic certainty. They anticipated confirming AD or altering pharmacological treatment following positive results more than excluding AD following negative results. Experts familiar with aPET reported changes that were more consistent with appropriate use criteria and published evidence., Conclusions: Knowledge about aPET strongly influenced effects on diagnostic certainty and changed clinical practice. Dementia experts may need additional training to achieve optimal benefit from aPET.

Background: The Disease Severity Scoring System (DS3) is a validated measure for evaluating Gaucher disease type 1 (GD1) severity. We developed a new framework, consisting of health states, transition probabilities between those states, and preferences for those states (utilities) based on the DS3 to predict long-term outcomes of patients starting treatment. We defined nine mutually exclusive (alive) health states based on three DS3 categories: mild (0 ≤ DS3 ≤ 3.5) without symptoms of bone disease; mild with bone pain, mild with severe skeletal complications (SSC) defined as lytic lesions, avascular necrosis, or fracture; moderate (3.5 < DS3 ≤ 6.5) without SSC; moderate with SSC; marked (6.5 < DS3 ≤ 9.5) without SSC; marked with SSC; severe (9.5 < DS3 ≤ 19) without SSC; and severe with SSC. Health-state transition probabilities and utilities were estimated from a longitudinal sample of patients with GD1 who started enzyme replacement therapy (the DS3 Score Study). Age dependent GD1-specific mortality was derived from published data. We used a Markov state-transition model to illustrate how to estimate time spent in each health state., Results: The average predicted utilities for each health state ranged from 0.76 for mild disease with no clinical symptoms of bone disease to 0.52 with severe disease with SSC. Transition probabilities depended on disease severity (DS3 score) at treatment initiation and whether patients had undergone a total splenectomy or had an intact spleen/partial splenectomy prior to starting treatment. Patients who started treatment with intact or residual spleens spent more time in better health states than those who started treatment with total splenectomy., Conclusions: This new framework, which is based on the DS3, can be used to project the long-term outcomes of GD1 patients starting treatment. The framework could also be used to compare the long-term outcomes of different GD1 treatment options., Trial Registration: NCT01136304 . Registered: May 31, 2010 (retrospectively registered).

Background: Methotrexate (MTX) is the primary disease-modifying antirheumatic drug used for the treatment of rheumatoid arthritis (RA). Optimizing the use of oral and subcutaneous MTX may delay the use of expensive biologic therapies; the effect of such a delay on overall medical costs is currently unknown., Objective: To compare the 5-year healthcare costs of treatment pathways for patients with RA who initiate oral MTX in the United States., Methods: We identified patients with RA in the Symphony Health Solutions database (Integrated Dataverse) who initiated treatment with oral MTX in 2009 and had RA-related claims for each year through 2014. We then grouped the patients into 4 treatment cohorts, including those who (1) continued to use oral MTX, (2) switched to subcutaneous MTX, (3) switched to subcutaneous MTX and then added or switched to a biologic therapy, and (4) added or switched to a biologic therapy. The costs (in 2015 US dollars) for pharmaceuticals, office visits, hospitalizations, and emergency department visits were estimated for each cohort., Results: Of the total 35,640 patients in this study, 15,599 patients continued to use oral MTX, with an average cost of $47,464 per patient in the full study period; 1802 patients switched to subcutaneous MTX, with an average per-patient cost of $59,058; 711 patients switched to subcutaneous MTX and then added or switched to a biologic agent, with an average per-patient cost of $175,391 and a mean time to a biologic use of 1184 days; and 17,528 patients added or switched to a biologic from oral MTX, with an average per-patient cost of $212,595 and a mean time to a biologic use of 478 days. Biologic treatments were responsible for the cost differences between the cohorts; the nondrug costs were similar across the groups., Conclusion: Our findings that patients who switched to subcutaneous MTX incurred lower costs than patients who only used oral MTX before using biologics may provide useful information for patients and providers who are choosing between continued MTX use and adding or switching to a biologic based on treatment guidelines.

Objectives: Although much has been published about the demographic and clinical characteristics of elderly patients with overactive bladder (OAB) who were enrolled in clinical trials, very little is known about the general population of elderly Americans with OAB. We update this gap in the literature by using real-world data to describe this population., Methods: We used Medicare claims and the Medicare Current Beneficiary Surveys from 2006 to 2011 to identify patients with OAB. We describe the demographic characteristics, functional impairment and physical limitations, concurrent medical conditions, Charlson Comorbidity Index (CCI) scores, and concomitant medication use of patients with OAB; these characteristics are also described by sex and age group (65-74 vs. ≥75 years). We also compare the characteristics of OAB with non-OAB patients., Results: We identified 415 elderly patients with OAB (average age 79 years; 71% female) and 6868 without OAB (average age 77 years; 62% female). Patients with OAB reported high levels of functional impairment as measured by the Activities of Daily Living (44% vs. 33% for non-OAB patients), Instrumental ADL (53% vs. 40% for non-OAB patients), and physical functioning limitation (90% vs. 81% for non-OAB patients) scales. Elderly patients with OAB also experienced high levels of comorbidity burden, as measured by the number of medical conditions (18 vs. 11 for non-OAB patients), CCI (2.1 vs. 1.4 for non-OAB patients), and number of non-OAB-related concomitant medications used (11 vs. 8 for non-OAB patients)., Conclusions: Elderly patients with OAB in the general population have high levels of functional impairment and physical limitations, comorbidity, and concomitant medication use. These characteristics should be taken into consideration when managing OAB symptoms and designing future clinical studies. These results, which are representative of elderly patients with OAB in the general US population, should, however, be interpreted in light of the key limitations of the data we used: patients may have been misclassified and medical conditions overestimated due to artifacts of diagnosis coding and our results can only be generalized to patients who were enrolled in Medicare Parts A, B, and D for at least 12 continuous months.

Background: Approximately 593,000 to 780,000 people in the United States (US) have been diagnosed with Crohn's disease (CD), and an additional 33,000 are diagnosed yearly. Our objective was to estimate CD's impact on medical costs, lost earnings, work and school absences, health status, and health-related quality of life (HRQOL) in the US and to compute current and forecasted national costs., Methods: We used the nationally representative Medical Expenditure Panel Survey to match 539 respondents with CD to similar respondents without any inflammatory bowel disease (IBD). We estimated annual costs, work and school absences, and self-assessed health status. HRQOL was assessed by the SF-12 Physical Component Summary and Mental Component Summary (PCS and MCS) scores. CD prevalence rates, population counts, and costs were used to forecast total national costs., Results: CD is associated with higher medical costs ($13,446 versus $6029) and lost earnings ($1249 versus $644) and is responsible for $3.48 billion in total national costs (expected to increase to $3.72 billion in 2025). Respondents with CD were more likely to miss work (38% versus 33%) or school (64% versus 33%), less likely to report being in excellent or very good physical health (24% versus 63%), and experienced lower HRQOL measured by the Physical Component Summary (43.4 versus 48.5) and Mental Component Summary (48.6 versus 50.0) than those without IBD., Conclusions: CD is responsible for increased medical care costs and lower earnings, health status, and HRQOL. These data can serve as benchmarks when examining future CD-related costs and HRQOL.

There is growing awareness of the impact of human activity on the climate and the need to stem this impact. Public health care decision makers from Sweden and the United Kingdom have started examining environmental impacts when assessing new technologies. This article considers the case for incorporating environmental impacts into the health technology assessment (HTA) process and discusses the associated challenges. Two arguments favor incorporating environmental impacts into HTA: 1) environmental changes could directly affect people's health and 2) policy decision makers have broad mandates and objectives extending beyond health care. Two types of challenges hinder this process. First, the nascent evidence base is insufficient to support the accurate comparison of technologies' environmental impacts. Second, cost-utility analysis, which is favored by many HTA agencies, could capture some of the value of environmental impacts, especially those generating health impacts, but might not be suitable for addressing broader concerns. Both cost-benefit and multicriteria decision analyses are potential methods for evaluating health and environmental outcomes, but are less familiar to health care decision makers. Health care is an important and sizable sector of the economy that could warrant closer policy attention to its impact on the environment. Considerable work is needed to track decision makers' demands, augment the environmental evidence base, and develop robust methods for capturing and incorporating environmental data as part of HTA., (Copyright © 2016 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Objectives: Economic evaluation is becoming more common and important as new biologic therapies for rheumatoid arthritis (RA) are developed. While much has been published about how to design cost-utility models for RA to conduct these evaluations, less has been written about the sources of data populating those models. The goal is to review the literature and to provide recommendations for future data collection efforts., Methods: This study reviewed RA cost-utility models published between January 2006 and February 2014 focusing on five key sources of data (health-related quality-of-life and utility, clinical outcomes, disease progression, course of treatment, and healthcare resource use and costs). It provided recommendations for collecting the appropriate data during clinical and other studies to support modeling of biologic treatments for RA., Results: Twenty-four publications met the selection criteria. Almost all used two steps to convert clinical outcomes data to utilities rather than more direct methods; most did not use clinical outcomes measures that captured absolute levels of disease activity and physical functioning; one-third of them, in contrast with clinical reality, assumed zero disease progression for biologic-treated patients; little more than half evaluated courses of treatment reflecting guideline-based or actual clinical care; and healthcare resource use and cost data were often incomplete., Conclusions: Based on these findings, it is recommended that future studies collect clinical outcomes and health-related quality-of-life data using appropriate instruments that can convert directly to utilities; collect data on actual disease progression; be designed to capture real-world courses of treatment; and collect detailed data on a wide range of healthcare resources and costs.

Background and Objective: Past studies have found episodes of severe hypoglycemia (SH) to be serially dependent. Those studies, however, only considered the impact of a single (index) event on future risk; few have analyzed SH risk as it evolves over time in the presence (or absence) of continuing events. The objective of this study was to determine the dynamic risks of SH events conditional on preceding SH events among patients with type 2 diabetes (T2D) who have initiated basal insulin., Methods: We used an electronic health records database from the United States that included encounter and laboratory data and clinical notes on T2D patients who initiated basal insulin therapy between 2008 and 2011 and to identify SH events. We used a repeated-measures lagged dependent variable logistic regression model to estimate the impact of SH in one quarter on the risk of SH in the next quarter., Results: We identified 7235 patients with T2D who initiated basal insulin. Patients who experienced ≥1 SH event during any quarter were more likely to have ≥1 SH event during the subsequent quarter than those who did not (predicted probabilities of 7.4% and 1.0%, respectively; p < 0.01). This effect was stronger than the impact of history of SH before starting basal insulin (predicted probabilities of 1.0% and 3.2%, respectively; p < 0.01) or of a history of SH during the titration period (predicted probabilities of 1.1% and 2.8%, respectively; p < 0.01)., Conclusions: The risk of experiencing a SH event is highly dependent on a patient's immediate history of SH events and therefore the value of preventing one SH event may be substantial. These results can inform patient care by providing clinicians with dynamic data on a patient's risk of SH, which in turn can facilitate appropriate adjustment of the risk-benefit ratio for individualized patient care. These results should, however, be interpreted in light of the key limitations of our study: not all SH events may have been captured or coded in the database, data on filled prescriptions were not available, we were unable to adjust for basal insulin dose, and the post-titration follow-up period could have divided into time units other than quarters (3 month blocks) resulting in potentially different conclusions. Further real-world studies on how to best to identify patients at risk for SH events based on the presence of recent SH events, rather than on more distant 'prior' events, can help healthcare providers to better manage patients starting basal insulin.

Objective: This study estimated the economic burden of obesity-related comorbidities (ORCs) in the US, at both the person and population levels., Methods: The Geisinger Health System provided electronic medical records and claims between January 2004 and May 2013 for a sample of 153,561 adults (50% males and 97% white). Adults with < 2 years of data, who were underweight (body mass index (BMI) < 18.5 kg/m(2)), or had diseases causing major weight change (e.g., malignancy) during the study period (i.e., continuous enrollment in health plans) were excluded. A total of 21 chronic conditions, with established association with obesity in the literature, were identified by diagnosis codes and/or lab test results. The total healthcare costs were measured in each year. The association between annual costs and ORCs was assessed by a regression, which jointly considered all the ORCs. The per-person incremental costs of a single comorbidity, without any of the other ORCs, were calculated. The population-level economic burden was the product of each ORC's incremental costs and the annual prevalence of the ORC among 100,000 individuals. The prevalence of ORCs was stratified by obesity status to estimate the economic burden among 100,000 individuals with obesity and among those without., Results: This study identified 56,895 adults (mean age = 47 years; mean BMI = 29.6 kg/m(2)). The annual prevalence of ORCs ranged from 0.5% for pulmonary embolism (PE) to 41.8% for dyslipidemia. The per-person annual incremental costs of a single ORC ranged from $120 for angina to $1665 for PE. Hypertensive diseases (HTND), dyslipidemia, and osteoarthritis were the three most expensive ORCs at the population level; each responsible for ≥$18 million annually among 100,000 individuals. HTND and osteoarthritis were much more costly among individuals with obesity than those without obesity., Limitations: Data were from a small geographic region., Conclusions: ORCs are associated with substantial economic burden, especially for those requiring continuous treatments.

Objectives: To derive current real-world data on the rates and costs of severe hypoglycemia (SH) for people with type 2 diabetes mellitus (T2D) who have initiated basal insulin therapy and to examine differences in SH rates and costs stratified by history of prior SH events., Methods: We used a nation-wide electronic health records database that included encounter and laboratory data, as well as clinical notes, to estimate the rates and costs of SH events among adults with T2D who initiated basal insulin between 2008 and 2011. Unadjusted and regression-adjusted rates and quarterly costs were calculated for all patients as well as stratified by history of a SH event before starting basal insulin and history of a SH event during the basal insulin titration period., Results: We identified 7235 incident cases of basal insulin use among patients with T2D who did not use insulin during the previous 12 months. Regression-adjusted incidence and total event rates were 10.36 and 11.21 per 100 patient-years, respectively. A history of SH events during the pre-index baseline and post-index titration periods were statistically significantly associated with both the incidence and total event rates (p < 0.01). Regression-adjusted total healthcare and diabetes-related costs were statistically significantly (p < 0.01) higher in those quarters when a SH event occurred than in those quarters without any SH events ($3591 vs. $487 and $3311 vs. $406, respectively). A history of previous SH or SH events during the titration period were not statistically significantly associated with costs., Conclusions: These results suggest that the real-world burden of SH is high among people with T2D who start using basal insulin and that history of previous SH events, both before starting insulin and during the insulin titration period, influences future SH. These results can also provide insights into interventions that can prevent or delay SH. These results should, however, be interpreted in light of the key limitations of our study: not all SH events may have been captured or coded in the database, data on filled prescriptions were not available, and the post-titration follow-up period could have been divided into time units other than quarters (3 month blocks) resulting in potentially different conclusions. Further real-world studies on the frequency and costs of SH, using methods to identify as many SH events as possible, can allow healthcare providers to make more informed decisions on the risks and benefits of basal insulin therapy in T2D patients.

Introduction: Clinical trials have shown that liraglutide effectively lowers glycated hemoglobin A1c (A1C) levels in adult patients with type 2 diabetes (T2D). However, no studies have evaluated the effectiveness of liraglutide by body mass index (BMI) in the United States (US) in clinical practice. This study examined liraglutide's clinical effectiveness to lower A1C and body weight after 6 months in T2D patients stratified by baseline BMI., Methods: This was a retrospective cohort study using the General Electric Centricity electronic medical records database. Adult patients with T2D (≥18 years and BMI≥ 25 kg/m(2)) and A1C >7% at baseline who started liraglutide between January 1, 2010 and January 31, 2013 and who did not use insulin or a glucagon-like peptide-1 analog 12 months before initiating liraglutide (N = 3,005) were selected. Changes from baseline, stratified by BMI, in A1C, body weight, A1C <7% goal attainment, and incidence of severe hypoglycemia at 6-month follow-up were examined., Results: After 6 months, A1C levels decreased on average by 0.95%, 1.02%, 0.99%, and 0.84% for BMI categories 25.0-29.9 (n = 333), 30.0-34.9 (n = 793), 35.0-39.9 (n = 821), and ≥40.0 kg/m(2) (n = 1,058), respectively (P = 0.30). The proportions of patients achieving A1C <7% at 6 months were 38.2%, 37.0%, 40.9%, and 41.0% (P = 0.54). The absolute body weight decreased by 1.5 to 4.0 kg across BMI and the rate of severe hypoglycemia (0.2%) was low., Conclusion: Patients with T2D experienced statistically significant decreases in A1C and body weight after initiating liraglutide regardless of their BMI. Liraglutide reduced A1C equally well across baseline BMI in clinical practice in the US.

Objectives: Obesity is a known risk factor for type 2 diabetes (T2D). We conducted a case-control study to assess the association between body mass index (BMI) and the risk of being diagnosed with T2D in the United States., Methods: We selected adults (≥ 18 years old) who were diagnosed with T2D (defined by ICD-9-CM diagnosis codes or use of anti-diabetic medications) between January 2004 and October 2011 ("cases") from an electronic health records database provided by an integrated health system in the Middle Atlantic region. Twice as many individuals enrolled in the health system without a T2D diagnosis during the study period ("controls") were selected based on age, sex, history of cardiac comorbidities or hyperinflammatory state (defined by C-reactive protein and erythrocyte sedimentation rate), and use of psychiatric or beta blocker medications. BMI was measured during one year prior to the first observed T2D diagnosis (for cases) or a randomly assigned date (for controls); individuals with no BMI measure or BMI < 18.5 kg/m2 were excluded. We assessed the impact of increased BMI (overweight: 25-29.9 kg/m2; Obesity Class I: 30-34.9 kg/m2; Obesity Class II: 35-39.9 kg/m2; Obesity Class III: ≥40 kg/m2), relative to normal BMI (18.5-24.9 kg/m2), on a T2D diagnosis using odds ratios (OR) and relative risks (RR) estimated from multiple logistic regression results., Results: We included 12,179 cases (mean age: 55, 43% male) and 25,177 controls (mean age: 56, 45% male). We found a positive association between BMI and the risk of a T2D diagnosis. The strength of this association increased with BMI category (RR [95% confidence interval]: overweight, 1.5 [1.4-1.6]; Obesity Class I, 2.5 [2.3-2.6]; Obesity Class II, 3.6 [3.4-3.8]; Obesity Class III, 5.1 [4.7-5.5])., Conclusions: BMI is strongly and independently associated with the risk of being diagnosed with T2D. The incremental association of BMI category on the risk of T2D is stronger for people with a higher BMI relative to people with a lower BMI.