Racial and ethnic differences in disease course Medicare expenditures for beneficiaries with dementia.

BACKGROUND: Research on racial and ethnic disparities in costs of care during the course of dementia is sparse. We analyzed Medicare expenditures for beneficiaries with dementia to identify when during the course of care costs are the highest and whether they differ by race and ethnicity. METHODS: We analyzed data from the 2000-2016 Health and Retirement Study (HRS) linked with corresponding Medicare claims to estimate total Medicare expenditures for four phases: (1) the year before a dementia diagnosis, (2) the first year following a dementia diagnosis, (3) ongoing care for dementia after the first year, and (4) the last year of life. We estimated each patient's phase-specific and disease course Medicare expenditures by using a race-specific survival model and monthly expenditures adjusted for patient characteristics. We investigated healthcare utilization by service type across races/ethnicities and phases of care. RESULTS: Adjusted mean total Medicare expenditures for non-Hispanic (NH) Black ($165,730) and Hispanic beneficiaries with dementia ($160,442) exceeded corresponding expenditures for NH Whites ($136,326). In the year preceding and immediately following initial dementia diagnosis, mean Medicare expenditures for NH Blacks ($26,337 and $20,429) exceeded expenditures for Hispanics and NH Whites ($21,399-23,176 and 17,182-18,244). The last year of life was responsible for the greatest cost contribution: $51,294 (NH Blacks), $47,469 (Hispanics), and $39,499 (NH Whites). These differences were driven by greater use of high-cost services (e.g., emergency department, inpatient and intensive care), especially during the last year of life. CONCLUSIONS: NH Black and Hispanic beneficiaries with dementia had higher disease course Medicare expenditures than NH Whites. Expenditures were highest for NH Black beneficiaries in every phase of care. Further research should address mechanisms of such disparities and identify methods to improve communication, shared decision-making, and access to appropriate services for all populations.

Life-Sustaining Treatments Among Medicare Beneficiaries with and without Dementia at the End of Life.

BACKGROUND: Older adults with dementia including Alzheimer's disease may have difficulty communicating their treatment preferences and thus may receive intensive end-of-life (EOL) care that confers limited benefits. OBJECTIVE: This study compared the use of life-sustaining interventions during the last 90 days of life among Medicare beneficiaries with and without dementia. METHODS: This cohort study utilized population-based national survey data from the 2000-2016 Health and Retirement Study linked with Medicare and Medicaid claims. Our sample included Medicare fee-for-service beneficiaries aged 65 years or older deceased between 2000 and 2016. The main outcome was receipt of any life-sustaining interventions during the last 90 days of life, including mechanical ventilation, tracheostomy, tube feeding, and cardiopulmonary resuscitation. We used logistic regression, stratified by nursing home use, to examine dementia status (no dementia, non-advanced dementia, advanced dementia) and patient characteristics associated with receiving those interventions. RESULTS: Community dwellers with dementia were more likely than those without dementia to receive life-sustaining treatments in their last 90 days of life (advanced dementia: OR = 1.83 [1.42-2.35]; non-advanced dementia: OR = 1.16 [1.01-1.32]). Advance care planning was associated with lower odds of receiving life-sustaining treatments in the community (OR = 0.84 [0.74-0.96]) and in nursing homes (OR = 0.68 [0.53-0.86]). More beneficiaries with advanced dementia received interventions discordant with their EOL treatment preferences. CONCLUSIONS: Community dwellers with advanced dementia were more likely to receive life-sustaining treatments at the end of life and such treatments may be discordant with their EOL wishes. Enhancing advance care planning and patient-physician communication may improve EOL care quality for persons with dementia.

Alzheimer's disease medication use and adherence patterns by race and ethnicity.

BACKGROUND: We examined racial and ethnic differences in medication use for a representative US population of patients with Alzheimer's disease and related dementias (ADRD). METHODS: We examined cholinesterase inhibitors and memantine initiation, non-adherence, and discontinuation by race and ethnicity, using data from the 2000-2016 Health and Retirement Study linked with Medicare and Medicaid claims. RESULTS: Among newly diagnosed ADRD patients (n = 1299), 26% filled an ADRD prescription ≤90 days and 36% ≤365 days after diagnosis. Among individuals initiating ADRD-targeted treatment (n = 1343), 44% were non-adherent and 24% discontinued the medication during the year after treatment initiation. Non-Hispanic Blacks were more likely than Whites to not adhere to ADRD medication therapy (odds ratio: 1.50 [95% confidence interval: 1.07-2.09]). DISCUSSION: Initiation of ADRD-targeted medications did not vary by ethnoracial group, but non-Hispanic Blacks had lower adherence than Whites. ADRD medication non-adherence and discontinuation were substantial and may relate to cost and access to care. HIGHLIGHTS: Initiation of anti-dementia medications among newly diagnosed Alzheimer's disease and related dementias (ADRD) patients was low in all ethnoracial groups. ADRD medication non-adherence and discontinuation were substantial and may relate to cost and access to care. Compared to Whites, Blacks and Hispanics had lower use, poorer treatment adherence, and more frequent discontinuation of ADRD medication, but when controlling for disease severity and socioeconomic factors, racial disparities diminish. Our findings demonstrate the importance of adjusting for socioeconomic characteristics and disease severity when studying medication use and adherence in ADRD patients.

Racial and Ethnic Differences in Hospice Use and Hospitalizations at End-of-Life Among Medicare Beneficiaries With Dementia.

Importance: The pool of studies examining ethnic and racial differences in hospice use and end-of-life hospitalizations among patients with dementia is limited and results are conflicting, making it difficult to assess health care needs of underresourced racial and ethnic groups. Objective: To explore differences in end-of-life utilization of hospice and hospital services among patients with dementia by race and ethnicity. Design, Setting, and Participants: This cohort study used national survey data from the Health and Retirement Study linked with Medicare and Medicaid claims that reflected a range of socioeconomic, health, and psychosocial characteristics. Eligible participants were Medicare fee-for-service beneficiaries aged 65 years or older diagnosed with dementia who died between 2000 and 2016. Analyses were performed from June to December 2021. Exposures: Race and ethnicity. Main Outcomes and Measures: We examined the frequency and costs of hospice care, emergency department (ED) visits, and hospitalizations during the last 180 days of life among Medicare decedents with dementia. We analyzed the proportion of dementia decedents with advance care planning and their end-of-life care preferences. Results: The cohort sample included 5058 beneficiaries with dementia (mean [SD] age, 85.5 [8.0] years; 3038 women [60.1%]; 809 [16.0%] non-Hispanic Black, 357 [7.1%] Hispanic, and 3892 non-Hispanic White respondents [76.9%]). In adjusted analysis, non-Hispanic Black decedents (odds ratio [OR], 0.65; 95% CI, 0.55-0.78), nursing home residents (OR, 0.81; 95% CI, 0.71-0.93), and survey respondents represented by a proxy (OR, 0.84; 95% CI, 0.71-0.99) were less likely to use hospice, whereas older decedents (age 75-84 vs 65-74 years: OR, 1.39; 95% CI, 1.12-1.72; age ≥85 vs 65-74 years: OR, 1.39; 95% CI, 1.13-1.71), women (OR, 1.19; 95% CI, 1.05-1.35), and decedents with higher education (high school vs less than high school: OR, 1.17; 95% CI, 1.01-1.36; more than high school vs less than high school: OR, 1.32; 95% CI, 1.13-1.54), more severe cognitive impairment (OR, 1.51; 95% CI, 1.02-2.23), and more instrumental activities of daily living limitations (OR, 1.07; 95% CI, 1.01-1.12) were associated with higher hospice enrollment. A higher proportion of Black and Hispanic decedents with dementia used ED (645 of 809 [79.7%] and 274 of 357 [76.8%] vs 2753 of 3892 [70.7%]; P < .001) and inpatient services (625 of 809 [77.3%] and 275 of 357 [77.0%] vs 2630 of 3892 [67.5%]; P < .001) and incurred roughly 60% higher inpatient expenditures at the end of life compared with White decedents (estimated mean: Black, $23 279; 95% CI, $20 690-$25 868; Hispanic, $23 471; 95% CI, $19 532-$27 410 vs White, $14 609; 95% CI, $13 800-$15 418). A higher proportion of Black and Hispanic than White beneficiaries with dementia who were enrolled in hospice were subsequently admitted to the ED (56 of 309 [18.1%] and 22 of 153 [14.4%] vs 191 of 1967 [9.7%]; P < .001) or hospital (48 of 309 [15.5%] and 17 of 153 [11.1%] vs 119 of 1967 [6.0%]; P < .001) before death. The proportion of dementia beneficiaries completing advance care planning was lower among Black (146 of 704 [20.7%]) and Hispanic (66 of 308 [21.4%]) beneficiaries compared with White beneficiaries (1871 of 3274 [57.1%]). A higher proportion of Black and Hispanic decedents with dementia had written instructions choosing all care possible to prolong life (30 of 144 [20.8%] and 12 of 65 [18.4%] vs 72 of 1852 [3.9%]), whereas a higher proportion of White decedents preferred to limit care in certain situations (1708 of 1840 [92.8%] vs 114 of 141 [80.9%] and 51 of 64 [79.7%]), withhold treatments (1448 of 1799 [80.5%] vs 87 of 140 [62.1%] and 41 of 62 [66.1%]), and forgo extensive life-prolonging measures (1712 of 1838 [93.1%] vs 120 of 138 [87.0%] and 54 of 65 [83.1%]). Conclusions and Relevance: The results of this cohort study highlight unique end-of-life care utilization and treatment preferences across racial and ethnic groups among patients with dementia. Medicare should consider alternative payment models to promote culturally competent end-of-life care and reduce low-value interventions and costs among the population with dementia.

When are breakthrough therapies cost-effective?

BACKGROUND: An increasing proportion of novel drug approvals use accelerated pathways, with notable growth in the US Food and Drug Administration-designated breakthrough pathway in recent years. Breakthrough therapy (BT) designation suggests that these therapies offer substantial potential to improve health outcomes but their value for money is not fully understood, as BTs typically cost more than non-BTs (NBTs). OBJECTIVE: To assess the economic value of BTs and factors associated with their reported value. METHODS: Using the Tufts Medical Center Cost-Effectiveness (CE) Analysis Registry, we (1) summarized the CE of BTs, as measured by cost per quality-adjusted-life-year (QALY); (2) compared the CE of BTs and NBTs in the United States; and (3) identified factors associated with BT CE using general estimating equation models across US willingness-to-pay (WTP) benchmarks ($50K-$150K/QALY). RESULTS: Between 2013 and 2018, the US Food and Drug Administration approved 279 drugs, designating 83 (32%) as BTs. Incremental costs and health gains (QALYs) were higher for BTs relative to NBTs ($29,000 vs $20,000 and 0.7 vs 0.2 QALYs, respectively), and BTs had more favorable CE ratios compared with NBTs (median values $38,000/QALY vs $50,000/QALY, respectively). For BTs, hepatitis C treatments had the most favorable CE ratios, which may be driven by the curative nature of some hepatitis C therapies. Furthermore, BT CE ratios for new molecular entities (NMEs) were about 40% lower than ratios for non-NME BTs on average, which may signal more value for money when the BT has a new active molecule. Regression analysis to identify trends driving CE found that BT drugs compared with active comparators (instead of best supportive care) were less likely to be cost-effective at standard US WTP thresholds (odds ratio [OR] = 0.1-0.6) and that BTs in the neoplasm space also trended less likely to be cost-effective (OR = 0.12-0.43). CE ratios reported by studies with industry funding were also more likely to be cost-effective than ratios from studies with other funding sources (OR = 4.3-4.5), though this finding was not significant at WTP thresholds over $50,000/QALY gained. CONCLUSIONS: Evidence from published, peer-reviewed CE studies suggests that BTs may confer greater health benefits than NBTs in terms of overall QALYs. Our analysis supports that the US Food and Drug Administration BT designation may be associated with increased value for money for these BTs. However, factors such as the disease area, NME status, and comparator (active vs standard of care) will also influence whether these therapies are cost-effective. DISCLOSURES: Dr Cohen reports grants or contracts from PhRMA Foundation, National Pharmaceutical Council, AstraZeneca, Bristol-Myers Squibb, Eli Lilly and Company, Gilead Sciences, Regeneron, Pfizer, Merck, Johnson & Johnson, Vir Biotechnology, Moderna, Amgen, and Lundbeck; consulting fees from AbbVie, Biogen, IQVIA, Novartis, Partnership for Health Analytic Research, Pharmerit, Precision Health Economics, Sage, Sanofi, and Sarepta; and stock or stock options from Bristol-Myers Squibb, Johnson & Johnson, and Merck. Ms Kowal is an employee and stockholder of Genentech, Inc. Dr Yeh is an employee and stockholder of Roche, Inc.

The Impact of Broader Value Elements on Cost-Effectiveness Analysis: Two Case Studies.

OBJECTIVES: This study aimed to explore the impact of including broader value elements in cost-effectiveness analyses by presenting 2 case studies, one on human papillomavirus (HPV) infection and one on early-stage Hodgkin's lymphoma (ESHL). METHODS: We identified broader value elements (eg, patient and caregiver time, spillover health effects, productivity) from the Second Panel's Impact Inventory and the ISPOR Special Task Force's value flower. We then evaluated the cost-effectiveness of HPV vaccination versus no vaccination (case 1) and combined modality therapy (CMT) versus chemotherapy alone for treatment of adult ESHL (case 2) using published simulation models. For each case study, we compared incremental cost-effectiveness ratios considering health sector impacts only (the "base-case" scenario) with incremental cost-effectiveness ratios incorporating broader value elements. RESULTS: For vaccination of US girls against HPV before sexual debut versus no vaccination, the base-case result was $38 334 per disability-adjusted life-year averted. Including each broader value element made cost-effectiveness progressively more favorable, with HPV vaccination becoming cost-saving (ie, reducing costs and averting more disability-adjusted life-years) when the analysis incorporated productivity costs. For CMT versus chemotherapy alone in patients with ESHL, the base-case result indicated that CMT was cost-saving. Including all elements made this treatment's net monetary benefits (the sum of its averted resource costs and the net value of its health impacts) less favorable, even as the contribution from CMT's near-term health benefits grew. CONCLUSIONS: Including broader value elements can substantially influence cost-effectiveness ratios, although the direction and the magnitude of their impact can differ across interventions and disease context.

Targeting of the diabetes prevention program leads to substantial benefits when capacity is constrained.

OBJECTIVE: Approximately 84 million people in the USA have pre-diabetes, but only a fraction of them receive proven effective therapies to prevent type 2 diabetes. We estimated the value of prioritizing individuals at highest risk of progression to diabetes for treatment, compared to non-targeted treatment of individuals meeting inclusion criteria for the Diabetes Prevention Program (DPP). METHODS: Using microsimulation to project outcomes in the DPP trial population, we compared two interventions to usual care: (1) lifestyle modification and (2) metformin administration. For each intervention, we compared targeted and non-targeted strategies, assuming either limited or unlimited program capacity. We modeled the individualized risk of developing diabetes and projected diabetic outcomes to yield lifetime costs and quality-adjusted life expectancy, from which we estimated net monetary benefits (NMB) for both lifestyle and metformin versus usual care. RESULTS: Compared to usual care, lifestyle modification conferred positive benefits and reduced lifetime costs for all eligible individuals. Metformin's NMB was negative for the lowest population risk quintile. By avoiding use when costs outweighed benefits, targeted administration of metformin conferred a benefit of $500 per person. If only 20% of the population could receive treatment, when prioritizing individuals based on diabetes risk, rather than treating a 20% random sample, the difference in NMB ranged from $14,000 to $20,000 per person. CONCLUSIONS: Targeting active diabetes prevention to patients at highest risk could improve health outcomes and reduce costs compared to providing the same intervention to a similar number of patients with pre-diabetes without targeted selection.

Comparing inpatient costs of heart failure admissions for patients with reduced and preserved ejection fraction with or without type 2 diabetes.

Both heart failure (HF) and diabetes mellitus (DM) account for major healthcare expenditures. We evaluated inpatient expenditures and cost drivers in patients admitted with HF with and without DM. METHODS: We created a retrospective cohort of acutely decompensated HF patients, using linked data from cost accounting systems and electronic medical records. We stratified patients by LVEF into reduced ejection fraction (HFrEF, LVEF ≤40%) and preserved ejection fraction (HFpEF, LVEF >40%) groups and by DM status at admission. RESULTS: Our population had 544 people: 285 HFrEF patients (43.5% with DM) and 259 HFpEF patients (43.6% with DM). Patients with HFrEF and DM had the longest hospital stay (5.10 ± 5.21 days). Patients with HFrEF and DM had the highest hospitalization cost ($11 576 ± 15 818). HFrEF and HFpEF patients with DM had the highest cost, and cost per day alive was highest for HFpEF patients with DM [$3153 (95% CI 2332, 4262)]. CONCLUSION: Overall cost was higher for patients with DM, whether or not they were admitted with acute HF due to HFrEF or HFpEF. Cost per day alive for patients with DM continued to exceed corresponding costs for patients without DM, with HFpEF patients with DM having the highest cost.

Two-year outcomes and cost for heart failure patients following discharge from the hospital after an acute heart failure admission.

BACKGROUND: Follow-up costs among heart failure (HF) patients place a heavy burden on healthcare. We sought to quantify follow-up costs after a HF hospitalization, as well as, length of stay (LOS), time to readmission and mortality for patients with preserved (HFpEF) versus reduced (HFrEF) left ventricular ejection fractions. METHODS: We developed a retrospective cohort of consecutively admitted acutely decompensated HF patients who had at least 1 follow-up visit. We extracted baseline clinical data from electronic medical records, and collected follow-up data on number of readmissions, length of stay, mortality over a 2-year period, and cost data from our hospital's accounting system. Kaplan-Meier curves and log rank tests were used to evaluate survival and time to first readmission in the two HF groups. RESULTS: Our study included 444 patients, of whom 224 (51%) had HFrEF. Patients with HFrEF compared to HFpEF had higher median total costs per month alive during the 2-year follow-up period ($1684 vs. $1496, p = 0.02), and median inpatient costs per month alive ($1368 vs. $991, p = 0.03). HFrEF patients had higher total mean LOS per month alive post index hospitalization discharge (1.6 vs 1 days, p = 0.003). Similarly, HF related mean LOS per month alive was higher for HFrEF patients than for HFpEF patients (1.3 vs 0.3 days, p = 0.003). Patients with HFrEF had a trend for shorter time to first readmission and survival compared to those with HFpEF. CONCLUSIONS: At 2 years following their index HF admission, patients with HFrEF have higher costs and longer LOS compared to HFpEF patients.

Cost comparison across heart failure patients with reduced and preserved ejection fractions: Analyses of inpatient decompensated heart failure admissions.

BACKGROUND: Heart failure (HF) is the leading cause of inpatient admissions in the US for adults aged over 65 years and accounts for more than $17 billion in Medicare expenditures annually. There are limited published data on factors influencing expenditure and the relationship between cost and hospital length of stay. We sought to describe institutional costs of HF hospitalization, as well as demographic and clinical predictors of higher hospitalization costs in an academic hospital setting. METHODS AND RESULTS: Demographic and clinical information was collected retrospectively for 564 unique consecutive patients with a decompensated HF admission during 2010-2013. Forty-six percent had a baseline LVEF >40%, categorized as HF with preserved ejection fraction (HFpEF). Forty-three percent were female and the mean age was 71 years. Patients with reduced ejection fraction (HFrEF) were predominantly male, younger and had a lower burden of baseline comorbidities than HFpEF patients. Length of stay was longer for HFrEF (median 4 days) than HFpEF (median 3 days, p = 0.01). Mean total hospitalization cost was $9521. Mean costs trended higher for HFrEF patients than for HFpEF patients ($10,286 versus $8858, p = 0.07). Room and board contributed more than half of all costs. CONCLUSIONS: In this single-center study, we observed a trend towards higher HF hospitalization costs for patients with HFrEF, compared to HFpEF, even though patients with HFpEF are older and had more comorbid conditions. Costs were largely driven by length of stay, with higher heart rate at admission, lower systolic blood pressure, and higher creatinine associated with higher inpatient costs.

Patient Variability Seldom Assessed in Cost-effectiveness Studies.

BACKGROUND: Cost-effectiveness analysis (CEA) estimates can vary substantially across patient subgroups when patient characteristics influence preferences, outcome risks, treatment effectiveness, life expectancy, or associated costs. However, no systematic review has reported the frequency of subgroup analysis in CEA, what type of heterogeneity they address, and how often heterogeneity influences whether cost-effectiveness ratios exceed or fall below conventional thresholds. METHODS: We reviewed the CEA literature cataloged in the Tufts Medical Center CEA Registry, a repository describing cost-utility analyses published through 2016. After randomly selecting 200 of 642 articles published in 2014, we ascertained whether each study reported subgroup results and collected data on the defining characteristics of these subgroups. We identified whether any of the CEA subgroup results crossed conventional cost-effectiveness benchmarks (e.g., $100,000 per QALY) and compared characteristics of studies with and without subgroup-specific findings. RESULTS: Thirty-eight studies (19%) reported patient subgroup results. Articles reporting subgroup analyses were more likely to be US-based, government funded (v. drug industry- or nonprofit foundation-funded) studies, with a focus on primary or secondary (v. tertiary) prevention (P < 0.05 for comparisons). One or more patient characteristics were used to stratify CEA results 68 times within the 38 studies, with most stratifications using one characteristic (n = 47), most commonly age (n = 35). Among the 23 stratifications reported alongside average ratios in US studies, 13 produced subgroup ratios that crossed a conventional CEA ratio benchmark. CONCLUSIONS: Most CEAs do not report any subgroup results, and those that do most often stratify only by patient age. Over half of the subgroup analyses reported could lead to different value-based decision making for at least some patients.

Understanding the Value of Individualized Information: The Impact of Poor Calibration or Discrimination in Outcome Prediction Models.

BACKGROUND: Risk prediction models allow for the incorporation of individualized risk and clinical effectiveness information to identify patients for whom therapy is most appropriate and cost-effective. This approach has the potential to identify inefficient (or harmful) care in subgroups at different risks, even when the overall results appear favorable. Here, we explore the value of personalized risk information and the factors that influence it. METHODS: Using an expected value of individualized care (EVIC) framework, which monetizes the value of customizing care, we developed a general approach to calculate individualized incremental cost effectiveness ratios (ICERs) as a function of individual outcome risk. For a case study (tPA v. streptokinase to treat possible myocardial infarction), we used a simulation to explore how an EVIC is influenced by population outcome prevalence, model discrimination (c-statistic) and calibration, and willingness-to-pay (WTP) thresholds. RESULTS: In our simulations, for well-calibrated models, which do not over- or underestimate predicted v. observed event risk, the EVIC ranged from $0 to $700 per person, with better discrimination (higher c-statistic values) yielding progressively higher EVIC values. For miscalibrated models, the EVIC ranged from -$600 to $600 in different simulated scenarios. The EVIC values decreased as discrimination improved from a c-statistic of 0.5 to 0.6, before becoming positive as the c-statistic reached values of ~0.8. CONCLUSIONS: Individualizing treatment decisions using risk may produce substantial value but also has the potential for net harm. Good model calibration ensures a non-negative EVIC. Improvements in discrimination generally increase the EVIC; however, when models are miscalibrated, greater discriminating power can paradoxically reduce the EVIC under some circumstances.

The peculiar economics of life-extending therapies: a review of costing methods in health economic evaluations in oncology.

Published literature lacks consensus, and most guidelines lack definitive recommendations as to whether cost-effectiveness analyses (CEAs) should include all "future" costs or distinguish between related and unrelated medical costs. This systematic review of oncology CEAs evaluated cost methods used and the impact on the cost-effectiveness of incorporating different cost categories, including costs due to study intervention, related medical costs of the treated condition, and unrelated medical costs. Of the 59 studies reviewed, none included medical costs unrelated to the treated condition and 14 studies (32%) excluded direct medical costs related to the condition but not the evaluated intervention. Recomputing ICERs using different cost categories altered overall cost-effectiveness conclusions. The authors propose conventional CEA methods may implicitly penalize therapies that add "expensive" life years for chronically ill patients. Presenting ICERs computed with and without disease-attributable costs can help better convey how much the treatment itself contributes to overall costs.

Potential role of network meta-analysis in value-based insurance design.

OBJECTIVES: Value-based insurance design (V-BID) has emerged as an approach to improve health outcomes and contain healthcare costs by encouraging use of high-value care. We estimated the impact of a V-BID for osteoporosis treatments using comparative effectiveness evidence and real-world data from a California health insurance plan to estimate the benefits of the design's implementation. METHODS: This study consisted of 4 steps. First, we reviewed randomized clinical trials including osteoporosis treatments-alendronate, ibandronate, risedronate, raloxifene, and teriparatide-reported in a recent Agency for Health Research Quality systematic review. Second, we performed a network meta-analysis to synthesize data from the clinical trials and estimate the comparative effectiveness of included treatments. Third, we implemented a V-BID by removing co-payments for the most effective treatments. Fourth, using a Monte Carlo simulation, we estimated the impact of the V-BID in terms of fracture reduction and cost-savings. RESULTS: Thirty-two randomized controlled trials were included in the network meta-analysis. We estimated that alendronate, risedronate, and teriparatide have the highest probability of being most effective across each fracture type-vertebral, hip, and nonvertebral/ nonhip. After eliminating co-payments, (ie, reducing them to zero), for these treatments, we estimated the health plan would experience a 7% (n = 287) decrease in fractures and an 8% ($6.8 million) decrease in costs. CONCLUSIONS: Our study illustrates the benefits of comparative effectiveness evidence in V-BID development. We show that where clinical trials are lacking, network meta-analysis can provide valuable insights into the potential clinical and economic benefits of V-BID.

The economic burden of treatment-resistant depression.

BACKGROUND: Major depressive disorder (MDD) is a leading cause of disability, morbidity, and mortality worldwide. The lifetime prevalence in the United States is estimated at 17%. Treatment-resistant depression (TRD) is generally defined as failure to achieve remissions despite adequate treatment. About 30% of patients do not achieve remission after 4 different antidepressant treatment trials. A few studies have examined the economic burden of TRD, but none has investigated the cost associated with more chronic and extensive forms of TRD characterized by nonresponse to ≥4 treatment trials. OBJECTIVE: The objective of this study was to compare the health care utilization (HCU) and direct medical expenditures of TRD patients with those of chronic MDD patients. METHODS: Patients with chronic MDD (defined as ≥2 years of continuous treatment) and patients with TRD (defined as undergoing at least 4 different qualifying antidepressant therapy trials) were identified in the PharMetrics Patient-centric Database. The association between TRD and medical expenditures was measured by using multivariate regression analysis. RESULTS: The classification of TRD had a clinically meaningful and statistically significant association with increased medical expenditures. Holding all else equal, the classification of TRD was associated with a 29.3% higher costs (P < 0.001) in medical expenditures compared with patients not meeting the study definition of TRD. CONCLUSIONS: These results demonstrate that TRD is associated with significantly higher per-patient medical costs due to higher HCU. The findings suggest that the development of treatment alternatives for TRD is warranted. Limitations related to the use of secondary administrative data are noted.

A role for research: an observation on preventive services for women.

BACKGROUND: The Patient Protection and Affordable Care Act of 2010 (ACA) added preventive services for women, recommended by the IOM, to healthcare coverage requirements beginning in August 2011. PURPOSE: The current review provides evidence on the economic impact of services that will be covered under the ACA, focusing on IOM-recommended measures that address women's health. METHODS: This review analyzed the cost-effectiveness literature related to these services using the Tufts Medical Center Cost-Effectiveness Analysis Registry (www.cearegistry.org), which catalogs detailed information on cost-effectiveness studies published in English in the peer-reviewed literature. In order to keep the review relevant to current clinical practice, the analysis was restricted to studies published in 2000-2010. The data search and analysis were performed in 2011. RESULTS: Cost-effectiveness studies have evaluated a limited subset of the preventive measures available for women. Further, few cost-effectiveness studies have evaluated the recommended counseling and screening services for women. Of 16 relevant studies found, eight focused on HIV screening, with results varying substantially depending on the specific groups screened and the screening frequency. CONCLUSIONS: The current review underscores the finding that there is a substantial gap in the health economic literature on preventive care, especially with respect to screening and counseling of women in the primary care setting. There is some evidence that better access to preventive services can be maintained at a reasonable cost to the healthcare system, and that certain services may even lower healthcare costs.

Assessment of the influence of test characteristics on the clinical and cost impacts of methicillin-resistant Staphylococcus aureus screening programs in US hospitals.

OBJECTIVE: To compare the impacts of different methicillin-resistant Staphylococcus aureus (MRSA) screening test options (eg, polymerase chain reaction [PCR], rapid culture) and program characteristics on the clinical outcomes and budget of a typical US hospital. METHODS: We developed an Excel-based decision-analytic model, using published literature to calculate and compare hospital costs and MRSA infection rates for PCR- or culture-based MRSA screening and then used multivariate sensitivity analysis to evaluate key variables. Same-day PCR testing for a representative 370-bed teaching hospital in the United States was assessed in different populations (high-risk patients, intensive care unit [ICU] patients, or all patients) and compared with other test options. RESULTS: Different screening program populations (all patients, high-risk patients, ICU patients, or patients with previous MRSA colonization or infection only) represented a potential savings of $12,158-$76,624 per month over no program ($188,618). Analysis of multiple test options in high-risk population screening indicated that same-day PCR testing of high-risk patients resulted in fewer infections over 1,720 patient-days (2.9, compared with 3.5 for culture on selective media and 3.8 for culture on nonselective media) and the lowest total cost ($112,012). The costs of other testing approaches ranged from $113,742 to $123,065. Sensitivity analysis revealed that variations in transmission rate, conversion to infection, prevalence increases, and hospital size are important to determine program impact. Among test characteristics, turnaround time is highly influential. CONCLUSION: All screening options showed reductions in infection rates and cost impact improvement over no screening program. Among the options, same-day PCR testing for high-risk patients slightly edges out the others in terms of fewest infections and greatest potential cost savings.

Economic impact of switching to bivalirudin for a primary percutaneous coronary intervention in a US hospital.

BACKGROUND: The addition of glycoprotein IIb/IIIa inhibitors (GPIs) to heparin in percutaneous coronary intervention (PCI) procedures has been demonstrated to reduce ischemic complications; however, GPI use is known to increase the risk of bleeding events, which are linked to increased mortality, longer hospital length of stay, greater medical resource utilization, and increased costs. New antithrombotic therapies have the potential to improve clinical outcomes and decrease costs. The Harmonizing Outcomes with Revascularization and Stents in Acute Myocardial Infarction (HORIZONS-AMI) study of bivalirudin demonstrated significantly reduced clinical event rates (mortality and bleeding) compared with an unfractionated heparin (UFH)+GPI regimen. OBJECTIVE: The potential clinical and economic value of implementing a bivalirudin-based strategy for ST-segment elevation myocardial infarction (STEMI) patients receiving primary PCI (PPCI) is compared with current UFH+GPI-based practice from a US hospital perspective. METHODS: A budget impact model was developed to compare treatment of STEMI patients undergoing PPCI with a bivalirudin- or UFH+GPI-based strategy. Clinical data for the model were derived from the HORIZONS-AMI trial, and included 30-day event rates for major complications (eg, protocol bleeding, Q-wave MI, repeat PCI, and coronary artery bypass graft procedures). United States cost data and clinical practice data were derived from a Premier Perspective™ database analysis and published sources. RESULTS: Overall, average procedure costs per UFH+GPI-treated patient were $18,561. Treating patients with bivalirudin (incorporating 7.2% provisional GPI use per HORIZONS-AMI) may save $1690 per patient (average procedural cost, $16,872). In extrapolating these benefits to the American College of Cardiology/American Heart Association recommended institutional minimum of 36 PPCIs annually, 1 major bleeding event (3.7%) and 3 minor bleeding events (6.8%) could be averted with use of bivalirudin. In addition, introducing a bivalirudin-based strategy to treat a minimum cohort of 36 STEMI patients would save the hospital budget $60,807 (9%) per year. CONCLUSION: Using a bivalirudin-based strategy in STEMI patients undergoing PPCI is associated with favorable clinical and economic outcomes when compared with an UFH+GPI-based strategy in a US hospital setting.

Can we better prioritize resources for cost-utility research?

PURPOSE: We examined 512 published cost-utility analyses (CUAs) in the U.S. and other developed countries from 1976 through 2001 to determine: 1) the types of interventions studied; 2) whether they cover diseases and conditions with the highest burden; and, 3) to what extent they have covered leading health concerns defined by the Healthy People 2010 report. DATA AND METHODS: We compared rankings of the most common diseases covered by the CUAs to rankings of U.S. disease burden. We also examined the extent to which CUAs covered key Healthy People 2010 priorities. RESULTS: CUAs have focused mostly on pharmaceuticals (40%) and surgical procedures (16%). When compared to leading causes of DALYs, the data show overrepresentation of CUAs in cerebrovascular disease, diabetes, breast cancer, and HIV/AIDS, and underrepresentation in depression and bipolar disorder, injuries, and substance abuse disorders. Few CUAs have targeted Healthy People 2010 areas, such as physical activity. CONCLUSIONS: Published CUAs are associated with burden measures, but have not covered certain important health problems. These discrepancies do not alone indicate that society has been targeting resources for research inefficiently, but they do suggest the need to formalize the question of where each CUA research dollar might do the most good.

Quality of abstracts of papers reporting original cost-effectiveness analyses.

BACKGROUND: Although many peer-reviewed journals have adopted standards for reporting cost-effectiveness analyses (CEAs), guidelines do not exist for the accompanying abstracts. Abstracts are the most easily accessed portion of journal articles, yet little is known about their quality. The authors examined the extent to which abstracts of published CEAs include key data elements (intervention, comparator, target population, study perspective) and assessed the effect of journal characteristics on reporting quality. METHODS: Systematic review of the English-language medical literature from 1998 through 2001. The authors searched MEDLINE for original CEAs reported in costs per quality-adjusted life years(i.e., cost-utility analyses). Two independent readers abstracted data elements and met to resolve discrepancies. RESULTS: Among the 303 abstracts reviewed, a clear description of the intervention was present in 94%, comparator in 71%, target population in 85%, and study perspective in 28%. All 4 data elements were reported in 20% of abstracts, 3 elements in 49%, 2 in 22%, and 0 or 1 in 9%. In journals with CEA-specific abstract reporting requirements, structured abstract requirements, or impact factors>or=10, significantly more data were included in abstracts than in journals without these features (P<0.01 for all comparisons). CONCLUSIONS: Abstracts of published CEAs frequently omit data elements critical to proper study interpretation. An explicit core set of reporting standards is needed, based on the standards by the US Public Health Service's Panel on Cost-Effectiveness for reporting of CEAs, but specific to the accompanying abstracts.