Developing a cross-validation tool for evaluating economic evidence in rapid literature reviews.

Background: Rapid economic reviews efficiently summarize economic evidence. However, reporting main findings without assessing quality and credibility can be misleading. The objective of this study was to develop a rapid cross-validation screening tool to evaluate economic evidence when conducting rapid economic literature reviews. Methods: This article outlines our reasoning and the theoretical concepts for developing the screening tool. Results: This cross-validation tool is a qualitative approach under a Bayesian framework that uses prior health economic evidence to gauge the credibility of the rapid economic review's findings. This article describes an application of this tool and highlights practical considerations for its development and deployment. Conclusion: This tool can provide a valuable screening instrument to evaluate the quality and credibility of the economic evidence.

Developing Economic Models for Assessing the Cost-Effectiveness of Multiple Diagnostic Tests: Methods and Applications.

BACKGROUND: Clinical pathways with multiple diagnostic tests are complex to model, but problematic and simplistic approaches are often used in economic evaluations. METHODS: We analyzed statistical methods of handling multiple diagnostic tests and provided guidance on applying these methods in economic modeling. We first introduced a statistical model to quantify the correlations between 2 tests and how those correlations can be incorporated within an economic model. We also presented the general form of conditional dependence among multiple tests. We then introduced net reclassification improvement (NRI), a measure that evaluates the added value of a new risk factor (e.g., biomarker) for risk prediction. We further provided 2 examples to illustrate the application of these methods. RESULTS: Our first example illustrated how to model an add-on test to an existing test, in the absence of a perfect reference standard. After accounting for the imperfect nature of both tests and the conditional dependence between tests, the potential health benefits from the additional test were reduced. This led to differential cost-effectiveness results when comparing models using the perfect test and conditional independence assumptions. The second example illustrated how to evaluate the added value of a new risk factor using the NRI measure. Using the new risk classification provides greater precision in risk prediction, and in the example, the strategy using the new risk classification with treatment for selected individuals led to more favorable cost-effectiveness results. CONCLUSIONS: These innovative methods for handling multiple diagnostic tests have improved the methodology within the field and should be adopted to provide more accurate estimates within cost-effectiveness analyses. HIGHLIGHTS: Economic evaluations of multiple diagnostic tests often apply problematic simplistic approaches, such as ignoring conditional dependence between 2 tests or assuming a perfect final test in the diagnostic pathway. We provided guidance on how to apply improved methods for economic modeling.We introduced methods to model conditional dependence between 2 imperfect tests. We used an example to illustrate how assumptions about perfect diagnostic test accuracy and conditional independence between tests affect cost-effectiveness.Compared with the results of the area under the receiver-operating-characteristic curve, net reclassification improvement has distinct advantages in measuring the added value of a new risk factor for model-based economic evaluation.Economic evaluations that appropriately account for the complexities of diagnostic test pathways can help decision makers ensure efficient use of resources.

Economic modelling of costs associated with outcomes reported for type 2 diabetes mellitus (T2DM) patients in the CANVAS and EMPA-REG cardiovascular outcomes trials.

AIMS: To model direct medical costs associated with reductions in cardiovascular disease (CVD) events in T2DM patients reported in the CANVAS and EMPA-REG trials, which assessed the cardiovascular safety of canagliflozin and empagliflozin, respectively. MATERIALS AND METHODS: Costs were modeled from a US managed care organization (MCO) perspective for the CVD outcomes included in both trials: three-point major adverse cardiovascular event (MACE) and its components (cardiovascular-related death, nonfatal myocardial infarction, nonfatal stroke), as well as heart failure requiring hospitalization. The rate of CVD events averted (difference between study drug and placebo) was projected to the portion of an MCO T2DM population matching the respective trial's inclusion criteria. A targeted literature search for paid amounts directly associated with each CVD event provided the unit costs, which were applied to the projected number of events averted, to calculate costs avoided per member per year (PMPY). One-way sensitivity analyses were performed on events averted, unit costs, and percentages of trial-applicable patients. RESULTS: Based on three-point MACE events averted, costs avoided PMPY of $6.17 (range: $1.27-$10.94) for CANVAS and $2.75 ($0.19-$4.83) for EMPA-REG were estimated. Costs avoided for individual components of MACE ranged from $0.77 to $3.84 PMPY for CANVAS and from -$0.97 (additional costs) to $1.54 for EMPA-REG. PMPY costs avoided for heart failure were $2.72 for CANVAS and $1.32 for EMPA-REG. LIMITATIONS AND CONCLUSIONS: Models assumed independent, non-recurrent outcomes and were restricted to medical costs directly associated with the trial-reported events. The reductions in CVD events in T2DM patients reported for both CANVAS and EMPA-REG project to a positive cost avoidance for these events in an MCO population. The analysis did not include an assessment of the impact on total cost, as the costs associated with adverse events, drug utilization or other clinical outcomes were not examined.

Health characteristics of patients with asthma, COPD and asthma-COPD overlap in the NHANES database.

Introduction: Asthma and COPD have overlapping characteristics. As there are limited data on whether asthma-COPD overlap (ACO) represents a distinct condition, this study aimed to determine the similarities and differences of ACO with asthma and COPD. Methods: US population-based, cross-sectional study using National Health and Nutrition Examination Survey data (2009-2012) compared participants with ACO vs those with asthma or COPD, each as mutually exclusive disease states. Demographics, health status, disability/limitations, health care resource utilization, clinical characteristics, and peripheral blood eosinophil counts were analyzed. Results: A total of 1,609, 479, and 299 participants with asthma, COPD, and ACO, respectively, were included. An age-matched asthma subgroup included 299 participants from the asthma group. Compared with asthma and COPD, participants with ACO had worse health status, increased disease burden, and more comorbid conditions. The ACO, vs age-matched asthma subgroup, had lower percent predicted prebronchodilator forced expiratory volume in 1 second (82.1% vs 88.0%; P=0.017). The ACO group had significantly more asthma attacks in the past year than the age-matched asthma subgroup (49.8% vs 38.4%; P<0.001). The ACO group had more participants with postbronchodilator forced expiratory volume in 1 second <80% predicted (52.1%) vs COPD (30.8%; P=0.003) and more participants with blood eosinophil counts ≥400 cells/µL (16.9%) vs COPD (9.5%; P=0.007) and the asthma subgroup (6.7%; P=0.014). Conclusion: The ACO group represents an important subset of patients with chronic respiratory disease with an increased burden of disease over asthma and COPD individually. Early identification of this population will enable appropriate therapeutic interventions in a timely manner.

Economic Impact in Medicaid Beneficiaries with Schizophrenia and Cardiometabolic Comorbidities Treated with Once-Monthly Paliperidone Palmitate vs. Oral Atypical Antipsychotics.

OBJECTIVE: The objective of this study was to compare persistence, costs, and healthcare resource utilization in patients with schizophrenia and cardiometabolic comorbidities treated with once-monthly paliperidone palmitate or an oral atypical antipsychotic. METHODS: Medicaid data from six states (07/2009-03/2015) were used to identify adults with schizophrenia and cardiometabolic comorbidities initiated on once-monthly paliperidone palmitate or an oral atypical antipsychotic (index date) on 01/2010 or after. Persistence to index medication at 12 months (no gap ≥ 90 days) was compared between patients taking once-monthly paliperidone palmitate and an oral atypical antipsychotic using Chi-squared tests. The 12-month post-index healthcare costs and healthcare resource utilization were compared using multivariate ordinary least squares and Poisson regression, respectively. RESULTS: Selected patients taking once-monthly paliperidone palmitate (n = 371) were younger (mean age: 45.0 vs. 47.5 years, standardized difference = 24%) than patients taking oral atypical antipsychotics (n = 8296). Persistence at 12 months was higher in patients taking once-monthly paliperidone palmitate (40 vs. 33%, p = 0.006). Adjusted all-cause medical costs were lower in patients taking once-monthly paliperidone palmitate vs. patients taking oral atypical antipsychotics (mean monthly cost differences = US $ - 369, p = 0.004) while all-cause pharmacy costs were higher (mean monthly cost differences = US $279, p < 0.001), resulting in no significant difference in total costs (mean monthly cost differences = US $ - 90, p = 0.357). No significant difference was observed in cardiometabolic comorbidity-related pharmacy or medical costs. Compared with patients taking oral atypical antipsychotics, patients taking once-monthly paliperidone palmitate had more schizophrenia-related outpatient visits (incidence rate ratio = 1.44, p < 0.001) but fewer cardiometabolic comorbidity-related inpatient admissions (incidence rate ratio = 0.73, p < 0.001) with shorter lengths of stay (incidence rate ratio = 0.72, p = 0.020), and fewer cardiometabolic comorbidity-related long-term care admissions (incidence rate ratio = 0.56, p = 0.016). CONCLUSIONS: Medicaid beneficiaries with schizophrenia and cardiometabolic comorbidities who were initiated on once-monthly paliperidone palmitate had similar 12-month total healthcare costs compared with oral atypical antipsychotics. Cardiometabolic comorbidity-related utilization of inpatient and long-term care services was lower in patients taking once-monthly paliperidone palmitate.

Real-world adherence and economic outcomes associated with paliperidone palmitate versus oral atypical antipsychotics in schizophrenia patients with substance-related disorders using Medicaid benefits.

AIM: Compare medication utilization, costs and healthcare resource use in schizophrenia patients with substance-related disorders initiated on once-monthly paliperidone palmitate (PP1M) or an oral atypical antipsychotic (OAA). MATERIALS & METHODS: Data from six Medicaid states (07/2009-03/2015) were used to compare outcomes between PP1M and OAA patients. RESULTS: PP1M patients had higher 12-month antipsychotic adherence and persistence than OAA patients. PP1M patients had lower medical (mean monthly cost difference [MMCD] = US$-191, p = 0.020), higher pharmacy (MMCD = US$250, p < 0.001) and similar total costs (MMCD = US$59, p = 0.517) during the overall follow-up. PP1M patients had lower rates of outpatient visits and inpatient days but higher rates of mental health-related utilization. CONCLUSION: PP1M was associated with higher antipsychotic adherence and persistence, and similar total costs versus OAA.