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Biomedical subjects

P J Neumann

Publications and source records attributed to P J Neumann.

At least 19 recordsLinked to original sources

Predictors of hospital length of stay and cost in patients with intracerebral hemorrhage.

We used the 2002 Healthcare Cost and Utilization Project Nationwide Inpatient Sample to assess hospital length of stay (LOS) and cost among adults with a principal diagnosis of intracerebral hemorrhage (n = 13,239). Sixty-nine percent of patients were aged > or =65 years, and 31% died during hospitalization. Mean LOS (cost) was 7.7 days (15,256 dollars) (survivors: 9.6 days, 17,442 dollars). Patient, hospital, and payer characteristics accounted for 69.1% of variation in cost per discharge.

Adult↗

Cost-effectiveness of migraine prevention: the case of topiramate in the UK.

The aim of this study was to assess the cost-effectiveness of topiramate vs. no preventive treatment in the UK. Model inputs included baseline migraine frequency, treatment discontinuation and response, preventive and acute medical cost per attack [2005 GBP ( pound)] and gain in health utility. Outcomes included monthly migraines averted, acute and preventive treatment costs and cost per quality-adjusted life year (QALY). Topiramate was associated with 1.8 fewer monthly migraines and a QALY gain of 0.0384. The incremental cost of topiramate vs. no preventive treatment was about 10 UK pounds per migraine averted and 5700 UK pounds per QALY. Results are sensitive to baseline monthly migraine frequency, triptan use rate and the gain in utility. Incorporating savings from reduced work loss (about 36 UK pounds per month) suggests that topiramate would be cost saving compared with no preventive treatment. This analysis suggests that topiramate is a cost-effective treatment for migraine prevention compared with no preventive treatment.

Adult↗

Measuring Alzheimer's disease progression with transition probabilities: estimates from CERAD.

OBJECTIVES: To estimate annual transition probabilities (i.e., the likelihood that a patient will move from one disease stage to another in a given time period) for AD progression. Transition probabilities are estimated by disease stages (mild, moderate, severe) and settings of care (community, nursing home), accounting for differences in age, gender, and behavioral symptoms as well as the length of time a patient has been in a disease stage. METHODS: Using data from the Consortium to Establish a Registry for Alzheimer's Disease (CERAD), the authors employed a modified survival analysis to estimate stage-to-stage and stage-to-nursing home transition probabilities. To account for individual variability, a Cox proportional hazards model was fit to the CERAD data to estimate hazard ratios for gender, age (50 to 64, 65 to 74, and more than 75 years), and level of behavioral symptoms (low/high, according to responses to the Behavioral Rating Scale for Dementia) for each of the key stage-to-stage and stage-to-nursing home transitions. RESULTS: The transition probabilities underscore the rapid progression of patients into more severe disease stages and into nursing homes and the differences among population subgroups. In general, male gender, age under 65, and high level of behavioral symptoms were associated with higher transition probabilities to more severe disease stages. Disease progression is roughly constant as a function of the time a patient has spent in a particular stage. CONCLUSIONS: Transition probabilities provide a useful means of characterizing AD progression. Economic models of interventions for AD should consider the varied course of progression for different population subgroups, particularly those defined by high levels of behavioral symptoms.

Aged↗

Effectiveness of chemotherapy for advanced lung cancer in the elderly: instrumental variable and propensity analysis.

PURPOSE: To compare the effectiveness of chemotherapy given to elderly patients in routine practice for stage IV non-small-cell lung cancer (NSCLC) with the efficacy observed in randomized trials. PATIENTS AND METHODS: We used instrumental variable analysis (IVA) and propensity scores (PS) to simulate the conditions of a randomized trial in a retrospective cohort of patients over age 65 from the Survival, Epidemiology, and End Results (SEER) tumor registry. Geographic variation in chemotherapy use served as the instrument for the IVA analysis, and propensity scores were calculated with a logistic model based on patient disease and sociodemographic characteristics. RESULTS: Among 6,232 elderly patients, the instrumental variable estimate indicated an increase in median survival of 33 days and an improvement in 1-year survival of 9% attributable to chemotherapy. In a Cox regression model, chemotherapy administration was associated with a hazard ratio of 0.81 (95% confidence interval, 0.76 to 0.85). When survival was analyzed separately within propensity score quintiles, the hazard ratios were all similar, ranging from 0.78 to 0.85. These results are comparable with those of a large meta-analysis, which found a hazard ratio of 0.87 in the subgroup of patients over age 65. CONCLUSION: Chemotherapy for stage IV NSCLC seems to have effectiveness for elderly patients and those with comorbid conditions that is similar to the efficacy seen in randomized trials containing mostly younger, highly selected patients. All suitable patients should be given the opportunity to consider palliative chemotherapy for metastatic NSCLC.

Aged↗

The case for daily dialysis: its impact on costs and quality of life.

Research suggests daily hemodialysis may improve clinical outcomes. To date, a comprehensive review of its implications on quality of life has not been performed, and little is known about its economic impact. We conducted an economic evaluation comparing short daily or nocturnal hemodialysis with thrice-weekly conventional in-center dialysis. Data on the quality of life and clinical effects of daily dialysis were obtained from more than 60 reports from 13 daily dialysis programs around the world (n = 197). Cost data were derived principally from the US Renal Data System, Centers for Disease Control, and Medicare Payment Advisory Commission. Resource use during daily hemodialysis was modeled after two ongoing programs in the United States. Results suggest that patients feel better and direct treatment costs could be reduced with daily dialysis. Costs are sensitive to assumptions about the effect of daily dialysis on hospital days. Reductions of at least 8% in hospital days are required for these modalities to be cost saving compared with documented reductions of 30% to 100%. Larger well-controlled studies of daily versus conventional dialysis would be helpful to determine whether daily dialysis fulfills these promises. Medicare policy, which limits payment for most patients to three dialysis treatments weekly, poses a disincentive to more widespread adoption among dialysis centers. Given this constraint to broader acceptance, we address several policy options to gain a better understanding of the potential risks and benefits of daily dialysis.

Centers for Medicare and Medicaid Services, U.S.↗

Bayesian value-of-information analysis. An application to a policy model of Alzheimer's disease.

A framework is presented that distinguishes the conceptually separate decisions of which treatment strategy is optimal from the question of whether more information is required to inform this choice in the future. The authors argue that the choice of treatment strategy should be based on expected utility, and the only valid reason to characterize the uncertainty surrounding outcomes of interest is to establish the value of acquiring additional information. A Bayesian decision theoretic approach is demonstrated through a probabilistic analysis of a published policy model of Alzheimer's disease. The expected value of perfect information is estimated for the decision to adopt a new pharmaceutical for the population of patients with Alzheimer's disease in the United States. This provides an upper bound on the value of additional research. The value of information is also estimated for each of the model inputs. This analysis can focus future research by identifying those parameters where more precise estimates would be most valuable and indicating whether an experimental design would be required. We also discuss how this type of analysis can also be used to design experimental research efficiently (identifying optimal sample size and optimal sample allocation) based on the marginal cost and marginal benefit of sample information. Value-of-information analysis can provide a measure of the expected payoff from proposed research, which can be used to set priorities in research and development. It can also inform an efficient regulatory framework for new healthcare technologies: an analysis of the value of information would define when a claim for a new technology should be deemed substantiated and when evidence should be considered competent and reliable when it is not cost-effective to gather any more information.

Alzheimer Disease↗

FEV(1) is associated with risk of asthma attacks in a pediatric population.

BACKGROUND: FEV(1) is endorsed by the National Asthma Education and Prevention Program as a means for grading asthma severity. However, few data exist on the relationship between FEV(1) and asthma outcomes during long-term follow-up. OBJECTIVE: We explored the relationship between the percent predicted FEV(1) (FEV(1)%) and subsequent asthma attacks in a longitudinal study of pediatric lung health. METHODS: A retrospective cohort of 13,842 children (100,292 observations) seen annually over a 15-year interval was analyzed for measurement of pulmonary function, and a respiratory questionnaire was completed. Up to grade 9, a standard questionnaire was completed by a parent or guardian; thereafter it was completed by the patient. For each observation, the report of an attack during the past year was paired with FEV(1) recorded at the field survey 1 year earlier. RESULTS: A progressive decrease in the proportion of individuals reporting an attack was associated with increasing decile of FEV(1)%. Two categorization schemes for FEV(1)% were examined: a scheme based on the National Asthma Education and Prevention Program recommendations (<60%, 60%-80%, and >80%), and an alternative scheme (<80%, 80%-100%, and >100%). In multivariate models, FEV(1)% was an independent predictor of attacks: among the parental report group, the odds ratios were 2.1 (95% CI, 1.3-3.4) and 1.4 (95% CI, 1.2-1.6) for FEV(1)% < 60% and FEV(1)% of 60% to 80% compared with FEV(1)% > 80%, respectively; and among the self-report group, odds ratios were 5.3 (95% CI, 2.2-12.9) and 1.4 (95% CI, 1.2-1.7) for FEV(1)% < 60% and FEV(1)% of 60% to 80% compared with FEV(1)% > 80%, respectively. With the alternative classification scheme, the relationship was similar, but the difference in risk between categories of FEV(1)% decreased. CONCLUSION: The strong association between FEV(1)% and risk of asthma attack over the subsequent year supports an emphasis on objective measures of lung function in assessment of risk for adverse asthma outcomes.

Adolescent↗

Cost-effectiveness of inhaled corticosteroids in adults with mild-to-moderate asthma: results from the asthma policy model.

BACKGROUND: Inhaled corticosteroids remain underused among United States-based clinicians in treating mild-to-moderate adult asthma. OBJECTIVE: The purpose of this investigation was to estimate the clinical impact, health-related quality of life, cost, and cost-effectiveness of inhaled corticosteroid therapy in a population of patients aged 18 years and over with FEV(1) = 60% to 100% of predicted normal. METHODS: We performed a cost-effectiveness analysis of quick relievers (eg, short-acting beta-agonists) on an as-needed basis plus inhaled corticosteroid therapy versus quick relievers alone. A mathematical simulation model was developed to forecast symptoms, acute exacerbations, quality-adjusted life-years (QALYs), health care costs, and cost-effectiveness, measured in both dollars per QALY gained and dollars per symptom-free day gained. All evaluation outcomes were discounted at an annual rate of 3% and measured over a 10-year planning horizon. Data on the natural history of disease, drug efficacy, patient preferences, and economic costs were obtained from a variety of observational cohorts, randomized trials, and patient surveys. RESULTS: Over a 10-year period, use of inhaled corticosteroids increases total health costs from roughly $5,200 to $8,400 and improves QALYs from 6.8 to 7.0, implying an incremental cost of $13,500 per QALY gained. Costs per symptom-free day gained are $7.50. Both per-person acute exacerbations and hospitalizations are reduced by 33%. The cost-effectiveness findings are sensitive to the assumed efficacy and side-effects of inhaled corticosteroid therapy. CONCLUSIONS: Inhaled corticosteroids appear to deliver good comparative value in adults with mild-to-moderate asthma. Although more research is needed to understand their impact on preferences regarding side effects and compliance, these findings might be useful for priority-setting in limited resource situations.

Administration, Inhalation↗

Systematic overview of cost-utility assessments in oncology.

PURPOSE: Cost-utility analyses (CUAs) present the value of an intervention as the ratio of its incremental cost divided by its incremental survival benefit, with survival weighted by utilities to produce quality-adjusted life years (QALYs). We critically reviewed the CUA literature and its role in informing clinical oncology practice, research priorities, and policy. METHODS: The English-language literature was searched between 1975 and1997 for CUAs. Two readers abstracted from each article descriptions of the clinical situation and patients, the methods used, study perspective, the measures of effectiveness, costs included, discounting, and whether sensitivity analyses were performed. The readers then made subjective quality assessments. We also extracted utility values from the reviewed papers, along with information on how and from whom utilities were measured. RESULTS: Our search yielded 40 studies, which described 263 health states and presented 89 cost-utility ratios. Both the number and quality of studies increased over time. However, many studies are at variance with current standards. Only 20% of studies took a societal perspective, more than a third failed to discount both the costs and QALYs, and utilities were often simply estimates from the investigators or other physicians. CONCLUSION: The cost-utility literature in oncology is not large but is rapidly expanding. There remains much room for improvement in the methodological rigor with which utilities are measured. Considering quality-of-life effects by incorporating utilities into economic studies is particularly important in oncology, where many therapies obtain modest improvements in response or survival at the expense of nontrivial toxicity.

Clinical Trials as Topic↗

The quality of reporting in published cost-utility analyses, 1976-1997.

PURPOSE: Cost-utility analysis is a type of cost-effectiveness analysis in which health effects are measured in terms of quality-adjusted life-years (QALYs) gained. Such analyses have become popular for examining the health and economic consequences of health and medical interventions, and they have been recommended by leaders in the field. These recommendations emphasize the importance of good reporting practices. This study determined 1) the quality of reporting in published cost-utility analyses through 1997 and 2) whether reporting practices have improved over time. We examined quality of reporting by journal type and number of cost-utility analyses a journal has published. DATA SOURCES: Computerized databases were searched through 1997 for the Medical Subject Headings or text keywords quality-adjusted, QALY, and cost-utility analysis. Published bibliographies of the field were also searched. STUDY SELECTION: Original cost-utility analyses written in English were included. Cost-effectiveness analyses that measured health effects in units other than QALYs and review, editorial, or methodologic articles were excluded. DATA EXTRACTION: Each of the 228 articles found was audited independently by two trained readers who used a standard data collection form to determine the quality of reporting in several categories: disclosure of funding, framing, reporting of costs, reporting of preference weights, reporting of results, and discussion. RESULTS: The number of cost-utility analyses in the medical literature increased greatly between 1976 and 1997. Analyses covered a wide range of diseases and interventions. Most studies listed modeling assumptions (82%), described the comparator intervention (83%), reported sensitivity analysis (89%), and noted study limitations (84%). Only 52% clearly stated the study perspective; 34% did not disclose the funding source. Methods of reporting costs and preference weights varied widely. The quality of published analyses improved slightly over time and was higher in general clinical journals and in journals that published more of these analyses. CONCLUSIONS: The study results reveal an active and evolving field but also underscore the need for more consistency and clarity in reporting. Better peer review and independent, third-party audits may help in this regard. Future investigations should examine the quality of clinical and economic assumptions used in cost-utility analyses, in addition to whether analysts followed recommended protocols for performance and reporting.

Cost-Benefit Analysis↗

Cost-utility analyses of clinical preventive services: published ratios, 1976-1997.

BACKGROUND: Cost-effectiveness analyses of clinical preventive services are a potential means to aid public health resource allocation. Cost-utility analysis (CUA) is a specific form of cost-effectiveness analysis where results are expressed in terms of cost per quality-adjusted life year (QALY) gained. To increase the transparency and comparability of CUAs, standardization of methods has been recommended. OBJECTIVES: The purposes of this study were as follows: (1) identify published articles with original CUAs of primary and secondary clinical preventive services, (2) summarize the ratios found in these analyses, (3) identify articles employing comparable methods, and (4) explore analytic methods employed over time. METHODS: As part of a larger study we conducted a comprehensive search of published CUAs in the area of clinical preventive services and systematically collected data on the results of the analyses and analytic methods employed. Cost-effectiveness ratios were standardized and organized into a table. RESULTS: We found 50 CUAs pertaining to clinical preventive services (primary, n=22, 44%; and secondary, n=28, 56%) and 174 cost-effectiveness ratios. These ratios ranged from cost-savings up to $27,000,000/QALY, with a median of $14,000/QALY. Only three (6%) of the CUAs met minimum reference case requirements. There was no apparent improvement of methods over time. CONCLUSIONS: Immunizations and chemoprophylaxis have the most favorable cost-effectiveness ratios, and preventive services are more cost-effective when targeted at high-risk populations. However, there is wide variation in the methods used in these analyses. This study allows us to define where improvements in methodologic rigor need to occur, provides a base-line for future audits, and highlights disease areas in clinical preventive services that have been omitted or underevaluated.

Cost-Benefit Analysis↗

Measuring costs in cost-utility analyses. Variations in the literature.

OBJECTIVES: Although cost-utility analysis (CUA) has been recommended by some experts as the preferred technique for economic evaluation, there is controversy regarding what costs should be included and how they should be measured. The purpose of this study was to: a) identify the cost components that have been included in published CUAs; b) catalogue the sources of valuation used; c) examine the methods employed for estimating costs; and d) explore whether methods have changed over time. METHODS: We conducted a comprehensive search of the published literature and systematically collected data on the cost estimation of CUAs. We audited the cost estimates in 228 CUAs. RESULTS: In most studies (99%), analysts included some direct healthcare costs. However, the inclusion of direct non-healthcare and time costs (17%) was generally lacking, as was productivity costs (8%). Only 6% of studies considered future costs in added life-years. In general, we found little evidence of change in methods over time. The most frequently used source for valuation of healthcare services was published estimates (73%). Few studies obtained utilization data from RCTs (10%) or relied on other primary data (23%). About two-thirds of studies conducted sensitivity analyses on cost estimates. CONCLUSIONS: We found wide variations in the estimation of costs in published CUAs. The study underscores the need for more uniformity and transparency in the field, and continued vigilance over cost estimates in CUAs on the part of analysts, reviewers, and journal editors.

Cost-Benefit Analysis↗

The use of proxy respondents in studies of older adults: lessons, challenges, and opportunities.

OBJECTIVE: Proxies play a critical role as sources of health information for older persons with cognitive impairment and other chronic debilitating conditions. This paper reviews the validity of proxy responses for people older than age 60 in the following areas: functioning, physical and mental health, cognition, medical care utilization, and preferences for types of care and health states. DESIGN: A Medline review identified 24 clinical studies from 1990 to 1999 that use proxy data as a source of information about older adults. RESULTS: In general, studies report fairly good agreement between subjects and proxies in assessments of functioning, physical health, and cognitive status, and fair-to-poor agreement in assessments of psychological well-being. Proxies tend to describe more impairment in functioning and emotional well-being, relative to subjects, a pattern that is particularly marked among persons with cognitive impairment. In addition, proxies who report more caregiver responsibilities and subjective stress from caregiver duties provide more negative assessments of subjects' health and well-being. CONCLUSIONS: Findings tend to support the use of proxy ratings among older adults in many areas but not all when self-reports are not feasible. There is a need for more evaluation of proxy data in relation to other measures, such as performance assessments, medical records, and claims data, which may be less subject to respondent biases.

Activities of Daily Living↗

Preference-based measures in economic evaluation in health care.

Estimating preferences for states of health has been an active area of research in recent years. Unlike psychophysical approaches, which discriminate levels of health status, preference-based approaches incorporate values or utilities for health outcomes and can be used in cost-effectiveness analyses to aid resource allocation decisions. This chapter considers issues and controversies involved in using preference-based measures in economic evaluation in health care, with a particular emphasis on cost-utility analysis and the estimation of quality-adjusted life years. Topics considered include techniques for measuring preferences, the use of preference-based classification systems, the relationship between patient and community preferences, methods for obtaining utilities from clinical trials, mapping health status from health utilities, the development of "off-the-shelf" preference weights, and proposed alternatives to quality-adjusted life years. We also consider applications of cost-utility analyses to public health interventions. Although cost-utility analyses have become more popular recently, many challenges remain for the field. Widespread acceptance of the methodology likely awaits more consensus on measurement techniques, as well as educational efforts in the public health and medical communities on the usefulness of the approach.

Alzheimer Disease↗

Cost-effectiveness of functional imaging tests in the diagnosis of Alzheimer disease.

PURPOSE: To evaluate the cost-effectiveness of functional neuroimaging in the work-up of patients at specialized Alzheimer disease clinics. MATERIALS AND METHODS: A decision model was used to calculate costs and benefits (in quality-adjusted life-years [QALYs]) that accrued to hypothetical cohorts of patients at presentation to an Alzheimer disease center. Sensitivity analysis was performed to examine the effects of diagnostic test characteristics, therapeutic efficacy, disease severity, and costs on cost-effectiveness. RESULTS: The incremental cost-effectiveness ratio of dynamic susceptibility contrast material-enhanced magnetic resonance (MR) imaging was $479,500 per QALY (compared with the usual diagnostic work-up), while visual or quantitative single photon emission computed tomography (SPECT) was dominated (higher costs, lower effectiveness) by the usual diagnostic work-up. These results depend critically on the sensitivity and specificity of the standard diagnostic work-up, the effectiveness of drug treatment, and the disease severity. Varying these parameters resulted in estimates of incremental cost-effectiveness for dynamic susceptibility contrast-enhanced MR imaging of $24,680 to $8.6 million per QALY. SPECT either was dominated by the usual diagnostic work-up or had cost-effectiveness ratios of $180,200 to $6 million per QALY. CONCLUSION: The addition of functional neuroimaging to the usual diagnostic regimen at Alzheimer disease clinics is not cost-effective given the effectiveness of currently available therapies.

Alzheimer Disease↗

Who gets chemotherapy for metastatic lung cancer?

STUDY OBJECTIVES: To determine the prevalence and factors associated with chemotherapy use in elderly patients presenting with advanced lung cancer. DESIGN: A retrospective cohort study using administrative data. SETTING AND PATIENTS: We analyzed the medical bills for the 6,308 Medicare patients > 65 years old with diagnosed stage IV non-small cell lung cancer (NSCLC) in the 11 SEER (survival, epidemiology, and end results) regions between 1991 and 1993. The main outcome measure, chemotherapy administration, was identified by the relevant medical billing codes. Patient sociodemographic and disease characteristics were obtained from the SEER database and census data. RESULTS: Almost 22% of patients received chemotherapy at some time for their metastatic NSCLC. As expected, younger patients and those with fewer comorbid conditions were more likely to receive chemotherapy. However, several nonmedical factors, such as nonblack race, higher socioeconomic status, treatment in a teaching hospital, and living in the Seattle/Puget Sound or Los Angeles SEER regions, also significantly increased a patient's likelihood of receiving chemotherapy. CONCLUSION: Compared to previous reports, the prevalence of chemotherapy use for advanced NSCLC appears to be increasing. However, despite uniform health insurance coverage, there is wide variation in the utilization of palliative chemotherapy among Medicare patients, and nonmedical factors are strong predictors of whether a patient receives chemotherapy. While it is impossible to know the appropriate rate of usage, nonmedical factors should only influence a patient's likelihood of receiving treatment if they reflect patient treatment preference. Research to further clarify the costs, benefits, and patient preferences for chemotherapy in this patient population is warranted in order to minimize the effect of nonmedical biases on management decisions.

Aged↗

Barriers to using cost-effectiveness analysis in managed care decision making.

Managed care organizations would appear to be natural advocates for, and users of, cost-effectiveness analysis (CEA) as a tool for maximizing health outcomes for their covered populations within fixed budgets. There is, however, little evidence that CEA plays a major role in managed care decision making. The purpose of this paper is to identify barriers to both conducting and using CEA in managed care decision making. Lack of understanding about the value and applicability of CEA, and incentives that do not align with a lifetime perspective on either health outcomes or costs may be at least as important as perceived or real methodological limitations of the methodology. Research focused on ways to overcome these barriers, and thereby improve resource allocations, is recommended.

Cost-Benefit Analysis↗