Country-specific cost-effectiveness of early intervention with budesonide in mild asthma.

Early intervention with budesonide is an effective strategy for mild persistent asthma, which has been shown to provide additional clinical benefits at a low incremental cost using USA cost data. The present authors analysed whether this strategy would be cost-effective using cost data for other countries. Based on the 3-yr prospective, randomised, double-blind inhaled Steroid Treatment As Regular Therapy (START) in early asthma study (comparing budesonide and placebo combined with usual asthma therapy), the cost-effectiveness was estimated separately for eight different countries, from both healthcare payer and societal perspectives, of adding budesonide to usual asthma therapy. Local unit costs were applied to data for the total trial population. Incremental cost-effectiveness ratios (ICER) were estimated as cost per symptom-free day (SFD) gained. Budesonide increased SFDs by an average of 14.1 days annually. From a healthcare payer perspective, budesonide would reduce the total cost of asthma care in Australia. In Sweden, Canada, France, Spain, UK, China and the USA, the ICER ranged from US$2.4-11.3 per SFD. From a societal perspective, budesonide would be cost-saving in Australia, Canada and Sweden. In conclusion, for countries where costs with budesonide are higher, the policy implication has to be determined by that health system's willingness to pay for an additional symptom-free day. However, where budesonide therapy increases symptom-free days and reduces total costs, the policy conclusion clearly favours early intervention.

Design and analytic considerations in determining the cost-effectiveness of early intervention in asthma from a multinational clinical trial.

Demand for economic and outcomes data in support of drug formulary listing in private and government-sponsored health programs has led to fundamental changes in drug development. In part as a response to these pressures, the pharmaceutical industry has begun to include economic and quality-of-life endpoints in clinical trials with the hope of providing information to answer health policy questions on the economic value of its products. Here, the design and health economic techniques that will be used to analyze the START (inhaled Steroid Treatment As Regular Therapy in early asthma) study-a multinational (31 countries), randomized, placebo-controlled trial of 7240 patients with mild asthma over 3 years-will be presented. START compares the effect of once-daily administration of an inhaled glucocorticosteroid (Pulmicort Turbuhaler to conventional therapy in the management of newly diagnosed asthma, for which the use of this therapy is currently not the standard. The START study will examine both clinical effectiveness (measured as symptom-free days) and asthma-related costs for both treatment arms, aggregated for all patients across all countries. We believe that presenting the analytical plan prior to disseminating the results is an important way of increasing the credibility of economic evaluations. However, using clinical trials for collecting economic data poses several challenges, and the methods for conducting such evaluations are being developed. This paper will present and discuss several methodological options and the current state of the art for conducting economic evaluations alongside multinational clinical trials.

Health plans' use of asthma quality improvement projects to meet NCQA accreditation standards.

BACKGROUND: Managed care represents an important system of healthcare delivery in the United States and the primary source of care for many persons with asthma. OBJECTIVE: To characterize how US managed care health plans address the quality of asthma care through the use of disease-specific quality improvement (QI) programs complying with National Committee for Quality Assurance (NCQA) standards. METHODS: This study was a cross-sectional review of reports from all accreditation surveys conducted in 1996 and 1997 by the NCQA. Each accreditation report was reviewed for evidence of whether the health plan explored asthma care as a way to demonstrate compliance with NCQA accreditation standards. Asthma activity was examined with respect to health plan characteristics such as size of plan and Medicaid contracting. Types of asthma QI activity, use of guidelines, and application of different NCQA accreditation standards were also examined. RESULTS: Approximately 90% of 197 health plans undergoing NCQA accreditation surveys in 1996 and 1997 reported some form of asthma QI activity. There were no statistically significant differences in this activity in large vs small plans or in plans with vs without Medicaid members. Approximately two thirds of health plans used asthma QI activities to meet NCQA accreditation standards in health management systems, and three fifths monitored and evaluated important aspects of asthma care and service. CONCLUSIONS: During the study period, many US health plans conducted asthma care QI activities. The recently released NCQA asthma performance measure may provide the next assessment of how well managed care is contributing to improving asthma care in the United States.

Impact of inhaled antiinflammatory therapy on hospitalization and emergency department visits for children with asthma.

OBJECTIVE: Although the efficacy of inhaled antiinflammatory therapy in improving symptoms and lung function in childhood asthma has been shown in clinical trials, the effectiveness of these medications in real-world practice settings in reducing acute health care use has not been well-evaluated. This study examined the effect of inhaled antiinflammatory therapy on hospitalizations and emergency department (ED) visits by children for asthma. DESIGN: Defined population cohort study over 1 year. Setting. Three managed care organizations (MCOs) in Seattle, Boston, and Chicago participating in the Pediatric Asthma Care-Patient Outcome Research and Treatment II trial. Participants. All 11 195 children, between 3 to 15 years old, with a diagnosis of asthma who were enrolled in the 3 MCOs between July 1996 and June 1997. OUTCOME MEASURES: We identified children with 1 or more asthma diagnoses using automated encounter data. Medication dispensings were identified from automated pharmacy data. Multivariate logistic regression analysis was used to calculate effects of inhaled antiinflammatory therapy on the adjusted relative risk (RR) for hospitalization and ED visits for asthma. RESULTS: Over 12 months, 217 (1.9%) of children had an asthma hospitalization, and 757 (6.8%) had an ED visit. After adjustment for age, gender, MCO, and reliever dispensing, compared with children who did not receive controllers, the adjusted RRs for an ED visit were: children with any (>/=1) dispensing of cromolyn, 0.4 (95% confidence interval [CI]: 0.3, 0.5); any inhaled corticosteroid (ICS), 0.5 (95% CI: 0.4, 0.6); any cromolyn or ICS combined (any controller), 0.4 (95% CI: 0.3, 0.5). For hospitalization, the adjusted RR for cromolyn was 0.6 (95% CI: 0.4, 0.9), for ICS 0.4 (95% CI: 0.3, 0.7), and for any controller 0.4 (95% CI: 0.3, 0.6). A significant protective effect for both events was seen among children with 1 to 5 and with >5 antiinflammatory dispensings. When the analysis was stratified by frequency of reliever dispensing, there was a significant protective effect for controllers on ED visits for children with 1 to 5 and with >5 reliever dispensings and on the risk of hospitalization for children with >5 reliever dispensings. CONCLUSIONS: Inhaled antiinflammatory therapy is associated with a significant protective effect on the risk for hospitalization and ED visits in children with asthma. Cromolyn and ICSs were associated with similar effects on risks.asthma drug therapy, inhaled antiinflammatory agents, health maintenance organizations, hospitalization, emergency department.

Use of inhaled anti-inflammatory medication in children with asthma in managed care settings.

BACKGROUND: Many factors affect use of inhaled therapy in asthma. Relatively little is known about current patterns of use of anti-inflammatory medication in children with asthma and whether variations occur with age and use of bronchodilator medication. OBJECTIVE: To study the factors associated with dispensing of anti-inflammatory (controller) asthma medication to children in 3 managed care organizations (MCOs). METHODS: Using automated databases, a 1-year cross-sectional study of children with asthma aged 3 to 15 years cared for in 3 MCOs was used to evaluate the association of age and other factors with controller medication use. RESULTS: A total of 13 352 children were studied. Significantly fewer children aged 3 to 5 years were dispensed any (> or =1) controller medication than older children (P<.001). Among children dispensed 6 or more beta-agonists, only 39% also received 5 or more controller dispensings, with adolescents significantly less likely than younger children to receive 5 or more controllers (33%; P<.001). Significant differences were seen among MCOs in proportions of patients dispensed controller medication. In a multiple logistic regression model, controlling for frequency of beta-agonist dispensing and MCO, significantly lower dispensing of any controller medication was seen for those aged 3 to 5 years (odds ratio [OR], 0.8; 95% confidence interval [CI], 0.7-0.9) and for girls (OR, 0.9; 95% CI, 0.8-0.96). In contrast, for repeated (> or =5) controller dispensing there were significantly fewer dispensings to adolescents (OR, 0.7; 95% CI, 0.6-0.9) and girls (OR, 0.8; 95% CI, 0.7-0.9). CONCLUSIONS: There may be differences in the use of preventive asthma medication in children that are affected by age, sex, and health care organization. Few children with frequent symptoms are using controllers regularly, as is recommended by national guidelines.

A community-based study of near-fatal asthma.

BACKGROUND: The purpose of this study was to describe the community-based impact of near-fatal asthma within the District of Columbia (Washington, DC). METHODS: The design was a prospective cohort study. Subjects included all persons in 1993 who presented to Washington, DC hospitals alive, requiring intubation for respiratory failure (including subjects who subsequently died in the hospital). Washington, DC hospitals were contacted on a biweekly basis to identify subjects. Patients were contacted by mail, followed by an interview with the subject or proxy. RESULTS: Of the 35 case subjects identified, 31 (88.6%) were interviewed. Sixty-one percent of the subjects were female; 84% were African-American; and 45.2% were less than 18 years old. Forty-five percent had asthma for 10 or more years. Twenty-three percent reported the emergency department as their usual source of health care, and 32% saw a provider on a weekly basis. Fifty-two percent were taking four or more prescription medications, and 29% were taking no anti-inflammatory medications. In the 24 hours before the event, 77% reported difficulty breathing, but only 64% reported contacting a health care provider. CONCLUSIONS: Community-based investigation of near-fatal asthma may lead to a better characterization of risk factors associated with this event. Findings from this study suggest that some of the factors associated with near-fatal events may be different from those associated with fatal asthma and that up to one third of the events may have been preventable.

Health economics of asthma and rhinitis. II. Assessing the value of interventions.

Health care providers and payers are being asked to weigh data on the economic impact of new interventions along with clinical evidence when making decisions about the care of patients. The notion of incorporating formal health economic assessments into clinical and resource decisions is a difficult concept for many in the health care sector. However, it is the reality in today's environment. To effectively participate in these ongoing discussions, clinicians and other decision makers must be able to understand and critically assess the evidence on economic impact of medical interventions. This second of 2 articles describes the elements of comparative economic evaluations, reviewing the published literature on asthma and rhinitis in an attempt to critically appraise the studies from the perspective of one who might use data for decision making. Unfortunately, the quality of the economic evidence in these two disease states is not extensive. Until better economic analyses are conducted and made available, the allocation of resources for asthma and allergic rhinitis will continue to primarily rely on expert opinion rather than evidence-based literature.

The health economics of asthma and rhinitis. I. Assessing the economic impact.

As new health care strategies compete with existing ones for limited resources, the health care system and its providers are beginning to turn to health economic analyses to help inform choices in the delivery of care. This 2-part review examines the current health economic literature for asthma and rhinitis. This first installment of the review focuses on studies that characterize the economic burden of asthma and rhinitis and examines how resources are allocated to the care of persons with asthma and rhinitis. In 1998, asthma in the United States accounted for an estimated 12.7 billion dollars annually. Similarly, in 1994, allergic rhinitis was estimated to cost 1.2 billion dollars. Most of the costs for these conditions are attributed to direct medical expenditures, with medications emerging as the single largest cost component. Indirect costs also represent an important social effect. While cost-of-illness studies help to characterize the economic burden, comparative health economic studies evaluate the value of new and existing strategies for clinical care. The second part of this review will explore how comparative studies have contributed to understanding how to best diagnose and treat asthma and allergic rhinitis.

Asthma pharmacotherapy and utilization by children in 3 managed care organizations. The Pediatric Asthma Care Patient Outcomes Research Team.

BACKGROUND: Asthma is the most common chronic disease among children and the most frequent cause of hospitalization. Appropriate pharmacotherapy is a cornerstone of published national guidelines for the care of children with asthma. OBJECTIVE: The goal was to compare the baseline pharmacotherapy and health care utilization from 1996 to 1997 in children with asthma at managed care organizations (MCOs). METHODS: A common protocol was used to extract the study sample from 3 MCOs with automated claims and pharmacy databases. Children were selected if they were 3 to 15 years old as of June 1997 with 1 or more encounters (outpatient, emergency department visit, hospitalization) with an asthma diagnosis in the previous year. RESULTS: Of the 13,352 children studied, less than 40% were given controllers during the 12-month interval, with ranges of 15% to 77% by level of bronchodilator use, 31% to 44% by age, and 38% to 42% by MCO. Among children given 6 or more bronchodilators, controller dispensing ranged from 73% to 89% among the 3 MCOs. Variability was most evident for inhaled corticosteroids, for which dispensing ranged from 51% to 70%. Rates of asthma hospitalization and emergency department visits also differed among the MCOs, ranging from 21 to 37 per 1000 person-years and 37 to 142 per 1000 person-years, respectively. CONCLUSION: Five years after dissemination of national guidelines for care, the pattern of asthma therapy does not reflect guideline recommendations. Variation among health care organizations with respect to asthma therapy and utilization of health services exists. In addition, controller medications may not be used by all children who could benefit from them.

The relation of socioeconomic factors and racial/ethnic differences in US asthma mortality.

OBJECTIVES: This study described relations between socioeconomic factors and race/ethnicity as risk factors for asthma mortality. METHODS: A cross-sectional study was conducted of US mortality records from 1991 through 1996. RESULTS: Higher standardized mortality ratios were seen for Blacks vs Whites (3.34 vs 0.65), low vs high educational level (1.51 vs 0.69), and low vs high income (1.46 vs 0.71). Excess mortality for Blacks vs Whites was present in the highest and lowest quintiles of median county income and educational level. The disparity in asthma mortality rates according to median county income and education remained after control for race/ethnicity. CONCLUSIONS: Black race/ethnicity appears to be associated, independently from low income and low education, with an elevated risk for asthma mortality.

Self-reported physician practices for children with asthma: are national guidelines followed?

OBJECTIVE: To determine self-reported adherence to national asthma guidelines for children by primary care physicians in managed care; and, to analyze sources of variation in these practices by physician specialty and managed care practice type. DESIGN: A survey of 671 primary care physicians (pediatricians and family physicians) practicing in 3 geographically diverse managed care organizations (MCO). Domains of interest included asthma diagnosis, pharmacotherapy, patient education and follow-up, and indications for specialty referral. Item formats included self-reports of usual practice and responses to case vignettes. RESULTS: A total of 429 (64%) physicians returned surveys, 22 of whom did not meet criteria for inclusion in the analysis. Most respondents had both heard of (91%) and read (72%) the National Asthma Education and Prevention Program (NAEPP) guidelines. For diagnosis, 75% reported routine use of office peak flow measurement, but only 21% used spirometry routinely. Family physicians were more likely than pediatricians to use spirometry in diagnosis (odds ratio [OR] = 5.9), and less likely to recommend daily peak flow measurement (OR =.3). The median reported frequency of providing written care plans was only 50%. Though inhaled corticosteroids were deemed very safe or safe by 93%, almost half had specific concerns regarding at least 1 side effect, most commonly growth delay. Primary care physicians' criteria for referral to an asthma specialist differed from those of the NAEPP panel in choosing to manage more severe patients without asthma specialist input. Family physicians were more likely than pediatricians to refer a child after a single hospitalization, 2 to 3 emergency department visits, after 2 exacerbations, or if the child was <3 years old and required daily medications. Responses to vignettes showed generally appropriate initial use of antiinflammatory agents, but reluctance to increase the dose in response to continued symptoms, and less frequent follow-up than recommended by the NAEPP. CONCLUSION: Most physicians for children report having read and adopted NAEPP guideline recommendations for asthma treatment, including generally appropriate use of medications. Opportunities for improvement exist in specific areas such as the use of written care plans, optimizing antiinflammatory dosing, and providing routine follow-up. Although physicians show evidence of awareness of national guidelines and knowledge consistent with much of their content, additional work is required to promote the use of self-management tools in practice.

Use of the child health questionnaire in a sample of moderate and low-income inner-city children with asthma.

The Child Health Questionnaire (CHQ-PF50) is one of several recent efforts to gauge pediatric, health-related quality of life from the patient's (or parent's) perspective. Although tested extensively with healthy children, more information is needed about CHQ performance among children with chronic conditions such as asthma. The current study extends previous work by examining the CHQ's psychometric performance in a sample of children with asthma, overrepresenting those at high risk for poor outcomes. Seventy-four adult caregivers of children with asthma completed the CHQ. Internal consistency reliability was consistently high for all but one scale. Intraclass correlation coefficients ranged from a low of 0.37 to a high of 0.84. Tests of validity found CHQ scales better at distinguishing levels of disease severity as defined by symptom activity than medication use or pulmonary function tests. Performance of the CHQ-PF50 in a sample of low-income to moderate income inner-city parents of children with asthma presented mixed results. The instrument addresses a broad range of concepts but some scales may be more salient than others in assessing health status of children at highest risk for asthma morbidity. Future efforts must compare condition-specific and generic instruments to evaluate their relative strengths and weakness, as well as potential links between them.

Trends in the cost of illness for asthma in the United States, 1985-1994.

BACKGROUND: During the past decade, there have been notable changes in asthma prevalence, morbidity, and mortality. In this same time period, there have also been important national efforts to increase asthma awareness and improve asthma care. OBJECTIVE: The purpose of this study was to examine the changes in US cost of illness for asthma during the 10-year period from 1985-1994. METHODS: The study was a two-period (1985 and 1994), cross-sectional, cost-of-illness analysis. Cost estimates were based on US population and health care survey data available from the National Center for Health Statistics. RESULTS: The total US costs of asthma for 1994 were $10.7 billion. On the basis of 1985 estimates adjusted to 1994 dollars, total asthma costs increased by 54.1% and direct medical expenditures increased by 20.4% during the 10-year period. In 1985, hospital inpatient care represented the largest component cost of direct medical expenditures (44.6%). Hospital inpatient costs decreased to 29.5% of direct medical expenditures in 1994, primarily because of shorter lengths of stay, as opposed to a decrease in the total number of admissions. In 1994, medications represented the largest component cost of direct medical expenditures (40.1%, up from 30.0% in 1985). The largest component increase in indirect costs was due to loss of work. On the basis of adjusted dollars, estimated costs per affected person with asthma declined by 3.4% (decrease of 15.5% for children and an increase of 2.9% for persons 18 years and older) during this time period. CONCLUSION: Although the US costs of asthma increased during the 1985-1994 time period, estimated costs per person with asthma demonstrated a modest decline. These findings may represent a combination of reductions in hospital lengths of stay and increasing prevalence of persons with low consumption of asthma-related health care resources. In examining the component costs, it is unclear whether these changes can be attributed to the many local, regional, and national efforts aimed at controlling untoward asthma outcomes during the 1985-1994 time period.

Performance measurement through audit, feedback, and profiling as tools for improving clinical care.

Clinical audits and practice profiling have become popular tools in the attempt to change physician behavior to improve quality of care. Unfortunately, the growing need for information on quality of care has often outpaced the development of standard, valid, and reliable approaches to using these tools. The studies of performance measurement published in the literature to date demonstrate varying impact on ability to improve clinical care; few are randomized controlled trials. While performance measurement has become a common practice, the science surrounding this field is still in its early stages of development; while it seems promising, it should be viewed as largely experimental.

Reliability and validity of the Children's Health Survey for Asthma.

OBJECTIVE: Describe the psychometric properties of the Children's Health Survey for Asthma (CHSA)- a condition-specific, self-report, functional health measure for parents of children 5 to 12 years of age with chronic asthma. METHOD: Data from two cross-sectional and one longitudinal study were used to assess internal consistency reliability, test-retest reliability, and validity of the CHSA. Over 275 parents and guardians of children with asthma completed the CHSA in one of three studies. The combined samples included a heterogenous mix of respondents by child age and race/ethnicity and parental marital and socioeconomic status. Five domain scores were computed: physical health, activity (child), activity (family), emotional health (child), and emotional health (family). Raw scale scores were transformed from 0 to 100 with higher scores indicating better or more positive outcomes. RESULTS: Across the three samples, mean scale scores ranged from a low of 61.5 (emotional health of the child) to a high of 86.1 (activity [family]). Internal consistency reliability for each of the scales was high (Cronbach's alpha =.81-. 92), and test-retest reliability (correlation between forms) ranged from.62 to.86. Significant differences in mean scores for four of five scales were noted between those with low versus moderate to high recent symptom activity. CONCLUSION: In three tests, the CHSA displays strong reliability and validity. Descriptive statistics demonstrate a range of scale scores. Internal consistency is good to excellent and short-term test-retest reliability is good for each of the five scales. Construct validity is demonstrated by the ability of CHSA to distinguish levels of disease severity, defined by symptom activity.

The economic burden of asthma in US children: estimates from the National Medical Expenditure Survey.

BACKGROUND: Asthma is the leading chronic illness of childhood, is responsible for substantial pediatric morbidity, and has a significant impact on use of health resources. OBJECTIVE: Our purpose was to assess the per capita impact of pediatric asthma on medical care utilization and total expenditures. METHODS: A population-based national probability survey, the National Medical Expenditure Survey, was conducted in 1987 to determine the use and cost of health care services in the United States. We analyzed the responses for all children aged 1 to 17 years with (n = 667) and without (n = 6911) asthma. Children with asthma were identified with use of a population-based screening question. Frequency and cost of medications, ambulatory visits, emergency department care, and hospitalizations for all reasons, including asthma, were assessed. RESULTS: The period prevalence of childhood asthma in 1987 was 8.8% and the treated prevalence (any asthma medications) was 4.0%. Forty-one percent of families with asthmatic children were classified as having no primary insurance. Children with asthma used substantially more services in all categories of care: 3.1 times as many prescriptions, 1.9 times as many ambulatory provider visits, 2. 2 times as many emergency department visits, and 3.5 times as many hospitalizations. Only 10.7% of children with asthma were defined as chronic users of medications. Children with asthma incurred an average of $1129 (SD $5310) per child per year in total health care expenditures compared with $468 (SD $2960) for children without asthma, a 2.8-fold difference. CONCLUSION: Asthma has considerable impact on the use and costs of medical care services among US children. Data from the 1987 National Medical Expenditure Survey provide a useful baseline against which more recent, postguideline data should be compared.

The Chicago Asthma Surveillance Initiative: a community-based approach to understanding asthma care.

Nearly all of the asthma surveillance literature focuses on national-, regional-, or state-based estimates of prevalence, health-care utilization (specifically hospitalizations, emergency department, and ambulatory care visits), and mortality. Although these are important events, they reveal little about asthma's impact at the community level and provide little information that could be used to design specific interventions for improving clinical outcomes. A useful representation of asthma care across a community could guide an effective community response to the burden of asthma. The goal of the Chicago Asthma Surveillance Initiative (CASI) is to develop a community-wide surveillance program that characterizes and monitors asthma care in the Chicago area, beyond existing public health surveillance. To accomplish this, CASI surveyed Chicago-area hospitals, emergency departments, primary care physicians, specialty care physicians, pharmacists, managed care organizations, the general public, and persons or families affected by asthma to learn about asthma care and its outcomes. A variety of techniques (including brochures, slide kits, and the Internet) were used to achieve rapid public dissemination of study findings. The value of this comprehensive community-based data surveillance effort will rest on how the community uses this information to stimulate new efforts to improve asthma care and reduce untoward outcomes.