GENERAL ESSAYS
HEALTH CARE RESOURCE USE AND STROKE OUTCOME: Multinational Comparisons within the GAIN International trial
- Kjell Asplund, Sharron Ashburner, Kathy Cargill, Margaret Hux, Ken Lees, Michael Drummond for the GAIN International Investigators
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 267-277
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Background and Purpose: Outcome in patients hospitalized for acute stroke varies considerably between populations. Within the framework of the GAIN International trial, a large multicenter trial of a neuroprotective agent (gavestinel, glycine antagonist), stroke outcome in relation to health care resource use has been compared in a large number of countries, allowing for differences in case mix.
Methods: This substudy includes 1,422 patients in 19 countries grouped into 10 regions. Data on prognostic variables on admission to hospital, resource use, and outcome were analyzed by regression models.
Results: All results were adjusted for differences in prognostic factors on admission (NIH Stroke Scale, age, comorbidity). There were threefold variations in the average number of days in hospital/institutional care (from 20 to 60 days). The proportion of patients who met with professional rehabilitation staff also varied greatly. Three-month case fatality ranged from 11% to 28%, and mean Barthel ADL score at three months varied between 64 and 73. There was no relationship between health care resource use and outcome in terms of survival and ADL function at three months. The proportion of patients living at home at three months did not show any relationship to ADL function across countries.
Conclusions: There are wide variations in health care resource use between countries, unexplained by differences in case mix. Across countries, there is no obvious relationship between resource use and clinical outcome after stroke. Differences in health care traditions (treatment pathways) and social context seem to be major determinants of resource use. In making comparisons between countries, great care should be exercised in using outcome variables as indicators of quality of stroke care.
LIFETIME COSTS FOR MEDICAL SERVICES: A METHODOLOGICAL REVIEW
- Philip Jacobs, Kamran Golmohammadi, Teresa Longobardi
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 278-286
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Objectives: Guidelines for economic evaluation studies recommend that modeling be undertaken to estimate long-term, downstream costs. In this study, we conduct a review of a sample of studies that estimated the lifetime medical care costs for a variety of conditions.
Methods: We developed a categorization of the elements for a lifetime-costing study and based on these elements, we abstracted information from a sample of 33 papers in the following areas: study subject, purpose, scope, methods (including time profile, utilization, and cost), and results.
Results: We analyzed papers that were observational, models or that combined the two approaches. The time profiles were estimated from registry and published data. Utilization data were obtained from administrative data, chart reviews, and professional opinion. Costs were obtained from administrative and financial records and were estimated using all charges, allocated costs, and provider payments. We noted wide variations in methods and reporting practices.
Conclusions: Following current guidelines (CCOHTA), lifetime models can be more easily interpreted and applied if investigators are more clear in their study aims, if they incorporate assumptions that are based on current data, if they follow current methodological practices (such as deflation, discounting, and sensitivity analyses), and if reporting is more transparent.
IMPACT OF GENE PATENTS ON THE COST-EFFECTIVE DELIVERY OF CARE: THE CASE OF BRCA1 GENETIC TESTING
- Christine Sevilla, Claire Julian-Reynier, François Eisinger, Dominique Stoppa-Lyonnet, Brigitte Bressac-de Paillerets, Hagay Sobol, Jean-Paul Moatti
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 287-300
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Background: In 1994/95, two genes, BRCA1/2, associated with a predisposition to breast or ovarian cancer were identified. Genetic testing of deleterious BRCA1/2 mutations consequently can be proposed to individuals with a family history of breast or ovarian cancer to identify who is at risk. The granting of U.S. patents on BRCA1/2 to a privately owned company has led to the monopoly use of a unique technique (Direct Sequencing of the gene, DS) for BRCA1/2 testing in this country. Alternative strategies using prescreening techniques, however, have been experienced worldwide.
Methods: On the basis of data collected at three laboratories of French public hospitals, we carried out a cost-effectiveness study comparing DS to 19 alternative strategies with the number of deleterious BRCA1 mutations detected as the outcome.
Results: Results show that the DS strategy presents the highest average cost per mutation detected (9,882.5 °) and that there exist strategies using prescreening techniques that can reach similar effectiveness while reducing total costs. Moreover, other strategies can obtain a four- to sevenfold reduction in the average cost per mutation detected as soon as some rates of false negatives (2% to 13%) are deemed to be acceptable.
Conclusions: Results suggest that gene patents with a very broad scope, covering all potential medical applications, may prevent health care systems from identifying and adopting the most efficient genetic testing strategies due to the monopoly granted for the exploitation of the gene. Policy implications for regulatory authorities, in the current context of the extension of BRCA1/2 patents in other countries, are discussed.
CONFRONTING THE “GRAY ZONES” OF TECHNOLOGY ASSESSMENT: EVALUATING GENETIC TESTING SERVICES FOR PUBLIC INSURANCE COVERAGE IN CANADA
- Mita Giacomini, Fiona Miller, George Browman
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- 18 June 2003, pp. 301-316
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We describe an evaluation model to guide public coverage of new predictive genetic tests in Ontario, Canada. The model confronts common “gray zones” in evaluation and coverage policy for challenging new technologies. Analysis addresses three domains of the evaluation picture. The first specifies evaluative criteria (purpose, effectiveness, additional effects, unit cost, demand, cost-effectiveness). The second induces or deduces acceptable cutoffs for each criterion. The third domain addresses the need to make decisions under uncertainty and to respond to “gray” evaluations with conditional-coverage decisions. The evaluation criteria should be applied within sound decision-making processes.
HEALTH TECHNOLOGY ASSESSMENT: THE CONTRIBUTION OF QUALITATIVE RESEARCH
- Mark Leys
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 317-329
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The pragmatism in health care has made health technology assessment (HTA) restrict its scope to a particular set of problems, c.q. methods. The “multidisciplinary and comprehensive nature” of HTA, as the concept is presented in certain definitions, is lacking. Health care is also dominated by a positivistic-rationalistic approach of evaluation. In contrast, social studies of evaluations learn that a major difference has to be made between scientific research on (potential) impact of a technology and valuing these effects. In this contribution, we will discuss how the positivistic scientific bias of current HTA practice can be made up with other research traditions. More specifically, we focus on the question of how social scientists and particularly how qualitative research can contribute to HTA, complementary to positivistic studies of evidence and efficacy.
DAILY COST PREDICTION MODEL IN NEONATAL INTENSIVE CARE
- John A. F. Zupancic, Douglas K. Richardson, Bernie J. O'Brien, Barbara Schmidt, Milton C. Weinstein
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 330-338
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Objectives: One barrier to economic evaluation alongside neonatal randomized controlled trials is the expense of collecting detailed patient resource information. To reduce this data collection burden, we identified the key resource items that predict daily ancillary costs for extremely low birth weight infants.
Methods: Participants were 385 infants enrolled in the Trial of Indomethacin Prophylaxis for Preterms in nine tertiary level neonatal intensive care units in Canada. Information on eighty-nine nonpersonnel resource items was abstracted from the hospital chart from admission to tertiary hospital discharge. Unit costs were derived from a provincially standardized cost accounting system. Using stepwise linear regression, models correlating total daily ancillary costs with key resource items were constructed for each of five periods of admission. Models were derived in a randomly split half of the total sample of patient days and validated against the remainder.
Results: The 385 infants contributed resource information from 23,354 admission days. The regression model for weeks one to twelve included the covariates surfactant, chest radiograph, red blood cell transfusion, cranial ultrasound, abdominal radiograph, parenteral amino acid infusion, surgery, platelet transfusion, and echocardiogram and explained 91% of the variability in daily nonpersonnel costs (P<.0001). Models for other admission periods similarly included between four and eight covariates, were highly significant (P<.0001) and explained between 76% and 94% of daily ancillary cost variability. The regression equations showed excellent predictive power when applied to the second half of the patient data set.
Conclusions: Daily nonpersonnel costs for extremely low birth weight infants are driven by a limited number of key resource variables. The ability to predict total ancillary costs with minimal data collection will facilitate inclusion of economic evaluations in neonatal trials.
HYPERBARIC OXYGEN TECHNOLOGY: AN OVERVIEW OF ITS APPLICATIONS, EFFICACY, AND COST-EFFECTIVENESS
- Shien Guo, Michael A. Counte, James C. Romeis
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 339-346
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Objectives: To examine the growing evidence and the consensus in the medical community concerning the efficacy and cost-effectiveness of hyperbaric oxygen therapy (HBO2T) and to suggest future research areas to ensure the appropriate use of this technology.
Methods: A literature search of articles published between 1985 and 2000 was conducted using PubMed to describe the growth of HBO2T-related articles published over the past fifteen years. In addition, articles involving the qualitative synthesis of the efficacy and cost-effectiveness of HBO2T in thirteen major application areas were identified and compared with the changing view of the medical community toward the evidence of HBO2T.
Results: The total number of HBO2T-related articles published annually has steadily increased over the past fifteen years. This increase has gradually contributed to a consensus in the medical community concerning the evidence of efficacy in its major application areas. However, information regarding the cost-effectiveness of HBO2T is still extremely limited.
Conclusions: Although evidence concerning the efficacy of HBO2T has been growing in the past, more evidence is still needed for some of its major application areas. Moreover, there is an urgent need to shift part of the research focus on HBO2T to its cost-effectiveness to provide decision makers with relevant information to evaluate this technology objectively.
EFFECT OF INCLUDING (VERSUS EXCLUDING) FATES WORSE THAN DEATH ON UTILITY MEASUREMENT
- Duska M. Franic, Dev S. Pathak
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 347-361
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Objectives: Most studies typically measure health preferences excluding health states perceived as worse than death. The objective of this study is to test the impact of including (versus excluding) health states perceived to be worse than death on utility measurementusing standard gamble (SG) and visual analogue scale (VAS) methods.
Methods: By means of a cross-sectional descriptive study design, women were asked to rate the utility of three hypothetical breast cancer health states: cure, treatment, and recurrence (n=119). Preference weights were estimated, allowing for negative utilities with death (perfect health) scaled at zero (1.0).
Results: Unpaired t-test analysis showed significantly greater change in SG and VAS weights for individuals perceiving cancer recurrence as worse than death than those perceiving death as least desirable state. Excluding negative utilities from the study resulted in significantly smaller changes in utility. Study results show that preference elicitation methods can be successfully adapted to acquire negative utilities.
Conclusions: Changes in utility were greater when negative preferences were permitted. Addressing negative preference scores could significantly affect quality adjusted life year estimates in economic analyses.
COMPARING COMMUNITY-PREFERENCE–BASED AND DIRECT STANDARD GAMBLE UTILITY SCORES: EVIDENCE FROM ELECTIVE TOTAL HIP ARTHROPLASTY
- David Feeny, Christopher Blanchard, Jeffrey L. Mahon, Robert Bourne, Cecil Rorabeck, Larry Stitt, Susan Webster-Bogaert
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 362-372
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Objectives: Do utility scores based on patient preferences and scores based on community preferences agree? The purpose is to assess agreement between directly measured standard gamble (SG) utility scores and utility scores from the Health Utilities Index Mark 2 (HUI2) and Mark 3 (HUI3) systems.
Methods: Patients were assessed repeatedly throughout the process of waiting to see a surgeon, waiting for surgery, and recovery after total hip arthroplasty (THA). Group mean scores are compared using paired t-tests. Agreement is assessed using the intraclass correlation coefficient (ICC).
Results: The mean SG, HUI2, and HUI3 (SD) scores at assessment 1 are 0.62 (0.31), 0.62 (0.19), and 0.52 (0.21); n=103. At assessment 2, the means are 0.67 (0.30), 0.68 (0.30), and 0.58 (0.22); n=84. There are no statistically significant differences between group mean SG and HUI2 scores. Mean SG and HUI3 scores are significantly different. ICCs are low.
Conclusions: At the mean level for the group, SG and HUI2 scores match closely. At the individual level, agreement is poor. HUI2 scores were greater than HUI3 scores. HUI2 and HUI3 are appropriate for group level analyses relying on community preferences but are not a good substitute for directly measured utility scores at the individual leve.
ASSESSMENT OF DIAGNOSTIC TESTS TO INFORM POLICY DECISIONS-VISUAL ELECTRODIAGNOSIS
- Renea V. Johnston, Elizabeth Burrows, Alexandra Raulli
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 373-383
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Objectives: To conduct a systematic review of the evidence for the effectiveness of five visual electrodiagnostic tests to inform the Medical Services Advisory Committee (MSAC) of the Department of Health and Ageing (Australia) in its decision in allocating public funding for new technologies.
Methods: We searched the biomedical literature to identify English-language articles published from 1966 to September 2000. We assessed validity of methodology of included studies against the following criteria: investigators (i) compared test with an appropriate reference test; (ii) tested an appropriate spectrum of patients; (iii) masked assessment of study and reference tests; (iv) measured the study test independently of clinical information; and (v) measured the reference test before any interventions.
Results: Sixty-one articles met inclusion criteria for critical appraisal: nineteen were cross-sectional studies that compared a study test with another test, thirty-four were case-control studies that compared a test in a group of patients with an eye disease to a group of subjects without eye disease, and eight studies were case series. None of the included studies met all of the validity criteria. Only four studies provided enough information to calculate diagnostic characteristics but were flawed due to inclusion of patients already diagnosed with disease or lack of an appropriate reference test and, thus, overestimated test accuracy.
Conclusions: Identified studies did not provide sufficient valid evidence of the clinical value of the five visual tests in diagnosing diseases of the retina or optic nerve. Thus, MSAC recommended that the tests not be supported by public funding.
COSTS OF FLEXIBLE SIGMOIDOSCOPY SCREENING FOR COLORECTAL CANCER IN THE UNITED KINGDOM
- David K. Whynes, Emma J. Frew, Robert Edwards, Wendy S. Atkin
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 384-395
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Objectives: Colorectal cancer is one of the most commonly occurring cancers in industrialized countries, yet appears to be amenable to screening. Amongst the many possible protocols is once-only screening by means of flexible sigmoidoscopy. This protocol is currently being investigated in a UK multicenter trial and the study provides estimates of the expected resource costs.
Methods: The direct health care costs of sigmoidoscopy and of all subsequent procedures were estimated from an audit of resource use of approximately 40,000 patients at thirteen centers. Patient-borne costs were estimated from the results of surveys conducted at twelve of these centers.
Results: The health service costs of a flexible sigmoidoscopy was estimated at £56. The total costs of screening (including private costs) averaged £82 per person screened, although costs varied by center. The total health service costs of screening and subsequent management averaged approximately £91 per person screened, again with variations between centers.
Conclusions: Even within a strict trial protocol, intercenter variation in costs can be detected, ascribable to variability in local management practices, local yield, and local patient-borne costs. Other recent estimates of flexible sigmoidoscopy costs vary widely. As these costs form the basis of technology assessment simulation models which, in turn, inform policy, obtaining realistic cost estimates within the appropriate health care setting is of paramount importance.
COMPARISON OF HOSPITAL COSTING METHODS IN AN ECONOMIC EVALUATION OF A MULTINATIONAL CLINICAL TRIAL
- Shelby D. Reed, Joëlle Y. Friedman, Ari Gnanasakthy, Kevin A. Schulman
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 396-406
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Objectives: To develop and evaluate strategies for estimating hospitalization costs in multinational clinical trials.
Methods: Hospital cost estimates for eleven diagnoses were collected from twelve countries participating in a trial of therapies for congestive heart failure. Estimates were combined with U.S.-based diagnosis-related group weights to compute country-specific unit cost estimates for all reasons for hospitalization. Variations of hospital costing methods were developed. The unit cost method assigns a country-specific unit cost estimate to each hospitalization. The other methods adjust for length of stay using a daily cost (DC) estimate for each diagnosis, based on either the mean length of stay (DC-mean method) or the median length of stay (DC-median method) for each diagnosis in each country. Additional modifications were explored through adjustment of the distribution of daily costs incurred during a hospital stay.
Results: The mean cost for all hospitalizations was $10,242 (SD, 10,042) using the unit cost method, $10,242 (SD, 12,760) using the standard DC-mean method, and $13,967 (SD, 18,762) using the standard DC-median method. In comparisons of costs for all 5,486 hospitalizations incurred by a subset of 2,352 patients in the trial, the unit cost method provided 92% power to detect a $1,000 cost difference. The standard DC-mean method provided 76% power, and the standard DC-median method provided 44% power.
Conclusions: Hospital costing methods that adjust for differences in length of stay require a significantly larger sample to attain comparable statistical power as methods that assign unadjusted unit cost estimates to hospitalization events.
COST-EFFECTIVENESS ANALYSIS: CAN WE REDUCE VARIABILITY IN COSTING METHODS?
- Taghreed Adam, David B. Evans, Marc A. Koopmanschap
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 407-420
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Objectives: The need for consistency and standardization of methods for economic appraisals has been recognized for some time and has led to the development of several sets of guidelines for economic evaluations and for costs. Despite this, considerable diversity is still apparent in applied studies. Some of these diversities might be defensible, and some might not. The objectives of this study are to explore sources of variations in the methods used in applied studies and to discuss the nature of these variations and the possibility of reducing some of them.
Methods: We first use a systematic approach to identify the major sources of variation in costing methods used in applied economic evaluations. We then compare the methods used with the recommendations made in available guidelines.
Results: Four possible sources of variation are identified. The first is where guidelines do not agree in their recommendations; therefore, it is not surprising that applied studies use different methods. The second is where guidelines agree in principle but provide little detail on how to comply with their recommendations; and the third is where a particular methodological issue is not discussed in guidelines. The fourth reason is simply lack of compliance with accepted guidelines.
Conclusions: Variability in costing methods used in applied studies raises questions about the validity of their results and makes it difficult to compare the results of different studies. We discuss the implications for the transferability and generalizability of results and suggest ways to minimize the variability in the methods so that the results of costing studies and economic evaluations can be of more value to policy-makers.
EVIDENCE OF IMPROVING COST-EFFECTIVENESS OF PEDIATRIC COCHLEAR IMPLANTATION
- T. Sach, C. O'Neill, D.K. Whynes, S.M. Archbold, G.M. O'Donoghue
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- Published online by Cambridge University Press:
- 18 June 2003, pp. 421-431
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Objectives: To examine the cost-effectiveness of pediatric cochlear implantation over time.
Methods: A prospective study based on ninety-eight children implanted between 1989 and 1996 at Nottingham's Paediatric Cochlear Implantation Programme, UK. The influence of outcomes and other variables on total costs was examined using multivariate regression analysis.
Results: Having controlled for potential confounding variables, total cost was negatively related to year of implant and positively related to the number of hours of rehabilitation (p=.000).
Conclusions: Having controlled for outcomes (Categories of Auditory Performance and Speech Intelligibility Rating), the cost-effectiveness improved over time. This finding may be due to a learning curve and have policy implications.