Cost-effectiveness Of New Treatments Research Articles

Verteporfin Photodynamic Therapy Cohort Study: Report 3: Cost Effectiveness and Lessons for Future Evaluations

To report (1) the costs of verteporfin photodynamic therapy (VPDT) in routine treatment of neovascular age-related macular degeneration (nAMD), (2) the relationship between health and social service costs and best-corrected visual acuity (BCVA), (3) the cost-effectiveness of VPDT versus a best supportive care (BSC) group who were assumed to have no active treatment, and (4) lessons for future cost-effectiveness analyses (CEAs). The CEA of VPDT versus BSC that uses health-related quality of life (HrQoL), resource use, and visual acuity data from the United Kingdom (UK) VPDT Cohort Study. Data on VPDT use were collected from patients attending 45 ophthalmology provider units in the UK National Health Service, 15 units collected data on self-reported use of services. Incremental costs of VPDT versus BSC were calculated from treatment costs, change in cost associated with declining BCVA, and difference in BCVA previously attributed to VPDT. Similarly, incremental quality-adjusted life years (QALYs) were calculated from change in HRQoL associated with declining BCVA, giving an incremental cost per QALY of VPDT versus BSC over 2 years. Incremental costs (UK pounds [ pound]; United States dollars [$]); incremental QALYs; costs per QALY. The treatment costs of VDPT were pound 3026 ($4544) in year 1 and pound 845 ($1269) in year 2. For patients who used services, a 5-letter decrease in BCVA was associated with an increase in annual costs of approximately pound 110 ($165; 95% confidence intervals, approximately pound 48 [$72] to pound 174 [$261]). The incremental costs and QALYs for VPDT were pound 3514 ($5276) and 0.021, respectively, giving incremental costs per QALY gained of pound 170000 ($255000). Verteporfin photodynamic therapy is unlikely to be cost effective for patients with nAMD. This article provides realistic estimates of VPDT costs and the costs associated with declining vision. Future studies can follow this approach to assess accurately the cost effectiveness of new interventions for nAMD.

PIH43 EARLY ASSESSMENT OF A NEW TREATMENT FOR ADOLESCENT IDIOPATHIC SCOLIOSIS

OBJECTIVES: Adolescent idiopathic scoliosis (AIS) reduces the quality of life. It can lead to back pain, reduced back function and socio-psychological problems. Left untreated it may induce cardio-vascular and pulmonary problems, and even death. Current treatment of severe forms of idiopathic scoliosis are based on fusion of the vertebrae. These treatments restrict back function and growth of the adolescents. This study aims to conduct an early technology assessment of a new non-fusion scoliosis correction system. METHODS: The early assessment of this new treatment is supported by the Analytic Hierarchy Process (AHP), a technique for multi-criteria decision analysis. The AHP quantifies the comparison between the performances of decision alternatives under a finite set of decision criteria. An expert panel composed of six technological engineers and medical specialists compared the expected performance of the new non-fusion treatment to the performance of posterior fusion surgery for patients with severe AIS. They regarded criteria related to the quality of life, complications, user friendliness and costs. RESULTS: The expert panel considered the influence of the treatment on the quality of life to be most important (weight 0.46) followed by complications (0.35), user friendliness (0.11) and costs (0.09). They expected the new non-fusion treatment to particularly have a more positive influence on the quality of life (priority 0.73) than posterior surgery has (priority 0.27). Main disadvantage was considered to be the relative high costs of the new treatment (priority 0.37 versus 0.63). In overall, the new treatment is expected to slightly outperform the current treatment (priority 0.63 versus 0.37). CONCLUSIONS: In the absence of clinical evidence, this method provides a valuable means to systematically predict a new technology’s clinical value. The outcomes provide clear directions for medical industry to improve the future cost-effectiveness of new treatments.

The future of breast cancer research and practice in Asia, Latin America, and the Middle East/North Africa: a qualitative horizon scanning analysis.

Abstract Abstract #5095 Objective: To conduct a horizon-scanning analysis to identify future needs, challenges and trends relating to breast cancer research and practices globally with special emphasis on emerging and understudied regions.
 Methods: Following the design and pilot of the survey instrument, data was derived from key informant interviews with 221 thought leaders in breast cancer in Asia (n=97), Latin America (n=46), Middle East/North Africa (n=39) and Australia and Canada (n=39). Thought leaders were identified using a combination of purposive and snowballing sampling and included oncologists, surgeons, other breast cancer specialists, advocacy leaders and policy makers. Transcripts and field notes were then coded and compared, and a taxonomy of 20 issues was developed. The propensity of these themes were then analyzed and compared across the four regions.
 Results: In Asia the most prevalent issues were i) building capacity for clinical research, ii) more nurses for patient care/education, iii) keeping up to date with new information and iv) increased targeted/personalized treatment. In Latin America key themes were the need to i) increase targeted/personalized treatment, ii) address disparities among the underserved, iii) high cost to third party payers, iv) increase capacity for clinical research and v) control out of pocket costs for patients. In the Middle East/North Africa key themes were the need for i) building capacity for clinical research, ii) more nurses for patient care/education, iii) increased public education on screening and iv) increased capacity for early detection. In Australia and Canada issues were i) keeping up to date with new information, ii) weighing the cost effectiveness of new treatments, iii) the need for increased data sharing and iv) improving communication between stakeholders.
 Discussion: While a number of respondents expressed observations relating to country specific etiology of disease (such as women presenting at younger ages and with more aggressive tumors) the lack of national registries and local clinical/genetic research make it hard to confirm these anecdotes scientifically. Finally, increased focus on the development of effective advocacy and policy leadership are needed in these regions to draw attention and resources towards issues of community empowerment, survivorship and quality of life which were neglected by all but a handful of respondents. Citation Information: Cancer Res 2009;69(2 Suppl):Abstract nr 5095.

Estimating a Preference-Based Single Index from the Overactive Bladder Questionnaire

Objectives The aim of the study is to estimate a preference-based single index for calculating quality-adjusted life years for patients with overactive bladder (OAB), based on a survey of the UK general population using the 5-dimensional health classification system OAB-5D, derived from the validated Overactive Bladder Questionnaire (OAB-q). Methods An interview valuation survey of members of public in the South Yorkshire, UK was undertaken using the time-trade-off method. Each respondent was randomly allocated to one of 14 blocks, and valued seven states each plus the “pits” state, so that a total of 99 states were valued. A number of multivariate regression models were estimated for predicting a total of 3125 health state values defined by the classification. Models were compared and selected using a set of criteria, including overall diagnosis by adjusted R-squared, the sign and significance of individual parameter estimates, the relative size of coefficients within a given dimension and predictive ability. Results The mean model was recommended for use in economic evaluation. Conclusion This will permit the cost-effectiveness of new interventions to be assessed in patients with OAB using existing and future OAB-q data sets.

Can the Spanish care system assume the new costs of medications against cancer?

Cancer is a high incidence disease, forcing healthcare systems to assign a significant amount of resources to its treatment. New developments have arisen recently: development of new agents that act at specific steps of cellular differentiation and proliferation and identification of predictive genetic markers which allow sub-groups of patients that will benefit from these agents, alone or in combination with chemotherapy, to be targeted. The majority of new drugs coming to the market combine greater clinical benefit and higher costs. Constraints on healthcare budgets worldwide make it necessary to rationalise the expense by prioritising allocation of available resources to the most efficient interventions, so that the best possible clinical result can be obtained at a reasonable cost and with the best quality of life for the patient. Economic evaluation studies represent the only tool available to scientifically determine the cost-effectiveness of new treatments and the budgetary impact of their introduction to the therapeutic arsenal available for the treatment of cancer.

Measuring the burden of injury

The political response to any given health problem is strongly influenced by two particular metrics: the scale of the problem and the effectiveness of acceptable counter measures. Injury prevention advocates...

Health care costs for the treatment of breast cancer recurrent events: estimates from a UK-based patient-level analysis

Cost pressures and the need to demonstrate cost-effectiveness of new interventions require consideration of the costs of treating disease. This study presents analyses of resource use data covering 199 postmenopausal women who experienced a breast cancer recurrent event between 1991 and 2004 and were treated at the Western General Hospital, Edinburgh. Aggregate (5-year) treatment costs for alternative recurrent events were estimated, as well as the annual costs incurred by patients experiencing contralateral, locoregional, or distant recurrence, who remained alive without further recurrence for a year. The 95% confidence intervals for the 5-year costs of recurrence ranged from £10 000 to £37 000 for locoregional recurrence, and £14 500–£20 000 for distant recurrence. No evidence of significant variations in these costs across time periods between 1991 and 2004 was identified. Annual costs for patients remaining in the same health state showed high initial costs for contralateral and locoregional recurrence, with low costs in subsequent years, while costs associated with distant recurrence declined at a slower rate and plateaued at 4–5 years post-diagnosis. The cost estimates presented in this paper not only inform the magnitude of the resource consequences of breast cancer recurrences, but they are also better suited to informing cost-effectiveness analyses, which have a far greater role in allocating health-care resources.

Model to assess the cost-effectiveness of new treatments for depression

The objective of this study was to develop a model to assess the cost-effectiveness of a new treatment for patients with depression. A Markov simulation model was constructed to evaluate standard care for depression as performed in clinical practice compared with a new treatment for depression. Costs and effects were estimated for time horizons of 6 months to 5 years. A naturalistic longitudinal observational study provided data on costs, quality of life, and transition probabilities. Data on long-term consequences of depression and mortality risks were collected from the literature. Cost-effectiveness was quantified as quality-adjusted life-years (QALYs) gained from the new treatment compared with standard care, and the societal perspective was taken. Probabilistic analyses were conducted to present the uncertainty in the results, and sensitivity analyses were conducted on key parameters used in the model. Compared with standard care, the new hypothetical therapy was predicted to substantially decrease costs and was also associated with gains in QALYs. With an improved treatment effect of 50 percent on achieving full remission, the net cost savings were 20,000 Swedish kronor over a 5-year follow-up time, given equal costs of treatments. Patients gained .073 QALYs over 5 years. The results are sensitive to changes in assigned treatment effects. The present study provides a new model for assessing the cost-effectiveness of treatments for depression by incorporating full remission as the treatment goal and QALYs as the primary outcome measure. Moreover, we show the usefulness of naturalistic real-life data on costs and quality of life and transition probabilities when modeling the disease over time.

The Long-Term Cost Effectiveness of Treatments for Benign Prostatic Hyperplasia

Excellent treatment outcomes with long-term durability and few adverse effects are expectations of treatments for chronic conditions. The long-term cost effectiveness of newer treatments for benign prostatic hyperplasia (BPH), including high-energy transurethral microwave thermotherapy (TUMT) and combination pharmaceutical therapy, has not been sufficiently studied against existing alternatives. The objective of this study was to estimate the incremental cost effectiveness of BPH treatment alternatives. We employed a Markov model over a 20-year time horizon and the payer's perspective to evaluate the cost effectiveness of watchful waiting (WW), pharmaceuticals (alpha-adrenoceptor antagonists [alpha-blockers], 5-alpha-reductase inhibitors [5-ARIs], combination therapy), TUMT and transurethral resection of the prostate (TURP) in treating BPH. Markov states included improvement in symptoms, no improvement in symptoms, adverse effects and death. We used data from the published literature for outcomes, including systematic reviews whenever possible. Costs were estimated using a managed-care claims database and Medicare fee schedules, and were reported in Dollars US, 2004 values. Costs and effectiveness outcomes were discounted at a rate of 3% per year. Men (aged > or =45 years) with moderate to severe lower urinary tract symptoms and uncomplicated BPH were included in the analysis, and results were stratified by age and BPH symptom levels. Outcomes included costs, QALYs, incremental cost-utility ratios and cost-effectiveness acceptability curves. Sensitivity analysis was performed on important parameters, with an emphasis on probabilistic sensitivity analysis. alpha-Blockers and TUMT were cost effective for treating moderate symptoms using the threshold of Dollars US 50,000 per QALY. For example, at 65 years of age, the cost per QALY was Dollars US 16,018 for alpha-blockers compared with WW and Dollars US 30,204 for TUMT versus alpha-blockers. TURP was the most cost-effective treatment for severe symptoms (Dollars US 5824 per QALY ) versus WW. Model results were robust to changes in costs and sensitive to the assumed probabilities, utility weights, extent of improvement and life expectancy. Nevertheless, acceptability curves consistently demonstrated the same alternatives as most likely to be cost effective. Our model suggests that alpha-blockers and TURP appear to be the most cost-effective alternatives, from a US payer perspective, for BPH patients with moderate and severe symptoms, respectively. TUMT was promising for patients with moderate symptoms and the oldest patients with severe symptoms, but otherwise was dominated. Value of information analysis could be used to determine the net benefit of additional research.

A COST COMPARISON OF MEDICAL MANAGEMENT AND TRANSURETHRAL NEEDLE ABLATION FOR TREATMENT OF BENIGN PROSTATIC HYPERPLASIA DURING A 5-YEAR PERIOD

We performed an analysis comparing the cost of medical management with TUNA therapy for a 5-year period. Published costs for tamsulosin, finasteride, transurethral needle ablation (TUNA, Medtronic, Inc., Minneapolis, Minnesota) and transurethral resection of the prostate were used to construct a cost analysis model comparing medication with TUNA. The model analyzed monotherapy with an alpha-blocker (tamsulosin) and a 5alpha-reductase inhibitor (finasteride), combination therapy using both medications, and a mixed scenario using monotherapy and combination therapy. Published data were used to estimate the rate of surgical intervention in patients initially treated with medications or TUNA. Tamsulosin monotherapy was less expensive than TUNA for 5 years ($3,485 for tamsulosin vs $4,811 for TUNA year 5). Finasteride monotherapy reaches a break-even point with TUNA during year 5 ($4,867 for finasteride vs $4,811 for TUNA). Combination therapy reaches a break-even point with TUNA after approximately 2 years 7 months of treatment ($4,515 for combination therapy vs $4,572 for TUNA) and the mixed scenario breaks even with TUNA at approximately year 4 ($4,696 for medical management vs $4,645 for TUNA). The TUNA procedure compares favorably to combination medical therapy for the treatment of benign prostatic hyperplasia on a cost basis. alpha-Blocker monotherapy is less costly than TUNA while 5alpha-reductase inhibitor monotherapy is approximately equivalent to TUNA for 5 years. The TUNA procedure is less expensive than combination medical management for 5 years, with a break-even point at approximately 2 years 7 months.

Medicare costs and health resource utilisation associated with atrial fibrillation in the elderly

SummaryThe study objective was to quantify health care utilisation and medical costs for Medicare beneficiaries with atrial fibrillation (AF). Patients aged >65 years with an AF diagnosis were identified in the Medicare 5% sample Standard Analytic Files between July and December 1998 and followed for 1 year. Total and AF-related resource use and costs were measured from the Medicare payment perspective.Among 59,648 AF patients, over 50% had a hospital admission during follow-up, and 88% had a physician encounter with a principal AF diagnosis. AF patients incur significant AF-related medical costs, averaging approximately $670 per patient each year. Estimated direct treatment costs to the Medicare Program for AF (identified by claims with a principal AF diagnosis) were $797M, whilst overall 1-year costs for all AF patients totalled $14B. These findings have important policy implications for the cost-effectiveness of new treatments that eliminate or reduce the AF burden in Medicare patients.

The relationship between health perception and utility in heart failure patients in a clinical trial: results from an OVERTURE substudy

Background Cost-effectiveness analyses should be based on incremental years of life gained adjusted with a health status measure known as a utility. Measuring utilities for all subjects in a large-scale randomized trial, however, would be prohibitively cumbersome. We therefore sought to estimate utilities for all subjects from results obtained in a subset of patients. Methods and results We studied a subset of patients enrolled in a randomized trial of omapatrilat for the treatment of heart failure. Survey instruments (a time trade-off questionnaire, a visual analog scale [VAS] score of overall health perception, and the Duke Activity Status Index [DASI]) were administered to patients by mail and by telephone interviews. There was a significant ( P < .0001) relationship between VAS score and utility described by the power function u = 1–(1–v)q, where q = 2.17 (95% CI 1.76 to 2.58). There was a significant positive correlation (r = .17, P < .04) between the DASI and utility, and a significant negative correlation (r = –.26, P < .001) between utility and New York Heart Association functional class. Conclusion There is a significant relationship between the relatively easily obtainable health perception score by VAS with the more complex utility by time tradeoff for a subset of patients in a multicenter randomized clinical trial. This relationship may be helpful in examining the cost-effectiveness of new treatments for heart failure.

A software application for multiattribute utility models 1

Rationale and Objectives. A software application for the building of preference-weighted quality of life instruments was constructed. The purpose of this application is to make the process of building such instruments with multiattribute utility (MAU) methods more accessible to physician investigators. Our application, “multiattribute outcomes evaluator” or “MOE,” could facilitate the use of such methods for constructing disease-specific instruments for assessing the cost-effectiveness of new treatments. MOE could also be useful for building outcomes measures that are intended to aid individual decision making, assessment of patient satisfaction with care, or measuring of multiattribute outcomes that do not need to be scaled for cost-effectiveness analysis. Materials and Methods. MOE was programmed in Visual Basic and runs atop a Microsoft Access data base. Data from groups or individuals can be stored, and valuations of health states can be performed using standard gambles and rating scale methods. Solutions to MAU models with multiplicative or additive utility independence are possible with this application. Results. Initial tests of the program algorithms have been successful. Conclusion. We anticipate The MOE approach to MAU modeling will be a useful tool for health services researchers.

Cost-utility of interferon β 1b in the treatment of patients with active relapsing-remitting or secondary progressive multiple sclerosis

Economic evaluations of treatments for multiple sclerosis have focused either on relapsing-remitting or secondary progressive disease. None has addressed specifically the question of how treatments affect disease progression from diagnoses to severe disability; however, in both clinical and economic terms this is one of the key questions. This study therefore created a disease model that includes both relapsing and progressive multiple sclerosis and covers the full range of disease severity. In addition, the issue of covariates influencing the cost per QALY and the uncertainty in the estimates is addressed. The model is based on our earlier approach using a Markov model, where disease progression is measured as functional disability (EDSS), costs and utilities associated with different disability levels, and outcome expressed as QALY. Treatment is based on a subgroup of patients with active disease in two placebo-controlled clinical trials and open-label extensions with interferon-beta(1b) in relapsing-remitting or secondary progressive multiple sclerosis, and natural history data are used for the extrapolation beyond the trials. Disease progression (transition probabilities) was estimated using a probit model controlling for differences in patient and disease characteristics, and costs and utilities for different disability levels are taken from large population-based observational studies. Results are presented for Sweden and compared to the United Kingdom. When treatment is given for 36 months (adjusted for compliance) and no further effect is included, the cost per QALY is EURO 7800 (direct, indirect, and informal care costs included, discounted at 3%) over 10 years. The total number of QALYs increases from 3.45 in the no-treatment group to 3.64 in the treatment group. When treatment is prolonged to 54 months, the cost per QALY is EURO 38,700. Using the full range of costs and utilities, the probability that the cost per QALY over a 20-year time-frame is below EURO 50,000 for patients starting treatment at EDSS 3.0 is 80%. In view of the progression of MS over many years models are required to estimate the cost-effectiveness of new treatments. The model proposed here allows estimating disease progression, costs, and QALYs for patients with different characteristics and types of MS, and it can be adapted to different countries. It can hence be used to assess the cost-effectiveness of a defined intervention, such as interferon-beta(1b).

Modeling the progression of rheumatoid arthritis: a two-country model to estimate costs and consequences of rheumatoid arthritis.

Two simulation models were developed to analyze the cost-effectiveness of new treatments that affect the progression of rheumatoid arthritis (RA). We used data from 2 cohorts of patients with early RA who had been followed up since disease onset (up to 15 years). In the Swedish study, 183 patients were followed up for a mean of 11.3 years. In the UK study, 916 patients were followed up for a mean of 7.8 years. Disease progression over 10 years was modeled as annual transitions between disease states, defined by Health Assessment Questionnaire (HAQ) scores. A regression model was used to estimate transition probabilities conditional on age, sex, and time since onset of disease, in order to allow simulation of different patient cohorts. Costs and utilities associated with different HAQ levels were based on data from the cohort studies and cross-sectional surveys. Costs increase and quality of life decreases as RA progresses. In Sweden, total annual costs range from 4,900 dollars to 33,000 dollars per patient, compared with 4,900 dollars to 14,600 dollars in the UK. Cumulative costs over 10 years for patients starting in disease state 1 (HAQ < 0.6) are 54,600 dollars in Sweden and 26,600 dollars in the UK. The cumulative numbers of quality-adjusted life-years (QALYs) are 5.5 and 5.6, respectively. Both costs and QALYs were discounted at 3%. The 2 models, which were based on different patient cohorts, reach a similar conclusion in terms of the effect of RA over 10 years. They appear to accurately capture disease progression and its effects and can therefore be useful in estimating the cost-effectiveness of new treatments in RA.

Development of a Decision-Analytic Model of Stroke Care in the United States and Europe

Objective Stroke places a huge burden on society in terms of premature death, disability, and costs of care. Increasingly, the cost-effectiveness of new interventions needs to be demonstrated before their widespread implementation. Clinical trials are unable to measure the long-term impact of such new interventions in stroke care, and a modeling approach is necessary. The Stroke Outcome Model has been developed in four countries: France, Germany, the United Kingdom, and the United States as a flexible tool for this purpose. Method The decision-analytic model represents the management of acute stroke and long-term care and prevention of recurrence for stroke survivors. The latter consists of semi-Markov state-transition processes, with health states defined by therapy, disability, and occurrence of further stroke. Sources of clinical data include trials, meta-analyses, and prospective cohort studies such as the Oxfordshire Community Stroke Project and the Northern Manhattan Stroke Study. Resource use data were obtained from published sources and expert clinician panels. Outcome measures used were strokes averted, life years, and quality-adjusted life-years gained. Results The model has been used to undertake economic analyses of antiplatelet therapy for the prevention of recurrent strokes, and of stroke unit care and thrombolytic therapy in acute stroke. From a health-and social-care perspective, new interventions were found to be cost saving or to provide health benefits at modest additional cost. Results were sensitive to the cost perspective, time horizon, baseline risk of stroke recurrence, and choice of effectiveness measure. Conclusion The development of this model highlights the need for improved information on prognosis and resources used by stroke survivors and the importance of differentiating between economically distinct end points such as death, disabled survival and nondisabled survival, which may be combined as outcomes in clinical trials.

Costs, quality of life and disease severity in multiple sclerosis: a cross-sectional study in Sweden.

This study assessed the cost to society of multiple sclerosis (MS) in Sweden in 1998. The cost-of-illness method, based on the human capital theory, was used as the theoretical framework. The study used a cross-sectional approach, in which resource utilization data and quality-of-life data (utilities) were collected at a single time point. The total cost of MS was estimated at 4868 MSEK, or 586 MEUR, giving an annual cost of 442 500 SEK, or 53 250 EUR, per patient (1USD = 9.73 SEK, 1 EUR = 8.31 SEK, as of 21 September 2000). Direct costs accounted for about 67% of total cost, and they were dominated by the cost of personal assistants and drugs. Indirect costs (loss of production) accounted for about 33% of total costs. To these economic costs, intangible costs of 2702 MSEK (325 MEUR) should be added as well. Direct, indirect and informal care costs all rose significantly with increased disability and were higher during a relapse. Quality of life declined substantially with increased disability and was lower during a relapse. Multiple sclerosis was found to be associated with much higher costs to society than has been ascertained by former studies. The study also revealed a strong correlation between severity of the disease and quality of life. These results are crucial for further studies on the cost-effectiveness of new treatments aimed at preventing relapses and reducing progression of the disease.

Healthcare Resource Utilization in the Three Months after Acute Stroke: A Report on 1604 Patients from the Glycine Antagonist in Neuroprotection (GAIN) Americas Trial

P226 Background/objective: Data on healthcare resource utilization (RU) in acute stroke will assist in evaluation of cost-efficacy and cost-effectiveness of new treatments. We present results of a substudy of resource utilization within GAIN Americas, a multi-center, randomized, placebo-controlled, phase 3 clinical trial of the glycine antagonist GV150526 in acute stroke, conducted in the US and Canada. Previously reported clinical results showed no difference in clinical outcome by treatment at 3 months. Methods: RU data included pre-stroke and Month 3 residence and employment status, hospital length of stay (LOS) by ward type, discharge destination, and outpatient rehabilitation services. There were no treatment differences on RU endpoints, so data were combined across treatment groups. Results: The study population comprised 1604 acute stroke patients (85% ischemic; median age: 72; female: 48%; median NIHSS score: 12). Case fatality was 13% in hospital, 21% by Month 3. Median hospital LOS was 9 days (mean=15), including 3 days (mean = 4.5) in intensive care and 4.5 days (mean=6.8) in step-down. Discharge destinations included rehabilitation facility (49%), home (27%) and nursing home (11%). Pre-stroke, 87% lived in own home, but at 3 months only 62% lived at home. Outpatient rehabilitation services included occupational therapy (31%), physical therapy (39%), speech therapy (20%) and psychotherapy (2%). Pre-stroke, 21% were employed full- or part-time; at Month 3, 6% were employed. Of those employed pre-stroke, 60% reported being unable to work for health reasons at Month 3. Conclusion: These data describe the social and economic burden of stroke in a well-defined cohort. Such information should be valuable to care providers at many levels of the healthcare system and to health outcomes researchers. Prospective collection of detailed RU data from a large clinical trial is feasible and worthwhile.

Healthcare Resource Utilization in the Three Months after Acute Stroke: A Report on 1604 Patients from the Glycine Antagonist in Neuroprotection (GAIN) Americas Trial

P226 Background/objective: Data on healthcare resource utilization (RU) in acute stroke will assist in evaluation of cost-efficacy and cost-effectiveness of new treatments. We present results of a substudy of resource utilization within GAIN Americas, a multi-center, randomized, placebo-controlled, phase 3 clinical trial of the glycine antagonist GV150526 in acute stroke, conducted in the US and Canada. Previously reported clinical results showed no difference in clinical outcome by treatment at 3 months. Methods: RU data included pre-stroke and Month 3 residence and employment status, hospital length of stay (LOS) by ward type, discharge destination, and outpatient rehabilitation services. There were no treatment differences on RU endpoints, so data were combined across treatment groups. Results: The study population comprised 1604 acute stroke patients (85% ischemic; median age: 72; female: 48%; median NIHSS score: 12). Case fatality was 13% in hospital, 21% by Month 3. Median hospital LOS was 9 days (mean=15), including 3 days (mean = 4.5) in intensive care and 4.5 days (mean=6.8) in step-down. Discharge destinations included rehabilitation facility (49%), home (27%) and nursing home (11%). Pre-stroke, 87% lived in own home, but at 3 months only 62% lived at home. Outpatient rehabilitation services included occupational therapy (31%), physical therapy (39%), speech therapy (20%) and psychotherapy (2%). Pre-stroke, 21% were employed full- or part-time; at Month 3, 6% were employed. Of those employed pre-stroke, 60% reported being unable to work for health reasons at Month 3. Conclusion: These data describe the social and economic burden of stroke in a well-defined cohort. Such information should be valuable to care providers at many levels of the healthcare system and to health outcomes researchers. Prospective collection of detailed RU data from a large clinical trial is feasible and worthwhile.

The cost of medical care for patients with cystic fibrosis in a health maintenance organization.

Cystic fibrosis (CF) is the most common life-shortening genetic disorder among white individuals worldwide. Previous estimates of the costs of medical care have been based on expert opinion rather than observed costs. Accurate cost estimates are needed to enable evaluation of the cost-effectiveness of new interventions and prenatal genetic screening recommendations. To evaluate the cost of medical care for patients (N = 136) served by a health maintenance organization with a CF center. Retrospective analysis of data from computerized cost databases and the Cystic Fibrosis Foundation annual survey. Severity of disease was classified based on the percent predicted forced expiratory volume at 1 second. The annual cost of medical care in 1996 averaged $13 300 and ranged from $6200 among patients with mild disease to $43 300 among patients with severe disease. Of total costs, 47% were from hospitalization, 18% were from DNase (Pulmozyme), 12% were from clinic visits, and 10% were from outpatient antibiotics. When the observed costs were used to estimate the costs of medical care for the entire population of CF patients in the United States, these costs were estimated to be $314 million per year in 1996 dollars. We conclude that the cost of medical care for CF varies greatly with severity but is substantial even among patients with mild disease. These findings underscore the need for strategies to ensure good health insurance coverage and high quality care for all individuals with this condition.