Abstract
Over the past quarter century, as costs have become an important factor in medical decision making, indices of cost-effectiveness (CE) have increasingly been used in the evaluation of medical therapies. The traditional approach to cost-effectiveness analysis (CEA) utilizes decision-analytic models based on estimates of cost and effectiveness outcomes obtained from nonsampled secondary data (from the literature, insurance claims databases, and expert opinion) and employs sensitivity analysis to examine the variability in results as uncertain model inputs are varied over reasonable ranges. Throughout the past decade, there has been an increasing trend for economic studies to be incorporated into large clinical trials. This has allowed for cost-effectiveness to be evaluated directly, using primary patient-level data on both clinical outcomes and costs. The stochastic, or random, nature of this data, resulting from patient-to-patient (sampling) variability, allows for uncertainty associated with estimates of cost-effectiveness to be estimated using methods of statistical analysis. This chapter presents an overview of statistical and other methodological considerations in the evaluation of cost-effectiveness using experimentally obtained data from randomized controlled trials.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Preview
Unable to display preview. Download preview PDF.
References
Barber JA, Thompson SG. Analysis of cost data in randomized trials: an application of the non-parametric bootstrap. Stat Med 2000; 19: 3219–3236.
Kendall MG, S., A., The advanced theory of statistics. Volume I: Distribution Theory. Charles Griffin ( 110 ) Company Limited, London, 1969.
Efron B. Bootstrap methods: another look at the Jackknife. Ann Stat 1979; 7: 1–26.
Efron B. Non-parametric standard errors and confidence intervals (with discussion). Canadian J Stat 1981; 9: 139–172.
Efron B. Better bootstrap confidence intervals. J Am Stat Assoc 1987; 82: 171–200.
Altman DG, Bland JM. Absence of evidence is not evidence of absense. Br Med J 1995; 311: 485.
Duan N. Smearing estimate: a nonparametric retransformation method. J Am Stat Assoc 1983; 78: 605–610.
Manning WG, Mullahy J. Estimating log models: to transform or not to transform? J Health Econ 2001; 20: 461–494.
Kalbfleisch JG. Probability and statistical inference. Volume 1: Probability. Springer texts in statistics, vol. 1. Springer-Verlag, New York, 1985, p. 343.
Cox DR, Hinkley DV. Theoretical Statistics. Chapman and Hall, New York, 1986.
Black WC. The CE plane: a graphic representation of cost-effectiveness. Med Decis Making 1990; 10: 212–224.
Briggs A, Fenn P. Trying to do better than average: a commentary on `statistical inference for cost-effectiveness ratios’. Health Econ 1997; 6: 491–495.
O’Brien BJ, Drummond MF, Labelle RJ, Willan A. In search of power and significance: issues in the design and analysis of stochastic cost-effectiveness studies in health care. Med Care 1994; 32: 150–163.
Briggs A, Fenn P. Confidence intervals or surfaces? Uncertainty on the cost-effectiveness plane. Health Econ 1998; 7: 723–740.
Fieller EC. The distribution of an index in a normal bivariate population. Biometrika 1932; 56: 635–639.
Fieller EC. Some problems in interval estimation. J Royal Stat Society, Series B, 1954; 16: 175–183.
Chaudhary MA, Stearns SC. Estimating confidence intervals for cost-effectiveness ratios: an example from a randomized trial. Stat Med 1996; 15: 1447–1458.
Willan AR, O’Brien BJ. Confidence intervals for cost-effectiveness ratios: an application of Fieller’s theorem. Health Econ 1996; 5: 297–305.
Briggs AH, O’Brien BJ, Blackhouse G. Thinking Outside the Box: Recent advances in the analysis and presentation of uncertainty in cost-effectiveness studies. Annu Rev Public Health 2002; 23: 377–401.
Briggs AH, Mooney CZ, Wonderling DE. Constructing confidence intervals for cost-effectiveness ratios: an evaluation of parametric and non-parametric techniques using Monte Carlo simulation. Stat Med 1999; 18: 3245–3262.
Briggs AH, Wonderling DE, Mooney CZ. Pulling cost-effectiveness analysis up by its bootstraps: a non-parametric approach to confidence interval estimation. Health Econ 1997; 6: 327–340.
Polsky D, Glick HA, Willke R, Schulman K. Confidence intervals for cost-effectiveness ratios: a comparison of four methods. Health Econ 1997; 6: 243–252.
Glick HA, Polsky D. Assessing the uncertainty of the results of randomized controlled trials. In: Society for Medical Decision Making, Short Course #5: Prospective Economic Evaluation of Clinical Trials: Methods and Analysis, course handouts, Reno, NV, 1999.
Stinnett AA, Mullahy J. The negative side of cost-effectiveness analysis. JAMA 1997; 277: 1931–1933.
Stinnett AA, Mullahy J. Net health benefits: a new framework for the analysis of uncertainty in cost-effectiveness analysis. Med Decis Making 1998; 18 (2 Suppl): 568–580.
Heitjan DF, Moskowitz AJ, Whang W. Problems with interval estimates of the incremental cost-effectiveness ratio. Med Decis Making 1999; 19: 9–15.
Scharfstein JA. Cost-Effectiveness and AIDS: Methods and Application. Doctoral thesis. Harvard University, Boston, MA, 1997.
Mahoney EM, Jurkovitz CT, Chu H, et al. Cost and cost-effectiveness of an early invasive versus conservative strategy for the treatment of unstable angina and non-ST elevation myocardial infarction. JAMA 2002; 288: 1851–1858.
Cook JR, Heyse JF. Use of an angular transformation for ratio estimation in cost-effectiveness analysis. Stat Med 2000; 19: 2989–3003.
Jonsson B, Cook JR, Pedersen TR. The cost-effectiveness of lipid lowering in patients with diabetes: results from the 4S trial. Diabetologia 1999; 42: 1293–1301.
van Hout BA, Al MJ, Gordon GS, Rutten FF. Costs, effects and C/E-ratios alongside a clinical trial. Health Econ 1994; 3: 309–319.
Tambour M, Zethraeus N, Johannesson M. A note on confidence intervals in cost-effectiveness analysis. Int J Technol Assess Health Care 1998; 14: 467–471.
Heitjan DF, Moskowitz AJ, Whang W. Bayesian estimation of cost-effectiveness ratios from clinical trials. Health Econ 1999; 8: 191–201.
Heitjan DF. Fieller’s method and net health benefits. Health Econ 2000; 9: 327–335.
Briggs AH, Gray AM. Power and sample size calculations for stochastic cost-effectiveness analysis. Med Decis Making 1998; 18 (2 Suppl): S81 - S92.
Willan AR, O’Brien BJ. Sample size and power issues in estimating incremental cost-effectiveness ratios from clinical trials data. Health Econ 1999; 8: 203–211.
Gardiner JC, Huebner M, Jetton J, Bradley O. Power and sample assessments for tests of hypotheses on cost-effectiveness ratios. Health Econ 2000; 9: 227–234.
Al MJ, van Hout BA, Michel BC, Rutten FF. Sample size calculation in economic evaluations. Health Econ 1998; 7: 327–335.
Laska EM, Meisner M, Siegel C. Power and sample size in cost-effectiveness analysis. Med Decis Making 1999; 19: 339–343.
Briggs A. Economics notes: handling uncertainty in economic evaluation. Br Med J 1999; 319: 120.
Lothgren M, Zethraeus N. Definition, interpretation and calculation of cost-effectiveness acceptability curves. Health Econ 2000; 9: 623–630.
Briggs AH. A Bayesian approach to stochastic cost-effectiveness analysis. Health Econ 1999; 8: 257–261.
Briggs AH. A Bayesian approach to stochastic cost-effectiveness analysis. An illustration and application to blood pressure control in type 2 diabetes. Int J Technol Assess Health Care 2001; 17: 69–82.
Briggs A, Gray A. Using cost effectiveness information. Br Med J 2000; 320: 246.
Weintraub WS, Thompson TD, Culler S, et al. Targeting patients undergoing angioplasty for thrombus inhibition: a cost-effectiveness and decision support model. Circulation 2000; 102: 392–398.
Mahoney EM, Thompson TD, Veledar E, et al. Cost-effectiveness of targeting patients undergoing cardiac surgery for therapy with intravenous amiodarone to prevent atrial fibrillation. J Am Coll Cardiol 2002; 40: 737–745.
Hoch JS, Briggs AH, Willan AR. Something old, something new, something borrowed, something blue: a framework for the marriage of health econometrics and cost-effectiveness analysis. Health Econ 2002; 11: 415–430.
Weinstein MC, Siegel JE, Gold MR, et al. Recommendations of the Panel on Cost-effectiveness in Health and Medicine. JAMA 1996; 276: 1253–1258.
Cannon CP, Weintraub WS, Demopoulos LA, et al. Comparison of early invasive and conservative strategies in patients with unstable coronary syndromes treated with the glycoprotein IIb/IIIa inhibitor tirofiban. N Engl J Med 2001; 344: 1879–1887.
Sonnenberg FA, Beck JR. Markov models in medical decision making: a practical guide. Med Decis Making 1993; 13: 322–338.
Beck JR, Pauker SG. The Markov process in medical prognosis. Med Decis Making 1983; 3: 419–458.
Kuntz KM, Weinstein MC. Modeling in economic evaluation. In: Drummond M, McGuire A (eds.), Economic Evaluation in Health Care: Merging Theory with Practice. Oxford University Press, Inc., NY, 2001, pp. 141–171.
Doubilet P, Begg CB, Weinstein MC, et al. Probabilistic sensitivity analysis using Monte Carlo simulation. A practical approach. Med Decis Making 1985; 5: 157–177.
Briggs AH, Gray AM. Handling uncertainty in economic evaluations of healthcare interventions. Br Med J 1999; 319: 635–638.
Peeters A, Mamun AA, Willekens F, Bonneux L. A cardiovascular life history. A life course analysis of the original Framingham Heart Study cohort. Eur Heart J 2002; 23: 458–466.
Mark DB, Harrington RA, Lincoff AM, et al. Cost-effectiveness of platelet glycoprotein IIb/IIIa inhibition with eptifibatide in patients with non-ST-elevation acute coronary syndromes. Circulation 2000; 101: 366–371.
Mark DB, Lee TH. Conservative management of acute coronary syndrome: cheaper and better for you? Circulation 2002; 105: 666–668.
Willke RJ, Glick HA, Polsky D, Schulman K. Estimating country-specific cost-effectiveness from multinational clinical trials. Health Econ 1998; 7: 481–493.
Drummond MF, Bloom BS, Canin G, et al. Issues in the cross-national assessment of health technology. Int J Technol Assess Health Care 1992; 8: 671–682.
Drummond M, Pang F. Transferability of economic evaluation results. In: Drummond A (ed.), Economic Evaluation in Health Care. Oxford University Press Inc., NY, 2001, pp. 256–276.
Jonsson B, Weinstein MC. Economic evaluation alongside multinational clinical trials. Study considerations for GUSTO IIb. Int J Technol Assess Health Care 1997; 13: 49–58.
Willke RJ, Glick HA, Polsky D, Schulman K. Estimating country-specific cost-effectiveness from multinational clinical trials. Health Econ 1998; 7: 481–493.
Laupacis A, Feeny D, Detsky AS, Tugwell PX. How attractive does a new technology have to be to warrant adoption and utilization? Tentative guidelines for using clinical and economic evaluations. Can Med Assoc J 1992; 146: 473–481.
Hirth RA, Chernew ME, Miller E, et al. Willingness to pay for a quality-adjusted life year: in search of a standard. Med Decis Making 2000; 20: 332–342.
Garber AM, Phelps CE. Economic foundations of cost-effectiveness analysis. J Health Econ 1997; 16: 1–31.
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2003 Humana Press Inc., Totowa, NJ
About this chapter
Cite this chapter
Mahoney, E.M., Chu, H. (2003). Cost-Effectiveness Analysis Alongside Clinical Trials. In: Weintraub, W.S. (eds) Cardiovascular Health Care Economics. Contemporary Cardiology. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-59259-398-9_9
Download citation
DOI: https://doi.org/10.1007/978-1-59259-398-9_9
Publisher Name: Humana Press, Totowa, NJ
Print ISBN: 978-1-4684-9784-7
Online ISBN: 978-1-59259-398-9
eBook Packages: Springer Book Archive