When should decision-analytic modeling be used in the economic evaluation of health care?

  • Uwe SiebertMDH, MPH

More than a quarter of a century ago Weinstein and Stason [1] introduced to the medical community the concept and methods of cost-effectiveness analysis. Although economic evaluations were not widely used at first, today they are a standard tool in the assessment of health care technologies. Health care resources continue to be limited, and new and more effective technologies often come with increased costs and bear different risks than the "standard" technology. Therefore the application of formal methods considering all dimensions relevant to the patient and society will continue to be of paramount importance in the future.

When faced with choosing between a variety of available procedures, physicians and patients may want to choose those that offer the best trade-off between potential harm and potential benefit in hopes of recommending the treatment with the maximum expected health benefit for the patient. Therefore health policy makers and health insurers must decide which...


  1. 1.
    Weinstein MC, Stason WB (1977) Foundations of cost-effectiveness analysis for health and medical practices. N Engl J Med 296:716–721PubMedGoogle Scholar
  2. 2.
    Raiffa H (1968) Decision analysis: introductory lectures on choices under uncertainty, 1st edn. Addison Wesley: ReadingGoogle Scholar
  3. 3.
    Pauker SG, Kassirer JP (1975) Therapeutic decision making: a cost-benefit analysis. N Engl J Med 293:229–234PubMedGoogle Scholar
  4. 4.
    Weinstein MC, Fineberg HV, Elstein AS et al. (1980) Clinical decision analysis, 1st edn. Saunders: PhiladelphiaGoogle Scholar
  5. 5.
    Keeney RL (1982) Decision analysis: an overview. Operations Res 30:803–838Google Scholar
  6. 6.
    Kassirer JP, Moskowitz AJ, Lau J, Pauker SG (1987) Decision analysis: a progress report. Ann Intern Med 106:275–291PubMedGoogle Scholar
  7. 7.
    Sox H, Blatt MA, Higgins MC, Marton KI (1988) Medical decision making. Butterworths: BostonGoogle Scholar
  8. 8.
    Chapman GB, Sonnenberg FA (2000) Decision making in health care: theory, psychology, and applications. Cambridge Series on Judgment and Decision Making. Cambridge University Press: New YorkGoogle Scholar
  9. 9.
    Hunink MG, Glasziou PP, Siegel JE et al. (2001) Decision making in health and medicine. Integrating evidence and values. Cambridge University Press: CambridgeGoogle Scholar
  10. 10.
    Buxton MJ, Drummond MF, Van Hout BA et al. (1997) Modelling in economic evaluation: an unavoidable fact of life. Health Econ 6:217–227Google Scholar
  11. 11.
    Weinstein MC, Toy EL, Sandberg EA et al. (2001) Modeling for health care and other policy decisions: uses, roles, and validity. Value Health 4:348–361CrossRefPubMedGoogle Scholar
  12. 12.
    Sheldon TA (1996) Problems of using modelling in the economic evaluation of health care. Health Econ 5:1–11Google Scholar
  13. 13.
    Weinstein MC, O'Brien B, Hornberger J et al. (2003) Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR Task Force on Good Research Practices-Modeling Studies. Value Health 6:9–17CrossRefPubMedGoogle Scholar
  14. 14.
    National Research Council (1991) Improving information for social policy decisions: the uses of microsimulation modelling, vol 1. National Academy Press: WashingtonGoogle Scholar
  15. 15.
    Box GEP, Hunter WG, Hunter JS (1978) Statistics for experimenters: an introduction to design, data analysis, and model building. Wiley: New YorkGoogle Scholar
  16. 16.
    Eddy DM (1990) Clinical decision making: from theory to practice. Designing a practice policy. Standards, guidelines and options. JAMA 263:3077CrossRefPubMedGoogle Scholar
  17. 17.
    Richardson WS, Detsky AS (1995) Users' guides to the medical literature. VII. How to use a clinical decision analysis. A. Are the results of the study valid? Evidence Based Medicine Working Group. JAMA 273:1292–1295CrossRefPubMedGoogle Scholar
  18. 18.
    Drummond MF, O'Brien B, Stoddart GL, Torrance GW (1997) Methods for the economic evaluation of health care programmes, 2nd edn. Oxford University Press: New YorkGoogle Scholar
  19. 19.
    Beck JR, Pauker SG (1983) The Markov process in medical prognosis. Med Decis Making 3:419–458PubMedGoogle Scholar
  20. 20.
    Sonnenberg FA, Beck JR (1993) Markov models in medical decision making: a practical guide. Med Decis Making 13:322–338PubMedGoogle Scholar
  21. 21.
    Anderson RM, May RM (1991) Infectious diseases of humans: dynamics and control. Oxford University Press: New YorkGoogle Scholar
  22. 22.
    Gold MR, Siegel JE, Russell LB, Weinstein MC (1996) Cost-effectiveness in health and medicine. Oxford University Press: New YorkGoogle Scholar
  23. 23.
    Tengs TO, Adams ME, Pliskin JS et al. (1995) Five-hundred life-saving interventions and their cost-effectiveness. Risk Anal 15:369–390PubMedGoogle Scholar
  24. 24.
    Deuffic S, Buffat L, Poynard T, Valleron AJ (1999) Modeling the hepatitis C virus epidemic in France. Hepatology 29:1596–1601PubMedGoogle Scholar
  25. 25.
    Wong JB, McQuillan GM, McHutchison JG, Poynard T (2000) Estimating future hepatitis C morbidity, mortality, and costs in the United States. Am J Public Health 90:1562–1569PubMedGoogle Scholar
  26. 26.
    Salomon JA, Weinstein MC, Hammitt JK, Goldie SJ (2002) Empirically calibrated model of hepatitis C virus infection in the United States. Am J Epidemiol 156:761–773CrossRefPubMedGoogle Scholar
  27. 27.
    Freeman AJ, Law MG, Kaldor JM, Dore GJ (2003) Predicting progression to cirrhosis in chronic hepatitis C virus infection. J Viral Hepat 10:285–293PubMedGoogle Scholar
  28. 28.
    Manns MP, McHutchison JG, Gordon SC et al. (2001) Peginterferon alfa-2b plus ribavirin compared with interferon alfa-2b plus ribavirin for initial treatment of chronic hepatitis C: a randomised trial. Lancet 358 958–965Google Scholar
  29. 29.
    Fried M, Shiffman M, Reddy R et al. (2002) Peginterferon alfa-2a plus ribavirin for chronic hepatitis C virus infection. N Engl J Med 347:975–982CrossRefPubMedGoogle Scholar
  30. 30.
    Krahn MD, Mahoney JE, Eckman MH et al. (1994) Screening for prostate cancer. A decision analytic view. JAMA 272:773–780PubMedGoogle Scholar
  31. 31.
    Coley CM, Barry MJ, Fleming C et al. (1997) Early detection of prostate cancer. II. Estimating the risks, benefits, and costs. Ann Intern Med 126:468–479PubMedGoogle Scholar
  32. 32.
    Harris R, Lohr KN (2002) Screening for prostate cancer: an update of the evidence for the U.S. Preventive Services Task Force. Ann Intern Med 137:917–929PubMedGoogle Scholar
  33. 33.
    Neumann PJ, Hermann RC, Kuntz KM et al. (1999) Cost-effectiveness of donepezil in the treatment of mild or moderate Alzheimer's disease. Neurology 52:1138–1145PubMedGoogle Scholar
  34. 34.
    Rogers SL, Farlow MR, Doody RS et al. (1998) A 24-week, double-blind, placebo-controlled trial of donepezil in patients with Alzheimer's disease. Donepezil Study Group. Neurology 50:136–145PubMedGoogle Scholar
  35. 35.
    Greiner W, Schöffski O, Graf von der Schulenburg JM (2000) Transferability of international results on national research questions (in German). In: Schöffski O, Graf von der Schulenburg JM (eds) Gesundheitsökonomische Evaluationen, 2nd edn. Springer: Berlin Heidelberg New York, pp 403–420Google Scholar
  36. 36.
    Graham DY, Agrawal NM, Roth SH (1988) Prevention of NSAID-induced gastric ulcer with misoprostol: multicentre, double-blind, placebo-controlled trial. Lancet II 1277–1280Google Scholar
  37. 37.
    Hillman AL, Bloom BS (1989) Economic effects of prophylactic use of misoprostol to prevent gastric ulcer in patients taking nonsteroidal anti-inflammatory drugs. Arch Intern Med 149:2061–2065CrossRefPubMedGoogle Scholar
  38. 38.
    Baltussen R, Ament A, Leidl R (1996) Making cost assessments based on RCTs more useful to decision-makers. Health Policy 37:163–183PubMedGoogle Scholar
  39. 39.
    Baltussen R, Leidl R, Ament A (1996) The impact of age on cost-effectiveness ratios and its control in decision making. Health Econ 5:227–239Google Scholar
  40. 40.
    Leidl R, von der Schulenburg J-M, Wasem J (1999) Approaches and methods of economic evaluation-an international perspective (in German). Health Technology Assessment, vol 9. Nomos: Baden-BadenGoogle Scholar
  41. 41.
    Bennett WG, Inoue Y, Beck JR et al. (1997) Estimates of the cost-effectiveness of a single course of interferon-alpha 2b in patients with histologically mild chronic hepatitis C. Ann Intern Med 127:855–865PubMedGoogle Scholar
  42. 42.
    Wong JB, Poynard T, Ling MH et al. (2000) Cost-effectiveness of 24 or 48 weeks of interferon alpha-2b alone or with ribavirin as initial treatment of chronic hepatitis C. International Hepatitis Interventional Therapy Group. Am J Gastroenterol 95:1524–1530CrossRefPubMedGoogle Scholar
  43. 43.
    Sennfält K, Reichard O, Hultkrantz R et al. (2001) Cost-effectiveness of interferon alfa-2b with and without ribavirin as therapy for chronic hepatitis C in Sweden. Scand J Gastroenterol 36:870–876CrossRefPubMedGoogle Scholar
  44. 44.
    Sagmeister M, Wong JB, Mullhaupt B, Renner EL (2001) A pragmatic and cost-effective strategy of a combination therapy of interferon alpha-2b and ribavirin for the treatment of chronic hepatitis C. Eur J Gastroenterol Hepatol 13:483–488CrossRefPubMedGoogle Scholar
  45. 45.
    Stein K, Rosenberg W, Wong J (2002) Cost effectiveness of combination therapy for hepatitis C: a decision analytic model. Gut 50:253–258CrossRefPubMedGoogle Scholar
  46. 46.
    Wong JB, Nevens F (2002) Cost-effectiveness of peginterferon alfa-2b plus ribavirin compared to interferon alfa-2b plus ribavirin as initial treatment of chronic hepatitis C in Belgium. Acta Gastroenterol Belg 65:110–111PubMedGoogle Scholar
  47. 47.
    Siebert U, Sroczynski G, Rossol S et al. (2003) Cost effectiveness of peginterferon alpha-2b plus ribavirin versus interferon alpha-2b plus ribavirin for initial treatment of chronic hepatitis C. Gut 52:425–432CrossRefPubMedGoogle Scholar
  48. 48.
    Buti M, Medina M, Casado MA et al. (2003) A cost-effectiveness analysis of peginterferon alfa-2b plus ribavirin for the treatment of naive patients with chronic hepatitis C. Aliment Pharmacol Ther 17:687–694PubMedGoogle Scholar
  49. 49.
    Canadian Coordinating Office for Health Technology Assessment (1997) Guidelines for economic evaluation of pharmaceuticals, 2nd edn. CCOHTA: OttawaGoogle Scholar
  50. 50.
    Schulenburg JM Graf von der, Hoffmann C (2000) Review of European guidelines for economic evaluation of medical technologies and pharmaceuticals. Eur J Health Econ 1:2–8Google Scholar
  51. 51.
    Mason J, Drummond MF, Torrance G (1993) Some guidelines on the use of cost effectiveness league tables. BMJ 306:570–572PubMedGoogle Scholar
  52. 52.
    Pauker SP, Pauker SG (1987) The amniocentesis decision: ten years of decision analytic experience. Birth defects. Original Article Series 23:151–169Google Scholar
  53. 53.
    Pauker SP, Pauker SG (1979) The amniocentesis decision: an explicit guide for parents. Birth defects. Original Article Series 15:289–324Google Scholar
  54. 54.
    Krahn MD, Mahoney JE, Eckman MH et al. (1994) Screening for prostate cancer. A decision analytic view. JAMA 272:773–780PubMedGoogle Scholar
  55. 55.
    Sonnenberg A, Delco F (2002) Cost-effectiveness of a single colonoscopy in screening for colorectal cancer. Arch Intern Med 162:163–168CrossRefPubMedGoogle Scholar
  56. 56.
    Eddy DM (1987) The frequency of cervical cancer screening. Comparison of a mathematical model with empirical data. Cancer 60:1117–22PubMedGoogle Scholar
  57. 57.
    Goldie SJ, Kuhn L, Denny L et al. (2001) Policy analysis of cervical cancer screening strategies in low-resource settings: clinical benefits and cost-effectiveness. JAMA 285:3107–3115 (erratum appears in 286:1026)Google Scholar
  58. 58.
    Bech GJ, De Bruyne B, Pijls NH et al. (2001) Fractional flow reserve to determine the appropriateness of angioplasty in moderate coronary stenosis: a randomized trial. Circulation 103:2928–2934PubMedGoogle Scholar
  59. 59.
    Kuntz KM, Fleischmann KE, Hunink MGM, Douglas PS (1999) Cost-effectiveness of diagnostic strategies for patients with chest pain. Ann Intern Med 130:709–718PubMedGoogle Scholar
  60. 60.
    McHutchison JG, Manns M, Patel K et al. (2002) Adherence to combination therapy enhances sustained response in genotype-1-infected patients with chronic hepatitis C. Gastroenterology 123:1061–1089CrossRefPubMedGoogle Scholar
  61. 61.
    Claxton K, Neumann PJ, Araki S, Weinstein MC (2001) Bayesian value-of-information analysis. An application to a policy model of Alzheimer's disease. Int J Technol Assess Health Care 17:38–55CrossRefPubMedGoogle Scholar
  62. 62.
    Weinstein MC, Coxson PG, Williams LW et al. (1987) Forecasting coronary heart disease incidence, mortality, and cost: the Coronary Heart Disease Policy Model. Am J Public Health 77:1417–1426PubMedGoogle Scholar
  63. 63.
    Goldman L, Weinstein MC, Williams LW (1989) Relative impact of targeted versus populationwide cholesterol interventions on the incidence of coronary heart disease. Projections of the Coronary Heart Disease Policy Model. Circulation 80:254–260PubMedGoogle Scholar
  64. 64.
    Edelson JT, Weinstein MC, Tosteson AN et al. (1990) Long-term cost-effectiveness of various initial monotherapies for mild to moderate hypertension. JAMA 263:407–413CrossRefPubMedGoogle Scholar
  65. 65.
    Goldman L, Weinstein MC, Goldman PA, Williams LW (1991) Cost-effectiveness of HMG-CoA reductase inhibition for primary and secondary prevention of coronary heart disease. JAMA 265:1145–1151CrossRefPubMedGoogle Scholar
  66. 66.
    Tsevat J, Weinstein MC, Williams LW et al. (1991) Expected gains in life expectancy from various coronary heart disease risk factor modifications. Circulation 83:1194–1201PubMedGoogle Scholar
  67. 67.
    Hunink MG, Goldman L, Tosteson AN et al. (1997) The recent decline in mortality from coronary heart disease, 1980–1990. The effect of secular trends in risk factors and treatment. JAMA 277:535–542CrossRefPubMedGoogle Scholar
  68. 68.
    Tosteson AN, Weinstein MC, Hunink MG et al. (1997) Cost-effectiveness of populationwide educational approaches to reduce serum cholesterol levels. Circulation 95:24–30PubMedGoogle Scholar
  69. 69.
    Goldman L, Coxson P, Hunink MG et al. (1999) The relative influence of secondary versus primary prevention using the National Cholesterol Education Program Adult Treatment Panel II guidelines. J Am Coll Cardiol 34:768–776CrossRefPubMedGoogle Scholar
  70. 70.
    Prosser LA, Stinnett AA, Goldman PA et al. (2000) Cost-effectiveness of cholesterol-lowering therapies according to selected patient characteristics. Ann Intern Med 132:769–779PubMedGoogle Scholar
  71. 71.
    Phillips KA, Shlipak MG, Coxson P et al. (2000) Health and economic benefits of increased beta-blocker use following myocardial infarction. JAMA 284:2748–2754CrossRefPubMedGoogle Scholar
  72. 72.
    Goldman L, Phillips KA, Coxson P et al. (2001) The effect of risk factor reductions between 1981 and 1990 on coronary heart disease incidence, prevalence, mortality and cost. J Am Coll Cardiol 38:1012–1017CrossRefPubMedGoogle Scholar
  73. 73.
    Tice JA, Ross E, Coxson PG et al. (2001) Cost-effectiveness of vitamin therapy to lower plasma homocysteine levels for the prevention of coronary heart disease: effect of grain fortification and beyond. JAMA 286:936–943CrossRefPubMedGoogle Scholar
  74. 74.
    Tsevat J (1992) Impact and cost-effectiveness of smoking interventions. Am J Med 93:43S–47SPubMedGoogle Scholar
  75. 75.
    Goldman L, Gordon DJ, Rifkind BM et al. (1992) Cost and health implications of cholesterol lowering. Circulation 85:1960–1968PubMedGoogle Scholar
  76. 76.
    Tosteson AN, Weinstein MC, Williams LW, Goldman L (1990) Long-term impact of smoking cessation on the incidence of coronary heart disease. Am J Public Health 80:1481–1486PubMedGoogle Scholar
  77. 77.
    Goldman L, Goldman PA, Williams LW, Weinstein MC (1993) Cost-effectiveness considerations in the treatment of heterozygous familial hypercholesterolemia with medications. Am J Cardiol 72:75D–79DPubMedGoogle Scholar
  78. 78.
    Stinnett A, Mittleman A, Weinstein M et al. (1996) The cost-effectiveness of dietary and pharmacologic therapy for cholesterol reduction in adults. In: Gold MR, Siegel JE, Russell LB, Weinstein MC (eds) Cost-effectiveness in health and medicine: report on the panel of cost-effectiveness in health and medicine. Oxford University Press: New York, 349–391Google Scholar
  79. 79.
    Anonymous (1993) Summary of the second report of the National Cholesterol Education Program (NCEP) Expert Panel on Detection, Evaluation, and Treatment of High Blood Cholesterol in Adults (Adult Treatment Panel II). JAMA 269:3015–3023PubMedGoogle Scholar
  80. 80.
    Tengs TO, Osgood ND, Lin TH (2001) Public health impact of changes in smoking behavior: results from the Tobacco Policy Model. Med Care 39:1131–1141CrossRefPubMedGoogle Scholar
  81. 81.
    Tengs TO, Osgood ND, Chen LL (2001) The cost-effectiveness of intensive national school-based anti-tobacco education: results from the tobacco policy model. Prev Med 33:558–570CrossRefPubMedGoogle Scholar
  82. 82.
    Perleth M, Busse R (2000) Health technology assessment in Germany. Status, challenges, and development. Int J Technol Assess Health Care 16:412–428CrossRefPubMedGoogle Scholar
  83. 83.
    Sassi F (2000) The European way to health technology assessment. Lessons from an evaluation of EUR-ASSESS. Int J Technol Assess Health Care 16:282–290CrossRefPubMedGoogle Scholar
  84. 84.
    Russell LB, Gold MR, Siegel JE et al. (1996) The role of cost-effectiveness analysis in health and medicine. Panel on Cost-Effectiveness in Health and Medicine. JAMA 276:1172–1177PubMedGoogle Scholar
  85. 85.
    Marckmann G, Siebert U (2002) Kosteneffektivität als Allokationskriterium in der Gesundheitsversorgung. Z Med Ethik 48:171–190Google Scholar

Copyright information

© Springer-Verlag 2003

Authors and Affiliations

  1. 1.Harvard Center for Risk AnalysisHarvard School of Public HealthBostonUSA
  2. 2.Bavarian Public Health Research and Coordinating Center, Institute of Medical Informatics, Biometry, and EpidemiologyLudwig Maximilian University MunichGermany
  3. 3.Harvard Center for Risk AnalysisBostonUSA

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