Prediction models used in support of clinical and health policy decision making often need to consider the course of a disease over an extended period of time, and draw evidence from a broad knowledge base, including epidemiologic cohort and case control studies, randomized clinical trials, expert opinions, and more. This paper is a brief introduction to these complex decision models, their relation to Bayesian decision theory, and the tools typically used to describe the uncertainties involved. Concepts are illustrated throughout via a simplified tutorial.
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References
1.
Evidence-Based Medicine Working Group . Evidence-based medicine: A new approach to teaching the practice of medicine . JAMA1992; 268: 2420-2420 .
2.
Sackett DL , Rosenberg WMC , Gray JAM , Haynes RB , Richardson WS.Evidence based medicine: what it is and what it isn’t . British Medical Journal1996; 312: 71-72 .
3.
Matchar DB , Duncan P , Samsa Get al.The stroke prevention patient outcomes research team: goals and methods . Stroke1993; 24 (12): 2135-2142 .
4.
Samsa GP , Reutter RA , Parmigiani G , Ancukiewicz M , Lipscomb J , Matchar DB.Performing cost-effectiveness analyses by integrating randomized trials with a comprehensive decision model: application to the treatment of acute ischemic stroke . Journal of Clinical Epidemiology1999; 52: 259-271 .
5.
Weinstein MC , Coxson PG , Williams LW , Pass TM , Stason WB , Goldman L.Forecasting coronary heart disease incidence, mortality, and cost: the coronary heart disease policy model . American Journal of Public Health1987; 77 (11): 1417-1426 .
6.
Matchar DB , Samsa GP , Matthews JRet al.The stroke prevention policy model (SPPM): Linking evidence and clinical decisions . Annals of Internal Medicine1997; 127(8S): 704-711 .
7.
Parmigiani G.Modeling in medical decision making. Chichester: Wiley , 2002.
8.
Raiffa H , Schleifer R.Applied statistical decision theory. Boston: Harvard University Press , 1961.
9.
DeGroot MH.Optimal statistical decisions. McGraw-Hill: New York , 1970.
10.
Lindley DV.Making decisions, 2nd edn.John Wiley & Sons: New York , 1985.
11.
O’Hagan A.Kendall’s advanced theory of statistics. Volume 2B: Bayesian Inference. Cambridge: Cambridge University Press , 1994.
12.
Berry DA , Stangl DK (eds.) Bayesian Biostatistics, volume 151 of Statistics: Textbooks and Monographs. Marcel Dekker: New York1996.
13.
Parmigiani G , Samsa GP , Ancukiewicz M , Lipscomb J , Hasselblad V , Matchar DB.Assessing uncertainty in cost-effectiveness analyses: Application to a complex decision model . Medical Decision Making1997; 17: 390-401 .
14.
Manton KG , Stallard E.Chronic Disease Modelling. Charles Griffin: London , 1988.
15.
Bhat UN.Elements of Applied Stochastic Processes, 2nd edn.John Wiley & Sons: New York , 1984.
16.
Keeney RL , Raifa HA , Meyer RFC.Decisions with Multiple Objectives: Preferences and Value Tradeoffs. John Wiley & Sons: New York , 1976.
17.
Draper D.Assessment and propagation of model uncertainty (disc: P71-97) . Journal of the Royal Statistical Society, Series B, Methodological1995; 57: 45-70 .
18.
Fishburn PC.Utility Theory for Decision Making. R. E. Krieger Pub. Co.: New York , 1979.
19.
Chapman GB , Sonnenberg F.Decision Making in Health Care: Theory, Psychology, and Applications. Boston: Cambridge University Press , 2000.
20.
Pliskin JS , Shepard D , Weinstein MC.Utility functions for life years and health status: Theory, assessment, and application . Operations Research1980; 28: 206-224 .
21.
Torrance G. A generalized cost-effectiveness model for the evaluation of health programs, Doctoral dissertation, State University of New York at Buffalo, 1971.
22.
Stinnett AA , Mullahy J.Net health benefits: a new framework for the analysis of uncertainty in cost-effectiveness analysis . Medical Decision Making1998; 18(2 Suppl): S68-S80 .
23.
O’Hagan A , Stevens JW.The probability of cost-effectiveness . BMC Medical Research Methodology2002; 2: 5-5 .
24.
Parmigiani G.On optimal screening ages . Journal of the American Statistical Association1993; 88: 622-628 .
25.
Doubilet P , Weinstein MC , McNeil BJ.Use and misuse of the term ‘cost effective’ in medicine . New England Journal of Medicine1986; 314(4): 253-256 .
26.
Sloan F.Valuing Health Care. Cambridge: Cambridge University Press , 1995.
27.
Kaplan R.Utility assessment for estimating quality-adjusted life years. In Sloan, F ed., Valuing health care, Cambridge University Press: Cambridge , 1995, pp. 157-190.
28.
Weinstein MC , Manning Jr WG.Theoretical issues in cost-effectiveness analysis . Journal of Health Economics1997; 16: 121-128 .
29.
Manning WG , Fryback DG , Weinstein MC.Reflecting uncertainty in cost-effectiveness analyses. In: Gold et al., Cost-effectiveness in health and medicine, Oxford University Press: Oxford , 1996.
30.
Thompson SG , Barber JA.How should cost data in pragmatic randomised trials be analysed?British Medical Journal2000; 320: 1197-1200 .
31.
Gold MR , Siegel JE , Russell LB , Weinstein MC.Cost-Effectiveness in Health and Medicine. Oxford: Oxford University Press , 1996.
32.
Mullahi J , Manning WG.Statistical issues in cost-effectiveness analyses. In: Gold et al., 1996.
33.
Siegel C , Laska E , Meisner M.Statistical methods for cost-effectiveness analyses . Controlled Clinical Trials1996; 17: 387-406 .
34.
Bernardo JM , Smith AFM.Bayesian Theory. Wiley: New York , 1994.
35.
Kreps DM.Notes on the Theory of Choice. Westview Press: Boulder , 1988.
36.
Claxton K.The irrelevance of inference: a decision-making approach to the stochastic evaluation of health care technologies . Journal of Health Economics1999; 18: 341-364 .
37.
Parmigiani G , Ancukiewicz M , Matchar DB.Decision models in clinical recommendations development: the Stroke Prevention Policy Model. In: Berry DA , Stangl DK , eds. Bayesian Biostatistics, volume 151 of Statistics: Textbooks and Monographs. Marcel Dekker: New York , 1996, pp. 207-233.
38.
Gilks WR , Richardson S , Spiegelhalter DJ.Markov Chain Monte Carlo in Practice. London: Chapman and Hall , 1996.
39.
Gardiner J , Hogan A , Holmes-Rovner M , Rovner D , Griffith L , Kupersmith J.Confidence intervals for cost-effectiveness ratios . Medical Decision Making1995; 15: 254-263 .
40.
Bult JR , Hunink MGM , de Vries JA , Weinstein MC.A fully stochastic cost-effectiveness analysis . Medical Decision Making1995; 15: 415-415 .
41.
Doubilet P , Begg CB , Weinstein MC , Braun P , McNeil BJ.Probabilistic sensitivity analysis using Monte Carlo simulation. A practical approach . Medical Decision Making1985; 5(2): 157-177 .
42.
Critcheld GC , Willard KE.Probabilistic analysis of decision trees using Monte Carlo simulation . Medical Decision Making1986; 6(2): 85-92 .
43.
Briggs AH.A Bayesian approach to stochastic cost-effectiveness analysis . Health Economics8: 257-261 .
44.
O’Hagan A , Stevens JW.A framework for cost-effectiveness analysis from clinical trial data . Health Economics2001; 10: 302-315 .
45.
Oakley J , O’Hagan A.Probabilistic sensitivity analysis of complex models: a Bayesian approach. Report 525=02, Department of Probability and Statistics, University of Sheffield, Sheffield , 2002.
46.
Saltelli A , Chan K , Scott M.Sensitivity Analysis. Wiley: New York , 2000.
47.
O’Hagan A , Stevens JW , Montmartin J.Inference for the C=E acceptability curve and C=E ratio . Pharmaco Economics2000; 17: 339-349 .
48.
O’Hagan A , Stevens JW , Montmartin J.Bayesian cost effectiveness analysis from clinical trial data . Statistics in Medicine2001; 20: 733-753 .
49.
Heitjan DF , Moskowitz AJ , Whang W.Bayesian estimation of cost-effectiveness ratios from clinical trials . Health Economics1999; 8(3): 191-201 .
50.
van Hout BA , Al MJ , Gordon GS , Rutten F.Costs, effects and C=E ratios alongside a clinical trial . Health Economics1994; 3: 309-319 .
