Economic endpoints in clinical trials

Statistical Methods in Medical Research

Volumn 13, Issue 2, 2004, Pages 157-176

  Cook, John ^a   Drummond, Michael ^b   Heyse, Joseph F ^a  

^a MERCK RESEARCH LABORATORIES   (United States)

^b UNIVERSITY OF YORK   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ENALAPRIL; PLACEBO;

ANALYTIC METHOD; CLINICAL STUDY; CLINICAL TRIAL; CONGESTIVE HEART FAILURE; COST BENEFIT ANALYSIS; COST EFFECTIVENESS ANALYSIS; DATA ANALYSIS; HEALTH CARE PLANNING; HEALTH CARE UTILIZATION; HUMAN; METHODOLOGY; REVIEW; SAMPLE SIZE; STATISTICAL ANALYSIS;

CLINICAL TRIALS; COST-BENEFIT ANALYSIS; DATA COLLECTION; ENDPOINT DETERMINATION; HUMANS; UNITED STATES;

EID: 1642292544     PISSN: 09622802     EISSN: None     Source Type: Journal    
DOI: 10.1191/0962280204sm359ra     Document Type: Review

References (48)

1. Hjelmgren J, Berggren F, Andersson F. Health economic guidelines - similarities, differences and some implications. Value in Health 2001; 4 (3): 447-54.
2. National Institute for Clinical Excellence. Guidance for manufacturers and sponsors. Technology appraisals process series no 5. London: National Institute for Clinical Excellence, 2001.
3. Academy of Managed Care Pharmacy. Format for formulary submissions. Version 2.0. AMCP, October 2002.
4. Coyle D, Davies LM, Drummond MF. Trials and tribulations: emerging issues in designing economic evaluations alongside clinical trials. International Journal of Technology Assessment in Health Care 1998; 14 (1): 135-44.
5. Drummond MF, O'Brien BJ, Stoddart GL, Torrance GW. Methods for the economic evaluation of healthcare programmes, Chapter 3. Oxford: Oxford University Press, 1997.
6. Rabin R, De Charro F. EQ-5D: A measure of health status from the EuroQoL Group. Annals of Medicine 2001; 33: 337-43.
7. Furlong WJ, Feeny DH, Torrance GW, Barr RD. The Health Utilities Index (HUI) system for assessing health-related quality of life in clinical studies. Annals of Medicine 2001; 33: 375-84.
8. Manca A, Sculpher MJ, Ward K, Hilton P. A cost-utility analysis of tension-free vaginal tape versus colposuspension for primary urodynamic stress incontinence. British Journal of Obstetrics and Gynaecology 2003; 110: 255-62.
9. Drummond MF, Pang F. Transferability of economic evaluation results. In: Drummond MF, McGuire AL, eds. Economic evaluation of health care: Merging theory tvith practice. Oxford: Oxford University Press, 2001, pp 256-76.
10. Cook JR, Drummond MF, Glick H, Heyse JF. Assessing the appropriateness of combining economic data from multinational clinical trials. Statistics in Medicine 2003; 22: 1955-76.
11. Efron B, Tibshirani RJ. An introduction to the bootstrap. New York: Chapman & Hall, 1993.
12. Westfall PH, Young SS. Resampling-based multiple testing. New York: John Wiley & Sons, 1993.
13. Lambert D. Zero-inflated Poisson regression, with an application to defects in manufacturing. Technometrics 1992; 34 (1): 1-14.
14. Mullahy J. Heterogeneity, excess zeros and the structure of count data. Journal of Applied Econometrics 1997; 1: 337-50.
15. Liang KY, Zeger SL. Longitudinal data analysis using generalized linear models. Biometrika 1996; 73: 13-22.
16. Stokes ME, Davies CS, Koch GG. Categorical data analysis using the SAS® system. North Carolina, USA: SAS Institute, 1995.
17. Itzler R, Dasbach E, Koch G, Heyse JF. Using longitudinal models for the analysis of resource use data: The case of asthma. Economic assessment in clinical trials. Drug Information Association Meeting, Orlando, Florida, November 1999.
18. Chang MN, Guess HA, Heyse JF. Reduction in burden of illness: a new efficacy measure for prevention trials. Statistics in Medicine 1994; 13: 1807-14.
19. Duan N, Manning WG, Morris CN, Newhouse JP. A comparison of alternative models for the demand for medical care. Journal of Business and Economic Statistics 1993; 1: 115-26.
20. Lipscomb J, Ancukiewicz M, Parmigiani G, Hasselblad V, Samsa G, Matchar DB. Predicting the cost of illness: A comparison of alternative models applied to stroke. Medical Decision Making 1998; 18 (Suppl): S39-S56.
21. Jimenez-Martin S, Labeaga JM, Martinez-Granado M. Latent class versus two-part models in the demand for physician services across the European Union. Health Economics 2002; 11: 301-21.
22. Dudley RA, Harrell FE, Smith LR, Mark DB, Califf RM, Pryor DB, Glower D, Lipscomb J, Hlatky M. Comparison of analytic models for estimating the effect of clinical factors on the cost of coronary artery bypass graft surgery. Journal of Clinical Epidemiology 1993; 46: 261-71.
23. Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. Journal of the American Statistical Association 1958; 53: 457-81.
24. Fenn P, McGuire A, Phillips V, Backhouse M, Jones D. The analysis of censored treatment cost data in economic evaluation. Medical Care 1995; 33: 851-63.
25. Carides GW, Heyse JF. Nonparametric estimation of the parameters of cost distributions in the presence of right-censoring. ASA Proceedings of the Biopharmaceutical Section. Alexandria, VA: American Statistical Association, 1996, pp 186-91.
26. Etzioni RD, Feuer EJ, Sullivan S, Lin D, Hu C, Ramsey SD. On the use of survival analysis techniques to estimate medical care costs. Journal of Health Economics 1999; 18: 365-80.
27. Carides GW. Estimation of mean treatment costs in the presence of right-censoring. PhD dissertation, Department of Statistics, Temple University, 1998.
28. Lin DY, Fewer EJ, Etzioni R, Wax Y. Estimating medical costs from incomplete follow-up data. Biometrics 1997; 53: 113-28.
29. Carides GW, Heyse JF, Iglewicz B. A regression-based method for estimating mean treatment cost in the presence of right-censoring. Biostatistics 2000; 1: 299-313.
30. Eubanks RL. Nonparametric regression and spline smoothing, 2nd edition, chapters 1 and 5. New York: Marcel Dekker, 1999.
31. Bang H, Tsiatis AA. Estimating medical costs with censored data. Biometrika 2000; 87: 329-43.
32. Herman WH, Shzhinfar S, Carides GW, Dasbach EJ, Gerth WC, Alexander CM, Cook JR, Keene WF, Brenner BM, for the RENAAL Investigators. Losartan reduces the cost associated with diabetic end-stage renal disease. Diabetes Care 2003; 26: 683-87.
33. O'Brien BJ, Drummond MF, LaBelle RJ, William A. In search of power and significance: Issues in the design and analysis of stochastic cost-effectiveness studies in health care. Medical Care 1994; 32: 150-63.
34. Wakker P, Klaassen MP. Confidence intervals for cost/effectiveness ratios. Health Economics 1995; 4: 373-81.
35. Chaudhary MA, Stearns SC. Estimating confidence intervals for cost effectiveness ratios: An example from a randomized trial. Statistics in Medicine 1996; 15: 1447-58.
36. Laska EM, Meisner M, Siegel C. Statistical inference for cost-effectiveness ratios. Health Economics 1997; 6: 229-42.
37. Fieller EC. Some problems in interval estimation. Journal of the Royal Statistical Society, Series B 1954; 16: 175-85.
38. Polsky D, Glick HA, Willke R, Schulman R. Confidence intervals for cost-effectiveness ratios: A comparison of four methods. Health Economics 1997; 6: 243-52.
39. Cook JR, Heyse JF. Use of an angular transformation for ratio estimation in cost-effectiveness analysis. Statistics in Medicine 2000; 19: 2989-3003.
40. vanHout BA, Al MJ, Gordon GS, Rutten FFH. Costs, effects and C/E-ratios alongside a clinical trial. Economic Evaluations 1994; 3: 309-19.
41. Stinnett AA, Mullahy J. Net health benefits: A new framework for the analysis of uncertainty in cost-effectiveness analysis. Medical Decision Making 1998; 18 (Suppl): S68-S80.
42. The SOLVD Investigators. Effect of enalapril on survival in patients with reduced left ventricular ejection fractions and congestive heart failure. New England Journal of Medicine 1991; 325: 303-10.
43. Glick H, Cook JR, Kinosian B, Pitt B, Bourassa MG, Pouleur H, Gerth W. Costs and effects of enalapril therapy in patients with symptomatic heart failure: An economic analysis of the Studies of Left Ventricular Dysfunction (SOLVD) treatment trial. Journal of Cardiac Failure 1995; 1: 3171-80.
44. Heitjan DF, Moskowitz AJ, Whang W. Bayesian estimation of cost-effectiveness ratios from clinical trials. Health Economics 1999; 8: 191-201.
45. Briggs AH. A Bayesian approach to stochastic cost-effectiveness analysis. Health Economics 1999; 8: 257-61.
46. O'Hagan A, Stevens JW, Montmartin J. Bayesian cost-effectiveness analysis from clinical trial data. Statistics in Medicine 2001; 20: 733-53.
47. O'Hagan A, Stevens JW. Bayesian methods for design and analysis of cost-effectiveness trials in the evaluation of health care technologies. Statistical Methods in Medical Research 2002; 11: 469-90.
48. Willke RJ, Glick HA, Polsky D, Schulman K. Estimating country-specific cost-effectiveness from multinational clinical trials. Health Economics 1998; 7: 481-93.