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Impact of uncertainty on cost-effectiveness analysis of medical strategies: The case of high-dose chemotherapy for breast cancer patients

Published online by Cambridge University Press:  04 August 2005

Patricia Marino
Affiliation:
University of Méditerranée and INSERM U379
Carole Siani
Affiliation:
University of Méditerranée and INSERM U379
Henri Roché
Affiliation:
Institut Claudius Régaud
Jean-Paul Moatti
Affiliation:
University of Méditerranée and INSERM U379

Abstract

Objectives: The object of this study was to determine, taking into account uncertainty on cost and outcome parameters, the cost-effectiveness of high-dose chemotherapy (HDC) compared with conventional chemotherapy for advanced breast cancer patients.

Methods: An analysis was conducted for 300 patients included in a randomized clinical trial designed to evaluate the benefits, in terms of disease-free survival and overall survival, of adding a single course of HDC to a four-cycle conventional-dose chemotherapy for breast cancer patients with axillary lymph node invasion. Costs were estimated from a detailed observation of physical quantities consumed, and the Kaplan–Meier method was used to evaluate mean survival times. Incremental cost-effectiveness ratios were evaluated successively considering disease-free survival and overall survival outcomes. Handling of uncertainty consisted in construction of confidence intervals for these ratios, using the truncated Fieller method.

Results: The cost per disease-free life year gained was evaluated at 13,074€, a value that seems to be acceptable to society. However, handling uncertainty shows that the upper bound of the confidence interval is around 38,000€, which is nearly three times higher. Moreover, as no difference was demonstrated in overall survival between treatments, cost-effectiveness analysis, that is a cost minimization, indicated that the intensive treatment is a dominated strategy involving an extra cost of 7,400€, for no added benefit.

Conclusions: Adding a single course of HDC led to a clinical benefit in terms of disease-free survival for an additional cost that seems to be acceptable, considering the point estimate of the ratio. However, handling uncertainty indicates a maximum ratio for which conclusions have to be discussed.

Type
GENERAL ESSAYS
Copyright
© 2005 Cambridge University Press

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References

Agro K, Bradley C, Mittmann N, et al. 1997 Sensitivity analysis in health economic and pharmacoeconomic studies: An appraisal of the literature. Pharmacoeconomics. 11: 7588.Google Scholar
Astier M, Mayordomo J, Abad J, et al. 2000 Cost Analysis of high-dose chemotherapy and blood stem-cell support in patients with solid tumors. Ann Oncol. 11: 603606.Google Scholar
Bercez C, Lebrun T, Bonneterre ME, et al. 1999 Advanced breast cancer: An evaluation of the cost of recurrence. Bull Cancer. 86: 585590.Google Scholar
Briggs A, Sculpher M, Buxton M. 1994 Uncertainty in the economic evaluation of health care technologies: The role of sensitivity analysis. Health Econ. 3: 95104.Google Scholar
Briggs AH, Fenn P. 1998 Confidence intervals or surfaces? Uncertainty on the cost-effectiveness plane. Health Econ. 7: 723740.Google Scholar
Briggs AH, Sculpter M. 1995 Sensitivity analysis in economic evaluation: The review of published studies. Health Econ. 4: 355371.Google Scholar
Briggs AH, Wonderling DE, Mooney CZ. 1997 Pulling cost-effectiveness analysis up by its bootstrap: A non-parametric approach to confidence interval estimation. Health Econ. 6: 327340.Google Scholar
Bull AA, Meyerowitz BE, Hart S, et al. 1999 Quality of life in women with recurrent breast cancer. Breast Cancer Res Treat. 54: 4757.Google Scholar
Cella DF, Mahon SM, Donovan MI. 1990 Cancer recurrence as a traumatic event. Behav Med. 16: 1522.Google Scholar
Chaudhary MA, Stearns SC. 1996 Estimating confidence intervals for cost-effectiveness ratios: An example from a randomized trial. Stat Med. 15: 14471458.Google Scholar
De Rosa L, Lalle M, Pandolfi A, Pescador L. 2001 Costs of high-dose chemotherapy and peripheral blood progenitor cell autograft for breast cancer. Bone Marrow Transplant. 27: 10311035.Google Scholar
Drummond M. 1995 Economic analysis alongside clinical trials: Problems and potential. J Rheumatol. 22: 14031407.Google Scholar
Drummond M, O'Brien B. 1995 Economic analysis alongside clinical trials: Practical considerations. The Economics Workgroup. J Rheumatol. 22: 14181419.Google Scholar
Drummond M, O'Brien B, Stoddart G. 1997. Methods for the economic evaluation of health care programs. 2nd ed. Oxford: Oxford Medical Publications, Oxford University Press;
Finkler SA. 1982 The distinction between cost and charges. Ann Intern Med. 96: 102109.Google Scholar
Garber AM. Advances in CE analysis of health interventions. In: Culyer AJ, Newhouse JP, eds. Handbook of health economics, Vol. 1A. Amsterdam: Elsevier; 2000 181221.
Gold MR, Siegel JE, Russell LB, Weinstein MC. 1996. Cost-effectiveness in health and medicine. Oxford: Oxford University Press;
Hanson Frost M, Suman VJ, Rummans TA, et al. 2000 Physical, psychological and social well-being of women with breast cancer: The influence of disease phase. Psychooncology. 9: 221231.Google Scholar
Hillner BE. 1998 Review of cost-effectiveness assessments of chemotherapy in adjuvant and advanced breast cancer. Anticancer Drugs. 9: 843847.Google Scholar
Hillner BE, Smith TJ, Desch CE. 1993 Assessing the cost effectiveness of adjuvant therapies in early breast cancer using a decision analysis model. Breast Cancer Res Treat. 25: 97105.Google Scholar
Jenkins PL, May VE, Hughes LE. 1991 Psychological morbidity associated with local recurrence of breast cancer. Int J Psychiatry Med. 21: 149155.Google Scholar
Johannesson M. 1996. Theory and methods of economic evaluation of health care. Amsterdam: Kluwer Academic Publishers;
Le Corroller A, Moatti J, Chabannon C, et al. 1999 Optimization of peripheral blood stem cell collection by leukopheresis. Interaction between economic and clinical assessment of an innovation. Int J Technol Assess Health Care. 15: 161172.Google Scholar
Meisenberg BR, Miller WE, McMillan R, et al. 1997 Outpatient high-dose chemotherapy with autologous stem-cell rescue for hematologic and nonhematologic malignancies. J Clin Oncol. 15: 1117.Google Scholar
O'Brien BJ, Drummond MF, Llabelle BJ, Willan A. 1994 In search of power and significance: Issues in the design and analysis of stochastic cost-effectiveness studies in health care. Med Care. 32: 150163.Google Scholar
Peters WP, Ross M, Vredenburgh JJ, et al. 1994 The use of intensive clinic support to permit outpatient autologous bone marrow transplantation for breast cancer. Semin Oncol. 21: 2531.Google Scholar
Pinguet F, Savelli K, Petit I, et al. 2001 Pharmaco-economic study of intensive chemotherapy with peripheral blood progenitor cell support for advanced breast cancer. Oncol Rep. 8: 10131017.Google Scholar
Republique Francaise. Decrêt, arrêtés, circulaires. Journal Officiel de la République Française. 1999 13281329.
Rodenhuis S. 2000 The status of high-dose chemotherapy in breast cancer. Oncologist. 5: 369375.Google Scholar
Rodenhuis S, Bontenbal M, Beex LV, et al. 2003 High-dose chemotherapy with hematopoietic stem-cell rescue for high-risk breast cancer. N Engl J Med. 349: 716.Google Scholar
Sculpher M, Drummond M, Buxton M. 1997 The iterative use of economic evaluation as part of the process of health technology assessment. J Health Serv Res Policy. 2: 2630.Google Scholar
Siani C, Moatti J. 2003 The handling of uncertainty in economic evaluations of health care strategies. Rev Epidemiol Sante Publique. 51: 255276.Google Scholar
Smith TJ, Hillner BE. 2000 Tamoxifen should be cost-effective in reducing breast cancer risk in high-risk women. J Clin Oncol. 18: 284286.Google Scholar
Sutherland HJ, Lockwood GA, Boyd NF. 1990 Ratings of the importance of quality of life variables: Therapeutic implications for patients with metastatic breast cancer. J Clin Epidemiol. 43: 661666.Google Scholar
Towse A, Prirchard C, Devlin N. 2002. Cost-effectiveness thresholds. Economic and ethical issues. London: King's fund and Office of Health Economics;
UCANSS. 1999. Nomencalture Générale des Actes Professionnels. Paris.
Van Hout BA, Al MJ, Gordon GS, Rutten FF. 1994 Costs, effects and C/E ratios alongside a clinical trial. Health Econ. 3: 309319.Google Scholar
Willan AR, O'Brien BJ. 1996 Confidence intervals for cost-effectiveness ratios: An application of Fieller's theorem. Health Econ. 5: 297305.Google Scholar