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Sample size and power issues in estimating incremental cost-effectiveness ratios from clinical trials data

  • Andrew R. Willan

    (Department of Clinical Epidemiology and Biostatistics, McMaster University and Centre for Evaluation of Medicines, St. Joseph's Hospital, Canada)

  • Bernie J. O'Brien

    (Department of Clinical Epidemiology and Biostatistics, McMaster University and Centre for Evaluation of Medicines, St. Joseph's Hospital, Canada)

Registered author(s):

    It is becoming increasingly more common for a randomized controlled trial of a new therapy to include a prospective economic evaluation. The advantage of such trial-based cost-effectiveness is that conventional principles of statistical inference can be used to quantify uncertainty in the estimate of the incremental cost-effectiveness ratio (ICER). Numerous articles in the recent literature have outlined and compared various approaches for determining confidence intervals for the ICER. In this paper we address the issue of power and sample size in trial-based cost-effectiveness analysis. Our approach is to determine the required sample size to ensure that the resulting confidence interval is narrow enough to distinguish between two regions in the cost-effectiveness plane: one in which the new therapy is considered to be cost-effective and one in which it is not. As a result, for a given sample size, the cost-effectiveness plane is divided into two regions, separated by an ellipse centred at the origin, such that the sample size is adequate only if the truth lies on or outside the ellipse. Copyright © 1999 John Wiley & Sons, Ltd.

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    Article provided by John Wiley & Sons, Ltd. in its journal Health Economics.

    Volume (Year): 8 (1999)
    Issue (Month): 3 ()
    Pages: 203-211

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    Handle: RePEc:wly:hlthec:v:8:y:1999:i:3:p:203-211
    Contact details of provider: Web page: http://www3.interscience.wiley.com/cgi-bin/jhome/5749

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    1. Alan Diener & Bernie O'Brien & Amiram Gafni, 1998. "Health care contingent valuation studies: a review and classification of the literature," Health Economics, John Wiley & Sons, Ltd., vol. 7(4), pages 313-326.
    2. Daniel Polsky & Henry A. Glick & Richard Willke & Kevin Schulman, 1997. "Confidence Intervals for Cost-Effectiveness Ratios: A Comparison of Four Methods," Health Economics, John Wiley & Sons, Ltd., vol. 6(3), pages 243-252.
    3. Andrew H. Briggs & David E. Wonderling & Christopher Z. Mooney, 1997. "Pulling cost-effectiveness analysis up by its bootstraps: A non-parametric approach to confidence interval estimation," Health Economics, John Wiley & Sons, Ltd., vol. 6(4), pages 327-340.
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