On the probability of cost-effectiveness using data from randomized clinical trials.
BACKGROUND
Acceptability curves have been proposed for quantifying the probability that a treatment under investigation in a clinical trial is cost-effective. Various definitions and estimation methods have been proposed. Loosely speaking, all the definitions, Bayesian or otherwise, relate to the probability that the treatment under consideration is cost-effective as a function of the value placed on a unit of effectiveness. These definitions are, in fact, expressions of the certainty with which the current evidence would lead us to believe that the treatment under consideration is cost-effective, and are dependent on the amount of evidence (i.e. sample size).


METHODS
An alternative for quantifying the probability that the treatment under consideration is cost-effective, which is independent of sample size, is proposed.


RESULTS
Non-parametric methods are given for point and interval estimation. In addition, these methods provide a non-parametric estimator and confidence interval for the incremental cost-effectiveness ratio. An example is provided.


CONCLUSIONS
The proposed parameter for quantifying the probability that a new therapy is cost-effective is superior to the acceptability curve because it is not sample size dependent and because it can be interpreted as the proportion of patients who would benefit if given the new therapy. Non-parametric methods are used to estimate the parameter and its variance, providing the appropriate confidence intervals and test of hypothesis.
Willan
A R
AR
Department of Clinical Epidemiology and Biostatistics, HSC-2C, McMaster University, 1200 Main Street West, Hamilton, ON, L8N 3Z5, Canada. willana@mcmaster.ca
D003362:Cost-Benefit Analysis / Q000706:statistics & numerical data*; D006801:Humans; D015233:Models, Statistical; D011336:Probability; D016032:Randomized Controlled Trials as Topic / Q000191:economics* / Q000706:statistics & numerical data*; D018709:Statistics, Nonparametric