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미국
The probability of cost-effectiveness
Background The study of cost-effectiveness comparisons between competing medical interventions has led to a variety of proposals for quantifying cost-effectiveness. The differences between the various approaches can be subtle, and one purpose of this article is to clarify some important distinctions. Discussion We discuss alternative measures in the framework of individual, patient-level, incremental net benefits. In particular we examine the probability of cost-effectiveness for an individual, proposed by Willan. Summary We argue that this is a useful addition to the range of cost-effectiveness measures, but will be of secondary interest to most decision makers. We also demonstrate that Willan's proposed estimate of this probability is logically flawed.
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On the probability of cost-effectiveness using data from randomized clinical trials
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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.
The use of percentage change from baseline as an outcome in a controlled trial is statistically inefficient: a simulation study
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Background Many randomized trials involve measuring a continuous outcome - such as pain, body weight or blood pressure - at baseline and after treatment. In this paper, I compare four possibilities for how such trials can be analyzed: post-treatment; change between baseline and post-treatment; percentage change between baseline and post-treatment and analysis of covariance (ANCOVA) with baseline score as a covariate. The statistical power of each method was determined for a hypothetical randomized trial under a range of correlations between baseline and post-treatment scores. Results ANCOVA has the highest statistical power. Change from baseline has acceptable power when correlation between baseline and post-treatment scores is high;when correlation is low, analyzing only post-treatment scores has reasonable power. Percentage change from baseline has the lowest statistical power and was highly sensitive to changes in variance. Theoretical considerations suggest that percentage change from baseline will also fail to protect from bias in the case of baseline imbalance and will lead to an excess of trials with non-normally distributed outcome data. Conclusions Percentage change from baseline should not be used in statistical analysis. Trialists wishing to report this statistic should use another method, such as ANCOVA, and convert the results to a percentage change by using mean baseline scores.
The costs and benefits of interventions in the area of mental health: a rapid review
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To inform the development of the draft strategic plan for mental health in NSW, a rapid review was commissioned to examine the evidence on the costs and benefits of interventions in the area of mental health. A rapid review was conducted with the assistance of an accredited librarian. A total of 50 studies were identified and included in this review.