Abstract
Economic evaluations are undertaken to help inform decision making, for example, to help determine which health care interventions to fund given limited health care budgets. A systematic approach is taken to compare alternative interventions in terms of their costs and consequences. Cost–effectiveness analysis (CEA) in particular compares the difference in costs and effects between two or more alternatives, reporting the incremental difference as a cost per unit of outcome, known as an incremental cost–effectiveness ratio (ICER). Alternatively a CEA will report the net monetary benefit of an intervention; however, ICERs are the most popular method for presenting CEA results. The larger the value of the ICER, the more it costs per unit of effectiveness and therefore the less cost-effective the intervention is in comparison to the alternative. The ICER value can be compared against a monetary threshold to help aid decisions regarding appropriate resource allocation.
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Notes
- 1.
The terminology progression–free survival is used in advanced stages of cancer while disease–free survival is used in cases where surgery has left the patient disease free. The X-ACT study was concerned with adjuvant treatment post-surgery and therefore disease free survival was the outcome of interest. The remainder of this chapter will discuss disease free survival.
- 2.
Non-parametric approaches will however assume multivariate normality on the log scale for the distribution of coefficients and for this reason the Cox proportional hazards model is often referred to as semi-parametric.
- 3.
While the survival curve slopes downwards by it very nature indicating a falling probability of survival over time; the corresponding hazard curves will slope upwards indicating an increasing hazard or probability of death over time.
- 4.
An accelerated failure time (AFT) model is a parametric alternative to the proportional hazards model. The AFT model assumes that the covariates affect the time scale, either accelerating or decelerating time to failure, as opposed to the proportional hazards model which assumes that the covariates have a multiplicative effect on the hazard function.
- 5.
Therefore, the sum of difference of the individual means does not equate to the mean difference between the two treatment arms total costs.
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Boyd, K.A., Briggs, A.H., Ducournau, P., Gyldmark, M., de Reydet, F., Cassidy, J. (2012). Statistical Issues with Trial Data and Economic Modeling for Cost-Effectiveness Evaluation. In: Tang, W., Tu, X. (eds) Modern Clinical Trial Analysis. Applied Bioinformatics and Biostatistics in Cancer Research. Springer, New York, NY. https://doi.org/10.1007/978-1-4614-4322-3_6
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