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Cost-effectiveness Analysis, Cost-Benefit Analysis 

Study Design and Statistics

Margaret A. Winker

and Stephen J. Lurie

Although a treatment or screening technique may be shown to be effective in an RCT, recommending it in general practice would not necessarily be rational. Such interventions may be prohibitively expensive, or they may benefit only a small number of people at the expense of a large number of people, or they may lead to significant “downstream” costs that would eventually negate any immediate savings or benefit. Thus, it is possible that interventions that appear less effective may actually lead to the greatest societal benefits over the long term. Cost-effectiveness and cost-benefit analyses comprise a set of mathematical techniques to

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