Cost‐effectiveness analysis based on the number‐needed‐to‐treat: common sense or non‐sense?

Abstract This paper explores and critically discusses some of the methodological limitations of using the number‐needed‐to‐treat (NNT) in economic evaluation. We argue that NNT may be a straightforward measure of benefit when the effect of an intervention is immediate, but that serious problems arise when the effect is delay rather than avoidance of an adverse event. In this case, NNT is not a robust or accurate measure of effect, but will vary considerably and inconsistently over time. This weakness will naturally spill over onto any CEA based on NNT. A literature review demonstrated that CEAs based on NNT were all published within the last five years, and that all studies suffered from important limitations. A major weakness of using NNT is the imposed restrictions on the outcome measure, which can only be strictly uni‐dimensional and non‐generic. Using NNT in economic evaluations is obtained at a cost in terms of both methodological shortcomings, and a reduced ability for such evaluations to serve as a useful tool in decision making processes. The use of NNT in economic evaluations might be better avoided. To every complicated question, there is a simple, straightforward, easy – and probably wrong answer (Occam's Sledgehammer). Copyright © 2003 John Wiley & Sons, Ltd.