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04 June 2018

Conventional power calculations for a confirmatory clinical trial may lead to a sample size that is infeasible or even impossible to achieve if the trial is in a rare disease or other small population. This suggests that choice of an appropriate sample size should, in some way, reflect the size of the population for which the investigational intervention is intended. One method by which this might be achieved is the value-of-information approach whereby the cost of data collection can be weighed against the gains from the improved decision-making based on the information obtained. In this talk explores the use of such a method in the rare disease setting, showing how this approach can lead to justification of smaller trials when the patient population is smaller.

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