Evaluate the relative efficiency of a targeted clinical trial design to an untargeted design under the issue of cost

In this article, we evaluate the relative cost of a targeted design to an untargeted design for a randomized clinical trial comparing a new treatment to a control. We observed that when the ratio of screen cost per person and drug cost per person is very small, say 0.008, the trial cost for conducting a targeted design might be fewer than that for conducting an untargeted design. When the difference between the treatment effect for the targeted patients and the treatment effect for the untargeted patients is increased, the savings in trial cost for the targeted design would be increased significantly. The greater sensitivity and specificity for the screen tool will lead to greater savings in trial cost for the targeted design. The relative cost of a targeted design to an untargeted design is associated with the treatment effect difference between targeted and untargeted patients, the proportion of targeted patients in the population, the screen tool performance, and the ratio of drug cost per person and screen cost per person.