Weighing evidence: robustness vs quantity.

Many new cancer therapies target specific molecular subtypes. Methods for addressing the bias induced from augmenting the control-arm data in a randomized clinical trial with nonconcurrent data from patients that have received the control treatment outside the randomized trial have been proposed ([9]). There are obvious scenarios where the significance level could be relaxed, for example, in exploratory settings where confirmatory evidence is expected to come via future studies or in the context of serious diseases for which there is no proven effective treatment. Brown et al. ([10]) implemented an alternative strategy relaxing the statistical significance level to.15 in a trial evaluating treatments for newly diagnosed KMT2A-rearranged infant acute lymphoblastic leukemia. [Extracted from the article]