All Times EDT
Keywords: Early Phase Clinical Trials, Exploratory, Descriptive, Statistical Reviewer
Early phase clinical trials are generally exploratory in nature. Goals are to evaluate preliminary safety and early efficacy as demonstrated by biomarkers. Rarely the endpoints are clinical endpoints used for regulatory submission. Primary statistical methods employed in these studies are descriptive in nature, sometimes aided by models, either Bayesian or frequentist, that can elicit additional patterns either related to doses or time. The data are published in general science or medical journals such as Nature, Cell, Journal of Clinical Pharmacolgy, etc or disease specific journals such as Diabetes, Hepatology, etc. Most often the questions from statistical reviewers revolve around p-values, multiplicity adjustment and in some cases about multiple imputations, the methods employed in late phase trials. How do we educate the reviewers about the exploratory nature of these studies and change the dialogue to beyond p-values and adjustments for multiplicity?