|
Abstract:
|
We consider a simplified exploratory master protocol in oncology that intends to investigate multiple test drugs in the same tumor indication (i.e., an umbrella trial) or investigate the same test drug in multiple tumor indications (i.e., a basket trial).
The primary objective of an exploratory umbrella trial is to test whether any of the drugs is effective, whereas individual drug cohorts are analyzed independently. In contrast, the primary objective of an exploratory basket trial is to test whether the test drug is effective in any of the tumor indications, whereas data are subject to pooling. The distinction is less explored in literature and is often overlooked in practice. In this research note, we illustrate it with the pruning-and-pooling method that was first proposed for designing confirmatory basket trials. The method is extended to address a high-level strategic question: how many cohorts (number of drugs in an umbrella trial or number of tumor indications in a basket trial) should be considered in each master protocol under resource constraint?
|
