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Adaptive enrichment designs: applications and challenges

Noah Simon

Our improving understanding of disease biology has reinforced the idea that many diseases are heterogeneous collections with different causal mechanisms. As such, the biomedical field has focused on developing targeted therapies, effective in only some subset of the population with a given disease. However, characterizing these subsets has been a challenge – often there is insufficient information until well into largescale trials. In this manuscript, we will discuss adaptive enrichment designs: clinical trial designs that allow the simultaneous construction and use of biomarkers, during an ongoing trial. We detail three common scenarios where adaptive enrichment designs can be much more effective than classical designs. We discuss which adaptive strategies are most applicable in each scenario.