Bayesian Phase 1/2 trial designs and cellular immunotherapies: a practical primer

Bayesian Phase 1/2 trial designs remain underused in biomedical research and are virtually absent from the field of cellular therapy. In this review, we highlight the severe limitations of the maximum tolerated dose (MTD) concept and the traditional Phase 1/Phase 2 paradigm. Next, we introduce statistical concepts underlying most adaptive Bayesian trial designs. We use the EffTox design [1,2], one of many adaptive Bayesian designs, as an example to illustrate ‘state-of-the-art’ Phase 1/2 designs. We highlight how these designs can be helpful to the cellular therapy field specifically. Furthermore, we provide the reader with practical examples, links to freely available web applications, and R packages. We hope this will incentivize investigators to implement these designs for chimeric antigen-receptor-engineered T cell therapy trials, as well as other T cell-based therapies.

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