Bayesian methods for the analysis of early-phase oncology basket trials with information borrowing across cancer types

Stat Med. 2020 Nov 10;39(25):3459-3475. doi: 10.1002/sim.8675. Epub 2020 Jul 27.


Research in oncology has changed the focus from histological properties of tumors in a specific organ to a specific genomic aberration potentially shared by multiple cancer types. This motivates the basket trial, which assesses the efficacy of treatment simultaneously on multiple cancer types that have a common aberration. Although the assumption of homogeneous treatment effects seems reasonable given the shared aberration, in reality, the treatment effect may vary by cancer type, and potentially only a subgroup of the cancer types respond to the treatment. Various approaches have been proposed to increase the trial power by borrowing information across cancer types, which, however, tend to inflate the type I error rate. In this article, we review some representative Bayesian information borrowing methods for the analysis of early-phase basket trials. We then propose a novel method called the Bayesian hierarchical model with a correlated prior (CBHM), which conducts more flexible borrowing across cancer types according to sample similarity. We did simulation studies to compare CBHM with independent analysis and three information borrowing approaches: the conventional Bayesian hierarchical model, the EXNEX approach, and Liu's two-stage approach. Simulation results show that all information borrowing approaches substantially improve the power of independent analysis if a large proportion of the cancer types truly respond to the treatment. Our proposed CBHM approach shows an advantage over the existing information borrowing approaches, with a power similar to that of EXNEX or Liu's approach, but the potential to provide substantially better control of type I error rate.

Keywords: Bayesian hierarchical models; basket trials; between-indication similarity; early-phase oncology study; information borrowing across cancer types.

Publication types

  • Review

MeSH terms

  • Bayes Theorem
  • Computer Simulation
  • Humans
  • Medical Oncology*
  • Neoplasms* / drug therapy
  • Research Design