Increasing the efficiency of oncology basket trials using a Bayesian approach

Contemp Clin Trials. 2017 Dec;63:67-72. doi: 10.1016/j.cct.2017.06.009. Epub 2017 Jun 16.


With the rapid growth of targeted and immune-oncology therapies, novel statistical design approaches are needed to increase the flexibility and efficiency of early phase oncology trials. Basket trials enroll patients with defined biological deficiencies, but with multiple histologic tumor types (or indications), to discover in which indications the drug is active. In such designs different indications are typically analyzed independently. This, however, ignores potential biological similarities among the indications. Our research provides a statistical methodology to enhance such basket trials by assessing the homogeneity of the response rates among indications at an interim analysis, and applying a Bayesian hierarchical modeling approach in the second stage if the efficacy is deemed reasonably homogenous across indications. This increases the power of the study by allowing indications with similar response rates to borrow information from each other. Via simulations, we quantify the efficiency gain of our proposed approach relative to the conventional parallel approach. The operating characteristics of our method depend on the similarity of the response rates between the different indications. If the response rates are comparable in most or all indications after treatment with the investigational drug, a substantial increase in efficiency as compared to the conventional approach can be obtained as fewer patients are required or a higher power is attained. We also demonstrate that efficacy again decreases if the response rates vary considerably among tumor types but it is still better than the conventional approach.

MeSH terms

  • Bayes Theorem*
  • Computer Simulation
  • Decision Support Techniques
  • Drugs, Investigational
  • Humans
  • Medical Oncology / methods*
  • Models, Statistical*
  • Research Design*


  • Drugs, Investigational