Clinical Trials with Biologic Primary Endpoints in Immuno-oncology: Concepts and Usage

Clin Cancer Res. 2022 Jan 1;28(1):13-22. doi: 10.1158/1078-0432.CCR-21-1593. Epub 2021 Jul 26.


Clinical trials that have a pharmacokinetic or a pharmacodynamic immunologic mechanism of action-based primary outcome could substantially improve the validity and efficiency of early development of immuno-oncology agents. Here, we outline different trial design options in this area, review examples from the literature and their unique immunologic aspects, and highlight how these trials have been underutilized. We illustrate how new technologies and translationally focused approaches can be successfully used to develop different classes of immunotherapeutic agents.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Humans
  • Immunologic Factors / therapeutic use
  • Immunotherapy*
  • Medical Oncology
  • Neoplasms* / drug therapy


  • Immunologic Factors