Light and shadow on innovative clinical trial designs: reflections from the EORTC-PAMM course on 'preclinical and early-phase clinical pharmacology'

Expert Rev Clin Pharmacol. 2019 Nov;12(11):1033-1036. doi: 10.1080/17512433.2019.1683446. Epub 2019 Oct 30.

Abstract

Introduction: In the era of 'precision' oncology, novel clinical trial designs have emerged, in order to better address the final goal of translating the above-mentioned preclinical discoveries into the clinic. Nonetheless, in aiming to achieve the greatest clinical benefit to patients, some limitations of these novel approaches from the statistical, methodological and practical point of view need to be overcome.Areas covered: In the present review, a short overview of basket trials, umbrella trials and platform trials are discussed, in particular advantages and disadvantages of such experimental approaches.Expert opinion: Master protocols represent the future of clinical oncology research. The possibility of investigating multiple biomarkers and therapeutic regimens under one study is a strong advantage over traditional trials, and it can lead to quick implementation of new, promising treatments or biomarkers into the clinic.

Keywords: Clinical trial; basket trial; precision oncology; umbrella trial.

Publication types

  • Review

MeSH terms

  • Biomedical Research / methods
  • Clinical Trials as Topic / methods*
  • Humans
  • Neoplasms / therapy*
  • Precision Medicine / methods
  • Research Design*
  • Translational Research, Biomedical / methods