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Updated: May 5, 2026

Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
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Published on: February 6, 2015

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Evaluating many treatments and biomarkers in oncology: a new design

Richard Kaplan1, Timothy Maughan, Angela Crook

  • 1Richard Kaplan, Angela Crook, David Fisher, Louise Brown, and Mahesh Parmar, Medical Research Council Clinical Trials Unit, London; Timothy Maughan, University of Oxford, Oxford; and Richard Wilson, Queen's University Belfast, Belfast, United Kingdom.

Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology
|November 20, 2013
PubMed

Insights

This study introduces an efficient clinical trial design for biomarker-stratified cancer treatments. The FOCUS4 protocol rapidly evaluates novel therapies and biomarkers in advanced colorectal cancer patients.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Translational Research

Background:

  • Biomarker-stratified clinical trials require efficient designs to evaluate novel therapies.
  • Current trial designs may not optimally integrate biomarker evaluation with treatment assessment.

Purpose of the Study:

  • To introduce and describe the FOCUS4 protocol, a novel approach for biomarker-stratified clinical trials.
  • To evaluate the efficacy and safety of novel targeted therapies in advanced colorectal cancer based on specific biomarkers.
  • To assess if treatment benefits are restricted to biomarker-defined patient populations.

Main Methods:

  • The FOCUS4 protocol employs a parallel, population-enriched, biomarker-stratified randomized trial design.
  • Patients with metastatic colorectal cancer are assigned to cohorts based on biomarkers (e.g., BRAF, AKT, KRAS/NRAS).
  • A multistaged approach with interim analyses allows for adaptation based on treatment activity.

Main Results:

  • FOCUS4 is the first trial to assign all metastatic colorectal cancer patients to parallel biomarker-stratified randomized trials.
  • This design enables efficient evaluation of multiple novel therapies and their associated biomarkers.
  • The protocol facilitates rapid assessment of treatment efficacy and safety in targeted patient groups.

Conclusions:

  • The FOCUS4 protocol offers an efficient strategy for evaluating novel therapies in biomarker-defined populations.
  • This approach accelerates the process of answering key research questions regarding targeted treatments.
  • Biomarker assessment within this trial design aids in targeting treatment to specific patient groups, optimizing outcomes.

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