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Discussion of Trial Designs for Biomarker Identification and Validation Through the Use of Case Studies

Fang-Shu Ou1, Ming-Wen An2, Amy S Ruppert3

  • 1Mayo Clinic, Rochester, MN.

JCO Precision Oncology
|March 20, 2020
PubMed

Insights

Precision medicine tailors treatments to individuals, moving beyond single-biomarker trials. This article explores five key trial designs—enrichment, umbrella, basket, subgroup, and window of opportunity—crucial for advancing personalized cancer care.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Biomarker Discovery

Background:

  • The National Cancer Institute's Precision Medicine Initiative (2015) spurred a shift towards personalized healthcare.
  • Traditional trial designs (one-trial/one-biomarker-at-a-time) are being replaced by more adaptive approaches.
  • Identifying and validating biomarkers is essential for effective targeted therapy selection.

Purpose of the Study:

  • To discuss five distinct precision medicine trial designs.
  • To detail the characteristics, case studies, recent advances, and pros/cons of each design.
  • To provide a comprehensive overview of innovative clinical trial strategies for personalized medicine.

Main Methods:

  • Review and discussion of five trial designs: enrichment, umbrella, basket, subgroup, and window of opportunity.
  • Analysis of design characteristics and application through case studies.
  • Exploration of recent advancements and comparative advantages/disadvantages.

Main Results:

  • Detailed descriptions of enrichment, umbrella, basket, subgroup, and window of opportunity trial designs.
  • Illustrative examples from ongoing or completed clinical trials for each design.
  • Discussion of the strengths and limitations inherent in each approach.

Conclusions:

  • These five trial designs are pivotal for realizing the potential of precision medicine.
  • Understanding these designs is critical for optimizing patient selection and therapeutic efficacy.
  • The article serves as a guide to navigating the evolving landscape of cancer clinical trials.

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