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Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
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Early-Phase Platform Trials: A New Paradigm for Dose Finding and Treatment Screening in the Era of Precision Oncology

Mei-Yin C Polley1, Ying Kuen Cheung2

  • 1Mayo Clinic School of Medicine, Rochester, MN.

JCO Precision Oncology
|September 14, 2020
PubMed

Insights

Early-phase cancer trial designs, including dose-finding and screening, are evolving for targeted therapies. Master protocols offer efficient platforms for evaluating novel molecularly targeted cancer treatments.

Area of Science:

  • Oncology
  • Biostatistics
  • Clinical Trial Design

Background:

  • Statistical designs for early-phase cancer trials have evolved since the 1980s, primarily for cytotoxic agents.
  • Precision medicine advances necessitate novel designs for molecularly targeted cancer therapies.

Purpose of the Study:

  • To review early-phase cancer trial design concepts and methodologies.
  • To contextualize these designs within master protocols for targeted therapies.
  • To explore the relevance of traditional designs for novel targeted cancer treatments.

Main Methods:

  • Review of statistical methodologies in early-phase cancer trials.
  • Focus on dose-finding and treatment screening.
  • Examination of master protocol frameworks (platform, basket, umbrella, N-of-1 trials).

Main Results:

  • Traditional statistical designs remain relevant for targeted cancer therapies.
  • Master protocols provide an efficient platform for evaluating novel targeted therapies.
  • Integration of established principles with novel designs is crucial.

Conclusions:

  • Early-phase cancer trial designs are adapting to precision medicine.
  • Master protocols are key for efficient evaluation of molecularly targeted therapies.
  • Bridging traditional and novel designs optimizes cancer drug development.

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