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Updated: Jul 27, 2025

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
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Early phase clinical trials in oncology: Realising the potential of seamless designs

Thomas Jaki1, Abigail Burdon2, Xijin Chen2

  • 1MRC Biostatistics Unit, University of Cambridge, UK; University of Regensburg, Germany.

European Journal of Cancer (Oxford, England : 1990)
|June 10, 2023
PubMed
Abstract

Insights

Novel statistical designs can improve oncology drug development efficiency by optimizing dosage and identifying promising treatments faster. These methods help terminate poor drug candidates early and accelerate successful ones.

Area of Science:

  • Pharmaceutical Science
  • Clinical Trial Design
  • Oncology Drug Development

Background:

  • The pharmaceutical industry faces declining productivity, characterized by high attrition rates and reduced regulatory approvals.
  • Oncology drug development is particularly challenging, with low approval rates for novel treatments compared to other therapeutic areas.
  • Efficiently establishing treatment potential and optimal dosage is crucial for successful drug development.

Purpose of the Study:

  • To explore novel statistical designs for improving efficiency in early oncology drug development.
  • To discuss seamless strategies for early oncology development, highlighting their strengths and weaknesses.
  • To provide best practice recommendations and identify future opportunities in oncology drug development.

Main Methods:

  • Review and discussion of various seamless strategies for early oncology development.
  • Illustration of strategy strengths and weaknesses using real clinical trial examples.
  • Analysis of missed opportunities and future potential in early oncology treatment development.

Main Results:

  • Novel statistical designs offer a reliable approach to establishing optimal dosage and treatment potential.
  • These designs enhance the efficiency of the drug development pathway by making effective use of collected data.
  • The paper discusses practical applications and provides insights into improving early oncology development.

Conclusions:

  • Modern dose-finding methods can significantly shorten and enhance the drug development process.
  • Implementing these modern statistical approaches requires only minor adjustments to current practices.
  • There is substantial potential to improve early oncology treatment development through the adoption of these advanced methods.
Keywords:
Dose-determinationEfficient trialsModel-based designs

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