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Improving early phase oncology clinical trial design: An opportunity for statisticians

Alan Phillips1, Shalini Mondal1

  • 1Centre for Pharmaceutical Medicine Research, Faculty of Life Sciences and Medicine, King's College London, London, UK.

Pharmaceutical Statistics
|December 27, 2022
PubMed

Insights

Rule-based clinical trial designs are common in early phase oncology, but newer methods offer better performance. Statisticians can improve drug development by adopting efficient designs for optimal biological dose estimation.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Biostatistics

Background:

  • Rule-based designs, exemplified by the '3+3' study design, remain prevalent in early-phase oncology drug development.
  • These traditional designs exhibit suboptimal performance compared to more contemporary approaches.

Purpose of the Study:

  • To highlight the continued use of rule-based designs in early phase oncology.
  • To advocate for the adoption of advanced study designs for improved efficiency and dose estimation.

Main Methods:

  • This communication analyzes the application of rule-based versus model-based designs in early phase oncology.
  • It emphasizes the statistical opportunities for optimizing clinical trial methodologies.

Main Results:

  • Rule-based designs are frequently employed despite evidence of their inferior efficacy.
  • Model-based and model-assisted designs demonstrate superior performance in dose-finding studies.

Conclusions:

  • There is a significant opportunity for biostatisticians to enhance early phase oncology trials.
  • Implementing advanced, efficient study designs can lead to better estimation of the Optimal Biological Dose (OBD).
Keywords:
model-assistedmodel-basedoncologyrule-based

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