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Updated: Sep 4, 2025

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Optimal single-arm two-stage designs with consideration of dependency on efficacy and safety

Huan Yin1, Weizhen Wang2,3, Zhongzhan Zhang3

  • 1School of Mathematics and Physics, 47840North China Electric Power University, Beijing, People's Republic of China.

Statistical Methods in Medical Research
|July 15, 2022
PubMed

Insights

This study introduces new hypotheses and optimal two-stage designs for phase II cancer trials, effectively assessing treatment efficacy and safety simultaneously. The methods ensure strict error control and minimize sample size for better clinical trial design.

Area of Science:

  • Clinical Trials
  • Biostatistics
  • Oncology

Background:

  • Phase II clinical trials for cancer treatments require simultaneous evaluation of efficacy and safety.
  • Existing statistical hypotheses may not adequately address this dual objective.

Purpose of the Study:

  • To introduce novel hypotheses and optimal two-stage designs for phase II cancer trials.
  • To effectively assess treatment efficacy and safety concurrently.

Main Methods:

  • Development of two new sets of hypotheses considering the association between efficacy and safety factors.
  • Construction of optimal two-stage designs based on these hypotheses.
  • Provision of an algorithm for design computation and R-codes.

Main Results:

  • The proposed designs strictly control the type I error rate at a nominal level.
  • Minimum power is maintained at a specified level.
  • The designs achieve the smallest expected total sample size under the null hypothesis.

Conclusions:

  • The new statistical framework and designs offer an effective approach for simultaneous efficacy and safety evaluation in phase II cancer trials.
  • These methods provide a statistically rigorous and efficient design for clinical research.
Keywords:
Dimensionality reductionexact testexpected sample sizemultinomial distributionphase II clinical trial

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