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Calculation of Phase 2 dose-finding study sample size for reliable Phase 3 dose selection.

Fang Liu1, Qing Zhao1, Anthony J Rodgers1

  • 1Biostatistics and Research Decision Sciences, Merck & Co., Inc., Rahway, New Jersey, USA.

Pharmaceutical Statistics
|August 8, 2023
PubMed
Summary
This summary is machine-generated.

Traditional Phase 2 study sample sizes lack precision for selecting Phase 3 doses. This study proposes a new sample size calculation method for dose-finding studies, enhancing precision in target dose identification.

Keywords:
MCP-ModPhase 3 dose selectiondose-finding studysample size determination

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Area of Science:

  • Biostatistics
  • Clinical Trial Design
  • Pharmacometrics

Background:

  • Phase 2 dose-finding studies typically use sample sizes based on power for detecting dose-response, which is insufficient for precise Phase 3 target dose selection.
  • Existing methods may not guarantee the accuracy needed for selecting optimal doses for subsequent clinical trials.

Purpose of the Study:

  • To propose a novel method for calculating sample sizes in Phase 2 dose-finding studies.
  • To enhance the precision of target dose selection for Phase 3 studies.

Main Methods:

  • Utilizing the multiple comparison and modeling procedure (MCP-Mod) to calculate sample size.
  • Focusing on the probability of identifying the target dose within a defined acceptable range (e.g., 80%-120%).
  • Comparing different Phase 2 dose-finding study design options.

Main Results:

  • The proposed approach provides a more precise method for sample size calculation in dose-finding studies.
  • Sensitivity analyses are recommended to ensure robustness against deviations from modeling assumptions.
  • Illustrates the application through a hypothetical Phase 2 study planning.

Conclusions:

  • The MCP-Mod approach offers improved precision for Phase 2 dose-finding sample size determination.
  • This method facilitates better-informed decisions for Phase 3 target dose selection.
  • The study provides accessible code for implementing the proposed methodology.