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LiMAP-curvature: a simple model-free approach for analysing dose-finding studies

  • Linxi Han
  • , Qiqi Deng
  • , Zhangyi He*
  • , Feng Yu*
  • *Corresponding author for this work

Research output: Contribution to journal › Article (Academic Journal) › peer-review

Abstract

MCP-Mod (Multiple Comparison Procedure-Modelling) is an efficient statistical method for the analysis of Phase II
dose-finding trials, although it requires specialised expertise to pre-specify plausible candidate models along with model

parameters. This can be problematic given limited knowledge of the agent/compound being studied, and misspecification of candidate models and model parameters can severely degrade its performance. To circumvent this challenge, in the work, we introduce LiMAP-curvature, a Bayesian model-free approach for the detection of the dose-response signal in
Phase II dose-finding trials. LiMAP-curvature is built upon a Bayesian hierarchical framework incorporating information
about the total curvature of the dose-response curve. Through extensive simulations, we show that LiMAP-curvature has
comparable performance to MCP-Mod if the true underlying dose-response model is included in the candidate model set
of MCP-Mod. Otherwise, LiMAP-curvature can achieve performance superior to that of MCP-Mod, especially when the
true dose-response model drastically deviates from candidate models in MCP-Mod.
Original languageEnglish
JournalNew England Journal of Statistics in Data Science
Early online date10 Sept 2025
DOIs
Publication statusE-pub ahead of print - 10 Sept 2025

Bibliographical note

Publisher Copyright:
© 2025 New England Statistical Society.

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