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.
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 language | English |
|---|---|
| Journal | New England Journal of Statistics in Data Science |
| Early online date | 10 Sept 2025 |
| DOIs | |
| Publication status | E-pub ahead of print - 10 Sept 2025 |
Bibliographical note
Publisher Copyright:© 2025 New England Statistical Society.
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