Comparison of hierarchical EMAX and NDLM models in dose-response for early phase clinical trials.

ABSTRACT:

Background

Phase II clinical trials primarily aim to find the optimal dose and investigate the relationship between dose and efficacy relative to standard of care (control). Therefore, before moving forward to a phase III confirmatory trial, the most effective dose is needed to be identified.

Methods

The primary endpoint of a phase II trial is typically a binary endpoint of success or failure. The EMAX model, ubiquitous in pharmacology research, was fit for many compounds and described the data well, except for a single compound, which had nonmonotone dose-response (Thomas et al., Stat Biopharmaceutical Res. 6:302-317 2014). To mitigate the risk of nonmonotone dose response one of the alternative options is a Bayesian hierarchical EMAX model (Gajewski et al., Stat Med. 38:31

SUBMITTER: Huang X 

PROVIDER: S-EPMC7370408 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature