Adjustment for inconsistency in adaptive phase 2/3 designs with dose optimization

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dc.contributor.authorChen, Cong
dc.contributor.authorHuang, Mo
dc.contributor.authorZhang, Xuekui
dc.date.accessioned2026-03-05T17:18:55Z
dc.date.available2026-03-05T17:18:55Z
dc.date.copyright2025
dc.description.abstractAdaptive Phase 2/3 designs hold great promise in contemporary oncology drug development, especially when limited data from Phase 1 dose-finding is insufficient for identifying an optimal dose. However, inconsistent results between Phase 2 and Phase 3 may raise regulatory and practical concerns. The imperfection in dose selection further complicates the issue. In this paper, we explicitly incorporate the concerns about inconsistency into the statistical analysis under three hypothesis testing strategies (conservative, aggressive, and neutral) by specifying an inconsistency cutoff and accounting for the probability of "picking-the-winner." This investigation illustrates how to balance regulatory caution, sponsor interests, and practical considerations in adaptive Phase 2/3 designs with dose optimization, which paves the way for further research in a less explored area.
dc.description.reviewstatusReviewed
dc.description.scholarlevelFaculty
dc.description.sponsorshipThis work was supported by Canada Research Chairs (CRC-2021-00232) and Michael Smith Health Research BC (SCH-2022-2553).
dc.identifier.citationChen, C., Huang, M., & Zhang, X. (2025). Adjustment for inconsistency in adaptive phase 2/3 designs with dose optimization. Pharmaceutical Statistics, 24(6), e70031. https://doi.org/10.1002/pst.70031
dc.identifier.urihttps://doi.org/10.1002/pst.70031
dc.identifier.urihttps://hdl.handle.net/1828/23422
dc.language.isoen
dc.publisherPharmaceutical Statistics
dc.rightsCC BY 4.0en
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/
dc.subjectadaptive design
dc.subjectdose optimization
dc.subjectinconsistency
dc.subjectphase 2/3 trial
dc.subjectseamless design
dc.subjecttype I error control
dc.subject.departmentDepartment of Mathematics and Statistics
dc.titleAdjustment for inconsistency in adaptive phase 2/3 designs with dose optimization
dc.typeArticle

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