Adaptive Phase 2/3 Design with Dose Optimization Article Swipe
Cong Chen
,
Mo Huang
,
Xuekui Zhang
·
YOU?
·
· 2024
· Open Access
·
· DOI: https://doi.org/10.48550/arxiv.2412.08439
YOU?
·
· 2024
· Open Access
·
· DOI: https://doi.org/10.48550/arxiv.2412.08439
FDA's Project Optimus initiative for oncology drug development emphasizes selecting a dose that optimizes both efficacy and safety. When an inferentially adaptive Phase 2/3 design with dose selection is implemented to comply with the initiative, the conventional inverse normal combination test is commonly used for Type I error control. However, indiscriminate application of this overly conservative test can lead to substantial increase in sample size and timeline delays, which undermines the appeal of the adaptive approach. This, in turn, frustrates drug developers regarding Project Optimus. The inflation of Type I error depends on the probability of selecting a dose with better long-term efficacy outcome at end of the study based on limited follow-up data at dose selection. In this paper, we discuss the estimation of this probability and its impact on Type I error control in realistic settings. Incorporating it explicitly into the two methods we have proposed result in improved designs, potentially motivating drug developers to adhere more closely to an initiative that has the potential to revolutionize oncology drug development.
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Metadata
- Type
- preprint
- Language
- en
- Landing Page
- http://arxiv.org/abs/2412.08439
- https://arxiv.org/pdf/2412.08439
- OA Status
- green
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W4405306761
All OpenAlex metadata
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- DOI
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https://doi.org/10.48550/arxiv.2412.08439Digital Object Identifier
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Adaptive Phase 2/3 Design with Dose OptimizationWork title
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preprintOpenAlex work type
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enPrimary language
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2024Year of publication
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2024-12-11Full publication date if available
- Authors
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Cong Chen, Mo Huang, Xuekui ZhangList of authors in order
- Landing page
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https://arxiv.org/abs/2412.08439Publisher landing page
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https://arxiv.org/pdf/2412.08439Direct link to full text PDF
- Open access
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YesWhether a free full text is available
- OA status
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greenOpen access status per OpenAlex
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https://arxiv.org/pdf/2412.08439Direct OA link when available
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Phase (matter), Computer science, Physics, Quantum mechanicsTop concepts (fields/topics) attached by OpenAlex
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0Total citation count in OpenAlex
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10Other works algorithmically related by OpenAlex
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