WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors
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arXiv
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| Main Authors: | , , , , , , , , , , |
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| Format: | Preprint |
| Published: |
2024
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| _version_ | 1866917904450060288 |
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| author | Sechidis, Konstantinos Sun, Sophie Chen, Yao Lu, Jiarui Zhang, Cong Baillie, Mark Ohlssen, David Vandemeulebroecke, Marc Hemmings, Rob Ruberg, Stephen Bornkamp, Björn |
| author_facet | Sechidis, Konstantinos Sun, Sophie Chen, Yao Lu, Jiarui Zhang, Cong Baillie, Mark Ohlssen, David Vandemeulebroecke, Marc Hemmings, Rob Ruberg, Stephen Bornkamp, Björn |
| contents | This paper proposes a Workflow for Assessing Treatment effeCt Heterogeneity (WATCH) in clinical drug development targeted at clinical trial sponsors. WATCH is designed to address the challenges of investigating treatment effect heterogeneity (TEH) in randomized clinical trials, where sample size and multiplicity limit the reliability of findings. The proposed workflow includes four steps: Analysis Planning, Initial Data Analysis and Analysis Dataset Creation, TEH Exploration, and Multidisciplinary Assessment. The workflow offers a general overview of how treatment effects vary by baseline covariates in the observed data, and guides interpretation of the observed findings based on external evidence and best scientific understanding. The workflow is exploratory and not inferential/confirmatory in nature, but should be pre-planned before data-base lock and analysis start. It is focused on providing a general overview rather than a single specific finding or subgroup with differential effect. |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2405_00859 |
| institution | arXiv |
| publishDate | 2024 |
| record_format | arxiv |
| spellingShingle | WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors Sechidis, Konstantinos Sun, Sophie Chen, Yao Lu, Jiarui Zhang, Cong Baillie, Mark Ohlssen, David Vandemeulebroecke, Marc Hemmings, Rob Ruberg, Stephen Bornkamp, Björn Applications This paper proposes a Workflow for Assessing Treatment effeCt Heterogeneity (WATCH) in clinical drug development targeted at clinical trial sponsors. WATCH is designed to address the challenges of investigating treatment effect heterogeneity (TEH) in randomized clinical trials, where sample size and multiplicity limit the reliability of findings. The proposed workflow includes four steps: Analysis Planning, Initial Data Analysis and Analysis Dataset Creation, TEH Exploration, and Multidisciplinary Assessment. The workflow offers a general overview of how treatment effects vary by baseline covariates in the observed data, and guides interpretation of the observed findings based on external evidence and best scientific understanding. The workflow is exploratory and not inferential/confirmatory in nature, but should be pre-planned before data-base lock and analysis start. It is focused on providing a general overview rather than a single specific finding or subgroup with differential effect. |
| title | WATCH: A Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors |
| topic | Applications |
| url | https://arxiv.org/abs/2405.00859 |