An IPCW Adjusted Win Statistics Approach in Clinical Trials Incorporating Equivalence Margins to Define Ties
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arXiv
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| Autores principales: | , , , , |
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| Formato: | Preprint |
| Publicado: |
2025
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| _version_ | 1866912411545501696 |
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| author | Cui, Ying Huang, Bo Dong, Gaohong Uozumi, Ryuji Tian, Lu |
| author_facet | Cui, Ying Huang, Bo Dong, Gaohong Uozumi, Ryuji Tian, Lu |
| contents | In clinical trials, multiple outcomes of different priorities commonly occur as the patient's response may not be adequately characterized by a single outcome. Win statistics are appealing summary measures for between-group difference at more than one endpoint. When defining the result of pairwise comparisons of a time-to-event endpoint, it is desirable to allow ties to account for incomplete follow-up and not clinically meaningful difference in endpoints of interest. In this paper, we propose a class of win statistics for time-to-event endpoints with a user-specified equivalence margin. These win statistics are identifiable in the presence of right-censoring and do not depend on the censoring distribution. We then develop estimation and inference procedures for the proposed win statistics based on inverse-probability-of-censoring {weighting} (IPCW) adjustment to handle right-censoring. We conduct extensive simulations to investigate the operational characteristics of the proposed procedure in the finite sample setting. A real oncology trial is used to illustrate the proposed approach. |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2506_03050 |
| institution | arXiv |
| publishDate | 2025 |
| record_format | arxiv |
| spellingShingle | An IPCW Adjusted Win Statistics Approach in Clinical Trials Incorporating Equivalence Margins to Define Ties Cui, Ying Huang, Bo Dong, Gaohong Uozumi, Ryuji Tian, Lu Methodology Applications 92B15 In clinical trials, multiple outcomes of different priorities commonly occur as the patient's response may not be adequately characterized by a single outcome. Win statistics are appealing summary measures for between-group difference at more than one endpoint. When defining the result of pairwise comparisons of a time-to-event endpoint, it is desirable to allow ties to account for incomplete follow-up and not clinically meaningful difference in endpoints of interest. In this paper, we propose a class of win statistics for time-to-event endpoints with a user-specified equivalence margin. These win statistics are identifiable in the presence of right-censoring and do not depend on the censoring distribution. We then develop estimation and inference procedures for the proposed win statistics based on inverse-probability-of-censoring {weighting} (IPCW) adjustment to handle right-censoring. We conduct extensive simulations to investigate the operational characteristics of the proposed procedure in the finite sample setting. A real oncology trial is used to illustrate the proposed approach. |
| title | An IPCW Adjusted Win Statistics Approach in Clinical Trials Incorporating Equivalence Margins to Define Ties |
| topic | Methodology Applications 92B15 |
| url | https://arxiv.org/abs/2506.03050 |