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| Autores principales: | , , , , , , , , , |
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| Formato: | Preprint |
| Publicado: |
2025
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| Materias: | |
| Acceso en línea: | https://arxiv.org/abs/2505.15735 |
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| _version_ | 1866914108062826496 |
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| author | Marks, Yanara Cunningham, Jessie Jiang, Arlene Li, Linke Lin, Yi-Shu McGrory, Abigail Ouyang, Yongdong Tran, Nam-Anh Wang, Yuning Heath, Anna |
| author_facet | Marks, Yanara Cunningham, Jessie Jiang, Arlene Li, Linke Lin, Yi-Shu McGrory, Abigail Ouyang, Yongdong Tran, Nam-Anh Wang, Yuning Heath, Anna |
| contents | Utilizing Bayesian methods in clinical trials has become increasingly popular, as they can incorporate historical data and expert opinions into the design and allow for smaller sample sizes to reduce costs while providing reliable and robust statistical results. Sample size determination (SSD) is a key aspect of clinical trial design and various methods for Bayesian sample size determination are available. However, it is unclear how these methods are being used in practice. A systematic literature review was conducted to understand how sample sizes for Bayesian randomized clinical trials (RCTs) are determined and inform the design of future Bayesian trials. We searched five databases in May 2023, and updated in January 2025, including efficacy RCTs in humans which utilized a Bayesian framework for the primary data analysis, published in English, and enrolled participants between 2009 and 2024. The literature search produced 19,182 records, of which 105 studies were selected for data extraction. Results show that the most common method for SSD in Bayesian RCTs was a hybrid approach in which elements of Bayesian and frequentist theory are combined. Many RCTs did not provide a justification for SSD, while fully Bayesian methods were rarely used in practice, despite significant theoretical development. Our review also revealed a lack of standardized reporting, making it challenging to review the SSD. The CONSORT statement for reporting RCTs states that sample size calculations must be reported, which was poorly adhered to. Among RCTs that reported SSD, relevant information was frequently omitted from the reports and discussed in poorly structured supplementary materials. Thus, there is a critical need for greater transparency, standardization and translation of relevant methodology in Bayesian RCTs. |
| format | Preprint |
| id |
arxiv_https___arxiv_org_abs_2505_15735 |
| institution | arXiv |
| publishDate | 2025 |
| record_format | arxiv |
| spellingShingle | A systematic review of sample size determination in Bayesian randomized clinical trials: full Bayesian methods are rarely used Marks, Yanara Cunningham, Jessie Jiang, Arlene Li, Linke Lin, Yi-Shu McGrory, Abigail Ouyang, Yongdong Tran, Nam-Anh Wang, Yuning Heath, Anna Applications Utilizing Bayesian methods in clinical trials has become increasingly popular, as they can incorporate historical data and expert opinions into the design and allow for smaller sample sizes to reduce costs while providing reliable and robust statistical results. Sample size determination (SSD) is a key aspect of clinical trial design and various methods for Bayesian sample size determination are available. However, it is unclear how these methods are being used in practice. A systematic literature review was conducted to understand how sample sizes for Bayesian randomized clinical trials (RCTs) are determined and inform the design of future Bayesian trials. We searched five databases in May 2023, and updated in January 2025, including efficacy RCTs in humans which utilized a Bayesian framework for the primary data analysis, published in English, and enrolled participants between 2009 and 2024. The literature search produced 19,182 records, of which 105 studies were selected for data extraction. Results show that the most common method for SSD in Bayesian RCTs was a hybrid approach in which elements of Bayesian and frequentist theory are combined. Many RCTs did not provide a justification for SSD, while fully Bayesian methods were rarely used in practice, despite significant theoretical development. Our review also revealed a lack of standardized reporting, making it challenging to review the SSD. The CONSORT statement for reporting RCTs states that sample size calculations must be reported, which was poorly adhered to. Among RCTs that reported SSD, relevant information was frequently omitted from the reports and discussed in poorly structured supplementary materials. Thus, there is a critical need for greater transparency, standardization and translation of relevant methodology in Bayesian RCTs. |
| title | A systematic review of sample size determination in Bayesian randomized clinical trials: full Bayesian methods are rarely used |
| topic | Applications |
| url | https://arxiv.org/abs/2505.15735 |