Incorporating historical two-arm data in clinical trials with binary outcome: A practical approach
The feasibility of a new clinical trial may be increased by incorporating historical data of previous trials. In the particular case where only data from a single historical trial are available, there exists no clear recommendation in the literature regarding the most favorable approach. A main prob...
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| Main Authors: | , , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
2020
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| In: |
Pharmaceutical statistics
Year: 2020, Volume: 19, Issue: 5, Pages: 662-678 |
| ISSN: | 1539-1612 |
| DOI: | 10.1002/pst.2023 |
| Online Access: | Verlag, kostenfrei, Volltext: https://doi.org/10.1002/pst.2023 Verlag, kostenfrei, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/pst.2023 
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| Author Notes: | Manuel Feißt, Johannes Krisam, Meinhard Kieser |
| Summary: | The feasibility of a new clinical trial may be increased by incorporating historical data of previous trials. In the particular case where only data from a single historical trial are available, there exists no clear recommendation in the literature regarding the most favorable approach. A main problem of the incorporation of historical data is the possible inflation of the type I error rate. A way to control this type of error is the so-called power prior approach. This Bayesian method does not “borrow” the full historical information but uses a parameter 0 ≤ δ ≤ 1 to determine the amount of borrowed data. Based on the methodology of the power prior, we propose a frequentist framework that allows incorporation of historical data from both arms of two-armed trials with binary outcome, while simultaneously controlling the type I error rate. It is shown that for any specific trial scenario a value δ > 0 can be determined such that the type I error rate falls below the prespecified significance level. The magnitude of this value of δ depends on the characteristics of the data observed in the historical trial. Conditionally on these characteristics, an increase in power as compared to a trial without borrowing may result. Similarly, we propose methods how the required sample size can be reduced. The results are discussed and compared to those obtained in a Bayesian framework. Application is illustrated by a clinical trial example. |
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| Item Description: | First published: 30 March 2020 Gesehen am 10.01.2021 |
| Physical Description: | Online Resource |
| ISSN: | 1539-1612 |
| DOI: | 10.1002/pst.2023 |