Practical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomes
<p>Bayesian analysis of a non-inferiority trial is advantageous in allowing direct probability statements to be made about the relative treatment difference rather than relying on an arbitrary and often poorly justified non-inferiority margin. When the primary analysis will be Bayesian, a Baye...
| Main Authors: | , , , , , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
Wiley
2023
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| _version_ | 1797109256445493248 |
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| author | Turner, RM Clements, MN Quartagno, M Cornelius, V Cro, S Ford, D Tweed, CD Walker, AS White, IR |
| author_facet | Turner, RM Clements, MN Quartagno, M Cornelius, V Cro, S Ford, D Tweed, CD Walker, AS White, IR |
| author_sort | Turner, RM |
| collection | OXFORD |
| description | <p>Bayesian analysis of a non-inferiority trial is advantageous in allowing direct probability statements to be made about the relative treatment difference rather than relying on an arbitrary and often poorly justified non-inferiority margin. When the primary analysis will be Bayesian, a Bayesian approach to sample size determination will often be appropriate for consistency with the analysis. We demonstrate three Bayesian approaches to choosing sample size for non-inferiority trials with binary outcomes and review their advantages and disadvantages.</p>
<p>First, we present a predictive power approach for determining sample size using the probability that the trial will produce a convincing result in the final analysis. Next, we determine sample size by considering the expected posterior probability of non-inferiority in the trial. Finally, we demonstrate a precision-based approach. We apply these methods to a non-inferiority trial in antiretroviral therapy for treatment of HIV-infected children.</p>
<p>A predictive power approach would be most accessible in practical settings, because it is analogous to the standard frequentist approach. Sample sizes are larger than with frequentist calculations unless an informative analysis prior is specified, because appropriate allowance is made for uncertainty in the assumed design parameters, ignored in frequentist calculations. An expected posterior probability approach will lead to a smaller sample size and is appropriate when the focus is on estimating posterior probability rather than on testing. A precision-based approach would be useful when sample size is restricted by limits on recruitment or costs, but it would be difficult to decide on sample size using this approach alone.</p> |
| first_indexed | 2024-03-07T07:39:14Z |
| format | Journal article |
| id | oxford-uuid:556cb257-6e0e-4501-8256-cc7bc8f06a44 |
| institution | University of Oxford |
| language | English |
| last_indexed | 2024-03-07T07:39:14Z |
| publishDate | 2023 |
| publisher | Wiley |
| record_format | dspace |
| spelling | oxford-uuid:556cb257-6e0e-4501-8256-cc7bc8f06a442023-04-12T10:04:00ZPractical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomesJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:556cb257-6e0e-4501-8256-cc7bc8f06a44EnglishSymplectic ElementsWiley2023Turner, RMClements, MNQuartagno, MCornelius, VCro, SFord, DTweed, CDWalker, ASWhite, IR<p>Bayesian analysis of a non-inferiority trial is advantageous in allowing direct probability statements to be made about the relative treatment difference rather than relying on an arbitrary and often poorly justified non-inferiority margin. When the primary analysis will be Bayesian, a Bayesian approach to sample size determination will often be appropriate for consistency with the analysis. We demonstrate three Bayesian approaches to choosing sample size for non-inferiority trials with binary outcomes and review their advantages and disadvantages.</p> <p>First, we present a predictive power approach for determining sample size using the probability that the trial will produce a convincing result in the final analysis. Next, we determine sample size by considering the expected posterior probability of non-inferiority in the trial. Finally, we demonstrate a precision-based approach. We apply these methods to a non-inferiority trial in antiretroviral therapy for treatment of HIV-infected children.</p> <p>A predictive power approach would be most accessible in practical settings, because it is analogous to the standard frequentist approach. Sample sizes are larger than with frequentist calculations unless an informative analysis prior is specified, because appropriate allowance is made for uncertainty in the assumed design parameters, ignored in frequentist calculations. An expected posterior probability approach will lead to a smaller sample size and is appropriate when the focus is on estimating posterior probability rather than on testing. A precision-based approach would be useful when sample size is restricted by limits on recruitment or costs, but it would be difficult to decide on sample size using this approach alone.</p> |
| spellingShingle | Turner, RM Clements, MN Quartagno, M Cornelius, V Cro, S Ford, D Tweed, CD Walker, AS White, IR Practical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomes |
| title | Practical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomes |
| title_full | Practical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomes |
| title_fullStr | Practical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomes |
| title_full_unstemmed | Practical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomes |
| title_short | Practical approaches to Bayesian sample size determination in non-inferiority trials with binary outcomes |
| title_sort | practical approaches to bayesian sample size determination in non inferiority trials with binary outcomes |
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