Bayesian Design in Clinical Trials

In the last decade, the number of clinical trials using Bayesian methods has grown dramatically. Nowadays, regulatory authorities appear to be more receptive to Bayesian methods than ever. The Bayesian methodology is well suited to address the issues arising in the planning, analysis, and conduct of...

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Format: Online
Language:English
Published: MDPI - Multidisciplinary Digital Publishing Institute 2022
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Online Access:ONIX_20220321_9783036533339_110
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description In the last decade, the number of clinical trials using Bayesian methods has grown dramatically. Nowadays, regulatory authorities appear to be more receptive to Bayesian methods than ever. The Bayesian methodology is well suited to address the issues arising in the planning, analysis, and conduct of clinical trials. Due to their flexibility, Bayesian design methods based on the accrued data of ongoing trials have been recommended by both the US Food and Drug Administration and the European Medicines Agency for dose-response trials in early clinical development. A distinctive feature of the Bayesian approach is its ability to deal with external information, such as historical data, findings from previous studies and expert opinions, through prior elicitation. In fact, it provides a framework for embedding and handling the variability of auxiliary information within the planning and analysis of the study. A growing body of literature examines the use of historical data to augment newly collected data, especially in clinical trials where patients are difficult to recruit, which is the case for rare diseases, for example. Many works explore how this can be done properly, since using historical data has been recognized as less controversial than eliciting prior information from experts’ opinions. In this book, applications of Bayesian design in the planning and analysis of clinical trials are introduced, along with methodological contributions to specific topics of Bayesian statistics. Finally, two reviews regarding the state-of-the-art of the Bayesian approach in clinical field trials are presented.
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language eng
publishDate 2022
publishDateRange 2022
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publisher MDPI - Multidisciplinary Digital Publishing Institute
publisherStr MDPI - Multidisciplinary Digital Publishing Institute
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spelling doab-20.500.12854ir-796742024-04-01T23:19:27Z Bayesian Design in Clinical Trials Berchialla, Paola Baldi, Ileana dose-escalation combination study modelling assumption interaction adaptive designs adaptive randomization Bayesian designs clinical trials predictive power target allocation Bayesian inference highest posterior density intervals normal approximation predictive analysis sample size determination bayesian meta-analysis clustering binary data priors frequentist validation Bayesian rare disease prior distribution meta-analysis sample size bridging studies distribution distance oncology phase I dose-finding dose–response bayesian inference prior elicitation latent dirichlet allocation clinical trial power-prior poor accrual Bayesian trial cisplatin doxorubicin oxaliplatin dose escalation PIPAC peritoneal carcinomatosis randomized controlled trial causal inference doubly robust estimation propensity score Bayesian monitoring futility rules interim analysis posterior and predictive probabilities stopping boundaries Bayesian trial design early phase dose finding treatment combinations optimal dose combination thema EDItEUR::N History and Archaeology::NH History thema EDItEUR::J Society and Social Sciences::JB Society and culture: general::JBF Social and ethical issues In the last decade, the number of clinical trials using Bayesian methods has grown dramatically. Nowadays, regulatory authorities appear to be more receptive to Bayesian methods than ever. The Bayesian methodology is well suited to address the issues arising in the planning, analysis, and conduct of clinical trials. Due to their flexibility, Bayesian design methods based on the accrued data of ongoing trials have been recommended by both the US Food and Drug Administration and the European Medicines Agency for dose-response trials in early clinical development. A distinctive feature of the Bayesian approach is its ability to deal with external information, such as historical data, findings from previous studies and expert opinions, through prior elicitation. In fact, it provides a framework for embedding and handling the variability of auxiliary information within the planning and analysis of the study. A growing body of literature examines the use of historical data to augment newly collected data, especially in clinical trials where patients are difficult to recruit, which is the case for rare diseases, for example. Many works explore how this can be done properly, since using historical data has been recognized as less controversial than eliciting prior information from experts’ opinions. In this book, applications of Bayesian design in the planning and analysis of clinical trials are introduced, along with methodological contributions to specific topics of Bayesian statistics. Finally, two reviews regarding the state-of-the-art of the Bayesian approach in clinical field trials are presented. 2022-03-21T16:30:22Z 2022-03-21T16:30:22Z 2022 book ONIX_20220321_9783036533339_110 9783036533339 https://directory.doabooks.org/handle/20.500.12854/79674 eng image/jpeg Attribution 4.0 International https://mdpi.com/books/pdfview/book/5059 https://mdpi.com/books/pdfview/book/5059 MDPI - Multidisciplinary Digital Publishing Institute 10.3390/books978-3-0365-3333-9 10.3390/books978-3-0365-3333-9 46cabcaa-dd94-4bfe-87b4-55023c1b36d0 9783036533339 190 Basel open access
spellingShingle dose-escalation
combination study
modelling assumption
interaction
adaptive designs
adaptive randomization
Bayesian designs
clinical trials
predictive power
target allocation
Bayesian inference
highest posterior density intervals
normal approximation
predictive analysis
sample size determination
bayesian meta-analysis
clustering
binary data
priors
frequentist validation
Bayesian
rare disease
prior distribution
meta-analysis
sample size
bridging studies
distribution distance
oncology
phase I
dose-finding
dose–response
bayesian inference
prior elicitation
latent dirichlet allocation
clinical trial
power-prior
poor accrual
Bayesian trial
cisplatin
doxorubicin
oxaliplatin
dose escalation
PIPAC
peritoneal carcinomatosis
randomized controlled trial
causal inference
doubly robust estimation
propensity score
Bayesian monitoring
futility rules
interim analysis
posterior and predictive probabilities
stopping boundaries
Bayesian trial design
early phase dose finding
treatment combinations
optimal dose combination
thema EDItEUR::N History and Archaeology::NH History
thema EDItEUR::J Society and Social Sciences::JB Society and culture: general::JBF Social and ethical issues
Bayesian Design in Clinical Trials
title Bayesian Design in Clinical Trials
title_full Bayesian Design in Clinical Trials
title_fullStr Bayesian Design in Clinical Trials
title_full_unstemmed Bayesian Design in Clinical Trials
title_short Bayesian Design in Clinical Trials
title_sort bayesian design in clinical trials
topic dose-escalation
combination study
modelling assumption
interaction
adaptive designs
adaptive randomization
Bayesian designs
clinical trials
predictive power
target allocation
Bayesian inference
highest posterior density intervals
normal approximation
predictive analysis
sample size determination
bayesian meta-analysis
clustering
binary data
priors
frequentist validation
Bayesian
rare disease
prior distribution
meta-analysis
sample size
bridging studies
distribution distance
oncology
phase I
dose-finding
dose–response
bayesian inference
prior elicitation
latent dirichlet allocation
clinical trial
power-prior
poor accrual
Bayesian trial
cisplatin
doxorubicin
oxaliplatin
dose escalation
PIPAC
peritoneal carcinomatosis
randomized controlled trial
causal inference
doubly robust estimation
propensity score
Bayesian monitoring
futility rules
interim analysis
posterior and predictive probabilities
stopping boundaries
Bayesian trial design
early phase dose finding
treatment combinations
optimal dose combination
thema EDItEUR::N History and Archaeology::NH History
thema EDItEUR::J Society and Social Sciences::JB Society and culture: general::JBF Social and ethical issues
topic_facet dose-escalation
combination study
modelling assumption
interaction
adaptive designs
adaptive randomization
Bayesian designs
clinical trials
predictive power
target allocation
Bayesian inference
highest posterior density intervals
normal approximation
predictive analysis
sample size determination
bayesian meta-analysis
clustering
binary data
priors
frequentist validation
Bayesian
rare disease
prior distribution
meta-analysis
sample size
bridging studies
distribution distance
oncology
phase I
dose-finding
dose–response
bayesian inference
prior elicitation
latent dirichlet allocation
clinical trial
power-prior
poor accrual
Bayesian trial
cisplatin
doxorubicin
oxaliplatin
dose escalation
PIPAC
peritoneal carcinomatosis
randomized controlled trial
causal inference
doubly robust estimation
propensity score
Bayesian monitoring
futility rules
interim analysis
posterior and predictive probabilities
stopping boundaries
Bayesian trial design
early phase dose finding
treatment combinations
optimal dose combination
thema EDItEUR::N History and Archaeology::NH History
thema EDItEUR::J Society and Social Sciences::JB Society and culture: general::JBF Social and ethical issues
url ONIX_20220321_9783036533339_110