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@ARTICLE{Calderazzo:157332,
author = {S. Calderazzo$^*$ and M. Wiesenfarth$^*$ and A.
Kopp-Schneider$^*$},
title = {{A} decision-theoretic approach to {B}ayesian clinical
trial design and evaluation of robustness to prior-data
conflict.},
journal = {Biostatistics},
volume = {23},
number = {1},
issn = {1468-4357},
address = {Oxford [u.a.]},
publisher = {Oxford Univ. Press},
reportid = {DKFZ-2020-01561},
pages = {328–344},
year = {2022},
note = {Volume 23, Issue 1, January 2022, Pages 328–344},
abstract = {Bayesian clinical trials allow taking advantage of relevant
external information through the elicitation of prior
distributions, which influence Bayesian posterior parameter
estimates and test decisions. However, incorporation of
historical information can have harmful consequences on the
trial's frequentist (conditional) operating characteristics
in case of inconsistency between prior information and the
newly collected data. A compromise between meaningful
incorporation of historical information and strict control
of frequentist error rates is therefore often sought. Our
aim is thus to review and investigate the rationale and
consequences of different approaches to relaxing strict
frequentist control of error rates from a Bayesian
decision-theoretic viewpoint. In particular, we define an
integrated risk which incorporates losses arising from
testing, estimation, and sampling. A weighted combination of
the integrated risk addends arising from testing and
estimation allows moving smoothly between these two targets.
Furthermore, we explore different possible elicitations of
the test error costs, leading to test decisions based either
on posterior probabilities, or solely on Bayes factors.
Sensitivity analyses are performed following the convention
which makes a distinction between the prior of the
data-generating process, and the analysis prior adopted to
fit the data. Simulation in the case of normal and binomial
outcomes and an application to a one-arm proof-of-concept
trial, exemplify how such analysis can be conducted to
explore sensitivity of the integrated risk, the operating
characteristics, and the optimal sample size, to prior-data
conflict. Robust analysis prior specifications, which
gradually discount potentially conflicting prior
information, are also included for comparison. Guidance with
respect to cost elicitation, particularly in the context of
a Phase II proof-of-concept trial, is provided.},
cin = {C060},
ddc = {610},
cid = {I:(DE-He78)C060-20160331},
pnm = {313 - Krebsrisikofaktoren und Prävention (POF4-313)},
pid = {G:(DE-HGF)POF4-313},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:32735010},
doi = {10.1093/biostatistics/kxaa027},
url = {https://inrepo02.dkfz.de/record/157332},
}