Bayesian survival analysis for early detection of treatment effects in phase 3 clinical trials.
Bayesian inference
Censored data
Clinical trial
Historical data
Journal
Contemporary clinical trials communications
ISSN: 2451-8654
Titre abrégé: Contemp Clin Trials Commun
Pays: Netherlands
ID NLM: 101671157
Informations de publication
Date de publication:
Mar 2021
Mar 2021
Historique:
received:
20
07
2020
revised:
04
12
2020
accepted:
04
01
2021
entrez:
29
1
2021
pubmed:
30
1
2021
medline:
30
1
2021
Statut:
epublish
Résumé
Despite appealing characteristics for the clinical trials setting, Bayesian inference methods remain scarcely used, especially in randomized controlled clinical trials (RCT). This is particularly true when dealing with a survival endpoint, likely due to the additional complexities to model specifications. We propose to use Bayesian inference to estimate the treatment effect in this setting, using a proportional hazards (PH) model for right-censored data. Implementation of such an estimation process is illustrated on two working examples from cancer RCTs, the ALLOZITHRO and the CLL7-SA trials, both originally analyzed using a frequentist approach. In these two different settings, we show that Bayesian sequential analyses can provide early insight on treatment effect in RCTs. Relying on posterior distributions and predictive posterior probabilities, we find that Bayesian sequential analyses of the ALLOZITHRO trial, which was terminated early due to an unanticipated deleterious effect of the intervention on survival, allow quantifying early that the treatment effect was opposite to what was expected. Then, incorporating historical data in the sequential analyses of the CLL7-SA trial would have allowed the treatment effect to be closer to the protocol hypothesis. These
Identifiants
pubmed: 33511301
doi: 10.1016/j.conctc.2021.100709
pii: S2451-8654(21)00011-9
pmc: PMC7817368
doi:
Types de publication
Journal Article
Langues
eng
Pagination
100709Informations de copyright
© 2021 The Authors.
Déclaration de conflit d'intérêts
The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
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