Monotonicity conditions for avoiding counterintuitive decisions in basket trials.
Bayesian statistics
basket trial
clinical trial design
information borrowing
Journal
Biometrical journal. Biometrische Zeitschrift
ISSN: 1521-4036
Titre abrégé: Biom J
Pays: Germany
ID NLM: 7708048
Informations de publication
Date de publication:
06 2022
06 2022
Historique:
revised:
11
02
2022
received:
17
09
2021
accepted:
05
03
2022
entrez:
12
6
2022
pubmed:
13
6
2022
medline:
15
6
2022
Statut:
ppublish
Résumé
In a basket trial, a new treatment is tested in different subgroups, called the baskets. In oncology, the baskets usually comprise patients with different primary tumor sites but a common biomarker. Most basket trials are uncontrolled phase II trials and investigate a binary endpoint such as tumor response. To combine the data of baskets that show a similar response to the treatment, many basket trial designs use Bayesian borrowing methods. This increases the power compared to a basketwise analysis. However, it can lead to posterior probabilities that are not monotonically increasing in the number of responses. We show that, as a consequence, two types of counterintuitive decisions can arise-one that occurs within a single trial and one that occurs when the results are compared between different trials. We propose two monotonicity conditions for the inference in basket trials. Using a design recently proposed by Fujikawa and colleagues, we investigate the case of a single-stage basket trial with equal sample sizes in all baskets and show that, as the number of baskets increases, these conditions are violated for a wide range of different borrowing strengths. We show that in the investigated scenarios pruning baskets can help to ensure that the monotonicity conditions hold and investigate how this affects type I error rate and power.
Identifiants
pubmed: 35692061
doi: 10.1002/bimj.202100287
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
934-947Informations de copyright
© 2022 The Authors. Biometrical Journal published by Wiley-VCH GmbH.
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