Cover Image

Extension of a conditional performance score for sample size recalculation rules to the setting of binary endpoints

Sample size calculation is a central aspect in planning of clinical trials. The sample size is calculated based on parameter assumptions, like the treatment effect and the endpoint’s variance. A fundamental problem of this approach is that the true distribution parameters are not known before the tr...

Full description

Saved in:
Bibliographic Details
Main Authors: Bokelmann, Björn (Author) , Rauch, Geraldine (Author) , Meis, Jan (Author) , Kieser, Meinhard (Author) , Herrmann, Carolin (Author)
Format: Article (Journal)
Language:English
Published: 19 January 2024
In: BMC medical research methodology
Year: 2024, Volume: 24, Pages: 1-14
ISSN:1471-2288
DOI:10.1186/s12874-024-02150-4
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1186/s12874-024-02150-4
Verlag, kostenfrei, Volltext: https://bmcmedresmethodol.biomedcentral.com/articles/10.1186/s12874-024-02150-4
Get full text
Author Notes:Björn Bokelmann, Geraldine Rauch, Jan Meis, Meinhard Kieser and Carolin Herrmann
Description
Summary:Sample size calculation is a central aspect in planning of clinical trials. The sample size is calculated based on parameter assumptions, like the treatment effect and the endpoint’s variance. A fundamental problem of this approach is that the true distribution parameters are not known before the trial. Hence, sample size calculation always contains a certain degree of uncertainty, leading to the risk of underpowering or oversizing a trial. One way to cope with this uncertainty are adaptive designs. Adaptive designs allow to adjust the sample size during an interim analysis. There is a large number of such recalculation rules to choose from. To guide the choice of a suitable adaptive design with sample size recalculation, previous literature suggests a conditional performance score for studies with a normally distributed endpoint. However, binary endpoints are also frequently applied in clinical trials and the application of the conditional performance score to binary endpoints is not yet investigated.
Item Description:Gesehen am 21.11.2024
Physical Description:Online Resource
ISSN:1471-2288
DOI:10.1186/s12874-024-02150-4

Similar Items