New insights into adaptive enrichment designs

Alessandro Baldi Antognini, Rosamarie Frieri () and Maroussa Zagoraiou ()
Additional contact information
Rosamarie Frieri: University of Bologna
Maroussa Zagoraiou: University of Bologna

Statistical Papers, 2023, vol. 64, issue 4, No 15, 1305-1328

Abstract: Abstract The transition towards personalized medicine is happening and the new experimental framework is raising several challenges, from a clinical, ethical, logistical, regulatory, and statistical perspective. To face these challenges, innovative study designs with increasing complexity have been proposed. In particular, adaptive enrichment designs are becoming more attractive for their flexibility. However, these procedures rely on an increasing number of parameters that are unknown at the planning stage of the clinical trial, so the study design requires particular care. This review is dedicated to adaptive enrichment studies with a focus on design aspects. While many papers deal with methods for the analysis, the sample size determination and the optimal allocation problem have been overlooked. We discuss the multiple aspects involved in adaptive enrichment designs that contribute to their advantages and disadvantages. The decision-making process of whether or not it is worth enriching should be driven by clinical and ethical considerations as well as scientific and statistical concerns.

Keywords: Continuous biomarker; Personalized medicine; Predictive biomarker; Stratified medicine; Subgroup identification (search for similar items in EconPapers)
Date: 2023
References: View references in EconPapers View complete reference list from CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s00362-023-01433-0 Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:stpapr:v:64:y:2023:i:4:d:10.1007_s00362-023-01433-0

Ordering information: This journal article can be ordered from
http://www.springer. ... business/journal/362

DOI: 10.1007/s00362-023-01433-0

Access Statistics for this article

Statistical Papers is currently edited by C. Müller, W. Krämer and W.G. Müller

More articles in Statistical Papers from Springer
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().