A Rescue Strategy for Handling Unevaluable Patients in Simon’s Two Stage Design

Lisa Belin, Philippe Broët and Yann De Rycke

PLOS ONE, 2015, vol. 10, issue 9, 1-14

Abstract: For phase II oncology trials, Simon’s two-stage design is the most commonly used strategy. However, when clinically unevaluable patients occur, the total number of patients included at each stage differs from what was initially planned. Such situations raise concerns about the operating characteristics of the trial design. This paper evaluates three classical ad hoc strategies and a novel one proposed in this work for handling unevaluable patients. This latter is called the rescue strategy which adapts the critical stopping rules to the number of unevaluable patients at each stage without modifying the planned sample size. blue Simulations show that none of these strategies perfectly match the original target constraints for type I and II error rates. Our rescue strategy is nevertheless the one which best approaches the target error rates requirement. A re-analysis of one real phase II clinical trials on metastatic cancer illustrates the use of the proposed strategy.

Date: 2015
References: View complete reference list from CitEc
Citations:

Downloads: (external link)
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0137586 (text/html)
https://journals.plos.org/plosone/article/file?id= ... 37586&type=printable (application/pdf)

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:plo:pone00:0137586

DOI: 10.1371/journal.pone.0137586

Access Statistics for this article

More articles in PLOS ONE from Public Library of Science
Bibliographic data for series maintained by plosone ().