Point estimation and p-values in phase II adaptive two-stage designs with a binary endpoint
Clinical trials in phase II of drug development are frequently conducted as single-arm two-stage studies with a binary endpoint. Recently, adaptive designs have been proposed for this setting that enable a midcourse modification of the sample size. While these designs are elaborated with respect to...
Saved in:
| Main Authors: | , |
|---|---|
| Format: | Article (Journal) |
| Language: | English |
| Published: |
15 March 2017
|
| In: |
Statistics in medicine
Year: 2017, Volume: 36, Issue: 6, Pages: 971-984 |
| ISSN: | 1097-0258 |
| DOI: | 10.1002/sim.7200 |
| Online Access: | Verlag, Volltext: http://dx.doi.org/10.1002/sim.7200 Verlag, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/sim.7200 
|
| Author Notes: | K. Kunzmann and M. Kieser |
| Summary: | Clinical trials in phase II of drug development are frequently conducted as single-arm two-stage studies with a binary endpoint. Recently, adaptive designs have been proposed for this setting that enable a midcourse modification of the sample size. While these designs are elaborated with respect to hypothesis testing by assuring control of the type I error rate, the topic of point estimation has up to now not been addressed. For adaptive designs with a prespecified sample size recalculation rule, we propose a new point estimator that both assures compatibility of estimation and test decision and minimizes average mean squared error. This estimator can be interpreted as a constrained posterior mean estimate based on the non-informative Jeffreys prior. A comparative investigation of the operating characteristics demonstrates the favorable properties of the proposed approach. Copyright © 2016 John Wiley & Sons, Ltd. |
|---|---|
| Item Description: | First published: 27 December 2016 Gesehen am 05.11.2018 |
| Physical Description: | Online Resource |
| ISSN: | 1097-0258 |
| DOI: | 10.1002/sim.7200 |