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Point and interval estimation in two-stage adaptive designs with time to event data and biomarker-driven subpopulation selection
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Kimani, Peter K., Todd, Susan, Renfro, Lindsay A., Glimm, Ekkehard, Khan, Josephine N., Kairalla, John A. and Stallard, Nigel (2020) Point and interval estimation in two-stage adaptive designs with time to event data and biomarker-driven subpopulation selection. Statistics in Medicine, 39 (19). pp. 2568-2586. doi:10.1002/sim.8557 ISSN 0277-6715.
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WRAP-point-interval-estimation-two-stage-adaptive-designs-time-event-data-biomarker-driven-subpopulation-selection-Kimani-2020.pdf - Published Version - Requires a PDF viewer. Available under License Creative Commons Attribution 4.0. Download (1113Kb) | Preview |
Official URL: http://dx.doi.org/10.1002/sim.8557
Abstract
In personalized medicine, it is often desired to determine if all patients or only a subset of them benefit from a treatment. We consider estimation in two‐stage adaptive designs that in stage 1 recruit patients from the full population. In stage 2, patient recruitment is restricted to the part of the population, which, based on stage 1 data, benefits from the experimental treatment. Existing estimators, which adjust for using stage 1 data for selecting the part of the population from which stage 2 patients are recruited, as well as for the confirmatory analysis after stage 2, do not consider time to event patient outcomes. In this work, for time to event data, we have derived a new asymptotically unbiased estimator for the log hazard ratio and a new interval estimator with good coverage probabilities and probabilities that the upper bounds are below the true values. The estimators are appropriate for several selection rules that are based on a single or multiple biomarkers, which can be categorical or continuous.
Item Type: | Journal Article | |||||||||
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Subjects: | Q Science > QH Natural history > QH426 Genetics | |||||||||
Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School |
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Library of Congress Subject Headings (LCSH): | Biochemical markers -- Research, Clinical trials -- Design -- Research, Clinical trials -- Statistical methods | |||||||||
Journal or Publication Title: | Statistics in Medicine | |||||||||
Publisher: | John Wiley & Sons Ltd. | |||||||||
ISSN: | 0277-6715 | |||||||||
Official Date: | 30 August 2020 | |||||||||
Dates: |
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Volume: | 39 | |||||||||
Number: | 19 | |||||||||
Page Range: | pp. 2568-2586 | |||||||||
DOI: | 10.1002/sim.8557 | |||||||||
Status: | Peer Reviewed | |||||||||
Publication Status: | Published | |||||||||
Access rights to Published version: | Open Access (Creative Commons) | |||||||||
Date of first compliant deposit: | 12 May 2020 | |||||||||
Date of first compliant Open Access: | 14 May 2020 | |||||||||
RIOXX Funder/Project Grant: |
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