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    Rights statement: The final, definitive version of this article has been published in the Journal, Statistical Methods in Medical Research, 25 (3), 2016, © SAGE Publications Ltd, 2016 by SAGE Publications Ltd at the Statistical Methods in Medical Research page: http://smm.sagepub.com/ on SAGE Journals Online: http://online.sagepub.com/

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A review of statistical designs for improving the efficiency of phase II studies in oncology

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A review of statistical designs for improving the efficiency of phase II studies in oncology. / Wason, James; Jaki, Thomas.

In: Statistical Methods in Medical Research, Vol. 25, No. 3, 06.2016, p. 1010-1021.

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Wason J, Jaki T. A review of statistical designs for improving the efficiency of phase II studies in oncology. Statistical Methods in Medical Research. 2016 Jun;25(3):1010-1021. Epub 2015 May 31. doi: 10.1177/0962280215588247

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Wason, James ; Jaki, Thomas. / A review of statistical designs for improving the efficiency of phase II studies in oncology. In: Statistical Methods in Medical Research. 2016 ; Vol. 25, No. 3. pp. 1010-1021.

Bibtex

@article{d391824e921b4846ac666ab3bb818aa9,
title = "A review of statistical designs for improving the efficiency of phase II studies in oncology",
abstract = "Phase II oncology trials are carried out to assess whether an experimental anti-cancer treatment shows sufficient signs of effectiveness to justify being tested in a phase III trial. Traditionally such trials are conducted as single-arm studies using a binary response rate as the primary endpoint. In this article, we review and contrast alternative approaches for such studies. Each approach uses only data that are necessary for the traditional analysis. We consider two broad classes of methods: ones that aim to improve the efficiency using novel design ideas, such as multi-stage and multi-arm multi-stage designs; and ones that aim to improve the analysis, by making better use of the richness of the data that is ignored in the traditional analysis. The former class of methods provides considerable gains in efficiency but also increases the administrative and logistical issues in running the trial. The second class consists of viable alternatives to the standard analysis that come with little additional requirements and provide considerable gains in efficiency.",
keywords = "augmented methods, continuous, multi-stage, phase II, RECIST, oncology",
author = "James Wason and Thomas Jaki",
note = "The final, definitive version of this article has been published in the Journal, Statistical Methods in Medical Research, 25 (3), 2016, {\textcopyright} SAGE Publications Ltd, 2016 by SAGE Publications Ltd at the Statistical Methods in Medical Research page: http://smm.sagepub.com/ on SAGE Journals Online: http://online.sagepub.com/ ",
year = "2016",
month = jun,
doi = "10.1177/0962280215588247",
language = "English",
volume = "25",
pages = "1010--1021",
journal = "Statistical Methods in Medical Research",
issn = "0962-2802",
publisher = "SAGE Publications Ltd",
number = "3",

}

RIS

TY - JOUR

T1 - A review of statistical designs for improving the efficiency of phase II studies in oncology

AU - Wason, James

AU - Jaki, Thomas

N1 - The final, definitive version of this article has been published in the Journal, Statistical Methods in Medical Research, 25 (3), 2016, © SAGE Publications Ltd, 2016 by SAGE Publications Ltd at the Statistical Methods in Medical Research page: http://smm.sagepub.com/ on SAGE Journals Online: http://online.sagepub.com/

PY - 2016/6

Y1 - 2016/6

N2 - Phase II oncology trials are carried out to assess whether an experimental anti-cancer treatment shows sufficient signs of effectiveness to justify being tested in a phase III trial. Traditionally such trials are conducted as single-arm studies using a binary response rate as the primary endpoint. In this article, we review and contrast alternative approaches for such studies. Each approach uses only data that are necessary for the traditional analysis. We consider two broad classes of methods: ones that aim to improve the efficiency using novel design ideas, such as multi-stage and multi-arm multi-stage designs; and ones that aim to improve the analysis, by making better use of the richness of the data that is ignored in the traditional analysis. The former class of methods provides considerable gains in efficiency but also increases the administrative and logistical issues in running the trial. The second class consists of viable alternatives to the standard analysis that come with little additional requirements and provide considerable gains in efficiency.

AB - Phase II oncology trials are carried out to assess whether an experimental anti-cancer treatment shows sufficient signs of effectiveness to justify being tested in a phase III trial. Traditionally such trials are conducted as single-arm studies using a binary response rate as the primary endpoint. In this article, we review and contrast alternative approaches for such studies. Each approach uses only data that are necessary for the traditional analysis. We consider two broad classes of methods: ones that aim to improve the efficiency using novel design ideas, such as multi-stage and multi-arm multi-stage designs; and ones that aim to improve the analysis, by making better use of the richness of the data that is ignored in the traditional analysis. The former class of methods provides considerable gains in efficiency but also increases the administrative and logistical issues in running the trial. The second class consists of viable alternatives to the standard analysis that come with little additional requirements and provide considerable gains in efficiency.

KW - augmented methods

KW - continuous

KW - multi-stage

KW - phase II

KW - RECIST

KW - oncology

U2 - 10.1177/0962280215588247

DO - 10.1177/0962280215588247

M3 - Journal article

VL - 25

SP - 1010

EP - 1021

JO - Statistical Methods in Medical Research

JF - Statistical Methods in Medical Research

SN - 0962-2802

IS - 3

ER -