Lancaster EPrints

Designing exploratory cancer trials using change in tumour size as primary endpoint

Jaki, Thomas and Andre, Valerie and Su, Ting-Li and Whitehead, John (2013) Designing exploratory cancer trials using change in tumour size as primary endpoint. Statistics in Medicine, 32 (15). pp. 2544-2554. ISSN 0277-6715

PDF - Draft Version
Download (560Kb) | Preview


    In phase III cancer clinical trials, overall survival is commonly used as the definitive endpoint. In phase II clinical trials, however, more immediate endpoints such as incidence of complete or partial response within 1 or 2 months or progression-free survival (PFS) are generally used. Because of the limited ability to detect change in overall survival with response, the inherent variability of PFS and the long wait for progression to be observed, more informative and immediate alternatives to overall survival are desirable in exploratory phase II trials. In this paper, we show how comparative trials can be designed and analysed using change in tumour size as the primary endpoint. The test developed is based on the framework of score statistics and will formally incorporate the information of whether patients survive until the time at which change in tumour size is assessed. Using an example in non-small cell lung cancer, we show that the sample size requirements for a trial based on change in tumour size are favourable compared with alternative randomized trials and demonstrate that these conclusions are robust to our assumptions.

    Item Type: Journal Article
    Journal or Publication Title: Statistics in Medicine
    Additional Information: The definitive version is available at
    Uncontrolled Keywords: cancer ; tumour size ; phase II ; design ; score statistic
    Departments: Faculty of Science and Technology > Mathematics and Statistics
    ID Code: 60471
    Deposited By: ep_importer_pure
    Deposited On: 06 Dec 2012 11:29
    Refereed?: Yes
    Published?: Published
    Last Modified: 24 May 2018 00:20
    Identification Number:

    Actions (login required)

    View Item