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Designing exploratory cancer trials using change in tumour size as primary endpoint.
- Source :
-
Statistics in medicine [Stat Med] 2013 Jul 10; Vol. 32 (15), pp. 2544-54. Date of Electronic Publication: 2012 Dec 19. - Publication Year :
- 2013
-
Abstract
- 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.<br /> (Copyright © 2012 John Wiley & Sons, Ltd.)
- Subjects :
- Biostatistics methods
Carcinoma, Non-Small-Cell Lung pathology
Carcinoma, Non-Small-Cell Lung therapy
Clinical Trials as Topic statistics & numerical data
Clinical Trials, Phase II as Topic methods
Clinical Trials, Phase II as Topic statistics & numerical data
Clinical Trials, Phase III as Topic methods
Clinical Trials, Phase III as Topic statistics & numerical data
Databases, Factual
Endpoint Determination methods
Endpoint Determination statistics & numerical data
Humans
Lung Neoplasms pathology
Lung Neoplasms therapy
Models, Statistical
Randomized Controlled Trials as Topic methods
Randomized Controlled Trials as Topic statistics & numerical data
Sample Size
Clinical Trials as Topic methods
Neoplasms pathology
Neoplasms therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1097-0258
- Volume :
- 32
- Issue :
- 15
- Database :
- MEDLINE
- Journal :
- Statistics in medicine
- Publication Type :
- Academic Journal
- Accession number :
- 23280944
- Full Text :
- https://doi.org/10.1002/sim.5716