Assessment of the value of confirming responses in clinical trials in oncology
Pérez-Gracia J.L. (a), Muñoz M. (b), Williams G. (c), Wu J. (d), Carrasco E. (b), García-Ribas I. (b), Peiro A. (b), López-Picazo J.M. (a), Gúrpide A. (a), Chopitea A. (a), Martín-Algarra S. (a), García-Foncillas J. (a), Blatter J. (e)
(a) Medical Oncology Department, Clínica Universitaria de Navarra, Avenida de Pío XII 36, 31008 Pamplona, Spain.
(b) Clinical Research Department, Eli Lilly and Company, Madrid, Spain.
(c) US Food and rug Administration, USA.
(d) Clinical Research Department, Eli Lilly and Company Indianapolis, USA.
(e) Clinical Research Department, Eli Lilly and Company, Frankfort, Germany.
The requirement for a second assessment to confirm initial tumour response is required by all response guidelines. Its rationale, however, is not clear. We have conducted this study to compare validity of response rate assessment determined with and without secondary confirmation.
Using specified criteria, nine trials of one single cytotoxic drug including 416 patients were selected from a pharmaceutical database. Objective response rates were determined by a single determination and by two separate determinations. 81 responses (19.5%, [15.8-23.6%]) were scored by the confirmation method and 97 responses (23.3% [19.3-27.7%]) by the no-confirmation method. The Kappa (kappa) coefficient of 0.89 indicates good agreement between both methods. This is the first study that systematically compares response rates calculated with and without performing response confirmation. Results show good agreement between both methods.
We suggest that assessing response without confirmation may be the preferred method. These results should be confirmed by additional studies in a variety of cancer settings.
CITATION Eur J Cancer. 2005 Jul;41(11):1528-32