Biomarkers in drug development: Friend or foe? A personal reflection gained working within oncology

Pharmaceutical Statistics

Volumn 6, Issue 4, 2007, Pages 253-260

  Carroll, Kevin J ^a  

^a ASTRAZENECA   (United Kingdom)

Author keywords

Biomarkers; Oncology; Predictive; Prognostic; Surgery

Indexed keywords

ANTINEOPLASTIC AGENT; EPIDERMAL GROWTH FACTOR RECEPTOR; EPIDERMAL GROWTH FACTOR RECEPTOR 2; PROSTATE SPECIFIC ANTIGEN; TRASTUZUMAB;

ARTICLE; BREAST CANCER; CANCER SURVIVAL; CLINICAL TRIAL; COLORECTAL CANCER; DEPRESSION; DISEASE MARKER; DRUG APPROVAL; DRUG EFFICACY; DRUG RESEARCH; DRUG RESPONSE; DRUG SAFETY; GENE MUTATION; HUMAN; LUNG NON SMALL CELL CANCER; NATIONAL INSTITUTES OF HEALTH STROKE SCALE; PATIENT SELECTION; PHARMACOGENETICS; POSITIVE AND NEGATIVE SYNDROME SCALE; PREDICTION; PROGNOSIS; PROSTATE CANCER; PROTEIN EXPRESSION; RISK REDUCTION; SENSITIVITY AND SPECIFICITY; STROKE; TREATMENT OUTCOME;

BIOMARKERS, PHARMACOLOGICAL; CLINICAL TRIALS AS TOPIC; DRUG DESIGN; ENDPOINT DETERMINATION; HUMANS; NEOPLASMS; PATIENT SELECTION; PROGNOSIS; RESEARCH DESIGN;

EID: 37349117673     PISSN: 15391604     EISSN: 15391612     Source Type: Journal    
DOI: 10.1002/pst.269     Document Type: Article

References (16)

1. De Gruttola VG, Clax P, DeMets DL et al. Considerations in the evaluation of surrogate end-points in clinical trials: summary of a National Institutes of Health Workshop. Controlled Clinical Trials 2001; 22:485-502.
2. Senn SJ. Individual response to treatment: is it a valid assumption? British Medical Journal 2004; 329:966-968.
3. Paez JG, Janne PA, Lee JC et al. EGFR mutations in lung cancer: correlation with clinical response to gefitinib therapy. Science 2004; 304:1497-1500.
4. Lynch TJ, Bell DW, Sordella R et al. Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small-cell lung cancer to gefitinib. New England Journal of Medicine 2004; 350:2129-2139.
5. Eberhard DA, Johnson BE, Amler LC et al. Mutations in the epidermal growth factor receptor and in KRAS are predictive and prognostic indicators in patients with non-small-cell lung cancer treated with chemotherapy alone and in combination with erlotinib. Journal of Clinical Oncology 2005; 23:5900-5909.
6. Blackwelder WC, Hastings BK, Lee ML, Deloria MA. Value of a run-in period in a drug trial during pregnancy. Controlled Clinical Trials 1990; 11:187-198.
7. Brittain E, Wittes J. The run-in period in clinical trials. The effect of misclassification on efficiency. Controlled Clinical Trials 1990; 11:327-338.
8. Schechtman KB, Gordon ME. A comprehensive algorithm for determining whether a run-in strategy will be a cost-effective design modification in a randomized clinical trial. Statistics in Medicine 1993; 12:111-128.
9. Maitournam A, Simon R. On the efficiency of targeted clinical trials. Statistics in Medicine 2005; 24:329-339
10. Prentice RL. Surrogate endpoints in clinical trials: definitions and operational criteria. Statistics in Medicine 1989; 8:431-440.
11. Freedman LS, Graubard BI, Schatzkin A. Statistical validation of intermediate endpoints for chronic diseases. Statistics in Medicine 1992; 11:167-178.
12. Buyse M, Molenberghs G. Criteria for the validation of surrogate endpoints in randomized experiments. Biometrics 1998; 54:1014-1029.
13. Molenberghs G, Geys H, Buyse M. Evaluation of surrogate endpoints in randomized experiments with mixed discrete and continuous outcomes. Statistics in Medicine 2001; 20:3023-3038.
14. Collette L, Burzykowski T, Carroll KJ, Newling D, Morris T, Schröder FH. Is prostate-specific antigen a valid surrogate end point for survival in hormonally treated patients with metastatic prostate cancer? Joint Research of the European Organisation for Research and Treatment of Cancer, the Limburgs Universitair Centrum, and AstraZeneca Pharmaceuticals. Journal of Clinical Oncology 2005; 23:6139-6148.
15. FDA Public Workshop on Clinical Trial Endpoints in Prostate Cancer, June 21-22, 2004. Available at: http://www.fda.gov/cder/drug/cancer_endpoints/ default.htm#prostate (last accessed 19 January 2007).
16. Buyse M, Burzykowski T, Carroll K, Michiels S, Pignon JP, Piedbois P, Meta-Analysis Group in Cancer (MAGIC). Progression-free survival (PFS) as a surrogate for overall survival (OS) in patients with advanced colorectal cancer: an analysis of 3159 patients randomized in 11 trials. Journal of Clinical Oncology, 2005. ASCO Annual Meeting Proceedings. Vol 23, No. 16S, Part I of II (June 1 Supplement), 2005, 3513pp. Available at: http://www.asco.org/portal/site/ASCO/ menuitem.34d60f5624ba07fd506fe310ee37a01d/