Early termination of a two-stage study to develop and validate a panel of biomarkers

Statistics in Medicine

Volumn 32, Issue 6, 2013, Pages 1027-1037

  Koopmeiners, Joseph S ^a,b   Vogel, Rachel Isaksson ^b  

^a UNIVERSITY OF MINNESOTA   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

Author keywords

Biomarker panel; Group sequential design; ROC curve

Indexed keywords

BIOLOGICAL MARKER;

ALTERNATIVE HYPOTHESIS; ARTICLE; CANCER DIAGNOSIS; CANCER PROGNOSIS; CANCER RESEARCH; EARLY TERMINATION OF CLINICAL TRIAL; METHODOLOGY; NULL HYPOTHESIS; PREDICTIVE VALUE; PROCESS DEVELOPMENT; PROSTATE CANCER; SAMPLE SIZE; SAMPLING ERROR; SEQUENTIAL ANALYSIS; SIMULATION; TWO STAGE DESIGN; VALIDATION STUDY;

COMPUTER SIMULATION; EARLY DETECTION OF CANCER; HUMANS; MALE; MODELS, STATISTICAL; NEOPLASM RECURRENCE, LOCAL; PREDICTIVE VALUE OF TESTS; PROSTATE-SPECIFIC ANTIGEN; PROSTATIC NEOPLASMS; REPRODUCIBILITY OF RESULTS; ROC CURVE; TUMOR MARKERS, BIOLOGICAL;

EID: 84873992447     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.5622     Document Type: Article

References (19)

1. Pepe MS. The Statistical Evaluation of Medical Tests for Classification and Prediction, Oxford Statistical Science Series, Vol.28. Oxford University Press: Oxford, 2003.
2. Pepe MS, Feng Z, Janes H, Bossuyt PM, Potter JD. Pivotal evaluation of the accuracy of a biomarker used for classification or prediction: standards for study design. Journal of the National Cancer Institute 2008; 100(20):1432-1438.
3. Mazumdar M, Liu A. Group sequential design for comparative diagnostic accuracy studies. Statistics in Medicine 2003; 22(5):727-739.
4. Mazumdar M. Group sequential design for comparative diagnostic accuracy studies: implications and guidelines for practitioners. Medical Decision Making 2004; 24(5):525-533. DOI: 10.1177/0272898X04269240.
5. Tang L, Emerson SS, Zhou XH. Nonparametric and semiparametric group sequential methods for comparing accuracy of diagnostic tests. Biometrics 2008; 64(4):1137-1145.
6. Heagerty PJ, Lumley T, Pepe MS. Time-dependent ROC curves for censored survival data and a diagnostic marker. Biometrics 2000; 56(2):337-344.
7. Tibshirani R. The lasso method for variable selection in the Cox model. Statistics in Medicine 1997; 16(4):385-395. DOI: 10.1002/(SICI)1097-0258(19970228)16:4385::AID-SIM3803.0.CO;2-3. (Available from: http://dx.doi.org/10.1002/(SICI)1097-0258(19970228)16:4385::AID-SIM3803.0.CO;2-3 [Accessed on May 2011]).
8. Simon N, Friedman JH, Hastie T, Tibshirani R. Regularization paths for Cox's proportional hazards model via coordinate descent. Journal of Statistical Software 2011; 39(5):1-13. (Available from: http://www.jstatsoft.org/v39/i05 [Accessed on May 2011]).
9. Jennison C, Turnbull BW. Group Sequential Methods with Applications to Clinical Trials. CRC Press Inc.: Boca Raton, Florida, USA, 2000.
10. O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1979; 35(3):549-556.
11. Pocock SJ. Group sequential methods in the design and analysis of clinical trials. Biometrika 1977; 64(2):191-199. DOI: 10.1093/biomet/64.2.191. (Available from: http://biomet.oxfordjournals.org/content/64/2/191.abstract [Accessed on May 2011]).
12. Lan KKG, DeMets DL. Discrete sequential boundaries for clinical trials. Biometrika 1983; 70(3):659-663. DOI: 10.1093/biomet/70.3.659. (Available from: http://biomet.oxfordjournals.org/content/70/3/659.abstract [Accessed on May 2011]).
13. Koopmeiners JS, Feng Z. Asymptotic properties of the sequential empirical ROC and PPV curves under case-control sampling. Annals of Statistics 2011; 39(6):3234-3261.
14. Hwang IK, Shih WJ, De Cani JS. Group sequential designs using a family of type I error probability spending functions. Statistics in Medicine 1990; 9(12):1439-1445.
15. Emerson SS, Fleming TR. Parameter estimation following group sequential hypothesis testing. Biometrika 1990; 77(4):875-892. DOI: 10.1093/biomet/77.4.875. (Available from: http://biomet.oxfordjournals.org/cgi/content/abstract/77/4/875 [Accessed on May 2011]).
16. Whitehead J. On the bias of maximum likelihood estimation following a sequential test. Biometrika 1986; 73(3):573-581. DOI: 10.1093/biomet/73.3.573. (Available from: http://biomet.oxfordjournals.org/cgi/content/abstract/73/3/573 [Accessed on May 2011]).
17. Troendle JF, Yu KF. Conditional estimation following a group sequential clinical trial. Communications in Statistics: Theory and Methods 1999; 28:1617-1634.
18. Pepe MS, Feng Z, Longton G, Koopmeiners J. Conditional estimation of sensitivity and specificity from a phase 2 biomarker study allowing early termination for futility. Statistics in Medicine 2009; 28(5):762-779.
19. Koopmeiners JS, Feng Z, Pepe MS. Conditional estimation after a group sequential diagnostic biomarker study that allows early termination for futility. Statistics in Medicine 2012; 31(5):420-435.