Conditional estimation of sensitivity and specificity from a phase 2 biomarker study allowing Early termination for futility

Statistics in Medicine

Volumn 28, Issue 5, 2009, Pages 762-779

  Pepe, Margaret Sullivan ^a   Feng, Ziding ^a   Longton, Gary ^a   Koopmeiners, Joseph ^a  

^a Fred Hutchinson Cancer Research Center   (United States)

Author keywords

Diagnostic test; Group sequential; Screening; True positive rate

Indexed keywords

TUMOR MARKER; BIOLOGICAL MARKER;

ARTICLE; BINOMIAL DISTRIBUTION; CANCER SCREENING; CLINICAL RESEARCH; CONTROLLED STUDY; DIAGNOSTIC TEST; EXPERIMENTAL DESIGN; FALSE POSITIVE RESULT; FEMALE; HUMAN; OVARY CANCER; PREDICTION; PROBABILITY; RISK ASSESSMENT; SENSITIVITY AND SPECIFICITY; STATISTICAL ANALYSIS; ALGORITHM; COMPUTER SIMULATION; CONFIDENCE INTERVAL; DIAGNOSTIC PROCEDURE; EPIDEMIOLOGY; NEOPLASM; NONPARAMETRIC TEST; SAMPLE SIZE; STATISTICAL MODEL;

ALGORITHMS; BIAS (EPIDEMIOLOGY); BIOLOGICAL MARKERS; COMPUTER SIMULATION; CONFIDENCE INTERVALS; DIAGNOSTIC TECHNIQUES AND PROCEDURES; HUMANS; MODELS, STATISTICAL; NEOPLASMS; PROBABILITY; SAMPLE SIZE; SENSITIVITY AND SPECIFICITY; STATISTICS, NONPARAMETRIC;

EID: 64549120274     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.3506     Document Type: Article

References (17)

1. Pepe MS, Etzioni R, Feng Z, Potter JD, Thompson M, Thornquist M, Winget M, Yasui Y. Phases of biomarker development for early detection of cancer. Journal of the National Cancer Institute 2001; 93:1054-1061. DOI: 10.1093/jnci/93.14.1054.
2. Institute of Medicine. Workshop Summary: Developing Biomarker-based Tools for Cancer Screening, Diagnosis, and Therapy - The State of the Science, Evaluation, Implementation, and Economics. National Academies Press: Washington, DC, 2006.
3. Mazumdar M. Group sequential design for comparative diagnostic accuracy studies: implications and guidelines for practitioners. Medical Decision Making 2004; 24:525-533. DOI: 10.1177/ 0272989X04269240.
4. Mazumdar M, Liu A. Group sequential design for comparative diagnostic accuracy studies. Statistics in Medicine 2003; 22:727-739. DOI: 10.1002/sim.1386.
5. Jung S-H, Kim K-M. On the estimation of the binomial probability in multistage clinical trials. Statistics in Medicine 2004; 23:881-896. DOI: 10.1002/sim.1653.
6. Bickel PJ, Doksum KA. Mathematical Statistics: Basic Ideas and Selected Topics. Holden-Day: San Francisco, 1977; 121.
7. Whitehead J. The Design and Analysis of Sequential Clinical Trials. Elis Horwood: Chichester, 1983.
8. Whitehead J. On the bias of maximum likelihood estimation following a sequential test. Biometrika 1986; 73:461-471. DOI: 10.1093/ biomet/73.3.573.
9. Pepe MS. The Statistical Evaluation of Medical Tests for Classification and Prediction. Oxford University Press: Oxford, 2003.
10. Brown LD, Cai TT, DasGupta A. Interval estimation for a binomial proportion. Statistical Science 2001; 16:101-133. DOI: 10.1214/ss/1009213286.
11. Armitage P. Sequential methods in clinical trials. American Journal of Public Health 1958; 48:1395-1402.
12. Jennison C, Turnbull BW. Confidence intervals for a binomial parameter following a multi-stage test with application to MIL-STD 105D and medical trials. Technometrics 1983; 25:49-58.
13. Emerson SS, Fleming TR. Parameter estimation following group sequential hypothesis testing. Biometrika 1990; 77:875-892. DOI: 10.1093/biomet/77.4.875.
14. Emerson SS. Computation of the uniform minimum variance unbiased estimator of a normal mean following a group sequential trial. Computational Biomedical Research 1993; 26:68-73. DOI: 10.1006/cbmr.1993.1004.
15. Emerson SS, Kittelson JM. A computationally simpler algorithm for the UMVUE of a normal mean following a group sequential trial. Biometrics 1997; 53:359-365.
16. Kiefer J. Conditional confidence statements and confidence estimators (with Discussion). Journal of the American Statistical Association 1977; 72:789-827.
17. Strickland PA, Casella G. Conditional inference following group sequential testing. Biometrical Journal 2003; 45:515-526.