324
Views
5
CrossRef citations to date
0
Altmetric
Original Articles

Estimation of Treatment Effect Following a Clinical Trial with Adaptive Design

, , &
Pages 700-718 | Received 16 Dec 2010, Accepted 16 Feb 2012, Published online: 31 May 2012

REFERENCES

  • Arjas , E. , Haara , P. ( 1984 ). A marked point process approach to censored failure data with complicated covariates . Scandinavian Journal of Statistics 11 : 193 – 209 .
  • Arjas , E. ( 1989 ). Survival models and martingale dynamics (with discussion) . Scandinavian Journal of Statistics 16 : 177 – 225 .
  • Amitage , P. ( 1958 ). Sequential methods in ckinical trials . American Journal of Public Health 48 : 1395 – 1402 .
  • Bauer , P. , Köhne , K. ( 1994 ). Evaluations of experiments with adaptive interim analyses . Biometrics 50 : 1029 – 1041 .
  • Box , G. E. P. , Jenkins , G. M. ( 1962 ). Some statistical aspects of adaptive optimization and control . In: Kotz , S. , Johnson , N. L. , eds. Breakthroughs in Statistics . Vol. II. New York , Inc. : Springer-Verlag , p. 370 .
  • CHMP/EWP/2459/02 . ( 2007 ). Reflection Paper on Methodological Issues in Confirmatory Clinical Trials Planned with an Adaptive Design. October 18 .
  • Chow , S. C. , Chang M. ( 2007 ). Adaptive Design Methods in Clinical Trials . Boca Raton , FL : Chapman & Hall .
  • Cox , D. R. , Lewis , P. A. W. ( 1972 ). Multivariate point processes . Proc. 6th Berkeley Symp. Math. Statist. Prob. 3 : 401 – 448 .
  • Cui , L. , Hung , H. M. J. , Wang , S.J. ( 1999 ). Modification of sample size in group sequential clinical trials . Biometrics 55 : 321 – 324 .
  • Emerson , S. S. , Fleming , T. R. ( 1990 ). Parameter estimation following group sequential hypothesis testing . Biometrika 77 ( 4 ): 875 – 892 .
  • Feng , J. ( 1999 ). Estimations in survival analysis – a stochastic filtering perspective Applied Probability Trust. October 28 .
  • Fisher , R. A. ( 1925 ). Statistical methods for research workers . In: Kotz , S. , Johnson , N. L. , eds. Breakthroughs in Statistics . Vol. II. New York : Springer-Verlag , pp. 66 – 81 .
  • Fisher , L. D. ( 1998 ). Self-designing clinical trials . Statistics in Medicine 17 : 1551 – 1562 .
  • Hu , F. , Rosenberger , W. F. ( 2003 ). Optimality, variability, power: evaluating response-adaptive randomization procedures for treatment comparisons . Journal of the American Statistical Association 98 ( 463 ): 671 – 678 .
  • Jennison , C. , Turnbull , B. W. ( 1999 ). Turnbull Group Sequential Methods with Applications to Clinical Trials . Boca Raton , FL : Chapman & Hall/CRC .
  • Johnson , N. L. , Kotz , S. ( 1970 ). Continuous Univariate Distributions-1 . New York : John Wiley & Sons, chap. 13 .
  • Li , G. , Zhu , J. , Ouyang , S. P. , Xie , J. , Deng , L. , Law , G. ( 2009 ). Adaptive design for interim dose selection . Statistics in Biopharmaceutical Research 1 ( 4 ): 366 – 376 .
  • Liu , Q. , Anderson , K. M. ( 2008 ). Theory of inference for adaptively extended group sequential designs with applications in clinical trials. Journal of the American Statistical Association, Supplemental Archive. http://www.amstat.org/publications/jasa/ supplemental_materials .
  • Liu , Q. , Chi , G. Y. H. ( 2001 ). On sample size and inference for two-stage adaptive designs . Biometrics 57 : 172 – 177 .
  • Liu , Q. , Proschan , M. A. , Pledger , G. W. (2002). A unified theory of two-stage adaptive designs. Journal of the American Statistical Association 97:1034–1041.
  • Luo , X. , Wu , S. , Xiong , J. ( 2010 ). Parameter estimation following an adaptive treatment selection trial design. Biometrical Journal. Doi: 10.1002/bimj.200900134 .
  • Martinussen , T. , Scheike , T. H. ( 2006 ). Dynamic Regression Models for Survival Data . New York , NY : Springer, Science + Business Media Inc .
  • Pocock , S. J. , Simon , R. ( 1975 ). Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial . Biometrics 31 : 103 – 115 .
  • Proschan , M. A. , Hunsberger , S. A. ( 1995 ). Designed extension of studies based on conditional power . Biometrics 51 : 1315 – 1324 .
  • Sampson , A. R. , Sill , M. W. ( 2005 ). Drop-the-losers design: normal case . Biometrical Journal 47 : 257 – 268 .
  • Sampson , A. R , Sill , M. W. ( 2009 ). Drop-the-losers design: binomial case . Computational Statistics and Data Analysis 53 : 586 – 595 .
  • Shen , L. ( 2001 ). An improved method of evaluating drug effect in a multiple dose clinical trial . Statistics in Medicine 20 : 1913 – 1929 .
  • Stallard , N. , Friede , T. ( 2008 ). A group-sequential design for clinical trials with treatment selection . Statistics in Medicine 27 : 6209 – 6227 .
  • Stein , C. ( 1945 ). A two sample test for a linear hypothesis whose power is independent of the variance . Annals of Mathematical Statistics 16 : 243 – 258 .
  • U.S. Food and Drug Administration. (2010). Guidance for Industry Adaptive Design Clinical Trials for Drugs and Biologics. February. Available at: www.fda.gov/DrugsGuidance- ComplianceRegulatoryInformation/Guidances/UCM201790.pdf
  • Wang , S. J. , O'Neill , R. T. , Hung , H. M. J. ( 2007 ). Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset . Pharmceutical Statistics 6 : 227 – 244 .
  • Zelen , M. ( 1969 ). Play the winner rule and the controlled clinical trial . Journal of the American Statistics Association 64 : 131 – 146 .
  • Zhang , L. , Hu , F. , Cheung , S. , Chan , W. ( 2007 ). Asymptotic properties of covariate-adjusted response-adaptive designs . Annals of Statistics 35 ( 3 ): 1166 – 1182 .

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.