Advertisement

A Review of Available Software and Capabilities for Adaptive Designs

  • Yevgen TymofyeyevEmail author
Chapter
Part of the Statistics for Biology and Health book series (SBH)

Abstract

This chapter provides a brief review of methodologies and software solutions for several types of adaptive designs: the traditional and adaptive group sequential designs including sample size reestimation, multistage adaptive designs with arm and subpopulation selection at interim analyses, and adaptive designs for dose-finding studies.

Keywords

Adaptive design software Simulations Group sequential Many-to-one comparison Arm selection Population enrichment Dose-ranging 

Notes

Acknowledgments

The author would like to thank Steve Ascher for his help with writing this chapter.

References

  1. Anderson KM (2007) Optimal spending functions for asymmetric group sequential designs. Biom J 49(3):337–345MathSciNetCrossRefGoogle Scholar
  2. Anderson KM, Clark JB (2010) Fitting spending functions. Stat Med 29(3):321–327MathSciNetGoogle Scholar
  3. Armitage P, McPherson CK, Rowe BC (1969) Repeated significance tests on accumulating data. J Roy Stat Soc A 132:235–244MathSciNetCrossRefGoogle Scholar
  4. Bauer P (1989) Multistage testing with adaptive designs. Biom Inform Med Biol 20:130–148Google Scholar
  5. Bauer P, Kieser M (1999) Combining different phases in the development of medical treatments within a single trial. Stat Med 18:1833–1848CrossRefGoogle Scholar
  6. Bauer P, Brannath W, Posch M (2001) Flexible two stage designs: an overview. Methods Inf Med 40:117–121Google Scholar
  7. Berry SM, Carroll RJ, Ruppert D (2002) Bayesian smoothing and regression splines for measurement error problems. J Am Stat Assoc 97:160–169MathSciNetCrossRefzbMATHGoogle Scholar
  8. Bornkamp B, Bretz F, Dmitrienko A, Enas G, Gaydos B, Hsu CH, Koenig F, Krams M, Liu Q, Neuenschwander B, Parke T, Pinheiro J, Roy A, Sax R, Shen F (2007) Innovative approaches for designing and analyzing adaptive dose-ranging trials (with discussion). J Biopharm Stat 17:965–995CrossRefGoogle Scholar
  9. Bornkamp B, Pinheiro JC, Bretz F (2009) MCPMod: an R package for the design and analysis of dose-finding studies. J Stat Software 29(7):1–23Google Scholar
  10. Bornkamp B, Bretz F, Dette H, Pinheiro JC (2011) Response-adaptive dose-finding under model uncertainty. Ann Appl Stat 5:1611–1631MathSciNetCrossRefzbMATHGoogle Scholar
  11. Brannath W, Posch M, Bauer P (2002) Recursive combination tests. J Am Stat Assoc 97:236–244MathSciNetCrossRefzbMATHGoogle Scholar
  12. Brannath W, Zuber E, Branson M, Bretz F, Gallo P, Posch M, Racine-Poon A (2009) Confirmatory adaptive designs with Bayesian decision tools for a targeted therapy in oncology. Stat Med 28:1445–1463MathSciNetCrossRefGoogle Scholar
  13. Bretz F, Pinheiro J, Branson M (2005) Combining multiple comparisons and modeling techniques in dose–response studies. Biometrics 61:738–748MathSciNetCrossRefzbMATHGoogle Scholar
  14. Bretz F, Koenig F, Brannath W, Glimm E, Posch M (2009) Adaptive designs for confirmatory clinical trials. Stat Med 28:1181–1217MathSciNetCrossRefGoogle Scholar
  15. Cui L, Hung HMJ, Wang S-J (1999) Modification of sample size in group sequential clinical trials. Biometrics 55:853–857CrossRefzbMATHGoogle Scholar
  16. Dmitrienko A, Tamhane AC (2009) Gatekeeping procedures in clinical trials. In: Dmitrienko A, Tamhane AC, Bretz F (eds) Multiple testing problems in pharmaceutical statistics. Chapman and Hall/CRC Press, New York, NYCrossRefGoogle Scholar
  17. Dragalin V, Bornkamp B, Bretz F, Miller F, Padmanabhan SK, Perevozskaya I, Pinheiro J, Smith JR (2010) A simulation study to compare new adaptive dose-ranging designs. Stat Biopharm Res 2:487–512CrossRefGoogle Scholar
  18. Dunnett CW (1955) A multiple comparison procedure for comparing several treatments with a control. J Am Stat Assoc 50:1096–1121CrossRefzbMATHGoogle Scholar
  19. Emerson SS, Fleming TR (1989) Symmetric group sequential test designs. Biometrics 45:905–923MathSciNetCrossRefzbMATHGoogle Scholar
  20. Friede T, Stallard N (2008) A comparison of methods for adaptive treatment selection. Biom J 50:767–781MathSciNetCrossRefGoogle Scholar
  21. Friede T, Parsons N, Stallard N, Todd S, Valdes-Marquez E, Chataway J, Nicholas R (2011) Designing a seamless phase II/III clinical trial using early outcomes for treatment selection: an application in multiple sclerosis. Stat Med 30:1528–1540MathSciNetGoogle Scholar
  22. Ivanova A (2006) Escalation, up-and-down and A + B designs for dose-finding trials. Stat Med 25:3668–3678MathSciNetCrossRefGoogle Scholar
  23. Ivanova A, Bolognese JA, Perevozskaya I (2008) Adaptive dose finding based on t-statistic for dose–response trials. Stat Med 27:1581–1592MathSciNetCrossRefGoogle Scholar
  24. Jennison C, Turnbull BW (2000) Group sequential methods with applications to clinical trials. Chapman & Hall/CRC, Boca RatonzbMATHGoogle Scholar
  25. Kieser M, Bauer P, Lehmacher W (1999) Inference on multiple endpoints in clinical trials with adaptive interim analyses. Biom J 41:261–277CrossRefzbMATHGoogle Scholar
  26. Kim K, DeMets DL (1987) Design and analysis of group sequential tests based on the Type I error spending rate function. Biometrika 74:149–154MathSciNetCrossRefzbMATHGoogle Scholar
  27. Koenig F, Brannath W, Bretz F, Posch M (2008) Adaptive Dunnett tests for treatment selection. Stat Med 27:1612–1625MathSciNetCrossRefGoogle Scholar
  28. Lachin JM (2005) A review of methods for futility stopping based on conditional power. Stat Med 24:2747–2764MathSciNetCrossRefGoogle Scholar
  29. Lan KKG, DeMets DL (1983) Discrete sequential boundaries for clinical trials. Biometrika 70:659–663MathSciNetCrossRefzbMATHGoogle Scholar
  30. Lehmacher W, Wassmer G (1999) Adaptive sample size calculations in group sequential trials. Biometrics 55:1286–1290CrossRefzbMATHGoogle Scholar
  31. Marcus R, Peritz E, Gabriel KR (1976) On closed testing procedures with special reference to ordered analysis of variance. Biometrika 63:655–660MathSciNetCrossRefzbMATHGoogle Scholar
  32. Muller H-H, Schafer H (2001) Adaptive group sequential designs for clinical trials: combining the advantages of adaptive and of classical group sequential approaches. Biometrics 57:886–891MathSciNetCrossRefGoogle Scholar
  33. Pampallona S, Tsiatis AA (1994) Group sequential designs for one-sided and two-sided hypothesis testing with provision for early stopping in favor of the null hypothesis. J Stat Plann Infer 42:19–35MathSciNetCrossRefzbMATHGoogle Scholar
  34. Parsons N, Friede T, Todd S, Valdes-Marquez E, Chataway J, Nicholas R, Stallard N (2012) An R package for implementing simulations for seamless phase II/III clinical trials using early outcomes for treatment selection. Comput Stat Data Anal 56:1150–1160MathSciNetCrossRefGoogle Scholar
  35. Posch M, Bauer P, Brannath W (2003) Issues in designing flexible trials. Stat Med 22:953–969CrossRefGoogle Scholar
  36. Posch M, Koenig F, Branson M, Brannath W, Dunger-Baldauf C, Bauer P (2005) Testing and estimation in flexible group sequential designs with adaptive treatment selection. Stat Med 24:3697–3714MathSciNetCrossRefGoogle Scholar
  37. Proschan MA, Hunsberger SA (1995) Designed extension of studies based on conditional power. Biometrics 51:1315–1324CrossRefzbMATHGoogle Scholar
  38. Robertson T, Wright FT, Dykstra RL (1988) Order restricted statistical inference. Wiley, New York, NYzbMATHGoogle Scholar
  39. Stallard N, Friede T (2008) A group-sequential design for clinical trials with treatment selection. Stat Med 27:6209–6227MathSciNetCrossRefGoogle Scholar
  40. Temple R (1994) Special study designs: early escape, enrichment, studies in non-responders. Comm Stat Theor Meth 23:499–531CrossRefzbMATHGoogle Scholar
  41. Temple R (2005) Enrichment designs: efficiency in development of cancer treatments. J Clin Oncol 23:4838–4839CrossRefGoogle Scholar
  42. Tukey JW, Ciminera JL, Heyse JF (1985) Testing the statistical certainty of a response to increasing doses of a drug. Biometrics 41:295–301CrossRefzbMATHGoogle Scholar
  43. Wang S-J, Hung HMJ, O'Neill RT (2009) Adaptive patient enrichment designs in therapeutic trials. Biom J 51(2):358–374MathSciNetCrossRefGoogle Scholar
  44. Wassmer G (2006) Planning and analyzing adaptive group sequential survival trials. Biom J 48:714–729MathSciNetCrossRefGoogle Scholar
  45. Wassmer G, Vandemeulebroecke M (2006) A brief review on software developments for group sequential and adaptive designs. Biom J 48(4):732–737MathSciNetCrossRefGoogle Scholar
  46. West M, Harrison J (1997) Bayesian forecasting and dynamic models, 2nd edn. Springer, New York, NYzbMATHGoogle Scholar
  47. Zhu L, Ni L, Yao B (2011) Group sequential methods and software applications. Am Stat 65(3):127–135MathSciNetCrossRefzbMATHGoogle Scholar

Copyright information

© Springer Science+Business Media New York 2014

Authors and Affiliations

  1. 1.Model Based Drug Development DepartmentJanssen Research and DevelopmentTitusvilleUSA

Personalised recommendations