Skip to main content
SpringerLink
Log in
Book cover

Practical Considerations for Adaptive Trial Design and Implementation pp 139–155Cite as

A Review of Available Software and Capabilities for Adaptive Designs

  • Chapter
  • First Online:

Part of the book series: Statistics for Biology and Health ((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.

This is a preview of subscription content, log in via an institution.

Chapter
USD   29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD   149.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD   199.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD   199.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Learn about institutional subscriptions

References

  • Anderson KM (2007) Optimal spending functions for asymmetric group sequential designs. Biom J 49(3):337–345

    Article  MathSciNet  Google Scholar 

  • Anderson KM, Clark JB (2010) Fitting spending functions. Stat Med 29(3):321–327

    MathSciNet  Google Scholar 

  • Armitage P, McPherson CK, Rowe BC (1969) Repeated significance tests on accumulating data. J Roy Stat Soc A 132:235–244

    Article  MathSciNet  Google Scholar 

  • Bauer P (1989) Multistage testing with adaptive designs. Biom Inform Med Biol 20:130–148

    Google Scholar 

  • Bauer P, Kieser M (1999) Combining different phases in the development of medical treatments within a single trial. Stat Med 18:1833–1848

    Article  Google Scholar 

  • Bauer P, Brannath W, Posch M (2001) Flexible two stage designs: an overview. Methods Inf Med 40:117–121

    Google Scholar 

  • Berry SM, Carroll RJ, Ruppert D (2002) Bayesian smoothing and regression splines for measurement error problems. J Am Stat Assoc 97:160–169

    Article  MathSciNet  MATH  Google Scholar 

  • 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 17:965–995

    Article  Google Scholar 

  • 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):1–23

    Google Scholar 

  • Bornkamp B, Bretz F, Dette H, Pinheiro JC (2011) Response-adaptive dose-finding under model uncertainty. Ann Appl Stat 5:1611–1631

    Article  MathSciNet  MATH  Google Scholar 

  • Brannath W, Posch M, Bauer P (2002) Recursive combination tests. J Am Stat Assoc 97:236–244

    Article  MathSciNet  MATH  Google Scholar 

  • 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 28:1445–1463

    Article  MathSciNet  Google Scholar 

  • Bretz F, Pinheiro J, Branson M (2005) Combining multiple comparisons and modeling techniques in dose–response studies. Biometrics 61:738–748

    Article  MathSciNet  MATH  Google Scholar 

  • Bretz F, Koenig F, Brannath W, Glimm E, Posch M (2009) Adaptive designs for confirmatory clinical trials. Stat Med 28:1181–1217

    Article  MathSciNet  Google Scholar 

  • Cui L, Hung HMJ, Wang S-J (1999) Modification of sample size in group sequential clinical trials. Biometrics 55:853–857

    Article  MATH  Google Scholar 

  • 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, NY

    Chapter  Google Scholar 

  • 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 2:487–512

    Article  Google Scholar 

  • Dunnett CW (1955) A multiple comparison procedure for comparing several treatments with a control. J Am Stat Assoc 50:1096–1121

    Article  MATH  Google Scholar 

  • Emerson SS, Fleming TR (1989) Symmetric group sequential test designs. Biometrics 45:905–923

    Article  MathSciNet  MATH  Google Scholar 

  • Friede T, Stallard N (2008) A comparison of methods for adaptive treatment selection. Biom J 50:767–781

    Article  MathSciNet  Google Scholar 

  • 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 30:1528–1540

    MathSciNet  Google Scholar 

  • Ivanova A (2006) Escalation, up-and-down and A + B designs for dose-finding trials. Stat Med 25:3668–3678

    Article  MathSciNet  Google Scholar 

  • Ivanova A, Bolognese JA, Perevozskaya I (2008) Adaptive dose finding based on t-statistic for dose–response trials. Stat Med 27:1581–1592

    Article  MathSciNet  Google Scholar 

  • Jennison C, Turnbull BW (2000) Group sequential methods with applications to clinical trials. Chapman & Hall/CRC, Boca Raton

    MATH  Google Scholar 

  • Kieser M, Bauer P, Lehmacher W (1999) Inference on multiple endpoints in clinical trials with adaptive interim analyses. Biom J 41:261–277

    Article  MATH  Google Scholar 

  • Kim K, DeMets DL (1987) Design and analysis of group sequential tests based on the Type I error spending rate function. Biometrika 74:149–154

    Article  MathSciNet  MATH  Google Scholar 

  • Koenig F, Brannath W, Bretz F, Posch M (2008) Adaptive Dunnett tests for treatment selection. Stat Med 27:1612–1625

    Article  MathSciNet  Google Scholar 

  • Lachin JM (2005) A review of methods for futility stopping based on conditional power. Stat Med 24:2747–2764

    Article  MathSciNet  Google Scholar 

  • Lan KKG, DeMets DL (1983) Discrete sequential boundaries for clinical trials. Biometrika 70:659–663

    Article  MathSciNet  MATH  Google Scholar 

  • Lehmacher W, Wassmer G (1999) Adaptive sample size calculations in group sequential trials. Biometrics 55:1286–1290

    Article  MATH  Google Scholar 

  • Marcus R, Peritz E, Gabriel KR (1976) On closed testing procedures with special reference to ordered analysis of variance. Biometrika 63:655–660

    Article  MathSciNet  MATH  Google Scholar 

  • 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 57:886–891

    Article  MathSciNet  Google Scholar 

  • 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 42:19–35

    Article  MathSciNet  MATH  Google Scholar 

  • 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 56:1150–1160

    Article  MathSciNet  Google Scholar 

  • Posch M, Bauer P, Brannath W (2003) Issues in designing flexible trials. Stat Med 22:953–969

    Article  Google Scholar 

  • 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 24:3697–3714

    Article  MathSciNet  Google Scholar 

  • Proschan MA, Hunsberger SA (1995) Designed extension of studies based on conditional power. Biometrics 51:1315–1324

    Article  MATH  Google Scholar 

  • Robertson T, Wright FT, Dykstra RL (1988) Order restricted statistical inference. Wiley, New York, NY

    MATH  Google Scholar 

  • Stallard N, Friede T (2008) A group-sequential design for clinical trials with treatment selection. Stat Med 27:6209–6227

    Article  MathSciNet  Google Scholar 

  • Temple R (1994) Special study designs: early escape, enrichment, studies in non-responders. Comm Stat Theor Meth 23:499–531

    Article  MATH  Google Scholar 

  • Temple R (2005) Enrichment designs: efficiency in development of cancer treatments. J Clin Oncol 23:4838–4839

    Article  Google Scholar 

  • Tukey JW, Ciminera JL, Heyse JF (1985) Testing the statistical certainty of a response to increasing doses of a drug. Biometrics 41:295–301

    Article  MATH  Google Scholar 

  • Wang S-J, Hung HMJ, O'Neill RT (2009) Adaptive patient enrichment designs in therapeutic trials. Biom J 51(2):358–374

    Article  MathSciNet  Google Scholar 

  • Wassmer G (2006) Planning and analyzing adaptive group sequential survival trials. Biom J 48:714–729

    Article  MathSciNet  Google Scholar 

  • Wassmer G, Vandemeulebroecke M (2006) A brief review on software developments for group sequential and adaptive designs. Biom J 48(4):732–737

    Article  MathSciNet  Google Scholar 

  • West M, Harrison J (1997) Bayesian forecasting and dynamic models, 2nd edn. Springer, New York, NY

    MATH  Google Scholar 

  • Zhu L, Ni L, Yao B (2011) Group sequential methods and software applications. Am Stat 65(3):127–135

    Article  MathSciNet  MATH  Google Scholar 

Download references

Acknowledgments

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

Author information

Authors and Affiliations

  1. Model Based Drug Development Department, Janssen Research and Development, Titusville, NJ, 08560, USA

    Yevgen Tymofyeyev

Authors
  1. Yevgen Tymofyeyev
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Yevgen Tymofyeyev .

Editor information

Editors and Affiliations

  1. Department of Clinical Biostatistics, Merck & Co., Inc., Rahway, New Jersey, USA

    Weili He

  2. Johnson & Johnson, Raritan, New Jersey, USA

    José Pinheiro

  3. Department of Clinical Biostatistics, Merck & Co., Inc., Rahway, New Jersey, USA

    Olga M. Kuznetsova

Rights and permissions

Reprints and permissions

Copyright information

© 2014 Springer Science+Business Media New York

About this chapter

Cite this chapter

Tymofyeyev, Y. (2014). A Review of Available Software and Capabilities for Adaptive Designs. In: He, W., Pinheiro, J., Kuznetsova, O. (eds) Practical Considerations for Adaptive Trial Design and Implementation. Statistics for Biology and Health. Springer, New York, NY. https://doi.org/10.1007/978-1-4939-1100-4_8

Download citation

Publish with us

Policies and ethics

Search

Navigation