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An R package for implementing simulations for seamless phase II/III clinical trials using early outcomes for treatment selection

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  • Parsons, Nick
  • Friede, Tim
  • Todd, Susan
  • Marquez, Elsa Valdes
  • Chataway, Jeremy
  • Nicholas, Richard
  • Stallard, Nigel

Abstract

Adaptive seamless phase II/III clinical trial designs allowing treatment selection at an interim analysis have gained much attention because of their potential benefits compared to more conventional drug development programmes with separate trials for individual phases. A scenario of particular interest is that in which the final outcome in the trial is based on long-term follow-up, but the interim analysis can only realistically be based on early (short-term) outcomes. A new software package (asd) for the statistical software R implements simulations for designs of this type, in addition to the simpler scenario where treatment selection is based on the definitive (final) outcome. The methodology is briefly described and two examples of proposed trial designs in progressive multiple sclerosis are provided, with R code to illustrate application of the methodology.

Suggested Citation

  • Parsons, Nick & Friede, Tim & Todd, Susan & Marquez, Elsa Valdes & Chataway, Jeremy & Nicholas, Richard & Stallard, Nigel, 2012. "An R package for implementing simulations for seamless phase II/III clinical trials using early outcomes for treatment selection," Computational Statistics & Data Analysis, Elsevier, vol. 56(5), pages 1150-1160.
    • Handle: RePEc:eee:csdana:v:56:y:2012:i:5:p:1150-1160
    DOI: 10.1016/j.csda.2010.10.027
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    References listed on IDEAS

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    1. Walter Lehmacher & Gernot Wassmer, 1999. "Adaptive Sample Size Calculations in Group Sequential Trials," Biometrics, The International Biometric Society, vol. 55(4), pages 1286-1290, December.
    2. Brannath W. & Posch M. & Bauer P., 2002. "Recursive Combination Tests," Journal of the American Statistical Association, American Statistical Association, vol. 97, pages 236-244, March.
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