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A New Clinical Trial Design Combining Phases 2 and 3: Sequential Designs with Treatment Selection and a Change of Endpoint

  • Clinical and Nonclinical Drug Development
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Abstract

This paper describes a method for designing a clinical trial to combine aspects of Phases 2 and 3 of the clinical development program. Specifically, a group sequential design is presented, which incorporates treatment selection based upon a short-term “provisional” endpoint, as is often undertaken in Phase 2 trials, followed by a comparison of the selected treatment with control in terms of a longer-term primary endpoint. An example is given illustrating the methodology and we discuss how this approach may reduce the total number of patients required in the evaluation process without compromising its integrity, leading to more ethical and efficient clinical studies.

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Authors and Affiliations

  1. Medical & Pharmaceutical Statistics Research Unit, The University of Reading, P O Box 240, Earley Gate, Reading, Berkshire, RG6 6FN, United Kingdom

    Susan Todd PhD (Senior Research Fellow) & Nigel Stallard PhD (Principal Research Fellow)

Authors
  1. Susan Todd PhD
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  2. Nigel Stallard PhD
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Corresponding author

Correspondence to Susan Todd PhD.

Additional information

Presented at the DIA 39th Annual Meeting, June 15–19, 2003, San Antonio, Texas.

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Todd, S., Stallard, N. A New Clinical Trial Design Combining Phases 2 and 3: Sequential Designs with Treatment Selection and a Change of Endpoint. Ther Innov Regul Sci 39, 109–118 (2005). https://doi.org/10.1177/009286150503900201

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  • DOI: https://doi.org/10.1177/009286150503900201

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