4.5 Article

Optimal adaptive designs for binary response trials

Journal

BIOMETRICS
Volume 57, Issue 3, Pages 909-913

Publisher

INTERNATIONAL BIOMETRIC SOC
DOI: 10.1111/j.0006-341X.2001.00909.x

Keywords

clinical trials; ethics; expected treatment failures; Neyman allocation; power; randomized play-the-winner rule; sequential design

Funding

  1. NCI NIH HHS [R01 CA087746] Funding Source: Medline
  2. NIDDK NIH HHS [3-R29-DK51017-05S1] Funding Source: Medline
  3. PHS HHS [1-R29-51017-05] Funding Source: Medline

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We derive the optimal allocation between two treatments in a clinical trial based on the following optimality criterion: for fixed variance of the test statistic, what allocation minimizes the expected number of treatment failures? A sequential design is described that leads asymptotically to the optimal allocation and is compared with the randomized play-the-winner rule, sequential Neyman allocation, and equal allocation at similar power levels. We find that the sequential procedure generally results in fewer treatment failures than the other procedures, particularly when the success probabilities of treatments are smaller.

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