Statistical Inference with Data-Dependent Treatment Allocation Rules

Abstract In comparing two treatments with dichotomous responses, the randomized play-the-winner rule (Wei and Durham 1978) tends to assign more study subjects to the better treatment. For ethical reasons, this property is desirable for studies on human subjects. The randomized play-the-winner rule, which is a modification of Zelen's play-the-winner rule (Zelen 1969), is not deterministic and is less vulnerable to experimental bias than other adaptive designs. Recently, this design has been used in a trial to evaluate extracorporeal membrane oxygenation (ECMO) for treating newborns with respiratory failures at the University of Michigan. In this article, exact conditional, exact unconditional, and approximate confidence intervals for the treatment difference are studied from a frequentist point of view with the randomized play-the-winner rule. For small and moderate-sized trials, the exact unconditional procedures perform much better than the conditional ones because of the adaptive nature of the designs. ...

[1]  William H. Press,et al.  Numerical recipes , 1990 .

[2]  J. Ware.  Investigating Therapies of Potentially Great Benefit: ECMO , 1989 .

[3]  L. J. Wei,et al.  Exact two-sample permutation tests based on the randomized play-the-winner rule , 1988 .

[4]  S L Beal,et al.  Asymptotic confidence intervals for the difference between two binomial parameters for use with small samples. , 1987, Biometrics.

[5]  W. Press,et al.  Numerical recipes in C. The art of scientific computing , 1987 .

[6]  William H. Press,et al.  Numerical recipes in C. The art of scientific computing , 1987 .

[7]  Robert H. Bartlett,et al.  Randomized play the winner clinical trials , 1986 .

[8]  J. Ware,et al.  Extracorporeal circulation in neonatal respiratory failure: a prospective randomized study. , 1985, Pediatrics.

[9]  R. G. Cornell,et al.  Extracorporeal circulation in neonatal respiratory failure: a prospective randomized study. , 1985, Pediatrics.

[10]  S. Wallenstein,et al.  Extracorporeal membrane oxygenation and the play the winner rule. , 1985, Pediatrics.

[11]  Samy Suissa,et al.  Exact Unconditional Sample Sizes for the 2 Times 2 Binomial Trial , 1985 .

[12]  P. Armitage.  The search for optimality in clinical trials , 1985 .

[13]  Samy Suissa,et al.  Exact unconditional sample sizes for the 2×2 binomial trial , 1985 .

[14]  T. Santner,et al.  Small-Sample Confidence Intervals for p1 p2 and p1/p2 in 2 2 Contingency Tables , 1980 .

[15]  L. J. Wei,et al.  The Generalized Polya's Urn Design for Sequential Medical Trials , 1979 .

[16]  L. J. Wei,et al.  The Randomized Play-the-Winner Rule in Medical Trials , 1978 .

[17]  R. Simon,et al.  Adaptive treatment assignment methods and clinical trials. , 1977, Biometrics.

[18]  Franklin A. Graybill,et al.  Introduction to the Theory of Statistics, 3rd ed. , 1974 .

[19]  M. Zelen,et al.  Play the Winner Rule and the Controlled Clinical Trial , 1969 .

[20]  J. Wolfowitz,et al.  An Introduction to the Theory of Statistics , 1951, Nature.