The search for optimality in clinical trials

Summary Much technical work has been devoted to the construction of schemes for optimizing some measure of the outcome of a clinical trial. The main ideas have been the use of data-dependent allocation, by which a majority of patients will tend to be assigned to the better treatment, and a decision-theoretic approach to the optimal allocation of treatment for a large 'horizon', including patients treated after the end of the trial. These ideas have rarely been used in practice, and some of the reasons for their neglect are outlined. It is suggested that more collaboration is needed between the proponents of these models and those statisticians concerned directly with the design and analysis of trials.

[1]  R. Maurice,et al.  A DIFFERENT LOSS FUNCTION FOR THE CHOICE BETWEEN Two POPULATIONS , 1959 .

[2]  F. J. Anscombe Sequential Medical Trials , 1963 .

[3]  T. Colton A Model for Selecting One of Two Medical Treatments , 1963 .

[4]  Peter Armitage,et al.  Sequential Medical Trials: Some Comments on F. J. Anscombe's Paper , 1963 .

[5]  T. Colton A Two-Stage Model for Selecting One of Two Treatments , 1965 .

[6]  A comparison of some sequential designs , 1969 .

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

[8]  Paul L. Canner,et al.  Selecting One of Two Treatments When the Responses are Dichotomous , 1970 .

[9]  Thomas A. Louis,et al.  Sequential treatment allocation in clinical trials , 1971 .

[10]  J. Lellouch,et al.  L'essai therapeutique: ethique individuelle ou ethique collective? , 1971 .

[11]  T. Louis,et al.  Sequential medical trials with data dependent treatment allocation , 1972 .

[12]  D. Berry A Bernoulli Two-armed Bandit , 1972 .

[13]  K McPherson,et al.  Statistics: the problem of examining accumulating data more than once. , 1974, The New England journal of medicine.

[14]  P. Meier,et al.  Statistics and medical experimentation. , 1975, Biometrics.

[15]  G. Weiss,et al.  A Survey of Adaptive Sampling for Clinical Trials , 1975 .

[16]  P. Armitage,et al.  Sequential medical trials. 2nd edition. , 1975 .

[17]  T. Louis Optimal allocation in sequential tests comparing the means of two Gaussian populations , 1975 .

[18]  G. J. G. Upton,et al.  The importance of the patient horizon in the sequential analysis of binomial clinical trials , 1976 .

[19]  E. Nordbrock An improved play-the-winner sampling procedure for selecting the better of two binomial populations , 1976 .

[20]  P. Armitage,et al.  Design and analysis of randomized clinical trials requiring prolonged observation of each patient. I. Introduction and design. , 1976, British Journal of Cancer.

[21]  T. Louis,et al.  Sequential allocation in clinical trials comparing two exponential survival curves. , 1977, Biometrics.

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

[23]  M. Pike,et al.  Design and analysis of randomized clinical trials requiring prolonged observation of each patient. II. analysis and examples. , 1977, British Journal of Cancer.

[24]  I. Olkin,et al.  Selecting and Ordering Populations: A New Statistical Methodology , 1977 .

[25]  S. Pocock Group sequential methods in the design and analysis of clinical trials , 1977 .

[26]  Donald A. Berry,et al.  Modified Two-Armed Bandit Strategies for Certain Clinical Trials , 1978 .

[27]  B. Iglewicz,et al.  An Extension of Colton's Model for Comparing Two Medical Treatments , 1978 .

[28]  Peter Armitage,et al.  THE DESIGN OF CLINICAL TRIALS , 1979 .

[29]  J. Gittins Bandit processes and dynamic allocation indices , 1979 .

[30]  P. Armitage The Analysis of Data from Clinical Trials , 1979 .

[31]  P. O'Brien,et al.  A multiple testing procedure for clinical trials. , 1979, Biometrics.

[32]  C. Begg,et al.  Sequential analysis of comparative clinical trials , 1979 .

[33]  L. Hayre Two-population sequential tests with three hypotheses , 1979 .

[34]  Sylvester Rj,et al.  Design of phase II clinical trials in cancer using decision theory. , 1980 .

[35]  B. W. Brown,et al.  Statistical controversies in the design of clinical trials—Some personal views* , 1980 .

[36]  B. Turnbull,et al.  A class of simple approximate sequential tests for adaptive comparison of two treatments , 1981 .

[37]  Herman Chernoff,et al.  Sequential medical trials involving paired data , 1981 .

[38]  J. Bather Randomized Allocation of Treatments in Sequential Experiments , 1981 .

[39]  F. Mosteller,et al.  Timely topics in statistical methods for clinical trials. , 1982, Annual review of biophysics and bioengineering.

[40]  D. DeMets,et al.  Fundamentals of Clinical Trials , 1982 .

[41]  R. Burkhardt,et al.  Basic problems in controlled trials. , 1983, Journal of medical ethics.

[42]  D W Vere,et al.  Problems in controlled trials--a critical response. , 1983, Journal of medical ethics.

[43]  J. Whitehead,et al.  A FORTRAN program for the design and analysis of sequential clinical trials. , 1983, Computers and biomedical research, an international journal.

[44]  A decision theoretic approach to the design of phase II clinical trials in cancer , 1984 .

[45]  B. Turnbull,et al.  Repeated confidence intervals for group sequential clinical trials. , 1984, Controlled clinical trials.

[46]  P. Macaskill,et al.  Stopping rules for clinical trials incorporating clinical opinion. , 1984, Biometrics.

[47]  T. Louis,et al.  Clinical trials : issues and approaches , 1984 .

[48]  J. A. Bather,et al.  On the Allocation of Treatments in Sequential Medical Trials , 1985 .

[49]  M. Buyse,et al.  Cancer Clinical Trials: Methods and Practice. , 1985 .