Frequentist performance of Bayesian inference with response-adaptive designs.

In controlled clinical trials, where minimizing treatment failures is crucial, response-adaptive designs are attractive competitors to 1:1 randomized designs for comparing the success rates φ(1) and φ(2) of two treatments. In these designs each new treatment assignment depends on previous outcomes through some predefined rule. Here Play-The-Winner (PW), Randomized Play-The-Winner (RPW), Drop-The-Loser, Generalized Drop-the-Loser and Doubly adaptive Biased Coin Designs are considered for new treatment assignments. As frequentist inference relies on complex sampling distributions in those designs, we investigate how Bayesian inference, based on two independent Beta prior distributions, performs from a frequentist point-of-view. Performance is assessed through coverage probabilities of interval estimation procedures, power and minimization of failure count. It is shown that Bayesian inference can be favorably compared to frequentist procedures where the latter are available. The power of response-adaptive designs is generally very close to the power of 1:1 randomized design. However, failure count savings are generally small, except for the PW and Doubly adaptive Biased Coin designs in particular ranges of the true success rates. The RPW assignment rule has the worst performance, while PW, Generalized Drop-the-Loser or Doubly adaptive Biased Coin Designs may outperform other designs depending on different particular ranges of the true success rates.

Assessment and monitoring in clinical trials when survival curves have distinct shapes: a Bayesian approach with Weibull modelling.

The comparison of Weibull distributions with unequal shape parameters, in the case of right censored survival data obtained from independent samples, is considered within the framework of Bayesian statistical methodology. The procedures are illustrated with the example of a mortality study where a new treatment is compared to a placebo. The posterior distributions about relevant parameters, which may provide support for a conclusion of clinical superiority of the treatment, and the predictive distributions, which may guide decision about early stopping at an interim analysis, are considered for a class of appropriate priors.