Bayesian adaptive trials have a lot of potential to cut down sample sizes in the dose-ranging trials and enable better selection of the best dose to take into pivotal trials. The canonical example is the ASTIN trial, published in Clinical Trials in 2005.
The power of the Bayesian adaptive trial as it is used in the ASTIN trial is that data from all subjects is used to find the dose of choice (in the case of ASTIN, the ED95, or the dose that gives 95% of the efficacy beyond the control). This is in contrast to most parallel-group multi-dose trials, where only trials from a particular treatment group are used to estimate the treatment effect at that dose, and also different from most dose-effect models such as Emax where the dose-response curve is assumed to have a certain shape. For example, the ASTIN trial was able to detect non-monotone dose-response curve (and good thing, too!).
What is notable about the ASTIN trial is that the literature is very transparent on the methodology and the operational aspects of the trial. Thus, the whole clinical trial project team can learn important lessons in the running of any adaptive trial, including modern flexible adaptive trials such as ASTIN.
Though a little heavy on the math, I recommend any clinical trial professional check out the literature on the ASTIN trial (ignoring the math if necessary and concentrating on the overall idea), starting with the article linked above.
The power of the Bayesian adaptive trial as it is used in the ASTIN trial is that data from all subjects is used to find the dose of choice (in the case of ASTIN, the ED95, or the dose that gives 95% of the efficacy beyond the control). This is in contrast to most parallel-group multi-dose trials, where only trials from a particular treatment group are used to estimate the treatment effect at that dose, and also different from most dose-effect models such as Emax where the dose-response curve is assumed to have a certain shape. For example, the ASTIN trial was able to detect non-monotone dose-response curve (and good thing, too!).
What is notable about the ASTIN trial is that the literature is very transparent on the methodology and the operational aspects of the trial. Thus, the whole clinical trial project team can learn important lessons in the running of any adaptive trial, including modern flexible adaptive trials such as ASTIN.
Though a little heavy on the math, I recommend any clinical trial professional check out the literature on the ASTIN trial (ignoring the math if necessary and concentrating on the overall idea), starting with the article linked above.
