Our client was developing a pediatric version of a drug already on the market for adults. How could our client make the best use of the data that had already been collected when conducting studies for adults, to improve the quality and design of the pediatric trial?
The Cytel Solution:
(1) Cytel implemented a Bayesian framework of analysis which allowed the data that had been collected during the clinical trials for adults to serve as 'informed priors' for the pediatric trial. In Bayesian methodology, empirical evidence already available at the start of a trial is taken as the 'prior' and then methodically 'updated' throughout the course of the trial. Since Bayesian methods allow the use of data collected before a clincial trial commences, trial time diminishes.
(2) Using the powerful simulation technologies which propel East and Enforesys, Cytel determined that a 2-look adaptive clinical trial design would provide rigorous statistical power and control Type I error within a Bayesian framework.
The expected trial duration dropped by 20% to 40% as a consequence of the interim looks and Bayesian analysis. The interim looks gave rise to the possibility of early stopping, while Bayesian analysis ensures that data that had already been collected could add to the statistical rigor of the trial.
Additionally, the Bayesian design required fewer patients to be enrolled. Sample size dropped by 30%.