PAREXEL - The Benefits of Adaptive Trial Designs by Dr Sy Pretorius, Chief Scientific Officer
It is also important to understand the potential benefits of adaptive design, and, in my opinion, there are many. The most important benefit of adaptive design in my opinion is the fact that they provide more information per dollar invested. In other words, they are not necessarily cheaper or faster than traditional trial designs but because you have the opportunity to adjust course and make corrections for some inaccurate assumptions the information gained per dollar invested is typically a lot richer and valuable in these trials. A second, important benefit is the fact that adaptive trials increase flexibility in what is typically a very rigid clinical trial process, all of which can simplify the journey between science and new treatments for the patients who need them most. In other words, you have the flexibility to reduce the trial size or to stop or modify the trials as you go and, by doing so shorten development time and speed up the development process. All of these modifications are planned at the beginning of the trial. Importantly, there is also an ethical benefit to adaptive trials in my opinion. Let me explain: Under the traditional trial paradigm patients would sometimes be in a treatment arm that is clearly not effective; adaptive trials give us the opportunity to either stop subjecting these patients to ineffective, or possible harmful doses, or to move these patients from an ineffective arm to a more effective treatment arm. A very attractive advantage of adaptive trials is that they allow us to address one of the biggest areas of concern in clinical trials today – the high failure rate of Phase III clinical trials. Today, approximately 50% of trials in Phase III fail which have a tremendous economic and social cost. A major reason for this failure is that the wrong dose is used in the Phase III trial. Several commonly-used adaptive designs target getting much more detail on the dose-response relationship by using accumulating data to adjust randomization allocations to the ‘best’ doses – those yielding better results. The result is to give sponsors a better chance to select the right dose in Phase III and to significantly increase the probability of success in Phase III trials.