The use of adaptive trial design appears to be increasingly popular in the pharmaceutical industry but has not yet become common practice in academic research. It is used by industry as it can shorten the development time of drugs and moves away from the traditional phase I, II and III categories that we are familiar with. Obviously for pharma this has commercial potential. However for our setting of conducting clinical research that is often not about registering products perhaps this approach might be highly beneficial?
Adaptive design is a clinical study design that uses accumulating data to decide how to modify aspects of the study as it continues, without undermining the validity and integrity of the trial. The goal of adaptive designs is to learn from the accumulating data and to apply what is learned as quickly as possible. In such trials, changes are made “by design,” and not on an ad hoc basis; therefore, adaptation is a design feature aimed to enhance the trial. The point is that there is flexibility within the design and the study responds as it progresses to new data and outcomes of the study. The use of adaptive design could, should? Lead to better treatments and would lend particularly well to disease management studies where a concept needs to be proven. Typically this has involved what would normally be called a phase II trial, as first step. This trial is completed, the data analysis reported and published and then the next step of phase III is planned and rolled out to gather the necessary efficacy and safety data in larger numbers and probably more varied settings and patient populations. If adaptive design was used then two different trials would not have been needed. Imagine the reduction in cost and time? More intelligent and flexible use of data as it is accumulated would likely allow for the question to be answered in less time.
I suggest that many of our health issues in developing countries could greatly benefit from this approach. Does anyone have any experience of this or know of examples?
Dear Dr Sosa.
Thank you for this informative post. We are currently drafting a guidance article on Adaptive Trial Design that will be placed within the Guidance and Resources section within the next few days. We welcome any contributions for any researchers who have experience of designing or conducting clinical trials that have used adaptive design concepts. Some example protocols would be very helpful.