Over the past several years, regulators, including the US Food and Drug Administration (FDA), European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Device Agency (PMDA), have provided guidance on the use of adaptive design for clinical development, which has accelerated industry adoption of adaptive design trials.
The FDA’s Critical Path Initiative identifies adaptive design as key to improving trial efficiency. Early stopping, sample size optimization and changing allocation are based on interim estimates of dose and dose-response curve. The FDA encourages this approach because, “when properly implemented, [adaptive design] can reduce resource requirements, decrease time to study completion, and/or increase the chance of study success”.
The EMA welcomes studies utilizing adaptive design, recognizing that “such a design can speed up the process of drug development or can be used to allocate resources more efficiently without lowering regulatory standards.”
Read more about how FDA and EMA thinking on adaptive designs has progressed significantly, and support for the approach has strengthened, in the following brief history.