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. Adaptations include sample size optimization and changing allocation based on the optimal 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”[1]. The requirements for proper implementation are outlined in ICON’s white paper, Adaptive Design for Medical Device Development. 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.”[2]

FDA and EMA thinking on adaptive designs has progressed significantly, and support for the approach has strengthened, as can be seen in the brief history found in our full-length blog post. Download this blog to learn more.