DIA/FDA Biostatistics Industry and Regulator Forum

Any trial design that improves the speed, efficiency, or flexibility of drug development over the standard randomized controlled trial design could be described as innovative. Novel approaches to clinical trial designs may be needed to bring new treatments to patients faster, particularly in often rare and more targeted patient populations. Complex innovative trials may feature the use of early surrogate endpoints, adaptive designs permitting mid-study changes to the trial based on accruing data, Bayesian methods leveraging trial-external information, and master protocols studying several treatments or disease subtypes under the same overarching protocol. The use of the ICH E9(R1) estimand framework remains relevant for complex innovative designs when the objective is to estimate a treatment effect, where trials answering several questions will have multiple estimands which may evolve during the trial as a result of pre-planned adaptations. In this session, through discussion and case-studies, we will explore innovative approaches to clinical trial design and review how the estimand framework can be applied to complex innovative trials.