Adaptive trial designs in clinical drug development
Heinz Schmidli - Statistical Methodology - Novartis Pharma AG

The use of adaptive trial designs can help to accelerate drug development in the pharmaceutical industry. In contrast to traditional clinical trial designs, adaptive designs allow to change the ongoing trial, without compromising the integrity and validity of the study. Adaptations may include dose selection, the reassessment of the sample size, the selection of a subpopulation, and the early stopping for futility or success. As these interim adaptations can be complex, and have to be done quickly, it is important that the decision making is transparent. Bayesian methods are ideally suited to support these interim adaptations.

Clinical trials with adaptive designs are more complex than traditional clinical trials, and hence the planning of such design is more challenging. To evaluate its operation characteristics, clinical trial simulation are usually necessary.

Examples of adaptive trial designs from early to late stages of clinical drug development are presented to illustrate the concepts.