All Times EDT
Keywords: Early Clinical Development, Quantitative Decision Making, Bayesian Methods
In most pharmaceutical companies there is a separation between early and late stage clinical development. Questions that single studies in early clinical development address range from characterizing the PK-profile via finding and analyzing biomarkers and PD-parameters to providing first hints of efficacy in small trials in patients often termed Proof-of-Concept (resp. Mechanism) trials. Overall the key output of early clinical development is also the provision of robust planning assumptions for subsequent clinical trials ideally coupled with an assessment of the remaining uncertainty. Statisticians in this area work together with other quantitative scientists like pharmacometricians, biomarker specialists, and researchers. This often leads to study designs that are considered informative in early clinical development, but not in late stage clinical development often applying Bayesian approaches integrating data sources provided by these partners. Another aspect of the work a statistician in this area has to take care of is to prepare the handover to statisticians in Phase IIb/III clinical development including the design of an efficient phase-II-program in close collaboration with statisticians from late stage clinical development. From a methodological perspective there is still more uptake of Bayesian and adaptive methods to contribute to quantitative decision making in this area of drug development compared to Phase IIb/III clinical development. In contrast to the above sketched importance of early clinical development the big bulk of applied statistical literature and conferences focusses on applications in late stage clinical development. It is the aim of this roundtable to bring together statisticians working in early clinical development and to foster the exchange on a) statistical methodology, b) efficient planning of the early clinical development program, and c) working relationships that statisticians in this field encounter in particular.