Adaptive designs (ADs) allow pre-planned changes to an ongoing trial without
compromising the validity of conclusions and it is essential to distinguish pre-planned from …

Historical data are important for the design of a clinical trial. Yet these data are rarely used in
the analysis of the actual trial. While justifiable in certain situations, ignoring historical data …

Non-alcoholic steatohepatitis (NASH) is the progressive form of nonalcoholic fatty liver
disease (NAFLD) and a disease with high unmet medical need. Platform trials provide great …

Platform trials have become increasingly popular for drug development programs, attracting
interest from statisticians, clinicians and regulatory agencies. Many statistical questions …

Background In clinical trials with fixed study designs, statistical inference is only made when
the trial is completed. In contrast, group sequential designs allow an early stopping of the …

Stopping an ongoing clinical trial based on an interim analysis that shows poor outcomes,
often referred to as a judgment of “futility”, is a familiar feature in current clinical trials …

Background Pre-planned futility analyses are commonly used in oncology studies. The
LUME-Lung 2 study (NCT00806819; 1199.14) was stopped early based on a pre-planned …

Count data and recurrent events in clinical trials, such as the number of lesions in magnetic
resonance imaging in multiple sclerosis, the number of relapses in multiple sclerosis, the …

Evidence‐based quantitative methodologies have been proposed to inform decision‐
making in drug development, such as metrics to make go/no‐go decisions or predictions of …

Background Group sequential designs incorporating the option to stop for futility at the time
point of an interim analysis can save time and resources. Thereby, the choice of the futility …