All Times EDT
This presentation will describe how external controls impacted the clinical development of a new product intended for treatment of recurrent glioblastoma, a serious disease with high unmet medical need. I will present the creation of a propensity score based external control to align to a single arm phase 2 trial that supported the internal go/no-go development decision and provided an estimate of the magnitude of the treatment effect that subsequently was utilized in the design of the phase 3 study. The phase 3 confirmatory trial design will also be shared. It is a patient centric design with a hybrid randomized and external control created using propensity score methods that allows more patients to be assigned to the investigational arm in the setting of this very serious disease with an inadequate standard of care.