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New sources of patient level data, such as so-called real world data, are presenting new opportunities for advancing medical product development. Well balanced external controls created from such patient level data can enhance the interpretation of single arm trials and even be used to augment randomized controlled trials in certain indications.
This talk will present how external controls are being used in the development of a product to treat recurrent glioblastoma. This includes creation of a propensity score weighted external control aligned to a single arm phase 2 study and a planned design for the phase 3 confirmatory study utilizing a hybrid control that includes both randomized and external control patients that has been accepted in principle by regulators at an end-of-phase 2 meeting. Inverse probability treatment weighting for estimation of the average treatment effect on the treated (also called weighting by odds) is utilized and will be described.
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