Case Studies: How Patient Input Is Changing R&D
US FDA's patient-focused drug development meeting on sickle cell disease inspired Global Blood Therapeutics to develop a new patient-reported outcomes instrument, while an externally led meeting on amyloidosis confirmed for Alnylam that its pivotal trial was studying clinically meaningful secondary endpoints.
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Company revealed that US FDA agreed to accelerated approval pathway for voxelotor in sickle cell disease, with focus on stroke prevention. Updated data from a key trial was also released at ASH.
US agency has started transitioning to a new model for Patient-Focused Drug Development meetings, with agency officials attending four externally hosted meetings so far, with at least three more planned for the next few months. So far, the Neurology Products Division has been particularly active with the external meetings.
At the fifth disease area meeting under PDUFA V’s patient-focused drug development initiative, patients with sickle cell disease talk about how they want their physicians to be more aware of potential studies.