Duchenne Group's Presentation Is Milestone For Patient Involvement
Advocacy group gets portion of Sarepta's formal presentation period to present eteplirsen patient experience data, believed to be a first for an advisory committee meeting. Will it become commonplace?
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Patient advocates asked what they could do after US FDA’s complete response letter for the proposed Duchenne drug, but Sarepta's CEO said the company would get the product back on track to approval itself, a marked contrast to engaged role the community played the last time the firm ran into a roadblock at the agency.
Agency wants public comment on creating clinical outcome assessments that could be adaptable to multiple trials.
Patient Engagement Collaborative expected to invite stakeholders to discuss how patient interaction with FDA can improve.