Patients’ Preferred Endpoints May Not Satisfy Regulatory Needs, US FDA Says
Just because a measure is relevant to the patient community does not mean it is capable of capturing meaningful change in a clinical trial, US FDA officials say at a meeting on mitochondrial disease drug development.
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Lilly is positioning the ramucirumab/erlotinib combination as a first-line alternative for EGFR-positive non-small cell lung cancer that preserves AstraZeneca’s Tagrisso for second-line use; advisory committee members vote 6-5 for expanded indication but express skepticism about adoption.
Agency’s clinical experts favor granting access under regulations intended for investigational drugs but ‘lawyers want consistency under the law,’ FDA’s Woodcock says; use of the expanded access pathway for approved agents is generally unnecessary but may provide sponsors with some regulatory cover, external experts say.