Keytruda's New Tissue-Agnostic Indication Could Prompt Orphan Policy Changes
US FDA contemplating how to match orphan designation process with drug development moving to molecularly-targeted therapies.
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Orphan Designations For Cancer Drugs: US FDA Urged To Distinguish Tissue-Agnostic From Tumor-Specific Diseases
Generic and brand firms both seem to want ‘very black and white lines’ on orphan exclusivity, but FDA isn't so sure how to distinguish biomarker-targeted disease from histologically-defined disease.
US FDA's draft guidance closes loophole where sponsors could obtain an orphan designation for a pediatric subset and then be exempt from requirements to conduct pediatric studies.