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.
Agency's agreement with rare disease advocacy group will facilitate patient meetings with review divisions, as well as enhance communication of safety information.
US FDA's draft guidance closes loophole where sponsors could obtain an orphan designation for a pediatric subset and then be exempt from requirements to conduct pediatric studies.