What Is A Rare Pediatric Disease? US FDA's Orphan Products Office Hopes To Update Guidance On Definition
The Office of Pediatric Therapeutics is working with the Office of Orphan Products Development to make rare pediatric disease determinations, which can help sponsors obtain a priority review voucher.
You may also be interested in...
US Combo Product Designation Appeal Process Limited In Proposed Rule
Overhaul of reg on how combo products are designated to review centers eliminates the reconsideration appeal, but US FDA still allows appeals up the chain of command.
US FDA's Acting Orphan Products Development Director Has Gene Therapy Background
Ilan Irony will take over as acting director of the Office of Orphan Products Development later this month.
Priority Review Voucher Crash: Spark's Sale To Jazz Lowest Price Since 2014
Only the first publicly disclosed sale of a priority review voucher was lower than what Jazz Pharmaceuticals paid Spark Therapeutics for its coupon.