Cautious Optimism Surrounding Rare Diseases and Orphan Drugs

Wayne chats with Geoffrey Levitt, Head of Global Regulatory Law at argenx, and Frank Sasinowski, Director at Hyman, Phelps & McNamara. You'll hear their thoughts on the recently introduced plausible mechanism framework for individualized therapies in ultra-rare diseases; where FDA is making meaningful progress for rare-disease sponsors and where gaps persist; the agency's willingness to exercise regulatory flexibility in rare diseases; recent experiences in dealing with FDA on orphan drugs; how the agency has fared in translating concepts such as historical data, real-world evidence, and patient engagement into review decisions; and much more. In our headlines segment, Wayne highlights these major developments:  Pharma: FDA-backed proposals aim to entice pharma companies to test and produce drugs domestically  Devices: FDA launches READI-Home Innovation Challenge  Food: FDA seeking public input on potential market name change for 18 species of rockfish  Cosmetics: Processors must complete first biennial FDA facility registration <span class= "NormalTextRun SCXW938